News history

The HAS published the 2024 activity reports for all nine of its committees this April.

The HAS's highlights in 2024 include:

• Active participation in the development of the new European framework for health technology assessment, implemented on January 12, 2025
• Involvement on the system for assessing innovative procedures outside the nomenclature (RIHN)
• Strengthening its commitment to digital health: opening of the digital access for requests for coverage under procedures dedicated to digital medical devices (DMN) and updating of the framework for mobile health applications
• Publication of recommendations: diagnosis and management of ADHD, "long Covid" care pathways, etc.
• Prevention: neonatal screening for spinal muscular atrophy and VLCAD deficiency, simplification of the vaccination schedule, etc.

Focus on the Transparency Commission (TC) report:

• Renewal of 11 of the 29 members in November 2024
• Continued high activity of the TC, similar to previous years, with a total of 360 opinions issued, including 75 opinions for new drugs
• A significantly longer time for assessment: 111 days on average for registrations and extension of indication (107 and 103 days in 2022 and 2023, respectively)
• Continued high activity for early access requests: 129 requests (including 47 renewals) with the following distribution, 33 AP1, 83 AP2, and 13 continuity of care.
• The proportion of unfavorable opinions for early access is higher than previous years.

Proud to have contributed to this white paper presenting recommendations to strengthen innovation and access to treatments for rare diseases.

France Biotech, an association of healthcare innovation companies, in partnership with the Imagine Institute, a world leader in genetic diseases, organized a thematic day "Accelerating Innovation in Rare Diseases: From Research to Patient Access, Partnerships and Perspectives" on March 11 at the Imagine Institute.

On this occasion, several key recommendations for structuring the sector and accelerating innovation were presented:

1. Strengthen the funding continuum to stimulate innovation
2. Facilitate market access for treatments
3. Stimulate innovation and investment
4. Improve screening and diagnosis

The white paper is available; don't wait any longer to read it, hoping that some of these recommendations will become reality for the benefit of patients with rare diseases.

The public directory lists projects using health data of public interest. This tool ensures transparency and traceability of health data usage and allows for reporting on research, studies, and evaluations conducted in France. Each project is documented by a descriptive sheet, published before the project begins and enriched with results once the study is completed.

The published results include:

• posters summarizing analyses, such as inequalities in access to hospitalization or the costs of rare diseases;
• scientific articles detailing demographic and clinical data, for example, on lung cancer or vitiligo;
• reports on the treatment of rare cancers or the design of innovative algorithms to predict cardiovascular risks;
• slideshows illustrating themes such as the profile of patients hospitalized for COVID-19 or the activities of specialized facilities.

A collective and broad mobilization involving numerous stakeholders such as healthcare professionals, researchers, patients, manufacturers and public authorities, among others, enabled the realization of this new plan with the objective of optimizing overall care between 2025 and 2030.

This new plan is based on 3 pillars (care, research and industry/partnership), and is structured around 4 axes, each axis is accompanied by concrete measures.

• Strengthen the patient journey (life and care journey) around diagnostic wandering (extended access), multidisciplinary coordination, information and training
• Facilitate and accelerate diagnosis: Observatory for the diagnosis of rare diseases to be strengthened, integrate fetopathology, improve antenatal and neonatal screening, strengthen access to new technologies/research and optimize training
• Promote access to treatment: strengthen GRIOT to improve Medical Device and medication management, establish “horizon scanning”, develop links between GRIOT, ANSM, HAS, FSMR, etc. strengthen research on new therapies
• Develop databases and biobanks: promote the collection and use of health data, identify biobanks and create a national collection, strengthen data sharing with Europe

Funding is allocated for the implementation of all of these actions for a total budget of €223.5 million per year.

The Senate voted for the PLFSS 2025 in accordance with the National Assembly, this text is therefore adopted and awaiting the return of the Constitutional Council which should only be a formality and rapid.

Note the main changes:

• The safeguard clause M (Article 29, ex 9): 

1. The application of the calculation of M on the amounts reimbursed by Health Insurance is postponed by one year to 2027 under M 2026
2. For M 2025, a trigger threshold set at 27.25 billion
3. Maximum return on the clause set at 1.6 billion if expenses are contained and in line with forecasts
4. Application of a threshold of 1.75% of turnover for Generics and similar (instead of 2% in 2024)

• Avoid the supply disruptions for medical devices
• Creation of a report on the RIHN

The work on the 2026 PLFSS should begin soon.

During this summit, an inventory of actions undertaken in terms of artificial intelligence in health in France was published as well as the launch of work to build a strategic roadmap on AI in health.

The objective of this inventory is to accelerate AI in health for a more efficient, more human and more accessible health system.

This document presents the initiatives undertaken:

• Actions developed for patients and professionals: improvement of diagnoses, personalization of care and optimized management of resources, etc.
• Actions put in place to support innovation in projects with AI, as part of France 2030 investments and the “Digital Health” acceleration strategy (SASN).
• Actions to define a framework for evaluation, regulation and ethical principles to ensure trusted and secure AI, adapted to the requirements of the health sector.

France wishes to be a major player in the field of artificial intelligence beyond health thanks to structuring assets such as:

• A national strategy deployed from 2018, built on the excellence of French research, the development of computing capacities (supercomputers) and the massive adoption of AI in the economy 
• An ecosystem of 600 startups specializing in AI, which benefits from constantly growing fundraising 
• France is one of seven countries to participate in international benchmark initiatives on AI.

HAS achieved most of the objectives it set during the period of the previous 2019-2024 strategic project, in particular work on user engagement, the early access process to medicines and anticipated pathways to access to innovative medical devices.

The 2025-2030 strategic project is based on the main developments in the health system and the resulting challenges and opportunities: acceleration of health innovation, demographic and epidemiological developments, social and territorial inequalities, and also the financial sustainability of the health system.

3 priorities have been defined:

• Promote the integrated approach to patient’s life pathway
• Defend quality in a strained health system
• Prepare for the future to consolidate the French model

with key themes: prevention, mental health and psychiatry, digital technology and artificial intelligence in health.

For each project, HAS develops its ambitions.

HAS has also published a specific Health-Environment roadmap to identify the actions to be deployed within HAS to better take into account health-environment issues within the framework of its various missions and work.

This study is a first and the analysis will provide ideas for the implementation of a national strategy for the organization of care and support all stakeholders in the health system in order to make treatments available to patients with equitable access and in optimal conditions.

The most promising therapies are gene and cell therapies in the treatment of rare diseases and cancers. Messenger RNA vaccines, oligonucleotides and immunotherapies also represent notable advances.

Until 2027, many of these drugs are under development with more than 600 molecules, 16% of which are biotherapies such as gene therapy.

Cancer still represents a significant part of clinical development with 25% of molecules in development targeting cancers, particularly lung and breast cancers.

Gene therapies target around 60% of rare diseases.

To support these innovation challenges, LEEM proposes in this report solutions to improve access to these new treatments by anticipating and simplifying access procedures.

Its activity was sustained in 2024 with 114 marketing authorizations compared to 77 in 2023. 

Note that there were 46 new drugs compared to 39 in 2023, therefore innovation slowed down a little compared to the number of marketing authorizations in 2024. 

We also see stability regarding orphan drugs with 15 in 2024 vs 17 in 2023 and 1 ATMP each year. 

Note the first drug approved in Alzheimer's disease, 1 vaccine against Chikungunya and 2 antibiotics for severe infections have obtained marketing authorization.

The number of files closed in 2023 (285) is down 18% compared to 2022. This change results from a 32% drop in the number of first registration files closed for generics and a decline of 3% for non-generics.

The median time from receipt of the HAS opinion to publication in the JORF of the 279 files closed with agreement, processed by the CEPS in 2023 was 91 days. These deadlines were 130 days for princeps and 83 days for generics, biosimilars and hybrids.

The deadlines for innovative medicines, i.e. the situation for the 6% of files having an innovative character, are more heterogeneous, leading to median deadlines of 244 days for ASMR III and 275 days for ASMR IV.

For the 70 hospital registration files published in 2023, the median time between registration on the “list en sus” or the retrocession list and publication of the price in the official journal was 132 days compared to 114 in 2022. This time was shorter for generics, biosimilars and hybrids (124 days vs 135 for originator and non-specific)

The savings made by CEPS in terms of price reductions amount to €842 million (including €231 million for the list en sus) identical to those made in 2022.

During 2023, 74 price increase request files were studied by the Committee, three times more than in 2022, among which 24 were deemed eligible and the price increases granted represented a total amount of 11 million euros.

In this report published before Christmas, we note that the negotiation times are lengthening as expected and we especially regret the lack of information on product discounts, early access in 2023, while the figures were available since the companies received their M 2023 invoice mid-November 2024!!

A joint evaluation of health products with the adoption, in December 2021, of a new European regulation will change the practices of national evaluation agencies. 

As a reminder, the regulation on the joint assessment of health technologies published on December 22, 2021 in the Official Journal of the European Union must be gradually implemented with different application deadlines depending on the products:

• January 12, 2025 for the evaluation of new drugs in oncology, including those benefiting from orphan drug status and advanced therapies (advanced medicinal therapeutic products, ATMP)
• January 13, 2028 for the evaluation of orphan drugs (excluding oncology or advanced therapies)
• January 13, 2030 for all categories of new drugs

The objective of this evaluation is to reach a consensus on the clinical benefit of a product.

The regulation provides that States "take due account" of the published joint clinical assessment reports (JCA report) and that they annex to their own national opinions the documentation relating to the European assessment.

Member states will retain responsibility for setting reimbursement and prices.

A new guide (Procedural guidance for JCA) has just been published concerning the European assessment procedures, there are others such as Guidance on scoping process & PICO exercises, Guidance on appointment of assessor and co-assessor, Guidance on filling in the JCA folder template which should follow very soon.

It seems that everything will be ready to begin as planned in the European regulation, the HTA assessment on January 12, 2025.

You can already find the information on the HAS website with a dedicated page and also all the documents and file models related to this European assessment. 

The first files in this framework are expected by HAS in the second half of 2025.

Each year, in application of the social security financing law for 2018, a report is submitted to parliament to take stock of the experimentation with Article 51.

In 2024, it is the completion of 41 experimental projects, more than half of which were completed during the first eight months. This year is therefore decisive since it is a transition from an experimental phase to the integration of the most promising innovations into common law.

Of the 155 projects authorized among the 1,258 submitted, 61 experiments have been completed and more than €640 million have been committed.

The main treatments management completed concern:

Osteoarticular diseases and trauma, Clinical situations linked to aging, Visual sector, Diabetes, Mental health, cardiovascular diseases, cancer, dental health, sexual health, neurodevelopmental disorders, support for autonomy and neurodegenerative neurological diseases.

You will find the progress of all the projects in this very informative report.

The CNIL has just published a map of health data warehouses (EDS) in France.

EDS are databases intended to be reused mainly for purposes of management and research, studies and evaluations in the field of health. They can be constituted by public actors (public healthcare establishment) or private actors (e.g. a startup) but subject to respecting the legal framework.

Faced with the proliferation of DHSs and organizations wishing to create them, it is particularly useful to create a tool allowing both to understand the dynamics and to improve the transparency of the use of health data in the context of research.

This tool allows you to explore current research and find projects that may be relevant to your own research.

This mapping provides access to files including: the name of the actor who manages the EDS and its status, its geographical location, the description of the EDS and sometimes the networks of actors involved.

As of publication, there are approximately 100 EDS that have been implemented since inception in 2017.

Since the reform implemented on July 1, 2021, requests for early access have maintained a sustained rythm with an average of 90 decisions per year and more than 120,000 patients in therapeutic impasse who benefit from its treatments.

Compared to the 2-year assessment, we note:

• A still fairly balanced share between pre-MA and post-MA AP (26 and 33% respectively) and an increase in renewals (40%).
• Unfavorable opinions increase slightly from 22 to 30%.
• The common law assessment decrease slightly from 69% to 61%. 
• Regarding ASMR levels, they remain stable for both ASMR II to IV (79% vs 78%) and ASMR V (21% vs 20%).

Oncology remains the therapeutic area with the greatest number of requests/decisions representing approximately half of the files.

In September, Mario Draghi presented Ursula von der Leyen, President of the European Commission, with his report on the future of Europe's competitiveness.

This report on competitiveness was commissioned by the European Commission from Mario Draghi, former president of the European Central Bank (ECB), in the fall of 2023.

At a time when strong competition is raging between China and the United States, the EU risks finding itself, if it does nothing, torn between issues of sovereignty, environment and social protection.

As a preamble, the Draghi report recalls that Europe has all the necessary bases to establish itself as a power as competitive as China and the United States, with its own assets and which outline a model other than that of the two others: that of social, educational, green, redistributive power. 

However, Europe is undergoing a major competitive downgrade.

Draghi's report therefore proposes three main axes for reforming and relaunching sustainable growth:

1. Innovate and close the technological gap
2. Have a common plan for decarbonization and competitiveness
3. Strengthen security and reduce dependencies

The report makes 170 proposals which you will find in detail in the attached report.

The report gives numerous recommendations which should serve as a basis for discussions within the European Commission.

The social security financing bill (PLFSS) for 2025 aims to contribute to the recovery of public finances, in addition to the measures announced in the finance bill (PLF).

The PLFSS was submitted on October 10, 2024 in the Parliament. Members of parliament will examine the text in public session from October 28, 2024.

The social security deficit is expected to reach 18 billion euros (€ billion) in 2024, instead of the €10.5 billion initially planned by the LFSS 2024. This variation is largely due to the deficit in the health sector, estimated to €14.6 billion due to a significant increase in healthcare expenditure and lower revenue.

In this context, Health Insurance will have to make savings, in particular on: 

• cut price of medicines and medical devices (for €1.2 billion)
• a reduction for covering daily allowances in the event of sick leave (for 600 million euros 
• a reduction in the share of Health Insurance in the reimbursement of consultations with doctors and midwives (for €1.1 billion) and a proportional increase in the share of complementary health insurance
• capping the remuneration of paramedical temporary workers in hospitals and medico-social establishments  
• continuing the fight against fraud

A limited number of articles in the project of law concerning medicines and medical devices compared to previous years (articles 9,19 and 20) allowed a more in-depth analysis by the CAS, particularly around the safeguard clause, where there are still numerous tensions and a certain consensus on shortages. However, the CAS rejected the whole text.

From Monday October 28, the debates will take place in the parliament, to be continued!

This new 2024 edition, published by the National Cancer Institute (INCa), includes most of the recent cancer figures in France.

The main topics concern the epidemiology of all cancers and the main locations, as well as the prevention, screening and care of cancer with a common objective, reducing the incidence and mortality of cancers, while improving patient quality of lives.

In France, cancers represent the leading cause of death in men, and the second in women with 433,136 new cases of cancer in 2023, a doubling in 30 years.

This development is mainly linked to demographic changes and secondarily to an increase in the risk of cancer.

There are many risk factors for the appearance of cancer. They can be internal, linked, for example, to age or family history, or external, linked to behavior or the environment.

It is estimated that almost half of cancers could be prevented by avoiding or limiting the impact of external risk factors such as tobacco, alcohol, overweight/obesity and an unbalanced diet.

Screening makes it possible to diagnose cancer at an early stage, in order to better treat it and limit its after-effects. To date, there are 3 organized screening programs: cervical, breast and colorectal with participation still too low, a new program will be implemented soon for lung cancer.

The public consultation was posted online on the HAS website from February 1 to March 13, 2024 (see SESAM Consulting news from February, 2024)

This public consultation mainly aimed to assess acceptance of the conclusions obtained during the consultation of November 17, 2023 and to understand the precise expectations of the various stakeholders on the methods of transmitting information.

This report therefore presents the results of the public consultation of manufacturers and patient associations. It also makes known the positions of the HAS. It is also intended to constitute a robust basis for regulatory development with stakeholders.

The HAS declares in favor of a regulatory evolution towards opening up information to patient associations, while respecting business secrecy, but warns that in an already tense context this must not increase its workload or its responsibilities.

Such a development is now the initiative of the Social Security Direction, to whom this report was submitted.

To be followed…

The AFCROs 2024 barometer analyzes data from academic clinical research and those from health industries.

The data indicates that in 2023, 446 clinical studies (interventional + observational) were conducted by industry and 1,651 studies in the academic sector. Although France places behind its European competitors in terms of clinical research carried out by industry, it remains the leader in academic clinical research, ahead of Germany, Spain, Italy and the United Kingdom.

By integrating academic clinical research, France remains on the top step of the podium for clinical studies in oncology since 2019. It also has leadership in rare diseases. 

At the European level, this decline in clinical research has affected all countries.

For France to return to positive developments, AFCROs has made several proposals:

• Improve the readability and fluidity of the regulatory process
• Strengthen the efficiency of investigative centers
• Promote the most active investigative centers
• Promote the possibility of simplified access to the SNDS
• Develop transparency and information on studies for patients

Like every year, the ANSM publishes its activity report with a multitude of information on projects and figures as part of its missions (Surveillance, security, safer use, stock shortages, etc.)

Always intense activity concerning the access of innovative treatments to patients with:

• 57,176 compassionate access granted and 24,346 patients treated, 
• 25 positive opinions in the context of pre-MA early access requests
• 687 clinical trials authorized
• 15 MA files in centralized procedure allocated to France
• 99 pediatric investigation plans (PIPs) where France is rapporteur or co-rapporteur
• The Innovation and Orientation Guichet (GIO) has recorded more and more requests each year since 2020, when it was set up (377 in 2023)

The ANSM confirms its important role, both national and European, in responding to the challenge of tensions and disruptions in the supply of medicines and health products. In addition, the ANSM with its European partners has made it possible to prepare recent decisions at Union level, aimed at securing access to drugs of major therapeutic interest. 

DiGA: launched in 2019, the German Digital Health Apps (DiGA) program offers doctors the possibility of prescribing digital therapies to patients reimbursed by Public Health Insurance Funds.

To do this, several criteria must be respected:

• Have CE medical device marking;
• Be based on digital technologies; 
• Contribute to the recognition, monitoring, treatment or mitigation of illnesses, injuries or disabilities; 
• Be used only by the patient or by the patient and the healthcare professional, and not by the healthcare professional alone; 
• Achieve its medical objective mainly thanks to its digital function.

The DiGA registration process consists of two phases:

• Provisional registration (12 months) Companies submit a file to the BfArM (German medicines agency), demonstrating security, interoperability and data protection. During the first 12 months, the company sets the reimbursement rate. 
• Examination for permanent registration: the BfArM re-evaluates the file. In the event of insufficiently documented but promising results, provisional registration may be extended by 12 months. Permanent integration requires price negotiation with the public social security. 

This recent study (J Med Internet Res 2024 | vol. 26 | e59013 | p. 10) highlights how DiGAs have become an important device in modern medicine, with more than 73 million Germans having access to them.

As of July 1, 2024 (since 2019), 56 digital therapies (DTx) have benefited from the DiGA procedure and coverage by health insurance funds.

As every year, this report is very awaited by all stakeholders involved in health, it foreshadows what could be the next Social Security finance bill (PLFSS) for 2025. Discussions are underway for the PLFSS 2025 in order to submit the text to the Parliament before October 1.

This report recommends, unsurprisingly, savings measures amounting to €1.5 billion, like every year (but even more !), in order to reduce the Social Security deficit forecast at €11.4 billion for 2025.

For this, Health Insurance offers options such as: 

• Improve the relevance of care and prevention by always strengthening screening for cancer (breast, uterus and colon) and diabetes, cardiovascular diseases, endometriosis, etc.
• Reduction of drug consumption by reinforcing the proper use and prescription of biosimilars
• Reduction in days of compensated work stoppage
• Continue to control the costs of medical transport, fight against fraud, waste, etc.

In this voluminous report, an inventory and numerous analyzes were carried out to arrive at these proposals.

The PLFSS 2025 vote promises to be complicated in a climate of still great political uncertainty, this will require finding consensus in the event of a blockage.

To be followed soon...

This new report builds on experience previously gained during the period September 2021 to February 2023 to conduct regulatory studies using real-world (RWE) data to support the MA decision.

This report covers the period from February 8, 2023 to February 7, 2024, which corresponds to year 2 of DARWIN EU and provides an overview of the progress made in using RWE to establish their values in the regulatory decision by 2025. The RWE data will be integrated alongside the RCTs which remain the Gold Standard in the MA decision.

In this second report, all RWE data collected by the EMA have been taken into account as well as information on the use of RWE by the competent national authorities.

In total, during the 12 months covered by this report, 22 studies were carried out (9 DARWIN EU studies, 9 FWC (EMA FrameWork Contract) studies and 4 internal studies), and 18 studies were ongoing (10 DARWIN EU studies, 5 FWC studies and 3 internal studies).

The studies carried out focused on 11 different therapeutic areas (according to the ATC classification), and most of them studied anti-infectious, antineoplastic and immunomodulatory agents, as well as the digestive tract and metabolism disorders.

DARWIN EU has now entered its operational phase. The network has grown from 10 to 20 data partners, providing access to data from around 130 million patients from 13 European countries.

Of these studies, 13 are on the safety and effectiveness of vaccines, including public health emergencies.

Two pilot studies to test the integration processes of RWE analyzes by HTA were carried out on: the characterization of patients with multiple myeloma and the overall survival of patients with locally advanced or metastatic lung cancer treated with immunotherapies.

A concept proposal for RWE to assess the possibility of extrapolation from adults to pediatrics was developed.

Real-life data is becoming increasingly important in regulatory decisions.

Among the 4 criteria evaluated by the HAS, taking into account the “adapted clinical development plan” (presumed innovative nature of the drug) when evaluating the early access authorization (taking the bet) is of interest particularly for helping to make a favorable decision to early access for a drug whose clinical benefit is very uncertain, and remains to be established. Especially if the uncertainties about its real clinical benefit are not resolved due to lack of confirmatory data establishing it within a reasonable time frame.

The objective of this working group will be to better supervise the challenge made during the examination of the request for early access to the drug for a pre-MA or post-MA request if a conditional MA.

In its future doctrine, the HAS wishes to better define the minimum data required from the clinical development plan at the time of the bet (granting of an AP). The aim is to allow the CT and the HAS College to have, within a reasonable time, confirmatory data to remove uncertainty about the clinical benefit (removal of the bet).

To determine whether the confirmatory data expected as part of the clinical development plan proposed by the manufacturer (at the time of the bet) have an appropriate level of proof to assess the clinical benefit, the following will be analyzed: the study design, the relevance of the criteria defined by the PICO and the timetable for raising the bet.

The publication of HAS expectations is expected for the 1st quarter of 2025.

The Economic Committee for Health Products (CEPS) and several organizations representing manufacturers (Snitem, UFAT, Clinical Nutrition Companies and AFIDEO) have signed a framework agreement relating to the conventional determination and revision policy. prices of health products.

This framework agreement aims to give visibility to manufacturers and facilitate price negotiations while also taking into account the challenges of reindustrialization of France and health sovereignty.

The main measures for manufacturers are: 

• Benefit from “CSIS credits” and progress on several points during tariff negotiations. 
• Taking into account the specificities and developments of the medical device sector and the wide variety of products which are characterized by a cycle of rapid incremental innovations.
• A possibility of price revaluation.

The framework agreement sets up a Conventional Policy Monitoring Committee (CSPC) with a first meeting in September 2024.

To follow the implementation of this long-awaited framework agreement.

The role of the HAS is to characterize what constitutes progress by discerning new developments from real innovations, and to allow patients to access them quickly and safely, to avoid any loss of opportunity, after an evaluation based on scientific rigor. 

Some highlights:

• Following the assessment of the first 2 years of early access in the fall of 2023, the authorizations have benefited approximately 100,000 patients suffering from a serious disease and in therapeutic impasse.
• The evaluation of remote monitoring medical activities or the publication of a guide to help choose digital medical devices for professional use, including those integrating artificial intelligence.
• Creation of a commission dedicated to the evaluation of diagnostic, prognostic and predictive technologies (CEDiag) in May 2023

Focus on the report of the Transparency Commission (CT)

The Transparency Commission (CT) issued 462 opinions in 2023 (+ 2.6% compared to 2022), including 123 in the context of early access and 220 in the context of registration requests.

Among the 123 early access opinions, 28 concerned opinions on pre-MA early access requests, 81 on post-MA early access requests and 14 on early access continuity requests. Early access really constitutes an important lever for patients to accelerate access to innovative treatments in situations of therapeutic impasse.

The median processing time for applications for registration and extension of common law indication was 103 day less than 2022 (107 days). The median processing time for early access requests (all reasons) was 80 days.

The proportion of ASMR I to III and ASMR IV decreased compared to 2022, from 21 to 14% and 33 to 26% respectively.

When hearings were used, this led to a modification of the CT's conclusions regarding SMR and/or ASMR in 41% of cases (24/58 hearings).

Due to the large number of files to be evaluated, the CT maintains its prioritization method, namely prioritizing the medical needs of patients..

Published every year since 2020, the Doctrine therefore makes it possible to share technical developments in projects carried out by the Digital Health Roadmap.

This doctrine is a reference document for players in the e-health ecosystem, who develop digital services for the benefit of health system professionals and users. It is mainly aimed at digital health companies and more broadly at public or private structures which develop digital health services.

This doctrine presents, in the form of summary sheets, the major new developments since the previous edition, the ambition and the planned trajectory over the coming years.

Unicancer and INCa launched the AcSé program in 2013, focused on innovative clinical trials, in order to expand therapeutic strategies for the benefit of patients in therapeutic failure, with molecular screening. To date, nearly 1,300 patients have benefited from innovative treatments as part of the 5 trials of the 1st AcSé program.

At the end of May 2024, in the AcSé program, 2 new trials “Pan-MSI-AcSé” and “AcSé FGFR” have just been opened for inclusion. These 2 new trials continue to aim to evaluate drugs targeting often rare molecular abnormalities in unauthorized indications, to propose combinations of treatments, and to provide access to innovative therapies.

Pan-MSI-AcSé will focus on evaluating the effectiveness of dostarlimab (anti-PD1) as first-line treatment for certain metastatic or locally advanced unresectable dMMR/MSI cancers.

Microsatellite instabilities (MSI), initially associated with hereditary colon cancer, are found in several types of cancer (intestine, stomach, pancreas, adrenal gland, sarcomas, etc.), thus opening new treatment perspectives.

AcSé FGFR will test a molecule targeting alterations in FGFR genes pemigatinib (tyrosine kinase inhibitor), currently indicated in urothelial cancers and cholangiocarcinomas. This trial will be accessible to patients suffering from other cancers and presenting these same anomalies. This approach will make it possible to make significant advances in the understanding and treatment of these types of cancers.

This new program opens new perspectives for patients facing cancers with a poor prognosis or tumors with rare molecular abnormalities for which targeted therapies exist in other indications.

The HDH (Health Data Hub) publishes in its 2023 report the progress related to its roadmap and access to health data.

Concretely:

• 1 private medicine data warehouse project
• 10 databases available and 24 others expected which should be published during the 2nd half of 2024
• 1 multicenter data warehouse integrating the SNDS and data from 4 hospitals
• Integration into international projects like Quantum, Darwin…
• Setting up conferences, training, publications, etc.

With the objective of greater exploitation of data assets to improve the health of all by involving all partners in the ecosystem.

A serious adverse event associated with care (SAE) is an unexpected event with regard to the state of health and pathology of the person and the consequences of which are death, life threatening, probable occurrence of a permanent functional deficit, including a congenital anomaly or malformation (art. R. 1413-67 of decree no. 2016-1606 of November 25, 2016).

All adverse events are analyzed in order to understand the reasons for their occurrence and find a way to prevent them from happening again. In addition, serious events are declared in order to develop a sharing of experiences at the regional and national levels.

This report addresses SAEs associated with care in emergency departments, in connection with the tensions encountered by emergency departments which can have an impact on the quality and safety of care.

The analysis of SAEs will make it possible to identify their causes and consequences, to draw lessons and, above all, to propose solutions to healthcare establishments and professionals in order to reduce the frequency of occurrence of SAEs or to mitigate their consequences.

The risks identified in this analysis have been known for years (examples: workload, medication errors, etc.) and on which there have already been numerous publications. But the analysis carried out by the HAS shows that the same dysfunctions persist and that we must continue to work on these risks to improve patient safety in emergency departments:

• Continue to streamline the care pathway for patients before, during and after their treatment by emergency services.
• Ensure the safety of patients in unsuitable accommodation.
• Better train professionals in the specificities of emergency services (technical skills) and non-technical skills, particularly teamwork.
• Strengthen the sharing of information necessary for good patient care throughout their journey
• Raise professional awareness of the risks of diagnostic errors (delayed, incorrect or missed diagnosis) and implement diagnostic support tools for the highest risk situations.
• Accentuate the security of medication care and the use of medical devices.
• Better prevent actions by the patient against himself (suicide attempts, suicides, leaving the service without the knowledge).
• Strengthen the involvement of governance and department heads in the operation and organization of emergency services.

Since April 25, 2024, the pricing for Early Support for Digital devices has been published, this Official Journal publication was eagerly awaited by all stakeholders.

PECAN is a procedure recently implemented for presumed innovative digital medical devices to allow faster access to patients.

This coverage by Health Insurance for 1 year makes it possible to finalize the demonstration of the clinical and/or organizational benefit by the manufacturer and to be financed during this time, while awaiting reimbursement under common law (permanent reimbursement ).

The PECAN pricing for digital therapeutics is as follows:

• Initial package of € 435 including tax for the first 3 months, invoiced only once for the same patient.
• Monthly payment of € 38.3 including tax from the 4th month, with an invoicing frequency modulated according to the type of MD.
• Maximum amount of € 780 including tax per year and per patient.

Innovations in health are accelerating and the health supply has never been so important, yet many patients remain without a suitable therapeutic solution, particularly in cases where it is difficult to conduct so-called controlled and randomized clinical trials.

Technological advances could help accelerate the development of health products in these cases of poorly or unmet medical needs.

Based on this observation, a multidisciplinary association with public and private actors was formed to reflect on the use of Artificial Intelligence as an accelerator of innovation in response to these issues.

This white paper details the challenges and prospects offered by the use of “artificial patient” cohorts in clinical trials.

Concretely, “artificial” patients could be created on the basis of already existing clinical data, collected as part of treatment or previous research. It will thus be possible to optimize the conduct of clinical trials by limiting and simplifying patient recruitment needs, while guaranteeing the safety and level of proof of these trials.

The generation of artificial data is an example of a secondary use of health data collected during patient care.

This white paper aims to show how artificial data will play an important role in clinical research and the optimization of care.

In February, after 3 years of implementing this new ten-year strategy, INCa publishes the state of progress.

80% of actions have already been launched out of the 237 actions planned on 4 priority axes.

• Improve prevention: tobacco control program, vaccination campaign against HPV viruses, improve cancer screening, and a particular focus on lung cancer screening and reduce the impact of the environment (smoking, alcohol, UV rays, sedentary lifestyle, pollution, etc.)
• Limit sequela and improve the Quality of life: reduction of toxicity linked to radiotherapy and consider therapeutic de-escalation, early access implemented and positive results, reform of the RIHN
• Fight against cancers with poor prognoses: improve survival rates, actions on the streamlined care pathway, the AcSé research program, increase the imaging offer
• Ensuring that progress benefits everyone: mobilization on childhood and young adult cancers (creation of centers of excellence in pediatric oncology) and new clinical research projects.

A very significant advance in the fight against cancer in this 3rd report.

Since April 17, 2024, the ANSM has modified the conditions for prescribing and dispensing certain biotherapies administered subcutaneously in the treatment of chronic inflammatory diseases.

Since 2019, only the renewal of these drugs has been possible by private specialists concerned.

The possibility of being initiated by private physicians is based on the experience acquired in their use taking into account their safety profile and only applies to biotherapies for which the treatment is administered subcutaneously.

Attached the list of biotherapies affected by this modification and which can be initiated by private specialists:

• Immunosuppressors: Anti-TNFs: adalimumab, certolizumab pegol, étanercept, golimumab, infliximab (only Remsima is considered); Anti IL-1: anakinra, canakinumab; Anti IL-6: tocilizumab, sarilumab; Anti IL-17: bimekizumab, brodalumab, ixékizumab, sécukinumab; Anti IL-23: guselkumab, mirikizumab, risankizumab, tildrakizumab; Anti IL-12 et IL-23: ustékinumab; Anti lymphocytes T: abatacept.
• Others: Anti IL-5: benralizumab, mépolizumab, reslizumab; Anti IL-13: lébrikizumab, tralokinumab; Anti IL- 4 et IL-13: dupilumab; Anti IgE: omalizumab; Anti lymphopoïétine stromale thymique (anti-TSLP): tezepelumab.

RIHN, a dedicated national funding system for biology tests in France, has just been published and applicable the 1rst April 2024.

The eligibility conditions relating to the management of biological or anatomical pathology procedures must meet all of the following conditions:

1. Innovative not limited to technical development
2. In the early phase of distribution on the national territory and have not been the subject, in the indications considered, of an opinion from the HAS
3. The risks for the patient, and where applicable for the operator, linked to the implementation of these procedures have been previously characterized
4. Are likely, based on available data and taking into account any relevant comparators, to present a significant clinical or medico-economic benefit making it possible to satisfy a medical need not or insufficiently covered.

The management of acts is subject to:

• The production of a registry, which the applicant undertakes to organize and finance as soon as these acts are registered and to transmit to the HAS (6 months before the end of the registration on the list) in order to give an opinion on the subsequent payment or reimbursement of these acts
• And the oral and written information of each patient, carried out by the prescriber, on the early and transitional nature of this act.

The acts are covered for a period which cannot exceed six years.

The procedure for requesting registration is now open to manufacturers in addition to the National Professional Councils.

The HAS, once the file is complete, has 75 days to publish an opinion. If additional documentation is requested, this deadline may be extended by 15 days.

This opinion is sent to the Ministry of Health and to applicants before publication on the HAS website.

The ministry has 60 days to decide on registration on the RIHN list.

The amount of the maximum unit value of the act, the duration of the financial support, as well as the list of establishments in which the performance of this act will be covered will be published.

The value is based on the economic data provided and the cost of comparable acts.

From January 1, 2025, the maximum unit value applicable for the act will be applied, compared to the previous year, a reduction equal to 20% of the value set for the year 2024.

From December 7, 2023 until April 8, 2025 you can apply.

This call for projects aims to finance post-CE marking clinical evaluations. These evaluations must be ambitious and precise in order to constitute the final step before being taken over by national solidarity.

The objectives of the study must clearly define the nature of the benefit they seek to demonstrate in relation to the state of the art:

• economic superiority AND medical superiority
• or medical superiority
• or economic superiority (if the clinical value has already been demonstrated)
• or economic superiority AND medical non-inferiority.

The project leader must be a single structure, responsible for developing the device and launching it on the market (CE marking).

Total project expenses between €200,000 and €5 million, for a duration of 12 to 48 months, for medical devices whose innovation is mainly digital.

If you wish to propose a project, apply as quickly as possible before April 9, 2024, the continuity of the call for projects will depend on the remaining financial resources.

Contrast products used in MRI and scanner examinations will now be purchased directly by radiologists (with exceptions).

The complete list of products which will no longer be dispensed by pharmacists was published on March 5, 2024 in the “Official Journal”.

These products will be financed in the packages invoiced by the radiologists (technical packages for imaging procedures or by a billable supplement in addition to the technical package). They will nevertheless remain included on the list of specialties approved for use by hospital, allowing in particular their purchase in health establishments.

Manufacturers, depositaries, wholesalers, etc. will be able to directly supply contrast products to authorized structures.

The ATIH (Technical Agency for Information on Hospitalization) updates this list of expensive drugs each year, excluding the “list en sus” (supplementary list) and excluding AAP, AAC, CPC (Early access program), in HAD (Home Hospitalization).

In addition, a registry concerning these expensive drugs is put in place in order to collect consumption and price data for these drugs not appearing on the supplementary list (List en sus) but which represent a significant cost for HAD. 

Financial support is awarded if patients benefit from these treatments listed on this list.

The medicines covered by this list must meet several criteria:

• Not being discontinued
• Have a significant SMR in each indication concerned
• Have a daily treatment cost (CTJ) per day of HAD > 30% of the average daily amount
• The drug and its generics must have a CTJ higher than the reference CTJ
• The biological drug and its biosimilars must have a CTJ higher than the reference CTJ

These last 2 criteria are currently not taken into account (waiting for the new HAD financing model).

Publication of most of the implementing acts for EU joint HTA has been delayed by 3-6 months, according to the European Commission’s latest update to the rolling plan for implementation of the HTA, for example a delay of 3 months on the “Joint Clinical assessment for medicinal products” planned for Q4-2023 and finally will be available in Q1-2024, a delay of 3 to 6 months on the “cooperation by exchange of information with EMA” with a new deadline in Q2-Q3-2024 etc…

Given the degree of change involved in adopting joint HTA at both EU and national level, it may prove challenging to meet the initial deadlines specified in the HTAR and for all stakeholders to be fully prepared for the new procedures.

The delays will raise questions about the feasibility of being ready for the start of joint clinical assessment (JCA) on 12th January 2025.

France is currently in 2nd position in Europe behind the UK and ahead of Germany and Switzerland with:

• 584 biomedicines in development representing 20% of European biomedicines,
• 31 production sites,
• innovations for patients suffering from diseases that are often rare, incurable or in a situation of therapeutic impasse.

This acceleration is driven by stakeholders in the Biotherapies and Bioproduction sector.

To have a more complete vision, the Health Innovation Agency, France Biotech and France Biolead carried out a study.

The HAS/HTA body (Haute Autorité de Santé) wishes to strengthen patient engagement in the evaluation by proposing that the file submitted by manufacturers be made available to patients.

For this, a public consultation was put online from February 1 to 29 in order to collect opinions on the possible methods of transmitting the files.

The consultation is open to patient associations and manufacturers who must complete a questionnaire available online (HAS website) and submit their comments and suggestions.

The objective after analyzing the contributions is to publish the results in April 2024.

The EMA has recommended a total of 77 Marketing Authorizations (MA) in 2023, including 39 new active substances. Only 3 drugs benefited from the PRIME system (Elrexfio et Talvey in oncology and Casgevy), 8 conditional marketing authorizations for Columvi, Elrexfio, Jaypirca, Krazati, Lytgobi, Talvey and Tepkinly in oncology and Casgevy and 1 drug under exceptional circumstances (Loargys).

19 requests were rejected and 3 received a negative opinion from the CHMP.

1 single ATMP in 2023, this is Casgevy, the first medicine using CRISPR/Cas9 a novel gene-editing technology in the treatment of B-thalassemia and severe sickle cell disease. Two inherited rare diseases caused by genetic mutations that affect the production or function of haemoglobin, the protein found in red blood cells that carries oxygen around the body.

Casgevy also obtained a positive opinion from the HAS (December 20, 2023) in the context of pre-MA early access in beta-thalassemia.

A large part of these new treatments will be evaluated in 2024 by the Transparency Commission in order to obtain reimbursement. To be followed !

The Health Data Hub (HDH) published a report in January 2024 concerning the implementation of around fifteen projects.

These projects concern the provision of data in order to improve the care pathway and the health of French people.

You will find in this report the contribution of the HDH in the development of the data.ansm platform allowing access to all to pharmacovigilance declarations and stock shortages, in the fight against antibiotic resistance (BactHub), in the improvement of the patient care pathway, better prevent allergies, prevent traumatic risks, etc. with numerous projects in collaboration with patient associations and learned societies.

Good reading!

The social security financing law for 2024 (LFSS 2024) was promulgated on December 27, 2023 (JO no. 2023-1250 of December 26, 2023).

It presents a forecast social security balance for 2024 at -€11.3 billion with a deterioration compared to 2023 (-€9.5 billion).

The national health insurance spending objective (Ondam) for 2024 amounts to 254.9 billion euros. This amount is up 3.2% compared to the revised Ondam 2023, excluding crisis expenses.

The major objectives of this law concern:

- Strengthening prevention:

• Facilitate access to the papillomavirus vaccine from 11 years old
• Improve sexual health by facilitating access to condoms
• Fight against menstrual poverty by facilitating access to reusable periodic protection
• Through experiments: testing physical activity programs adapted for patients suffering from cancer and testing a dedicated care pathway for postpartum depression

- The transformation of the financing and organization of the health system:

• Reform of the contribution base for self-employed workers, or that of the financing of health establishments

- The continuation of policies to support autonomy with the construction of a society for aging well and the full inclusion of people with disabilities:

• The creation of a renewable right to the daily allowance for the next caregiver,
• The creation of an identification, diagnosis and early support service for all disability situations for children
• Full reimbursement of wheelchairs covered on the list of products and services.

- Many measures for medicines:

• The removal of the exemption regime for marketing authorizations for medicines derived from blood (Art 29)
• The postponement of the reform of the financing of contrast products until March 1, 2024 (Art 59)
• The new restrictions on the coverage of medicines with high public health or financial implications in a form (Art 73)
• Modifications to the conditions for compassionate use and early access (Art 76)
• The creation of a mechanism for authorizing the temporary use and coverage of cannabis-based medicines (Art 78)
• Dispensing certain medications without a prescription after carrying out a diagnostic orientation test (TROD)(Art 52)
• Substitution of biosimilars permitted 2 years after registration of the first biosimilar in the city (Art 54)
• Measures relating to supply disruptions: conditional dispensation, extension of the powers of the ANSM (Art 72)
• Measures on drugs of major therapeutic interest (MITM) (Art 77)
• Measures relating to safeguard clause M, amount M 2024 at 26.4 billion euros (Art 4, 28 and 29)

Most of these measures are not immediately applicable; implementing decrees are awaited for entry into force.

The Health Innovation Agency was created just 1 year ago as part of the Health Innovation 2030 plan.

This roadmap presents 12 objectives to accelerate access to innovations, whether new technologies, innovative medicines or advanced medical solutions, thus improving our Health System and promoting France internationally. 

These 12 objectives are consolidated into 5 main themes :

1. Anticipate innovations and medical needs to guide public policies
2. Facilitate, accelerate, simplify the journey of an innovation, from idea to availability to patients
3. Support innovative project leaders at each step of their journey
4. Invest to make France the leading innovative and sovereign European nation in Health
5. Integrate prevention as a pillar for a change of scale in terms of results and positive impacts for the health of French people: a major need

This roadmap is part of various current and future initiatives, including those resulting from "Borne report".

When the Transparency Committee (TC) evaluates a new medicine, it’s often faced with uncertainties about its use in current practice, its clinical benefit and adverse events.

To answer these questions, the TC asks the pharmaceutical companies to carry out a post-registration studies.

The pharma companies are responsible for proposing a study protocol to respond to post-registration study requests. The drug evaluation department assesses these protocols and ensures that they are able to answer the questions asked by the TC.

Thanks to this new dynamic dashboard, you have access to the list of drugs concerned by a current post-registration study and also the list of drugs for which the results of the post-registration study have been evaluated by the TC.

It’s possible to search by product name, therapeutic class and to select the objective of the study for example effectiveness, quality of life, conditions of prescription or use, impact on morbi/mortality, on care system, compliance, tolerance…

These lists will be updated every month.

Following a self-referral from the HAS on a proposal from its User Engagement Council (CEU), the HAS set up this project with a framework note validated in September 2023. The first meeting of the working group will take place at the end of November with reports publication expected by the end of 2024.

« Patient experience » is a subject of numerous definitions and covers many dimensions of the healthcare experience.

The objective of this project will be to clarify the notions of « patient experience » and « experiential knowledge » as well as theirs connections with user engagement to help people who act in favor of people being treated or supported : patient organizations, representatives in steering and governance committees, healthcare professsionals, decision-makers…

In this project, a literature review and a methodological guide are the 2 expected deliverables to answer, among others, these questions: How are these notions useful for developing user engagement? What conditions must be met for these concepts to be used?

The project has been operational since April 2018 and regular reviews have been published. 

The 6th update provides an overview by region as well as national experiments and ministerial initiatives. 

This Atlas is a complement to the 2022 Parliament report.

Currently, there are more than 350 projects being tested across the entire country.

In this report, you will find all the projects summarized in a sheet with the project description, project leaders, partners, patients included, experiment duration, financing etc…

Experimenting means innovating to provide better care!

HAS has just put in line a new dashboard concerning the evaluation of medicines in common law (DC) and also in the context of early access (AP). 

This dashboard is more dynamic and updated every month, unlike the previous one which was updated quarterly and included less information.

You can find all of this data : 

• The number of files currently being examined.
• The cumulative numbers of requests since the beginning of the year or previous years. The annual report is presented in 2 parts: common law (first registration and extensions/others) and early access (1rst request/others).
• The median evaluation time in days for DC and AP.
• Opinions or decisions rendered at the end of the evaluation (number of SMR/ASMR granted in DC and favorable decision or not in AP).
• The therapeutic areas concerned : an observation during this update with oncology drugs still by far the therapeutic area with the most evaluation in DC and AP.

Also note the results of early access since its implementation on July 1,2021.

Among the 125 decisions rendered, 98 were favorable, around 80 % with a median deadline of 77 days below the regulatory deadline of 90 days.

This is the 3rd edition of Cancers panorama in France produced by INCa. This new publication presents a summary of recent data concerning the epidemiology of all cancers and the main locations, as well as cancer prevention, screening and care.

New cases of cancer in 2023 amount to 433,136 and this is still growing due in large part to the demographic evolution of our country (Growth and aging of the population), but also to the increase in risk linked to our behavior and our lifestyles, hence the need for strong mobilization of all.

Half of cancers could be avoided thanks to changes in our behavior and lifestyle (tobacco, alcohol, unbalanced diet, sedentary lifestyle, exposure to UV). We could therefore prevent more than 170,000 new cases of cancer each year.

In France, cancers represent the leading cause of death in men and the second in women. Among women, 2 cancers in particular are showing a worrying increase : lung and pancreatic cancers.

Pediatric cancers with a 5-year survival rate now exceed 80 %. This is encouraging, but still need to progress.

Research, prevention and screening are essential to slow this progression.

Among the ANSM’s activities, we can highlight support in providing patients with access to innovative treatments.

In 2022, the ANSM made an initial assessment of the medical cannabis experimentation including 2.296 patients and continued its commitment to the SACHA study on new molecules in pediatric oncology. This study is mainly based on compassionate use data delivered for pediatric cancers.

Concerning Early Access Program (AAP), 30 favorable opinions were given for 21 drugs.

63.340 AAC (Compassionate Use Program) represent more than 27.000 patients treated and 293 drugs (60% of AACs represent 10 products), a large increase compared to previous years.

The ANSM has also invested a lot in managing the risk of stock shortages.

This new PLFSS is part of a complex economic environment at global and european level.

For France, the control of health expenditures is key and the objective is to reduce the Social Security deficit. 

4 Major objectives for this PLFSS 2024:

• Strengthen prevention and access to care
• Transform the financing and organization of the health system
• Fight against fraud
• The continuation of policies to support autonomy

An increase in the ONDAM of 3.2% is expected in 2024, an increase of € 8 billion on a constant basis compared to 2023.

However, € 3.5 billion in savings are expected:

• € 1.3 billion on health products with €1 billion in price reductions (€ 850 million on drugs and € 150 million on medical devices) and € 300 million on volumes
• € 1 million for care (biology acts, contrast products …)
• € 1.25 million on prescriptions 

The main measures concerning  health products are: 

• Stabilization of the mechanism for the safeguard clause M 2023 and 2024.
• Strengthening the obligation for manufacturers to find a buyer in the event of stop of marketing the drug with a major therapeutic interest (MITM).
• Strengthening levers for saving drugs in shortage of supply (delivery of medicines individually).
• Facilitating the registration in common law of organizational experiments article 51.
• Deployment of vaccination campaigns (HPV).
• Expanding the skills of pharmacists in prescribing antibiotics, after a rapid diagnostic orientation test (TROD) for tonsillitis and cystitis.

As every year, at the end of September/beginning of October, PLFSS is annual legislative meeting which sets budgetary constraints of the Social Security including the economic conditions of access to health products in France.

The PLFSS is examined, discussed, amended alternately by the parliament and the Senate during the fall, then the law will be voted on December 2023.

The PLFSS is still awaited by the companies in the health sector, especially regarding the budgetary constraints linked to the amounts of savings to be made each year.

This year, following « Borne » report, it’s expected that the current situation in this PLFSS will be taken into account, particularly with regard to the » safeguard clause » and its amount.

The government took into account the expectations of health compagnies and revealed ahead of PLFSS presentation, the yield of the safeguard clause for 2023 and 2024.

The yield of the safeguard clause will be lowered to 1.6 billion € for 2023 ( compared to the latest estimation around 2 billion €) and stabilized at this level for 2024. The M threshold has not yet been revealed.

The amount of price reductions on health products (drugs and medical devices) will be 1 billion € divided into 850 M€ for drugs and 150 M€ for medical devices (close to previous years).

Certains proposals will also come to: 

• Strengthen the attractiveness of production in France or Europe to enable relocation or avoid relocation;
• Avoid disruptions in supply to the French market;
• Make the rules of early access more flexible to allow innovations to arrive fastly in France.

The PLFSS will be presented to the government on Wednesday September 27 and to the parliament the following week.

The National Health Strategy allows to define the main orientations of health policy for the next 10 years. The analysis was carried out by the High Council for Public Health (HSCP) on the state of health of the population taking into account the Covid-19 sanitary crisis.

Several improvement objectives have been defined among them :

• Enable all citizens to live longer in good health, through prevention, health promotion and support at all ages of life;
• Meet the health needs of everyone, throughout the territory with an adapted health offer;
• Make our health system more resilient and better prepared to face ecological challenges and crises.

This public consultation open until October 2, 2023 will allow to collect the opinions of all French people on the priorities, directions and levers linked to these objectives.

The mission was launched in January 2023 by the Prime Minister in order to make proposals to the Government concerning the regulation and financing of Health products (drugs and medical devices).

This report representents a substantial work carried out by the 6 experts in charge of this analysis with an expected observation on the difficulties of our current regulatory system.

Many proposals (63) are made in this report with the objective that some of them will be a basis for the next PLFSS 2024 which should be availbale soon.

Among these proposals, it’s noted that market access procedures are increasingly complex and it’s necessary to simplify them by reviewing clinical and medico-economic assessments, pricing and conventional life to take into account diversity of Health products and innovation.

Another important point for manufacturers is financial regulation with the « Clause M », this tool should be limited.

All the other proposals could be part of France 2030 and Innovation Santé plans.

After this important milestone, we are awaiting deployment in the next few weeks or months!

The report submitted each year by Health Insurance to the Ministers responsible for Health and Social Security and to Parliament on the evolution of Health Insurance costs is intended to inform the preparation and debates of the Social Security Financing Law (PLFSS) and Health policies more broadly.

Access to healthcare remains a major issue and a daily concern for many people.

The population approach focuses on a population and its specific health needs. This year, the report focuses on 2 distinct populations : the young and elderdy.

The approach by pathologies wishes to lead priority approaches for : diabetes, heart failure, cancers and mental health.

To respond to these ambitious public health actions, Health Insurance formulated 30 innovative proposals.

A the end of its General Council in June 2023, the SNITEM (National Union of the Medical Technology Industry) published its annual report.

The report reviews the 12 major projects of the recent last months, the most important projects were : 

• The postponement of the transition period of the MDR regulation in order to ensure successful compliance, but also an effective evaluation of the products on the European market (postponement under conditions until the end of 2027 or the end of 2028)
• Participation to the evolution of the care management and the regulation mechanism and MD financing by proposing solutions to the Government (interministerial mission on health products)
• Challenges regarding additional costs linked to inflation by developing new organizational models 

Data collection in the context of early access program remains a major challenge. HAS has therefore adapted its model for drugs indicated in rare diseases by using the BNDMR data bank (National Rare Disease Data Bank) and by encouraging the use of pre-existing data sources.

This model is to be used since July 15, 2023.

The 10 reports concerning all the HAS commissions were published on June 29. HAS ‘s activity in 2022 was once again busy by continuing the post-COVID work and the assessments of large number of early access requests.

The report of the Transparency Committee highlights the major achievements of 2022 :

• Consolidation of the assessments of early access demand, involving the Transparency Committee (opinion) and the HAS College (for the decision) with a process review (Early access doctrine update)
• Review of the common law doctrine (published early 2023)
• Contributions from patients organizations during registration and early access procedures representing approximately 53% (157 open contributions)
• A median timelines for applications (registration and extension of indication) of 107 days, slightly longer than the regulatory time limits of 90 days
• The number of files increased by around 10% in 2022 vs 2021
• 66 new drugs received a favorable opinion for reimbursement with therapeutic progress identified for 34 clinical situations (ASMR II to IV).

Article 59 of the 2022 Social Security Financing Law (LFSS) allowed the creation of a new article in the Social Security Code to regulate the reimbursement of particular drugs:

• registered for at least one of their indication on the « liste en sus » (supplementary list);
• likely to be used in combination, concomitantly or sequentially with other drugs which have in these indications a Marketing Authorization (MA) from coverage or from an early access;
• but which do not have an approval or reimbursement in these indications where they are used in combination, concomitantly or sequentially.

It requires to the manufacturers to inform the Economic Committee (CEPS), no later than February 15, of the total turnover of their product for the previous year.

The reimbursement publication will be done in the Official Journal within 90 days after request to the ministers.

The decree details also the associated annual discounts (according to a progressive scale awaiting publication) and the end of reimbursement of a drug by order of the ministers:

• when the drugs is removed from the « liste en sus »;
• when the drugs with which it is used in combination are stopped or no longer covered by the early access or under common law;
• when the manufacturer does not pay the rebates.

Coverage also stops if the product is approved for hospital or obtained an early access authorization in the considered indications.

According to IQVIA report published recently, it should be noted that oncology continues to develop strongly with many innovations to come. 

The costs associated with treating more patients for longer and with more advanced therapies is bringing stress to healthcare budgets. 

More than 2,000 products are currently under development with 250 only with CAR T cell.

mRNA vaccines development in cancer is also increasing strongly from 8 in 2017 to 21 in 2022.

Chinese companies are playing an important rôle in this market with a contribution of 22% in the pipeline compared to only 5% 10 years ago.

These 5 types of tumors (breast, non small cell lung, prostate, multiple myeloma and kidney) currently represent 53% of sales. PD/PD-L1 inhibitors have strongly contributed to this evolution.

Between 2018-2023, the launch average is 23 anti-cancer treatments/year.

The campaign for acts evaluation has been open since June 1rst and until October 3, 2023 with the objective to be registered in the 2024 HAS work program.

The National Professional Councils (CNP) can submit a file on the EVActe platform. The request must be done only by CNP.

To complete the application file, you must:

• Fully completed application form
• Mandate to submit an application completed and signed by the applicant and the legal representative of CNP
• Articles/scientific studies/professional recommendations to justify the request

HAS proposes 3 support methods:

• Strategic orientation meetings
• Early meetings when your act is new
• Pre-submission appointements

All of these meetings are free, optional, confidential and non-binding.

The objective of this new platform is to facilitate access to drug data for everyone (individuals, healthcare professionals and manufacturers) and ensure greater transparency.

Data.ansm consolidates data from adverse event reports, medications errors and drug stock-out since 2014.

ANSM in partnership with the Health Data hub has developed this platform from 5 already existing databases: Open Medic, Codex database, medication errors database, pharmacovigilance and Trustmed for drug stock-outs.

Statistical data will be updated each year, in April for the previous year.

France, as many countries, is facing an aging population and an large increase of chronic diseases : people aged 75 and more will represent 16% of the population in 2050 and more than 20% of them have 3 pathologies with chronic medicines. This modify our health system, initially designed around the hospital care.

Ambulatory practices were initiated in France, twenty years ago, with the creation of day hospital (HDJ). These activities were subsequently structured with the emergence of outpatient surgery, but also with the development of home hospitalization care (HAD).

It’s essential to promote the patients return home after hospitalization and allow more care directly at home. These new health pathways include a large number of health actors, beyond the hospital.

The emergence of many innovations in recent years has contributed to significantly transforming healthcare pathways and offering new modes of care thanks to technical innovations (surgical robot which reduces the time of interventions, etc.), technological innovations (remote monitoring, etc.) and therapeutic innovations (oral cancer drugs, etc.).

Despite this, France is still struggling to initiate the ambulatory shift, it’s in this context that the study conducted by Institut Montaigne with a large number of health actors has published 6 operational proposals to build new care pathways, more efficient, more fluid and more balanced between ambulatory care, hospital and home.

The innovation package aims to facilitate patient access to innovative health technologies in the early development phase. The innovation package set up in 2015 has evolved during the time to better meet the manufacturers and healthcare professionals expectations. 

Since 2015, 16 health technologies (45%) have received a positive opinion from HAS (10 MD and 6 acts) for access to the innovation package.

To make the diagnosis of type 1 glucose transporter deficiency encephalopathy, hypoglycorachia must be used as a reference test, glycorachia requiring a lumbar puncture.

METAglut1™ is an in vitro diagnostic Medical Device (DM-DIV), carried out on a blood sample, to highlight a deficiency in the glucose transporter expression on the circulating erythrocytes surface.

In 2017, METAFORA Biosystems marketing the METAglut1™ test obtained a positive opinion from HAS to realize a clinical study as part of the innovation package. The clinical study aimed to evaluate the diagnostic performance of the METAglut1™ test in patients with type1 glucose transporter deficiency encephalopathy, thus making it possible to provide important clinical data to claim a reimbursement.

At the end of the clinical study in April 2022, the DGOS contacted the HAS to take into account the results of the clinical study. The results confirm the diagnostic performance of this test based on blood sample and are very similar to those of glycorachy with lumbar puncture.

METAglut1™ test has therefore been granted by the HAS a level II (ASA II), which will lead the Health Insurance to decide on its effective reimbursement within maximum period of 6 months.

Following the appointment of Professor Lionel Collet as President of the HAS College published in the Official Journal of April 20, Claire Compagnon, Anne-Claude Crémieux and Jean-Yves Grall were appointed members of the HAS College by decree of April 21.

They succeed within the College to Elisabeth Bouvet, Cédric Grouchka and Valérie Paris whose mandates ended on April 9th.

The mandates of the new members are for a period of 6 years.

Claire Compagnon is a lawyer specializing in Health and will take her new position on June 1.

Anne-Claude Crémieux is an infectious disease specialist, University Professor and hospital practitioner, she works at the Saint-Louis hospital Paris (AP-HP)

Jean-Yves Grall, cardiologist by training, has notably been Director of several regional health agencies (ARS)  and Director of Health (DGS).

Professor Lionel Collet is expected to be appointed President of the HAS College, the mandate of the current President Pr Dominique Le Guludec ending on April 9.

The President of the HAS, as its other members, is appointed by decree of the President of the Republic. The mandate is for 6 years.

Before this formal appointment, he must be auditioned by the social affairs committee of the parliament and the senate. Both committees voted in favor of his appointment.

Prof. Lionel Collet was a University Professor and Hospital Practitioner (PU-PH), ENT and psychiatrist at the Hospices Civils de Lyon (HCL) from 2000 to 2012 and the President of Claude Bernard University Lyon I from 2006 to 2011. He has also a position in the office of the MoH with Pr Agnès Buzyn, then held numerous positions as CSIS coordinator, INTS and CNPC President etc.

For HAS, he believes that the place of patients in the expertise will be strengthened and insists on the importance to give more expertise to human and social sciences.

The new HAS commission recently created is the Commission for the assessment of Diagnostic, prognostic and predictive Health Technologies (CEDiag)

This commission will assess all diagnostic, prognostic and predictive health technologies whether they are professional acts (radiology or nuclear medicines, medical biology and anatomocytopathology etc.), drugs for diagnostic purposes (radiopharmaceuticals, contrast product in imaging) or medical devices (self-test …)

It may be supported the other commissions for its expertise such as the TC, CNEDiMTS or the CEESP. 

The members of this commission have all been appointed from April 2023 to April 2026.

Following its recent publication of 2022 report (end of March 2023), the HDH has developed its 2023-2025 roadmap.

The major achievements for HDH are: 

• Selection of databases to improve the catalog (hospital, oncology databases …) and launch of a data warehouse project for private medicine
• Reduced access times to the health data thanks to training improvement and numerous projets (73 projects including 22 initiated in 2022)
• Improvement of the offer on the technical aspect
• Involvement of citizens in order to access to health data
• Contribution to the future European Health data space
• Establishement of more than hundred partnerships

As the 2019-2022 roadmap is coming to end, a new roadmap has been developed including the 4 strategic priorities already present in the 2019-2022 map but which are reinforced, clarified and prioritized.

The AFCROs barometer published on March 20 confirms France as a leader in clinical research in Europe.

French clinical research is still strongly oriented to interventional trials (1443 in 2022) in oncology and rare diseases even if slight decrease (10 %) has been noted in 2022.

Among observational studies (933 in 2022), it’s also a slight decrease (17%).

However, this decreased activity is also observed in other european countries. 

Clinical research had been strongly supported in recent years by COVID studies, which is no longer the case, and also impacted by many changes in 2022 and for the future (political and financial crises, new regulations, etc.).

The remote monitoring prices publication will be effective within a few weeks.

From July, 1rst, digital medical devices, accessories and associated medical services may be covered as part of remote monitoring activities.

The activities will be take in charge with a package for the digital medical devices, the remote medical follow-up and some possibilities to modulate the package under certain conditions. The package will be reimbursed at 100 %.

July 1st will be also the end of ETAPES experimentation.

Annouced in 2021, The « Health Innovation 2030 plan » provides 7.5 billion euros to promote France as a leading country in health innovation and contributing to be a leader in EU. and contributing to health sovereignty in Europe as well as a set of legislative and regulatory measures present in the various Social Security Financing Laws (LFSS).

The Health Innovation Agency (AIS), under the authority of the Prime Minister, contributes to continuing and accelerating access to innovations and manage the ecosystem of innovation locally. 

AIS’s mission is to be the dedicated interlocutor of of the health innovation actors and contributes to the development of new projects.

In this context, Paris Saclay Cancer Cluster is the first Biocluster set up with a excellence center involving private companies, care, research and breakthrough innovation, they aim to transform the French biomedical research landscape.

Following the BMJ EMB article publication*, The Transparency Committee updated its Doctrine for the medical assessment, in particular on the conditions of acceptability of studies comparing a single-arm trial to external data.

The double-blind randomized controlled studies remain the Gold standard and the reference for the evaluation of any drugs.

But in absence of a direct comparison, single-arm clinical trials with an external control arm or others indirect comparison may allow the assessment od the added value of these medicines only if these comparisons are of good methodological quality and can lead to an improvement of the ASMR level.

Data from real-life studies may also be considered.

On the other hand, we find in this new Doctrine a specific focus on pediatric drugs assessment and antibiotics in last line.

* Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health.

Since 2 years, G_NIUS, Guichet National de l'Innovation et des Usages en e-Santé developed  by the Ministry of Healthin collaboration with the Digital health Agency has been helping innovative companbies to better define their projects.

G_NIUS provides on their plateform various tools to help project leaders in the world of French digital health (Digital MDs in Europe, finacial aids, roadmaps by French regions, official documentation and reports etc.).

This call for projects concerning medical imaging is associated to a major changing sector with many innovations (Digital health, IA …) particularly in diagnosis to improve patient’s care pathway.

This project has an investment of €90 millions. The projects supported will then be rapidly developped in all hospitals. This call for projects is open until March 28, 2023.

With dugs innovation accelerating for rare diseases with restricted population reinforces the interest of real-life data (RWE) to complete clinical trials and reduce uncertainties for HTA agencies. However, to date, the impact of RWE data for obtaining reimbursement is still limited and hihgly variable among countries. The proportion of files integrating RWE has increased from 6% in 2011 to 39% in 2021.

The report published by IQVIA at the end of 2022 presents the impact of RWE in oncology drugs assessment. The work was presented at ISPOR Europe 2022 with 20 examples in oncology.

Based on this analysis, the report concludes with initiatives to be put in place to maximize the impact of RWE during evaluation across Europe. Among these initiatives, the report cited HAS guidelines published in 2021 for France, in Germany new rules for rare diseases and also NICE publication (RWE framework) in June 2022.

A report recently published by the French Health Insurance (CNAM) analyses the international scientific literature on the effectiveness of digital therapies in mental health and explores the methods of using these digital solutions. 

Following the COVID-19 crisis with a strong impact on mental health, health systems are increasingly interested in these tools to meet the challenges of care. Some countries have also begun to develop or even fund such tools. 

The CNAM has therefore studied the conditions under which these devices could effective and relevant to reinforce the more traditional methods of managing patients based on scientific literature review.

The 2 decrees allowing the entry into the common law of remote monitoring were published on December 30,2022:

• Décret no 2022-1767 du 30 décembre 2022 relatif à la prise en charge et au remboursement des activités de télésurveillance médicale https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000046849110
• Décret no 2022-1769 du 30 décembre 2022 relatif au contenu de la déclaration des activités de télésurveillance médicale aux ARS https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000046849231

2023 will be the year of the deployment of this new tool for patients and healthcare professionals.

Offices managed by the Digital Health Agency (ANS) will be open for certification from Q1-2023.

In this context, there will be a requirement for the interoperability and security reference system.

A HAS office will also be open in Q1-2023.

A decree is expected to set the tariff for Healthcare professionals and periodicity.

On December 6, OECD published its 2019 to 2021 report on the health of europeans and especially on the impact of COVID-19. Life expectancy in EU countries has decreased by 1.2 years on average, and by as much as -3.7 years in Bulgaria, -3 years in Slovakia and -2.7in Romania.

The COVID-19 crisis has also increased the health expenditure. The pandemic has disrupted the primary health care, cancer screening and treatment, continuity of care for patients with chronic disease and non emergency surgeries. 

Finally, the impact of the restrictions of recent years on the health of the youngest, particularly mental, is significant with, in September 2022, the proportion of young people showing symptoms of depression thus remained twice as high as before the COVID-19 crisis.

The HTA (HAS) has listed the registries/databases to be used by companies to meet HAS requests/expectations when assessing healthcare products.

An initial list of data sources used by HAS is already available since November. This list comes from a project launched in May 2022. 

The objectives of this research were to analyse the distribution of ASMR levels granted by HAS according to the expected innovation of the drugs on 2 criteria : early access program and the impact on public health and the distribution according the therapeutic areas.

The unmet or partially unmet medical need is an important factor in the HAS assessment, the HAS granted ASMR levels I to III in the large majority of drugs with an early access program (+72%) versus without early access program. This analysis also revealed an evolution in the therapeutic areas. Before 2020, the major innovations were in oncology, then from 2020, the share of orphan drugs has increased significantly.

This online conference aims to highlight the movement of user engagement, supported people and associations at HAS and elsewhere. It will also be an opportunity to discuss the prospects for development. It will be led by Christian SAOUT, member of the HAS College, chairman of the council for user engagement and of the commission in charge of social and medico-social matters at HAS. 

This report contains international R&D data and Global economic data. Key data for COVID-19 vaccines and treatments are presented, including development and manufacturing in historic quantities.

A summary of the number of molecules in development in 2021 by therapeutic areas shows that the fight against cancer, although great advancements have been made, remains one of the global health challenges followed by numerous developments in neurology, infectious , respiratory, digestive and autoimmune diseases. 

In order to guarantee access to medicinal products for patients, the ANSM may impose financial penalties on MA holders/operators who fail to comply with their obligations with regard to the anticipation and management of the risks of drug stock-outs. These financial sanctions may be accompanied by daily penalty payments.

The cases of non-compliance are subject to financial sanctions provided by law (LFSS 2020) and the amount of the sanctions applicable to such non-compliance has been increased.

The entry into force of the document entitled "Guidelines for the determination of financial penalties", its amended Annex 1 and its new Annex 3 is 1 October 2022. 

The 2022 work programme, which has already begun, will enable the implementation of numerous projects such as the experimentation of medical cannabis, the misuse of medicines, risk management, the implementation of European regulations (new European regulation on medical devices), the reform of early and compassionate access, stock shortages (decrees), the Covid-19 epidemic which is still present, and the current geopolitical tensions in Europe.

In summary, the main 2021 activities of the ANSM concerning access to innovative treatments:

• 277 scientific or regulatory support provided through the Innovation and Guidance Desk;
• 73 European scientific opinions awarded to France;
• 51,096 ATUn/AAC granted and 28,876 patients included;
• 27 new cohort ATUs granted and 7 favorable opinions on EAPs issued
• 1,098 patients included in medical cannabis trials;
• 855 clinical trials authorized for drugs and 80 for DM and DMDIV;
• 636 MAs and registrations issued by the ANSM (national procedure and decentralized European procedures and mutual recognition);
• 18 marketing authorization dossiers in the centralized procedure awarded to France;
• France rapporteur or co-rapporteur for 100 PIP (3rd position);
• France is the first Member State to release batches of vaccines on the French and European markets.

Oncology is today the largest and most dynamic market in the pharmaceutical industry with a large number of innovations to address current and future challenges.

The market for cancer treatments in 2021 was expected to grow by 12% to $185 billion. It could reach $307 billion (+66%) by 2026.

30 new active substances in oncology were launched worldwide in 2021, 104 in the last five years (21 per year on average) and 159 since 2012 (16 per year).

Clinical trials are also increasing rapidly, especially for rare cancers. 

However, it was found that diagnoses were still too late with advanced metastatic disease.

Within the framework of the Digital Health project, we can see that many projects have been launched, such as the Health Data Hub, My Health Space, the SI-DEP. (Système d'Informations de DEPistage), a secure platform where the results of Covid-19 tests are systematically recorded..... However, the challenge of the interoperability of the different software and applications still arises for health professionals.

In three years, significant progress has been made under difficult conditions, due to an unprecedented health crisis, making it possible to place digital technology at the heart of a modern health system.

3 digital platforms put in place:

• The Health Data Hub (HDH) (30 November 2019) 
• My Health Space (February 2022)
• The Bouquet de Services Pro (BSP): a trusted space for professionals around Mon Espace Santé. It is intended to bring together the historical services of the public and private players in the ecosystem, both national and regional, as well as new applications ready to offer innovative services to healthcare professionals

The BSP will offer professionals unique access via Pro Santé Connect to secure health messaging, to Health Insurance teleservices (Ameli Pro, INSi, e-prescription, etc.), as well as the possibility of exchanging data securely between their software and their patients' health space .

WAYFIND-R is a registry of approximately 15,000 patients diagnosed with a solid tumour and associated genomic data. This platform, in which the Institut Curie is heavily involved, is being published in the Journal of Clinical Oncology - Precision Oncology on 8 August 2022.

Registries including the natural history of cancers will make it possible to answer major questions that will accelerate the development and marketing authorisation of new treatments. The ambition is to offer increasingly personalised therapies.

The WAYFIND-R registry is expected to be set up initially in 12 countries and then expanded to other countries, in academic centres, hospitals and private clinics. 

An Approach to Solving the Complex Clinicogenomic Data Landscape in Precision Oncology: Learnings From the Design of WAYFIND-R, a Global Precision Oncology Registry. Christophe Le Tourneau, Camille Perret, Allan Hackshaw, Jean-Yves Blay, Christoph Nabholz, Jan Geissler, Thy Do, Martina von Meyenn et Rodrigo Dienstmann. JCO Precision Oncology. 

All digital remote monitoring devices are eligible, regardless of the pathology treated, provided they are CE marked.

A certificate of conformity must be issued by the ANS and then? 

An application must be made for a "generic line" (requesting an individual code from the MoH in order to be reimbursed on the generic line) or for a "brand name" (filing an application on the HAS PEGA platform, assessed by the HAS and the Ministry of Solidarity and Prevention (MSP)). 

Following the experiments that have been conducted as part of ETAPES (Expérimentations de Télémédecine pour l’Amélioration des Parcours en Santé), France will reimburse remote monitoring solutions under common law (Article 36 of Law No. 2021-1754 of December 23, 2021), either as a generic line or a brand name. 

A decree of application by the Council of State concerning the methods of evaluation, registration for reimbursement and reimbursement of remote medical monitoring activities is expected soon.

This decree will describe reimbursement conditions on obtaining a certificate, issued by the Agence du Numérique en Santé (ANS), ensuring compliance with an interoperability and security reference framework, the first version of which was published by decree on July 31, 2022.

In anticipation of the entry into force of the common law for remote monitoring, the desk is thus opened in advance to allow companies to anticipate their steps and start filing their evidence.

A webinar will be organized by the ANS on August 25 from 2 to 3 pm to answer any questions about the content or practical conditions of opening the desk.

The HAS College has just published a decision in June 23, 2022 concerning the significant impact on health insurance expenditure of the medico-economic evaluation of health products claiming an ASMR or an ASA of levels I, II or III.

The HAS modifies the eligibility criteria for a medico-economic evaluation taking into account:

• the forecast or actual sales of the product in the indication concerned by the application for reimbursement, and no longer "all indications combined", as previously, and in terms of sales excluding taxes, and no longer including all taxes. The forecast sales excluding taxes of the second year of marketing in case of first registration of the indication and the sales excluding taxes recorded during the 12 months preceding the application for renewal of the registration of the indication must be greater than or equal to 20 million euros annually excluding taxes;
• the fact that the product concerned is an advanced therapy medicinal product (ATMP), taking into account the impact that this type of medicinal product is likely to have on the organization of care, professional practices or the conditions of patient care;
• that the company claims an impact on the organization of care, professional practices or conditions of patient care.

The information requested at the time of filing is: 

• the claims concerning the impact on the organization of care, practices or the conditions of patient care;
• an estimate of the projected turnover per year over three years, in the indication concerned, in the case of first-time registration, or the actual turnover, in the indication concerned, in the case of registration renewal;
• an estimate of the projected population per year over three years in the indication concerned in the case of initial registration, or the population reached per year over three years in the indication concerned in the case of registration renewal.

This information transmitted to the HAS, in particular that relating to turnover and the population reached, must correspond to the information transmitted to the CEPS in the context of the economic note.

This is the same for medical devices. 

However, there are some exceptions for pediatric extensions, products that no longer have patent protection and an extension of indication that results in an increase in the population reached by the product of less than 5% over two years.

These new eligibility criteria should reduce the number of evaluations that were linked to sales > or equal to €20 million for all indications, but also oblige ATMPs, and therefore innovations, to submit a dossier even if the budget impact may be much lower than €20 million. 

As every year, the report of the Assurance Maladie is highly expected. 

Following the analysis of the 2021 data, the Assurance Maladie presents 30 proposals in its report to the Minister of Health and the Parliament in view of the preparation of the LFSS 2023 (Social Security financing law).

These proposals should improve the relevance and efficiency of care through better management.

The main axes to generate financial impacts in 2023 are:

• Strengthen the care pathway for heart failure with "specialized" care teams and the deployment of remote monitoring
• Develop digital technology for diabetic patients with connected innovations
• Improving the participation rate in cancer screening
• Improve vaccination rates
• Create an Observatory on access to innovative medicines on a European scale
• Improve the organization of and access to care
• Create a new status for "digital therapies" within "health products" (in addition to drugs and medical devices)
• Improve compliance, the relevance of prescribing and the delivery of health products by increasing the penetration of biosimilar medicines, strengthening the proper use of antibiotic therapy, etc..

The statement is based on an 18-month structured dialogue between the two organisations focused on identifying proposals that can improve access to orphan medicines across Europe.

This is the first time that EURORDIS-Rare Diseases Europe, as a patient organisation, has come together with the pharmaceutical industry’s European organisation EFPIA in such a structured dialogue. Discussions focused on how to improve patient access to orphan medicinal products, which remains inequitable across countries and routinely delayed.

EURORDIS believes that the proposals - increased equity of access for patients and solidarity between Member states, improved HTA and Pricing and Reimbursement (P&R) processes, and accelerated needs-led innovation - will bring about tangible change in access to medicines for people living with a rare disease.

EFPIA’s commitment to develop science for rare diseases”, is welcome given how much there is still to learn about rare diseases, especially in underserved areas. Funding in Public-Private Partnerships is a critical success factor in both research and healthcare, for clinical research before and after marketing authorisation.

EURORDIS and EFPIA invite other stakeholders to consider the proposals and to continue the debate, on the proposals discussed and beyond.

As every year, HAS publishes a general activity report and a specific report for each Committee

Regarding drugs, the report of the Transparency Committee is still awaited. 2021 has been a year of changes, HAS has taken into responsability the granting of pre and post-MA early access. 

The meetings of the Transparency Committee has had more numerous to ensure the assessments within a period of less than 3 months.

Patients organizations and experts were much more numerous in 2021 to support the assessment of the medicines. 

We can observe an increase in ASMR I to III at the highest level since 2010 with 31 drugs exceeding 2020 with 25 drugs. For ASMR IV it’s quite similar with 37 drugs.

The file processing times were slightly improved with 98,6 days. The transparency Committee granted 52 oral hearings stable for several years.

This report, made public at the end of May 2022, notes disappointing results in terms of participation in screenings. Despite the financial means implemented, the report points in particular to various organizational problems and delays in the evolution towards digital technology for breast cancer screening.

The national steering methods have often proved to be too complex (DGS, Inca, ARS, etc.) with multiple stakeholders. The transition from the departmental system to a regional system was done without support. Wanting to move, without investment in a single computer system, from separate management of screenings for different cancers to grouped management using 3 different programs, was more difficult and took much longer than expected.

The IGAS makes a series of recommendations to address the difficulties observed. For example, it recommends speeding up the digitization of negatives. It also recommends the transfer to the Health Insurance of the work of sending and following up invitations to screening in order to lighten the tasks of the CRCDCs which currently carry it out.

On 7 June 2022, EMA’s Medicines Shortages Steering Group (MSSG) adopted the list of critical medicines for the COVID-19 public health emergency . The medicines included in the list are authorised for COVID-19 and their supply and demand will be closely monitored to identify and manage potential or actual shortages.

The published list contains all the approved vaccines and therapeutics in the European Union (EU) to prevent or treat COVID-19. It will be updated regulalry.

For the first time, it’s now possible to know the number of patient identified for each rare disease, thanks to the National Rare Disease Data Base.

The national report from March 2022 provides alphabetical access to the number of patients affected.

These data are very important since up to 1 person in 2000 is affected by a rare disease and 80% of them are of genetic origin.

In total, nearly 8 000 genetic diseases have been identified and about 50 % of patients have no diagnosis and 85% do not yet have a dedicated treatment.

Launched in 2013 by the National Cancer Institute, AcSé (Secure access to innovative targeted therapies) clinical research program is evolving to enable greater number of patients to benefit innovative treatments with research program with multi-arm, multi-targets and multi-drugs.

This new program has identified the first 4 cohorts to respond to the cancers with poor prognonsis :

• Cohort targeting gene fusions for all cancers
• Cohort targeting abnormalities sensitive to PARP inhibitors in Off label solid tumors
• Cohort targeting ErBb2gene abnormalities for solid and hematological cancers
• Cohort targeting microsatellite instabilities not including colorectal and endometrial cancer.

The new European regulation 2017/746 relating to in vitro diagnostic medical devices comes into force on May 26, 2022.

L’ANSM held a training webinar on April 20, 2022 to present transitory steps, the schedule and the rules of classification concerning the entry into application of this European regulation.

PROMs (Patient Reported Outcome Measures) are questionnaires completed by patients to measure the outcome of care. These questionnaires are used both in routine clinical practice and in clinical research.

There are generic PROMs (the most used in France are EQ-5D, SF36, SF12) measuring the dimensions of the outcome of care used for all clinical situations and specific PROMs for targeted populations (ie cancer, asthma, depression …).

The survey set up by the HAS reveals that these PROMs are still little used in France in current clinical practice.

In order to develop their use, HAS has published a guideline to use PROMs in routine clinical practice.

Specific guidelines by pathology are also available : COPD, chronic coronary disease, chronic kidney disease.

EFPIA and IQVIA published in April the 2021 report on W.A.I.T. (Waiting to Access Innovative Therapies) survey shows the average time to patient access to treatments across EU and European Economic Area (EEA).

France is in 8th position regarding access to medicines for patients, less one place compared to last year.

For France, the time to availability is 497 days includes products under the ATU system (Early Access Program) for which the price negotiation process is usually longer.

Nevertheless, the average time to availability for medicine without ATU system is 240 days.

European Union average is 511 days.

was signed on April, 6th, revising article 15 to better adapt to the specificities of orphan drugs.

The main changes are : 

• stop the capping at €50,000/year/patient, but the possibility to negotiate conventionnally a « budget package » ;
• no possibility to use generics, Biosimilars or off label use drugs as a comparators for ASMR I to IV ;
• savings for ASMR V relating to the net price ;
• review of price conditions in the case of changes ;
• possibility for CEPS to hear experts during the negotiation phase.

31 decisions were rendered:

• 25 grants (80%) and 6 refusals (20%)
• 45% in onco-haematology and 26% in infectiology (COVID)
• 21 post-MA and 10 pre-MA decisions

Early access decision deadlines:

• Post-MA: 58.2 days
• Pre-MA: 43.5 days
• That is, on average around 50 days, including COVID products less than the 3 months provided for in the Law.

In summary:

• The situation should reverse with more pre-MA applications in 2022
• Priority given to early access requests during CT Commissions to meet the deadline of 3 months maximum
• + 30% of requests compared to the forecast based on previous ATU requests
• PUT-RD: the completeness of the data is set at 90%, but no sanctions provided for in the event of lower completeness.

*Data provided by the HAS during the meeting of March 17, 2022 at the AFCRO

A new framework agreement between the Economic Committee for Health Products (CEPS) and "associations representing patients and users of the approved health system and associations fighting against health inequalities", represented by France Assos Santé, has been signed on Wednesday 16 March.

The previous framework agreement, signed in December 2018, expired on November 6, 2021. This new version is very close to the previous agreement. To compensate for a lack of information, the CEPS General Secretariat communicates the list of CT or Cnedimts opinions of which it has received over the last four weeks and for which negotiations are about to begin.

Subject of experiment since 2014, remote monitoring will come into force in common law no later than July 1, 2022. A 1st step with the publication by HAS of the reference systems concerning the 5 following chronic pathologies:

• Diabetic patients;
• Patients with chronic renal failure;
• Patients with chronic respiratory failure;
• Patients with chronic heart failure;
• Patients with implantable cardiac prostheses for therapeutic purposes.

The regulation on joint health technology assessment published on December 22, 2021 in the Official Journal of the European Union (Regulation (EU) 2021/2282 of the European Parliament and of the Council of December 15, 2021) is to be gradually implemented on nine years, with different application deadlines depending on the product category:

• January 12, 2025 for the evaluation of new drugs in oncology, including those with orphan drug status and advanced therapies (advanced medicinal therapeutic products, ATMP)
• January 13, 2028 for the assessment of orphan drugs (excluding oncology or advanced therapies)
• January 13, 2030 for all categories of new drugs

Regarding medical devices, the regulation provides that some of them are subject to joint assessments, in particular those “which appear in the highest risk classes“.

Reform of early access to medicines in France: initial feedback, challenges and perspectives…

HAS published on November 10 the new composition of the Transparency Commission

In this report, the Montaigne Institute formulates five recommendations: invest in human; make health quality the objective of the system; focus on health data; facilitate access to e-health and encourage coordination between actors.

Since July 1, 2021, the HAS has been evaluating and authorizing drugs that are the subject of a request for reimbursement under "early access".

Each year, the Health Insurance presents to the Government and to Parliament its proposals relating to the evolution of expenses and income for the following year and to the measures necessary to achieve the balance provided for by the multiannual financial framework of expenditure of Health Insurance.

Agreement valid from March 05, 2021 until 2024.