Making Innovative Gene and Cell Therapies More Accessible: New Rules in France for 2023

Advanced Therapy Medicinal Products (ATMPs), such as gene and cell therapies, are revolutionary but extremely expensive, often costing millions of euros per dose. Because they are often one-time treatments for rare diseases, traditional drug-pricing models do not work well for them. This paper examines how the French Social Security Financing Act (LFSS) for 2023 introduced pioneering reforms to ensure patients can access these treatments without bankrupting the healthcare system. The most significant change is a “see before you pay” funding model, where the government pays in instalments only if the treatment proves effective in real life. These steps aim to balance fair rewards for innovation with sustainable healthcare costs.

The paper focuses primarily on France, while discussing its developments as a potential model for the broader European Union. The scientific discipline is law and regulation, specifically health and pharmaceutical law. The object covered is the regulatory and economic framework for ATMPs (gene therapies, cell therapies, and tissue-engineered products) and how France is managing their high costs and reimbursement.

• The Challenge of High Costs: ATMPs differ from traditional drugs because they are highly technical to manufacture and often target small numbers of patients with rare diseases, leading to price tags that can reach millions of euros.
• The 2023 Reform (LFSS 2023): The French Social Security Financing Act for 2023 aims to control "face prices" and make the pricing of these expensive therapies more transparent.
• Performance-Based Payments: A major innovation is the staged payment system. The state pays an initial sum, but subsequent payments are only made if the drug meets specific performance criteria in the patient. If the treatment fails or the patient dies, payments stop.
• Encouraging Full Coverage: To prevent companies from only seeking reimbursement for small, high-profit groups of patients, new rules mandate compulsory discounts if a manufacturer does not apply for reimbursement for the full scope of their marketing authorisation.
• Fair Evaluation: The paper details how French bodies like CEPS and HAS evaluate the "medical service rendered" (SMR) and its "improvement" (ASMR) to set fair prices based on therapeutic value rather than just production costs.
• European Cooperation: France’s reforms are viewed in the context of the new EU Regulation on Health Technology Assessment (HTA), which seeks to harmonise how the value of new medicines is assessed across Europe starting in 2025.

1 Introduction

2 Setting the Price of an ATMP in France and Its Challenges

     2.1 The Economic Committee for Health Products (CEPS) and Real-World Studies

     2.2 Transparency Commission, ASMR and SMR

     2.3 ITR, Economic Evaluation, Cost-Effectiveness Analysis and QALY

     2.4 European Price Guarantee

3 Towards the Solution

     3.1 Performance-Based Agreements and EUnetHTA

     3.2 The Social Security Financing Act (LFSS) for 2023

Summary

Full reference of the paper: 

Bartos, B. (2024). What are the Changes in 2023 for Access to Advanced Therapy Medicinal Products (ATMPs) in France?. European Journal of Health Law, 31(2), 187-208. https://doi.org/10.1163/15718093-bja10121

Open Access link to the paper:

What are the Changes in 2023 for Access to Advanced Therapy Medicinal Products (ATMPs) in France? in: European Journal of Health Law Volume 31 Issue 2 (2024)