BlueReg Group Comply with french regulations and take advantage of early access pathways for your medicinal product
Comply with french regulations and take advantage of early access pathways for your medicinal product
The European Medicines Agency (EMA) provides recommendations through the Committee for Medicinal Products for Human Use (CHMP), but these do not create a legal framework. Expanded access programs are coordinated and implemented by each Member States, which set their own rules and procedures.
Companies must follow the national framework within each European country and as such, the wide range of regulations between countries makes navigating the European landscape complex.Launching several country-specific compassionate use programs in Europe simultaneously requires a lot of preparation to shape a comprehensive strategy that can fulfill both the company’s strategic objectives and the national regulatory requirements.
France Market Specific Regulations
In addition to the European regulation, the French Market is closely monitored by the ANSM (Agence nationale de sécurité du médicament et des produits de santé – National Agency for Medicines and Health Products Safety) that oversees pharmaceutical surveillance.
This surveillance is carried out on the basis of French national legislation that defines which information must be provided to government Agencies, who are in charge of authorizing the marketing of drugs.
From 2021, the access to new medicines scheme in France has been simplified into two separate but linking processes; Early Access Authorisation (“accès précoce”) and Compassionate Access Authorisation (“accès compassionnel”).
Expanded Access is a treatment option that allows the use of an unauthorized medicine (investigational drugs). Under strict conditions, products in development (with ongoing clinical trials) can be made available to groups of eligible patients who have a disease with no satisfactory authorized therapies and who cannot enter clinical trials.
The Early Access Authorization (EAA) route is for drugs that are in the process of being developed and are to be put on the market for a given indication. The EAA is reserved for unapproved drugs for which the holder has submitted or undertakes to apply for a marketing authorization or an application for reimbursement. However, unlike the previous ATU pathway, early access will only be available for medicines that are presumed to be innovative.
The Compassionate Access Authorisation (CAA) route is for off-label drugs that are not being developed for a given indication in France. It replaces the previous nominative ATU pathway, and temporary recommendations for use. The CAA route is intended for drugs that are not necessarily innovative, that are not necessarily intended to obtain marketing authorisation in the indication concerned, but that respond satisfactorily to a therapeutic need.
The HAS stands for the Haute Autorité de Santé / French National Authority for, Health.
The Transparency Committee (TC) of the French National Authority for Health (HAS) is responsible for the scientific and medical assessment of medicinal products when pharmaceutical companies submit applications to the French Minister of Health for registration on the lists of medicinal products reimbursed by National Health Insurance.
The remit of the HAS was extended with the publication of the French Social Security Financing Act for 2021 which completely reformed the authorizing procedure for early and derogatory access to medicinal products.
The French National Agency for the Safety of Medicines and Health Products (ANSM) known as “Agence nationale de sécurité du médicament et des produits de santé” is one of the competent authorities in France.
The french agency ensures the safety of medicines and health products available on the market, while promoting rational prescription by elaborating clinical guidelines, carrying out studies on therapeutic effects, assessing risks related to a medicine use, conducting pharmaco-vigilance studies, etc.
The early access to medicinal products for innovative treatments is now overseen by HAS, French HTA body, in association with the French national agency for health product safety (ANSM). The decision of the HAS will result in both the granting of early access and derogatory reimbursement by the health insurance system.
For pre-MA early access, ANSM is still involved for the assessment of product safety and efficacy, whereas HAS is the only decision-maker for post-MA early access.
The ANSM oversees both compassionate access programs. The AAC is requested by the prescribing healthcare professional (“HCP”), and the CPC is requested by the Ministry of Health or set up on the ANSM’s own initiative.
Questions and dialogues
There are a wide variety of reasons and situations outside of submission and registration, where you may wish to engage with Health Authorities. Ensuring an early dialogue with Regulatory authorities and building a good, early relationship will improve chances of a smooth and successful access to the Market.
Meetings with the health authorities can be arranged by our team, we have a great deal of experience in guiding you through its process, providing advice on the process and also writing briefing packages, presentations and the questions themselves.
Applications - The exploitant status
Under the French legal framework, an operator that wants to market a medicinal product from and in France should hold an Exploitant status or partner with an Exploitant. The Exploitant operator is one of the pharmaceutical establishments authorized and regularly inspected by ANSM (French competent authority).
Exploitation is a pharmaceutical status that applies to commercial operations for medicinal products in France. In charge of placing the medicinal product on the market in France, the operator must ensure that all pharmaceutical activities associated with the products for which it is responsible are carried out in accordance with the applicable provisions.
Avoid all the pitfalls
Partnering with a team of consultants is highly recommended and will prevent you from falling on the following pitfalls :
1. Avoid typical planning errors of first timers
The planning element is absolutely essential and should be factored into the timing, allowing for time to prepare documents and contracts, assemble educational materials and establish treatment criteria. Actual timing and advanced preparation are important to ensure the early access program runs smoothly and that the patients can benefit as early as possible.
2. Not having enough French resources available when needed
This necessarily has an impact on the planning, but also on the submission time once companies have started the project (we usually foresee 3 months of preparation in our estimates, and we generally assign 6-7 consultants working on the early access dossier).
Indeed, the approval process of the early access program is specific to France, with a detailed and standardized dossier to be prepared and submitted. The submission and assessment process requires regular interactions with the appropriate authorities (HAS & ANSM).
3. Lack of a solid understanding of differences of regulations in EU
Differences in the regulatory landscape of the EAPs in the EU are more challenging to implement than in the US, therefore understanding the process needs to commence early on in the project.
4. Being unaware of changing regulations
The pharmaceutical industry has been thriving in France. With this significant growth comes new regulatory changes that will impact many biopharma organizations like simplification and modification of the procedure for temporary authorization for use (ATU) in 2021.
For those not prepared, these steps can be extremely difficult without outside support from third party providers like BlueReg.
Benefits of outsourcing
Outsourcing Exploitant activities has a few benefits and makes it a preferred option for most pharmaceutical companies. On top of having access to a very experienced team, knowledgeable about every aspect and intricacies of the Early Access Program Application process, you have :
PharmaBlue (a BlueReg company) is a partner “Exploitant” of pharmaceutical companies for the marketing in France.
PharmaBlue holds an “Etablissement Pharmaceutique Exploitant” license from ANSM and as such can take over the organization and supervision of the distribution in France of pharmaceutical products, including :
Avoid Low-cost partners
Partnering with an exploitant established and authorized in France is a requirement for early access program applications.
Many of our clients have faced an obstacle as they had selected their previous Exploitant partner based on pricing considerations. We strongly recommend having in mind that Exploitant responsibilities and activities are extremely demanding, especially if you are looking to distribute innovative products.
Therefore, you need to choose an Exploitant partner that truly understands your technology and most importantly has a flexible approach to be able to support you along the way.
A dedicated team of over 70 highly qualified consultants and project managers providing services in France.
The team has:
Our team works with many clients, from biotech start-ups to large pharma companies looking for local expertise or specific services for France. Our team has a highly successful track record of delivering all projects, from the simple to the complex.
Our regulatory affairs consulting services in France are designed to support clients during all the product development, registration, and commercialization phases (launch, communications, lifecycle management).
Please note that BlueReg is also able to provide regulatory solutions in all European countries. We support many pharmaceutical companies from various locations.
BlueReg services gives you instant access to :
whenever you feel ready and we will be happy to help you be on the right track for success.