How to market an orphan drug in France?
Because of the small number of patients, it is hard to recoup the costs of developing and marketing an orphan drug for a rare disease through sales, which explains why manufacturers are reluctant to develop them.
For patients to benefit from these treatments, the European Union has introduced legislation to create incentives for pharmaceutical companies to develop these medicinal products : Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products.
Definition
A medicinal product is said to be “orphan” when it is used to treat rare diseases, considered as such when they afflict under 1 person in 2000 in Europe.
Three types of cases occur:
- Product for the treatment of rare diseases
- Product removed from the market for economic or therapeutic reasons
- Non developed product (non-patentable research process or non-viable market)
Specificities and challenges of orphan drugs
European regulation has changed in recent years to push industrial companies to develop and sell medicinal products that tackle rare or ultra-rare diseases. These measures include credit allocation and clinical research support.
To be registered, all orphan drugs must go through a centralized procedure to obtain a European MA issued by the EMA. At the end of this process, a 10-year period of exclusivity is granted in Europe.
It should be noted that obtaining a centralized MA does not mean that this medicinal product will be marketed because it is up to MA holders to initiate the procedures at the national level for each Member State where they intend to market their product.
Orphan drugs and the French market 🇫🇷
For France, this means initiating administrative procedures for reimbursement and pricing of the medicinal product, which in the case of orphan drugs cannot be agreed under classic and usual marketing conditions. In the case of economically unviable arrangements, the MA holder may decide not to market the drug despite the validity of the MA in the territory.
European Situation
In Europe, approximately 30 million people suffer from a rare disease. In France, this represents a major public health issue with approximately 7,000 identified rare diseases affecting over 3 million people.
French Specificities
France is the first country in Europe to have developed and implemented a national plan to identify reference centres to collect and centralise data in order to make them accessible to patients and health professionals.
Cevidra’s Expertise
In its support during the pricing & reimbursement (P&R) process, Cevidra relies on public data sources and on patient associations, whose contribution is key when assessing the contribution of a new medicinal product compared to existing therapies.
Cevidra’s expertise
Cevidra is very active in conferences dedicated to market access and P&R challenges, which it uses to keep its knowledge up to date with evolving best practices and the state of the art in the field.
Thanks to our experience in orphan drugs, we offer customized support at all key stages of the marketing process:
Support and strategic advice during the MA registration process
- Support for local regulatory requirements (packaging and multilingual exemption, blue box)
- Management of pricing & reimbursement processes
- Assessment of eligibility for early access programs and implementation support, if applicable
- Communication with health authorities for the coordination of the product launch (marketing declaration, pharmaceutical databases update, submission of shortage management plans…)
Referral of the product to hospital purchasing platforms and wholesaler-distributors
- Management of public tenders for medicinal products
- Exploitation of medicinal products on the market, including medical information and local pharmacovigilance, promotion and sales activities
Secondary packaging to comply with local requirements (blue box, traceability labels)