Breakthrough Therapies in Europe: EMA Launches PRIME
[Posted on: Thursday, 10 March, 2016]

The Breakthrough Therapy designation is perhaps one the strongest incentives available to developers of innovative therapies in the US. Since implementation of the Breakthrough designation in 2012, FDA has designated about 110 products and approved 30 new products in this category. Now European Medicines Agency (EMA) has created its own version similar to the US regulatory pathway, called the Priority Medicines or “PRIME”. Breakthrough designation provides an accelerated pathway to market for products and is loved by both the patients and industry. Although some questions have been raised about the post-market commitments from FDA and industry for products approved under this extremely accelerated regulatory process, the utility of this pathway is undeniable. PRIME has several similarities with the US process. Like the US process, the PRIME designation requires two conditions. First, that the drug or biologic addresses an unmet medical need. An unmet medical need could be diseases with no treatment options or that the new treatment option provides a major therapeutic advantage over existing treatments. The second condition is availability of preliminary clinical evidence demonstrating that the drug addresses the said unmet medical need. Like the US program, PRIME designation provides accelerated review and additional ongoing advice by the regulators to speed the time to market. However, the PRIME program has additional incentives to small companies and academic researchers. Such applicants can be granted PRIME designation based on “compelling” non-clinical data and tolerability data from initial clinical trials. EMA would not charge its fee for the scientific advice meetings from PRIME holders who are small companies or academic researchers. The applicants have to fill an application form for the PRIME program and a justification form. These documents will be reviewed by a specially formed Scientific Advice Working Party (SAWP) which will meet once a month and will provide response within about 40 days from application receipt. PRIME is not available to manufacturers seeking new indications of previously approved products. EMA will also appoint a dedicated contact point who will assure timely responses and support. A major difference from the FDA process is the high transparency of the PRIME program. EMA will publish a monthly report listing all applications for the PRIME designation including the type of product (chemical, biological or advanced therapy), the intended indication, the type of data supporting the eligibility request,  and the type of applicant (small business, applicants from the academic sector or others. For products that successfully receive the PRIME designation, the name of the active substance/INN will also be made public, whilst it will not be published in case of negative outcome. This may scare companies that want to keep this information confidential. The EMA program seems to have learnt from the US program and improved on it. It is more structured than the FDA program. EMA requires a “kick-off” meeting for the PRIME designated products to chalk a development plan and milestones. EMA also requires annual progress reports and meetings at major development milestones. The PRIME designation can be withdrawn if data generated in the program shows that the product no longer meets the two conditions for PRIME. The PRIME program provides an additional incentive to companies that in the past used the breakthrough designation from the FDA to seek approval in both US and Europe in parallel. PRIME is ready for prime-time and soon we will find out how it compares to the US Breakthrough program in real-life.