Four years ago, the US Food and Drug Administration launched its “breakthrough designation” program, designed to speed the review and potential approval of new therapeutics deemed to offer the possibility of substantial improvement over existing options for patients with life-threatening or serious diseases. Since then, the FDA has approved more than 30 drugs in this category, many of which are cancer therapeutics. Research by the non-profit patient advocacy group Friends of Cancer Research has found that the FDA’s initiative has been successful in shortening the time to market for cancer drugs with breakthrough designation by more than two years.
On March 7 of this year, The European Medicines Agency (EMA) launched the PRIME (PRIority MEdicines) initiative, which is designed to achieve much the same goal in Europe of speeding the approval of innovative therapeutics. Drugs submitted for consideration must offer a major therapeutic advantage over existing treatments or benefit patients where no current treatment options exist for their disease. PRIME offers early, proactive and enhanced support to drug developers to optimize their clinical trials to generate robust data on drug benefits and risks, as well as to enable accelerated review and approval for drugs accepted for PRIME status. The EMA’s most recent report (July 27) cited a total of eight drugs that had been accepted for PRIME, out of a total of 37 applications. Four of these are in the area of oncology:
- KTE-C19 from Kite Pharmaceuticals, aimed at the treatment of diffuse large B cell lymphoma;
- CTL019 from Novartis, for the treatment of pediatric patients with relapsed or refractory B cell acute lymphoblastic leukemia;
- NY-ESO-1c6259T from Adaptimmune, for the treatment of inoperable or metastatic sarcoma in patients who are NY-ESO-1 positive; and
- DNX 2401 from DNATrix, for the treatment of recurrent glioblastoma where gross total resection is not possible or not advisable.
The other PRIME approvals included one drug in each of the areas of immunology/ rheumatology/ transplantation, neurology, and haemotology/haeomstaseology. PRIME approval was also granted to two applications in the area of vaccines, most recently Merck’s investigational vaccine for Ebola Zaire, V920 (rVSV∆G-ZEBOV-GP, live attenuated), which received FDA Breakthrough Designation at the same time.
The advent of PRIME is an important event for companies and a clear step toward further harmonization of regulatory policies aimed at speeding access to important drugs in both the United States and Europe. For products based on new technologies or those addressing indications where there has been little prior success, the opportunity to discuss the path forward in ongoing dialog with both the FDA and EMA is a major advantage. We believe it may ultimately help streamline the development process for many companies engaged in multi-regional development programs, as regulatory requirements and the ways in which regulators evaluate drugs for approval do not always translate between the two agencies.