EMA’s PRIority Medicines (PRIME) scheme has had a positive impact on the authorisation of new medicines that address patients’ unmet medical needs. The time to marketing authorisation was reduced for medicines that benefitted from PRIME support, giving patients earlier access to transformative treatments that can make a real difference to their health.

EMA launched the PRIME scheme in March 2016 to support the development of medicines that are expected to benefit patients with no current treatment options for their disease or offer a major therapeutic advantage over existing treatments. The scheme provides early and enhanced scientific and regulatory support to promising medicines.

A PRIME: Analysis of the first 5 years' experience, published today, presents results from the first five years of PRIME.

From March 2016 to June 2021, a total of 18 medicines that had PRIME support were approved in the European Union (EU). Among these, 10 received a conditional marketing authorisation (CMA) enabling them to access the market earlier and reach patients more quickly; seven are advanced therapy medicinal products (ATMPs), which have the potential to reshape the treatment of a wide range of conditions; and 16 concern rare diseases.

PRIME medicines represent significant progress in their therapeutic areas. They include innovative technologies such as CAR T-cells therapies, one-time curative gene therapies, treatments for rare cancers and a vaccine to protect against the Ebola virus.

Enhanced interaction with EU regulators through PRIME is particularly useful for developers of more complex products and for applications that rely on smaller datasets. This includes for example ATMPs and orphan diseases, which often present new scientific and regulatory challenges.

The analysis also showed that the support to PRIME products throughout the development resulted in a reduction of the clock-stop, i.e. the time required by the applicant to answer questions from EMA during the evaluation. This effect was more pronounced for small and medium-sized enterprises. Overall, this led to faster reviews and faster patient access to PRIME products.

At the time of publication of the report, 98 medicines have been accepted into the scheme. This also includes the first academia-led development, an ATMP intended to treat relapsed or refractory acute lymphoblastic leukaemia in adults over 25 years old, which was granted PRIME eligibility in December 2021. All academic developers are encouraged to interact with regulatory authorities to obtain early support for the development and clinical translation of their products.

For an overview of the five-year analysis, you can also check the PRIME: 5 years' experience.

Foreseen changes to optimise support and additional guidance for sponsors

The report also makes a number of recommendations for improvements to the scheme in three areas:

  • Timing for entering the PRIME scheme
  • Flexibility of scientific advice procedures for PRIME medicines
  • Leveraging the knowledge built during development for more robust marketing authorisation applications that can be assessed in an accelerated manner

In addition, proactive identification of promising developments and engagement on development and evaluation of PRIME products with other European decision makers and international partners should be strengthened to address patients’ unmet medical needs.

A toolbox with guidance for developers of medicines supported by EMA’s PRIME scheme on quality data is currently being finalised, following a public consultation that took place last year. The guidance is intended to help developers to generate robust quality data packages for EU marketing authorisation applications, which will support the rapid assessment of promising medicines and enable patients to benefit from these therapies as early as possible.

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