Two years of PRIME

7 May 2018

Driving innovation to where it is most needed

In the two years since its launch, the PRIority Medicines scheme (PRIME) of the European Medicines Agency (EMA) has succeeded in driving innovation and improved the efficiency of the development process in therapeutic areas with the most pressing unmet medical needs. The goal is to support and optimise medicine development, so that patients whose diseases cannot be treated or who need better treatment options have access to new medicines that enable them to live healthier lives.

The PRIME: a two-year overview, published today, presents results from the first two years of PRIME. It explains how the criteria for eligibility to the scheme have been applied and what type of support applicants have received so far.

“Our analysis of the first two years shows we have established a platform that promotes the development of promising medicines for unmet medical needs and at the same time addresses the complexity of medicine development,” said Guido Rasi, EMA's Executive Director. “Through PRIME, we offer early and enhanced dialogue to enable the generation of better data and more robust evidence on a medicine's benefits and risks.”

Since the launch of PRIME in March 2016, EMA has received and assessed a total of 177 requests for eligibility to the scheme. Of these, 36 (21%) have been accepted. The Agency has received requests across a wide range of therapeutic areas; oncology and haematology medicines make up the largest share, but there have also been notable submissions for medicines that cover indications in infectious diseases, neurology and psychiatric disorders.

The overview of the 36 medicines accepted for PRIME shows the focus of the scheme on therapeutic areas where the availability of new medicines could be particularly beneficial: 83% concern rare diseases and 44% are intended to treat paediatric patients.

22 of the medicines accepted for PRIME have, in the meantime, received scientific advice from the Agency (a total of 37 requests altogether, many including input from health technology assessment (HTA) bodies and patients). Scientific advice is one of the Agency's key instruments to support the development of high-quality, effective and safe medicines that meet patients' needs. This procedure can guide applicants to invest resources in viable developments and to avoid exposure of patients to possibly ineffective medicines.

Enhanced interaction with EU regulators available through PRIME is particularly useful for developers of innovative or novel types of medicines that often present new scientific and regulatory challenges. 40% of the medicines admitted into PRIME are advanced therapy medicinal products (ATMPs), which have the potential to reshape the treatment of a wide range of conditions. A large proportion of these medicines are being developed by small and medium-sized enterprises (SMEs), who often lack experience in the regulatory approval process.

Two years on, the Agency has already received the first three marketing authorisation applications for medicines that were accepted for PRIME. They are all currently are under evaluation with the first opinion expected later in 2018.

Multidisciplinary support

A key feature of the PRIME scheme are 'kick-off' meetings - a unique type of meeting for medicines that are eligible for PRIME. These multidisciplinary meetings bring together the rapporteur for the medicine as well as the chairs and experts of relevant EMA committees to ensure that all aspects of a medicine's life cycle are discussed early, including risk-management issues. The first kick-off meetings were organised in July 2016 and, to date, 31 meetings have taken place. The aim of the meetings is to agree on next steps on how best to address any identified issues and/or to identify issues to be discussed normally in the context of scientific advice.

Based on past experience the Agency is also publishing today new European Medicines Agency guidance on interactions in the context of PRIME which covers the preparation and conduct of kick-off meetings.

EMA is also publishing an updated version of its Enhanced early dialogue to facilitate accelerated assessment of PRIority MEdicines (PRIME), and the PRIME eligibility request: applicant’s justification template.