Improvements to EMA’s PRIME Designation Scheme

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The Priority Medicines (PRIME) scheme focuses on the development of novel medicines that address an unmet medical need, such as those that offer a major therapeutic advantage over existing treatments, or which benefit patients with no current treatment options for their disease (1). Launched in 2016, the European Medicines Agency (EMA) developed PRIME in line with the European Commission’s (EC) priorities and the common strategy to 2020 for the European medicines regulatory network (1).

PRIME is an entirely voluntary scheme that aims to optimize development plans and speed up evaluations through early and enhanced interaction between the regulator and developers of promising medicines with a view to achieving expedited marketing authorization approval (MAA) in the European Union (EU). The scheme gives medicine manufacturers an opportunity to open communication with EMA’s Committee for Medicinal Products for Human Use (CHMP), or the Committee on Advanced Therapies (CAT) early in the development process (2). Proof of a potential drug’s efficacy must be provided from preliminary clinical evidence which clearly demonstrates that the medicine has the potential to provide a clinically meaningful improvement in effectiveness or that it can improve patient mortality/morbidity rates.

From March 2016 to June 2021, a total of 18 medicines that had received PRIME support were approved in the EU. Among these, 10 received a conditional marketing authorization (CMA), facilitating earlier access to the market; seven are Advanced Therapy Medicinal Products (ATMPs), which have the potential to reshape the treatment of a wide range of conditions, and 16 are aimed at rare diseases (3). In December 2021, the first academia-led development of an ATMP, intended to treat relapsed or refractory acute lymphoblastic leukaemia in adults over 25 years old, was granted PRIME eligibility (3). EMA strongly encourages all academic developers to interact with regulatory authorities to obtain early support for the development and clinical translation of their products.

As of May 2022, the overall approval rate for PRIME sat at 25% of applications, 40% of which were granted to small and medium-sized enterprises (SMEs), 61% to others, and 4% to academia; while in terms of therapeutic area, oncology gained the highest number of approvals at 29% (4). According to the Regulatory Affairs Professionals Society, “PRIME medicines represent significant progress in their therapeutic areas as 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” (5).

Improvements to the PRIME pathway

In 2022, EMA published a report analysing the experiences of the first five years of the scheme, along with lessons learned (6). Accordingly, the report highlighted some opportunities for further strengthening the scheme, which aims to “facilitate and accelerate the generation of robust and relevant evidence for the evaluation of a MAA, which will give patients earlier access to transformative treatments that can make a real difference” (6). The measures include:

1. Regulatory roadmap and development tracker. A roadmap for each PRIME-designated product is being established alongside a product development tracker to optimize the early scientific and regulatory support provided to sponsors with promising medicines in the scheme. Starting as a pilot from March 2023, the roadmap and tracker will replace the PRIME annual update for any products that have not yet been discussed in a kick-off meeting. Under the new guidelines, applicants are required by EMA to maintain and update the regulatory roadmap and development tracker which includes information on planned regulatory submissions and interactions with regulators. The roadmap includes plans for scientific advice/protocol assistance requests and other regulatory interactions, with applicants urged to consider feedback received from regulators, while the development tracker aims to facilitate the efficient tracking of critical development aspects which may arise during the kick-off meeting or subsequent product development (7). The guidance states that “both tools will facilitate the continuous dialogue between regulators and developers as the progress of the development is continuously monitored and as critical aspects for further discussion can be identified throughout the development process” (8).

2. Expedited scientific advice. Expedited scientific advice can now be provided for PRIME designated products in instances where there are issues with a specific development programme that has already received comprehensive initial advice. To qualify for expedited scientific advice, the new guidance states that all the following criteria must be met:

• The initial scientific advice procedure has already been sought on the overall development (in the PRIME indication), that is the request is for follow-up advice.
• The advice concerns issues with a specific, well-defined scope (not limited to a single quality/non-clinical/clinical discipline).
• The advice is justifiably required more urgently than the standard scientific advice timelines allow (8).

The expedited scientific advice feature is being tested in a 12-month pilot that will run until March 2024. According to EMA, the scientific advice pilot programme is “meant to help significantly expedite the ability of sponsors to get answers to key queries from the agency in a faster timeframe” (8).

3. Submission readiness meetings. EMA offers a submission readiness meeting with the developer approximately 9–12 months ahead of the applicant submitting their MAA for purposes of discussing the development status and dossier maturity, application type, requirements for post-marketing evidence generation, and potential regulatory challenges (9). Prospective applicants would also be expected to present mature plans for post-marketing evidence generation, as applicable. According to the updated guidance, “applicants are asked to contact their PRIME scientific coordinator about 15 months before their expected application submission date to set up a submission readiness meeting with the PRIME Rapporteur and the assessment team, relevant national experts, as well as the EMA product team” (8).

Anticipated outcomes

The experience gained during the COVID-19 pandemic has given EMA greater insight into the types of tools and features that would better assist the acceleration, development, and approval of life-saving medicines. As a result, the new features aimed at bolstering the PRIME scheme are designed to address the perceived shortfalls in tools and support mechanisms needed to enhance and expedite the innovation and development process.

Furthermore, the changes also bring the PRIME scheme into closer alignment with the United Kingdom’s (UK) Innovation Licensing and Access Pathway (ILAP), which was initiated in 2021, and is sponsored by the Medicines and Healthcare Product’s Regulatory Agency (MHRA). The UK’s ILAP initiative more closely mirrors the United States Food and Drug Administration’s Fast-Track process, which enables rolling reviews and allows applicants to submit completed sections for review, rather than waiting until the entire application is complete (10).

With the improvements to the PRIME scheme brought about in 2023, it is hoped that medicine developers throughout the EU can expect a more transparent and easier drug development process, thereby benefiting patients with life-threatening illnesses to have earlier access to medicines. It is also hoped that improvements to the PRIME scheme will help drive greater harmonization across major pharmaceutical markets with regard to scientific advice and regulatory support for innovative products (10).

References

1. EMA. PRIME: Priority Medicines Fact Sheet. EMA.europa.eu, 2023.
2. ProPharma. 5 Benefits of Receiving EU PRIME Designation for Medicine Developers. Article, 20 July 2022.
3. PharmaLetter. EMA Says its PRIME Scheme Enables Earlier Availability of Life-changing Meds. News Release, 4 March 2022.
4. EMA. Recommendations on Eligibility to PRIME Scheme. EMA.europa.eu, 23 May 2022.
5. RAPS. EMA Lauds PRIME Priority Meds Scheme and Outlines Potential Revisions. Regulatory Focus, 8 March 2022.
6. EMA. PRIME: Analysis of the First Five Years’ Experience. EMA.europa.eu, 2022.
7. RAPS. EMA Updates PRIME Pathway with Roadmaps, Scientific Advice Pilot. Regulatory Focus, 4 April 2023.
8. EMA. European Medicines Agency Guidance for Applicants Seeking Access to PRIME Scheme. Revised Guidance. EMA.europa.eu, 30 March 2023.
9. European Confederation of Pharmaceutical Entrepreneurs. New Features Further Strengthen EMS’s Priority Medicines Scheme (PRIME). News Release, 5 April 2023.
10. PharmaLex. Changes Strengthen PRIME Scheme and Bring EMA in Closer Alignment with Innovation Programs in the UK and US. Blog Post. 2 May 2023.

About the author

Bianca Piachaud-Moustakis is lead writer at Pharmavision, Pharmavision.co.uk.

Article details

Pharmaceutical Technology Europe
Vol. 35, No. 7
July 2023
Pages: 7–8

Citation

When referring to this article, please cite it as Piachaud-Moustakis, B. Improvements to EMA’s PRIME Designation Scheme. Pharmaceutical Technology Europe, 2023, 35 (7), 7–8.