According to the European Medicines Agency (EMA) its PRIority Medicines (PRIME) scheme “has had a positive impact on the authorisation of new medicines that address patients’ unmet medical needs”, reducing the time to marketing authorisation. The EMA launched the PRIME scheme in March 2016 to provide early and enhanced scientific and regulatory support to medicines that are expected to benefit patients with no current treatment options, or which offer a major therapeutic advantage over existing treatments. PRIME medicines include CAR T-cell therapies, one-time potentially curative gene therapies, rare cancer treatments and a vaccine for the Ebola virus.
Key findings from an EMA report which presents results from the first five years of PRIME are as follows:
- In the period 7 March 2016 to 30 June 2021, a total of 384 requests for PRIME eligibility were received. Of these, 372 were validated and 95 granted, corresponding to an overall acceptance rate of 25%. The success rate across the therapeutic areas ranges from 20 to 30%, with the exception of vaccines (55% success rate over 11 submissions). Haematology is also an outlier with 57% of the 30 applications successful.
- Advanced therapy medicinal products (ATMPs) accounted for 46% of successful PRIME applications, biologics for 26%, chemical drugs for 23% and immunologics for 4%. Fifty-six per cent of PRIME products were orphan drugs.
- Medicines that benefited from PRIME support and were granted marketing authorisation had a consistent reduction of the clock-stop duration (the time required by the applicant to answer questions from EMA during the evaluation) compared to equivalent non-PRIME submissions. Indeed, a reduction of clock-stop and overall assessment time was observed, with an average (across all product/company types) of a 49-day (35%) reduction of the clock-stop duration for PRIME products compared to non-PRIME products.
- Despite their complexity, the 7 ATMPs that benefited from PRIME support and were granted marketing authorisation had on average shorter active assessment time and clock-stop duration than the average assessment time for all types of new active substances in 2020.
- PRIME products were also more likely to be granted accelerated assessment (17 out of 19 requests for accelerated assessment for PRIME products were granted) and maintain it during evaluation (7 out of 16 PRIME products maintained accelerated assessment until opinion), compared with equivalent non-PRIME submissions.
- There is a correlation between compliance with scientific advice, maintenance of accelerated assessment and a positive marketing authorisation procedure outcome, confirming the findings of previous studies.
- Regarding the type of marketing authorisation granted in the study period, PRIME products had a higher proportion of conditional marketing authorisations compared to non-PRIME; in most cases this was discussed during the Scientific Advice.
- Based on feedback from an industry-led survey, the window of opportunity to apply for PRIME eligibility is narrow, estimated to be between 6 and 9 months in the drug development lifecycle. This is due to the need to have sufficient supportive clinical data for the PRIME applications available and that the pivotal clinical plans are at sufficiently early stages to benefit from additional regulatory support.
The EMA concludes, “on the basis of the available experience during the first five years of operation of the PRIME scheme, all parameters suggest a trend for a positive impact of PRIME on evaluation times, which is more pronounced for SMEs [small and medium enterprises] and ATMPs. The majority of PRIME products are also orphan-designated. It would therefore appear that the scheme is well placed to have a positive impact on products that hold the potential to address an unmet medical need. This review also re-confirmed that provision of advice, and compliance of the company with that advice, enhances the MAA success rate.”
The EMA report can be found here.
www.ema.europa.eu, “PRIME enables earlier availability of life-changing medicine”, 3rd March 2022