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P34: Facilitated Regulatory Pathways (FRPs): Their Value and How it Could be Maximized to Ensure Timely Availability of Medicines

Poster Presenter

Magda Bujar

Senior Manager, Regulatory Programme and Strategic Partnerships
Centre for Innovation in Regulatory Science (CIRS)
United Kingdom

Objectives

Evaluate how EMA PRIME, FDA Breakthrough, Fast Track, and PMDA Sakigake accelerate the development and approval of medicines; Characterize the perceived value of the four FRPs based on industry experiences and identify how the FRPs may be evolved to further ensure timely availability of medicines.

Method

A survey was undertaken in 2019 across companies to evaluate the qualitative value of the FRPs - strengths, weaknesses, opportunities, and threats. Data on new active substances, including FRP use, was collected from EMA, FDA and PMDA websites to calculate development and approval time 2018-2020.

Results

Qualitative assessment of the value of FRPs: Survey results were returned by 11 companies. Based on their perceived value and impact, the evaluated FRPs seem to be generally recognized as helpful tools for ensuring timely development and review of important medicines. Overall, respondents felt that FRPs, particularly FDA Breakthrough, offer important benefits, most notably the opportunity for early information exchange, multi-stakeholder engagement and guidance available through these FRPs. These benefits are counterbalanced by the high level of time and workforce commitment required of the sponsor as well as a shift to the post-approval period for evidence collection, which can be labour-intensive and costly. The survey results highlighted common recommendations across all four FRPs - relating to resource optimization, education, alignment, and communication to improve effective use - as well as agency-specific recommendations, some of which are already being addressed by the regulators. Impact of FRPs on timelines: In 2018-2020, EMA approved 10 new active substances (NASs) through PRIME (10% of total EMA approvals); FDA approved 41 NASs which received Breakthrough (25%) and 37 which received Fast Track (23%) and PMDA approved 6 NASs which received Sakigake (6%). In general, a medicine which received one of the four FRPs also received another designation, such as a priority review or an orphan designation, and were generally anti-cancer medicines. The median approval time for medicines which received PRIME in 2018-2020 was 294 days compared to 433 for non-PRIME; for FDA it was 190 for Breakthrough and 241 for Fast Track compared to 335 for other NASs; and 172 for PMDA Sakigake compared to 320 days for non-Sakigake NASs. The median development time (from IND to regulatory submission) was also generally faster for medicines with one of the FRPs compared to non-FRP NASs. Analysis of common products across the three agencies was also undertaken.

Conclusion

Understanding both the perceived value and the impact associated with FRPs remains a topic of interest to both companies and authorities. This analysis confirms the value of PRIME, Fast Track, Breakthrough and Sakigake in reducing the time it takes to make new important medicines available. However, although the number of medicines which received Breakthrough or Fast Track from FDA is high, this is not the case for EMA Prime and PMDA Sakigake. This is in partially due to fact that EMA and PMDA only recently introduced their FRPs, and it will be of interest to see if their use will increase in the coming years with further experience. Another challenge in ensuring not only the availability but also expedited patient access to important new medicines and this is still difficult due to the fact that the outcomes of FRPs are not always widely embraced by HTAs and payers because of the uncertainty around the effectiveness of such treatments. In addition, the qualitative survey highlighted the perceived value of these pathways as seen by companies, where company respondents generally considered the studied FRPs as useful tools to facilitate timely development and review of important medicines and involvement of multiple stakeholders, especially the FDA Breakthrough and PMDA Sakigake. The findings and recommendations should be of interest to companies as they plan their development as well as to agencies as they look to develop and revise their FRP processes and ensure even greater efficiency and flexibility for important new medicines.