The second of two sessions for the Friends of Cancer Research (Friends) Annual Meeting was held on November 19th. Day 2 featured a shift from Modernizing Expedited Development Pathways to a focus on Optimizing the Use of Accelerated Approval. Following a few words of welcome from Friends President and CEO, Jeff Allen, a fireside chat between Kate Rawson of Prevision Policy; Patrizia Cavazzoni, Acting Director at the FDA’s Center for Drug Evaluation and Research (CDER); and Peter Marks, Director at the FDA’s Center for Biologics Evaluation and Research (CBER) kicked off the meeting. The conversation began with lessons learned from the ongoing fight against COVID-19, including how remote access, telemedicine, and more flexible monitoring could be used beyond the pandemic to expand clinical trials. Other topics discussed included new challenges in regulating cell and gene therapies, the upcoming Prescription Drug User Fee negotiations, and the role of patient experience data. They concluded the conversation talking about accelerated approval (AA) and its important utility in oncology.
After the fireside chat was a panel discussion around the working group findings on optimizing AA. The panel began with a presentation of the group’s white paper findings by panel moderator and working group member George Demetri from the Dana-Farber Cancer Institute. The presentation covered current challenges in the existing AA framework and potential solutions, such as how a benefit-risk framework could facilitate a more holistic assessment. The panel also consisted of Josh Bilenker of Loxo Oncology at Lilly, R. Angelo de Claro of FDA, Aaron Kesselheim from Harvard Medical School, and David Mitchell, a multiple myeloma patient. They discussed why AA is important for getting life-saving drugs to patients sooner. They also discussed the importance of balancing flexibility, as the FDA is bound by certain legislative language when it comes to AA, with clarity, which is important to industry when allocating resources and initiating drug development programs. They closed by discussing ways to help the AA process work better, such as incorporating real-world evidence and potentially giving the FDA more flexibility in confirmatory trial requirements.
The final segment of the Annual Meeting was a conversation between Jeff Allen and Richard Pazdur, Director of the FDA’s Oncology Center of Excellence (OCE). They discussed the importance of AA as a patient-centric approach and not an incentive program for industry. They also discussed the need to take a fresh look at the AA program, which is nearly 30 years old. Benefits of how the AA program is working, such as reductions in lung cancer mortality due in large part to AA drugs, were mentioned, as were issues where some approvals have to be shoehorned into AA even if they do not fit into the framework. Finally, they discussed several ways to potentially improve the program such as requiring companies to renew the accelerated approval on an annual basis while confirmatory studies are be conducted, which is a process utilized in Europe.
Richard Pazdur: “The whole idea of Accelerated Approval is it is patient-centric, it is around the patient. It is not an incentive program for the pharmaceutical industry, and I think that that has to be underlined."
The session and meeting concluded with a few words from Jeff Allen, thanking all the key contributors and staff involved with the Annual Meeting. Be sure to read the white papers for both Modernizing Expedited Development Programs and Optimizing the Use of Accelerated Approval and stay tuned for additional information and next steps here on our website and social media.