On Thursday, March 22, 2012, Friends of Cancer Research (Friends) held a congressional briefing titled “Expediting New Treatments to Patients: FDA Approval Mechanisms” to discuss and define the use of current Food and Drug Administration (FDA) mechanisms for accelerating drug development and explore the prospect of new and/or enhanced tools to bolster FDA’s ability to get drugs to market sooner and safer. The four-person panel featured:Dr. Janet Woodcock, Director, Center for Drug Evaluation and Research, FDA;Mr. Jonathan Leff, Managing Director, Warburg Pincus;Dr. Howard Scher, Chief, Genitourinary Oncology Service, Memorial Sloan-Kettering Cancer Center;Dr. Ellen Sigal, Chair, Friends of Cancer Research and was moderated byDr. Mark McClellan, former commissioner of the FDA and Director of the Engelberg Center for Health Care Reform at the Brookings Institution.
Dr. Sigal introducedSenator Michael Bennet (D-CO)as a “champion for FDA and Cancer Research.” Sen. Bennet, in delivering opening remarks, underscored the importance of discussing new approval pathways to get treatments to patients in a faster and safer way, particularly with the upcoming reauthorization of the Prescription Drug User Fee Act legislation in Congress. He praised the bipartisan nature of the discussion and highlighted the need for patients, as well as the scientific community, to have confidence in the future of drug approvals in a rapidly changing scientific environment.
Dr. Jeff Allen,FriendsExecutive Director, gave a brief overview of a proposal for a “Breakthrough Therapies” designation for the FDA. The designation, which originated from a panel at the annual Friends-Brookings Conference on Clinical Cancer Research, would trigger close communication between FDA and the drug sponsor, to develop trial designs to shorten or combine traditional phases of development, expedite the pathway to approval and avoid giving larger numbers of patients a potentially harmful or ineffective drug as part of a control arm.
Dr. Woodcock then began the panel discussion by summarizing some of the FDA’s current mechanisms for expediting drug development and review; accelerated approval, fast-track designation, and priority review status. Dr. Woodcock pointed out that 80 drugs have been approved under accelerated approval since 1992, 29 of them cancer drugs.
Dr. McClellan asked the panel where drug development is headed as a result of new scientific discoveries and progress. Dr. Scher stated that genetics and biomarkers have changed the way many have looked at and classified certain diseases, using prostate cancer, once thought of as a site specific disease but now known to comprise up to 30 different diseases, as an example.
The panel discussed current proposals to enhance the FDA, including enhanced accelerated approval and the breakthrough therapy designation. Dr. McClellan asked Dr. Woodcock what she thought of the proposal and whether a mechanism like “breakthrough” would be beneficial. Dr. Woodcock stated that it would be helpful and that there should be an “all hands on deck” by the FDA when significant benefit is seen early so that a potentialbreakthroughcan be tested and developed as rapidly as possible. She stressed the need for FDA and developers to design trials that are ethical and able to yield information as rapidly as possible. Dr. Scher agreed and said that anytime you can get everyone together and on the same page, things get done faster.
Dr. Sigal also reiterated this point and went further to say that “since people, especially in Washington, tend to work in silos, collaboration, communication and clarity are of the utmost importance to alleviating the confusion in all communities about what the agency will or will not do.”
Mr. Leff went on to discuss the opportunities and challenges to investors in light of these new discoveries. While the scientific developments present opportunities themselves, Mr. Leff said, the high costs and time delays getting drugs through the regulatory cycle aresignificantbarriers to new investment. He described a growing trend toward new areas of investment including health information technology and social media.
Asked later what investors are looking for to reverse this trajectory, Mr. Leff replied that looking at new approaches and ways to speed up the process is the right conversation to be having, but also cautioned that it is important not to forget about the many drugs that may not designated breakthrough therapies moving through the pipeline or lose focus on other treatments because they are not considered to be breakthroughs.
Dr. Sigal emphasized the importance of continued development of targeted therapies and getting safe and effective treatments to patients, who “at the end of the day want treatments options that will work for them.”