Experts Encourage Greater Use of Accelerated Approval Authority at FDA and Modernizing of Clinical Trial Processes
WASHINGTON, DC – The House Energy and Commerce Subcommittee on Health today held a hearing in the 21st Century Cures initiative to review the President’s Council of Advisors on Science and Technology (PCAST) Report on Drug Innovation. The hearing built on a recent white paper soliciting input on the recommendations made in the PCAST report and how those proposals, among others, can contribute to the 21st Century Cures goal of accelerating the discovery, development, and delivery cycle and ultimately get innovative new cures and treatments to patients more quickly. The subcommittee today heard from a panel of experts who contributed to the PCAST report.
“The president, in soliciting recommendations on this important topic, decided propelling drug innovation is a policy worthy of exploring. I could not agree more,” said full committee Chairman Fred Upton (R-MI). “In their report, the president’s advisors found that the nation’s biomedical innovation ecosystem is under significant stress, citing the patent cliff facing the pharmaceutical industry, declining investment from venture capital and decreasing research and development in critical areas, including Alzheimer’s. We have heard similar concerns in our discussions with patients, innovators, and thought leaders.”
Jeff Allen, the Executive Director of the Friends of Cancer Research, commented, “The state of science and medicine has never been as promising as it is today. … Aligning policies with the current state of science has the ability to accelerate the development of safe and effective therapies to improve the state of biomedical research and improve the lives of patients. The 21st Century Cures initiative can be the next step toward that goal.”
Frank Sasinowski, testifying on behalf of the National Organization for Rare Diseases, noted that from the mid-1980s through June 2013, only 19 drugs were approved under Accelerated Approval that were not for cancer or HIV/AIDS. He said, “Both PCAST and FDASIA recommended that FDA expand, beyond cancer and AIDS, use of its Accelerated Approval authority to approve medicines for those with serious diseases and no available therapies,” noting “to implement the PCAST and FDASIA recommendation to use this authority more often, FDA would not need to establish any new program or policies, but may only need to give this approval pathway more visibility and more frequent consideration.”
Garry A. Neil, M.D., Global Head of Research and Development of Medgenics, Inc., discussed the costs and regulations surrounding clinical trials. Neil said, “Clinical trails are the greatest cost driver of biologic and drug development. Today each clinical trial essentially requires establishing new infrastructure facilitating the creation of clinical trial networks involving academic investigators, patient advocacy groups and industry sponsors in close collaboration with FDA to increase cost efficiency, shorten time lines and allow more ‘plug and play’ clinical studies.” Neil offered several examples including, “the Clinical Trials Transformation Initiative/NIH Collaboratory project that has been established to support the design and rapid executive of several ‘Pragmatic Clinical Trial Demonstration Projects’ including performing randomized trials using electronic health records and the Cystic Fibrosis Foundation’s Therapeutic Development Network (TDN), the largest cystic fibrosis clinical trials network in the world and the Lung Cancer Master Protocol established under FNIH.”
Health Subcommittee Chairman Joe Pitts (R-PA) underscored the committee’s collaborative approach to reform, noting, “The first goal of this project is to solicit ideas. Congress does not have all the answers, but we do have a role to play in ensuring our nation’s laws and regulations keep pace and complement the biomedical research and innovation that is happening at lightening speed.”
Pitts also submitted for the record a recent piece authored by Dr. Janet Woodcock, Director of the FDA Center for Drug Evaluation and Research, regarding the FDA’s progress on the recommendations made by PCAST.
Responses to the questions posted in the white paper, 21st Century Cures: An Update on the President’s Council of Advisors on Science and Technology 2012 Report on Propelling Innovation, can be sent to email@example.com by June 10, 2014.