Session Synopsis: Patient advocacy plays an increasingly critical part in today’s health care delivery system. This session will facilitate a discussion that focuses on two examples of how patient involvement is shaping the course of therapies and decisions: driving the 21st Century Cures Act from concept to the finish line, and as a second example, expediting “conditional approval” of treatments that have been proven safe in clinical trials.
Session Chair Profile
Ph.D., Founder & Chairman, Cancer Commons
Marty Tenenbaum is a renowned computer scientist, Internet entrepreneur and cancer warrior. He is the Founder and Chairman of Cancer Commons, a non-profit network of physicians, scientists, and patients that Newsweek dubbed the “LinkedIn of Cancer.” He began his career in AI, leading elite research groups at SRI International and Schlumberger Ltd. Later, as an Internet commerce pioneer, he founded or co-founded five successful startups. Dr. Tenenbaum is a fellow and former board member of the American Association for Artificial Intelligence, a former Director of Commerce One, the Public Library of Science (PLoS) and Patients Like Me, and a former consulting professor of Computer Science at Stanford. He currently serves as a Director of CommerceNet and Efficient Finance. Dr. Tenenbaum holds B.S. and M.S. degrees in Electrical Engineering from MIT, and a Ph.D. from Stanford, and has received numerous awards for his contributions to AI and his work as a patient advocate.
Is An Additional Path to FDA Approval Possible?
This talk will explore the question: When faced with certain death, it is acceptable to accept less than 100% proven safety and efficacy? Patients with glioblastoma and certain other cancers with dire prognoses do not have the time to wait for new treatments to be fully approved by the FDA. Is it time to add “conditional approval” as an additional pathway to approval for these patients? In this session, a plan will be proposed by which the FDA could conditionally approve a treatment that has been proven safe in a clinical trial(s) with at least 25 patients, and has demonstrated biologic activity. Therefore, this treatment could not be denied by insurance as being “experimental”. However, the requires patients using these drugs to participate in a registry through which their physicians submit details on the treatments, side effects and outcomes. The FDA’s periodic review of the registry data would lead to a range of outcomes from full approval to the conditional approval being revoked.
Ph.D., Chairperson and Founder, Friends of Cancer Research (Friends)
Since Dr. Sigal founded Friends of Cancer Research, the organization has been instrumental in the creation and implementation of policies ensuring patients receive the best treatments in the fastest and safest way possible. Dr. Sigal is Vice Chair, and currently Acting Chair, of the inaugural board of directors of the Reagan-Udall Foundation. She serves on the Board of the Foundation for the National Institutes of Health, where she chairs its Public Private Partnerships Committee. In 2016 Dr. Sigal was named to Vice President Biden’s Cancer Moonshot Blue Ribbon Panel, to the Parker Institute for Immunotherapy Advisory Group and joined the inaugural board of advisors for the George Washington University’s Milken Institute of Public Health. She also holds leadership positions with the MD Anderson Cancer Center External Advisory Board, the Duke University Cancer Center Board of Overseers, and The Sidney Kimmel Comprehensive Cancer Center Advisory Council.
Patient Advocacy Role in the Development of the 21 Century Cures Act
The 21st Century Cures Act, passed and signed into law last year, was a landmark bipartisan bill years in the making. The Act was initially focused as a funding mechanism for the NIH, with the goal of accelerating treatments for patients through research. Yet, after numerous nationwide public meetings, including a significant movement driven by the patient advocacy community and Vice President Joe Biden, the Cures bill became the most groundbreaking healthcare innovation bill of the past decade. The final result is a monumental piece of legislation that enhances vital programs, improves health agencies, engages young scientists and addresses patient needs. We will explore how patient groups drove the 21st Century Cures Act from a concept to the finish line.