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Increasing Patient Access to Investigational Drugs Is Focus of Early Afternoon Session on Day 1 at Personalized Medicine World Conference (PMWC) 2017 at Duke

PMWC 2017 at Duke Co-Chair Ralph Snyderman, MD, introduced the two panelists for a discussion of “Increasing Patient Access to Investigational Drugs,” as the two leading people in the country working to make such drugs available to patients, and noting that for patients this is perhaps the best of times and the worst of times—best in that we are learning so much so rapidly and worst due to the difficulty of patients gaining access to potentially helpful drugs. One panelist was Ellen V. Sigal (photo), PhD, Founder of Friends of Cancer Research (https://www.focr.org/), an organization that 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 introduced herself by noting that her PhD is actually in history and she had worked as a real-estate developer until her sister was diagnosed with breast cancer and died two days after receiving a bone marrow transplant. Then, Dr. Sigel began her unstinting work to help cancer patients. Dr. Sigal is Vice Chair, and currently Acting Chair, of the Inaugural Board of Directors of the Reagan-Udall Foundation (http://www.reaganudall.org/), which was designed to be a vehicle for bringing an array of resources and perspectives to bear on high-priority FDA regulatory science projects. The foundation fosters collaborations between patient groups, industry, academia, and FDA scientists to design and conduct regulatory science research. Dr. Sigal also 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.

Dr. Sigal described how patient advocacy groups drove the The 21st Century Cures Act (https://www.congress.gov/bill/114th-congress/house-bill/6?r=9) from a concept to the finish line. The bill was passed and signed into law last year, and 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 ground-breaking 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.

The second panelist was Marty Tenenbaum (https://www.cancercommons.org/about/our-founders-story/), PhD, a renowned computer scientist, Internet entrepreneur, a cancer warrior, and a survivor of metastatic melanoma. He is the Founder and Chairman of Cancer Commons (https://www.cancercommons.org/), a non-profit network of physicians, scientists, and patients that Newsweek dubbed the “LinkedIn of Cancer.” Above all, Dr. Tenenbaum stresses the potential power of sharing as much information as possible by cancer patients.

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 BS and MS degrees in Electrical Engineering from MIT, and a PhD 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?

In the PMWC 2017 discussion, Dr. Tenenbaum focused on the question: When faced with certain death, is it 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 the session, he described a plan that 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 plan would require 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. Finally, in order to better achieve buy-in from pharma companies, the proposal would require that the FDA not be able to hold results of the conditional approval against a drug going through traditional clinical trials.

In closing, Dr. Sigal emphasized that all efforts in this area should ultimately be driven by striving to do what is best for the patient. For his part, Dr. Tenenbaum argued that the entire clinical trial system needs to be re-thought.

[PMWC 2017 Duke]