The BIO International Convention kicked off Monday with an interactive session organized by the BIO Board Standing Committee on Bioethics: The Bioethics of Drug Development—You Make the Call. Attendees engaged in a real-time polling exercise addressing the many difficult issues related to expanded access using a mock case study of a company – iCures – dealing with an expanded access request.
Moderator Steve Usdin, Senior Editor, BioCentury, led the panel through challenging issues in drug development and commercialization, including how to decide what indication to pursue, navigating the ethical issues around expanded access programs, and the trade-offs involved in making pricing decisions.
Panelists, who played the role of board members of iCures, included:
- Tim Mackey, MAS, PhD, Assistant Professor, UC San Diego, School of Medicine; Associate Director, MAS Program in Health Policy and Law, UC San Diego; Director, Global Health Policy Institute
- Russell Medford, MD, PhD, Managing Partner, The Salutramed Group, Inc.
- Richard Moscicki, MD, Center for Drug Evaluation and Research (CDER) Deputy Center Director for Science Operations, Food and Drug Administration (FDA)
- J. Leighton Read, MD, Venture Partner, Alloy Ventures
- Una Ryan, O.B.E., PhD, D.Sc., Chairwoman, The Bay Area BioEconomy Initiative
- Josh Sommer, Executive Director & Co-founder, Chordoma Foundation
Dr. Moscicki made it clear from the beginning of the session that the FDA is supportive of expanded access. Una Ryan chimed in that the approach must be fair, adding that she is ‘not in favor of giving a drug to whoever has the best media campaign.’ Furthermore, she asserted that ‘we are ethically obliged to continue to treat patients after granting expanded access.’ Ryan also emphasized the importance of avoiding diluting investment and considering the long-term impact on the drug development timeline.
Panelists shared several ideas for approaching the decision making process, including using a lottery system or appointing an external, independent entity to make the call. Dr. Mackey pointed out that transparency is key, saying that ‘it must be the hallmark of how we make decisions.’
Attendees split evenly when asked if the iCures company should provide expanded access to the patient if it did not impede the company’s ability to provide broader access expeditiously. Furthermore, the majority of attendees voted that a combination of government, insurers and manufacturers together are responsible for ensuring expanded access for patients.
Dr. Moscicki emphasized, “The first responsibility for a company is to conduct clinical trials.” Panelists and attendees mostly agreed that open label controlled trials provided the best design model for testing safety and efficacy. Josh Sommer commented that ‘a patients’ hope is a powerful resource, placebos do not offer hope, so therefore an active control is preferable to a placebo control.’
Turning to price, Usdin used the example of Gilead’s Sovaldi to highlight the balance between access and the need for return on investment. Ryan asserted that ‘this is a game-changing drug – investors took a risk from the beginning, the high price is fair and justified.’
Mackey added that access should be equitable and Dr. Moscicki emphasized that the main issue is sustainability based on value. Dr. Medford pointed out that the curve is a ‘rapid rise and rapid fall’ and companies take that into serious consideration when making pricing decisions.
Attendees mainly agreed with panelists that companies are justified in setting high prices for curative therapies by more than three to one.
Regulatory and market access issues will be key topics at this year’s BIO International Convention, which continues through Thursday.