The Biotechnology Innovation Organization (BIO) held an International Convention in San Francisco June 6 – 9, 2016. This year, the organization held two fireside chats, one of which was with Dr. Robert Califf, the Commissioner of the Food and Drug Administration (FDA). Dr. Califf discussed his commitment to strengthening programs and policies that enable the agency to carry out its mission to protect and promote the public health.
Dr. Califf noted that for the past few years, the FDA has been meeting with patients and patient advocates in an attempt to better understand their diseases and medical problems, and where regulatory efforts should be focused. Califf noted also that it is not just the regulator that should be thinking this way and increasing exposure to patient viewpoints, but also the FDA is “encourag[ing] industry to think that way” as well.
He believes that in particular, industry, patients sand the FDA ought to come together to better understand patients’ tolerance for risk. The spectrum varies widely, from rare diseases for which there are no effective treatments and for which patients are willing to take on more risk, to diseases that affect millions, where many times drugs already exist to treat aspects of the disease. However, Califf feels that there remains “a lot of work to be done about where we fall in between” those two extremes.
He discussed how in the past, the agency often spoke to small groups of representative advocates and now, they are beginning to enter this “very exciting, explosive era … with social media and broad use of the Internet, we can communicate directly.” He noted that the “best way to develop products in the future is likely going to be to involve a lot of people with diseases to have a handle on what their needs are, what their expectations are, and what their risk tolerance may be.”
Califf also touched upon FDA’s recent efforts to streamline the process by which patients can access experimental medicines under the “compassionate use” framework. He discussed the fact that FDA has been working on streamlining for years, and “not just because of a particular issue we were facing.” It was discussed that the previous system was onerous and hard to navigate, and that the new compassionate use process will likely reduce the amount of time physicians spend filling out requests for access to an investigational therapy. The FDA still doesn’t give people access, but rather the company that is developing the drug makes that decision.
Califf seemed to suggest that when it comes to off-label communications, if information was supportable and worth sharing, it should be on a drug’s label. The clinical trials construct, or “human experimentation,” as Califf referred to it, and its associated institutional review board, create a commitment to produce generalizable knowledge, “That, to me, means that if you have useful information from a human subject, you have a commitment to make that knowledge public. Then I ask what information do companies have that’s not public that they’d like to communicate properly to physicians and patients?”
Other topics discussed by Califf included: FDA staffing concerns, succession planning, new tools for evidence generation, and companies’ interest in communicating off-label information about drugs to physicians. Interestingly, Califf remarked that “I have great respect for industry, but in general, I don’t look to industry to be the least biased source of information.”