Industry should work with patients and their advocates to better understand their experiences, what they’re looking for in a therapy, and their tolerance for risk, US Food and Drug Administration commissioner Robert Califf emphasized during a fireside chat at the BIO International Convention this afternoon.
For the past few years FDA has been meeting with patients and patient advocates to better understand their diseases and medical problems and asking them where regulatory efforts should be focused, Califf noted. “And FDA is inculcating that into its way of thinking. It’s an exciting and explosive era,” and it’s one that has been accelerated thanks to technological tools including social media. But it’s not just the regulator that ought to be increasing its exposure to patient viewpoints, said Califf. “We encourage industry to think that way” as well, he said.
As patients, we shouldn’t just be recipients of the end process of what someone else determines is important, we should be involved from the beginning, said Califf. “What are the problems that need to be addressed?”
In particular industry, patients, and FDA ought to come to better understanding about patients’ tolerance for risk. This tolerance is fairly well understood at either end of the spectrum, Califf said. In the case of rare diseases for which there are no effective treatments, in general people are willing to take on more risk. But requirements are different for diseases that affect millions, like coronary disease, where in many situations drugs already exist to treat aspects of the disease.
“A lot of work to be done about where we fall in between” those two extremes, Califf said. “Industry should be involved, of course,” and patient advocacy groups are in a powerful position as well. “They can describe their needs and the level of risk that’s appropriate for their disease.”
Califf also elaborated on FDA’s recent efforts to streamline the process by which patients can access experimental medicines under so-called “compassionate use” framework. The announcement’s timing, coming so close to a hotly anticipated FDA decision about accelerated approval of a drug to treat a form of muscular dystrophy, had raised eyebrows. But “anyone who’s ever worked for the federal government would know right away that you couldn’t react to any situation that fast,” joked Califf.
FDA has been working on the streamlining for years, he said, “not just because of a particular issue we were facing.” The previous system was onerous and difficult to navigate, and the new compassionate use process should reduce the amount of time physicians must spend filling out requests for access to an investigational therapy. It’s still the case, however, that the FDA doesn’t give people access, but rather the company that’s developing the drug must make that decision. “Hopefully our recent changes are at least taking one step toward making it easier” to provide not-yet-approved drugs for compassionate use, “but it’s not the final solution,” said Califf. There are still “complicated issues but we’re very sincere about working on them.”
Califf touched on a variety of issues facing the agency during his session, including FDA staffing concerns, succession planning at the agency, new tools for evidence generation, and companies’ interest in communicating off-label information about drugs to physicians. With regard to the latter, Califf wryly acknowledged companies’ expertise. “I have great respect for industry, but in general I don’t look to industry to be the least biased source of information” about their products, he remarked.