The nation’s former Food and Drug Administration commissioner Robert Califf, appearing for the first time since he resigned last week, told a Silicon Valley crowd this morning that the FDA will have to be flexible in coming years, allowing greater patient input into drug evaluation and leaning on outside watchdogs for help weeding out “imposters.”
Califf’s brief appearance at a precision medicine conference near San Francisco comes as regulation across industries is in the bullseye of the new Trump administration. President Trump, at a gathering in Washington, told U.S. business leaders today that the federal government could cut regulation “by 75 percent.”
The FDA oversees products that make up about 20 percent of the American economy. Speculation about Califf’s replacement has centered mainly on critics of the agency’s evaluation of medicines. For example, one name that has surfaced is hedge fund manager Jim O’Neill, an associate of libertarian investor Peter Thiel. O’Neill said publicly in 2014 that the FDA should only evaluate drugs for safety, then let the open market determine whether a drug actually works.
(Derek Lowe, a pharma industry chemist and popular blogger, countered the talk of radical FDA drug deregulation this morning with a defense of the clinical trial system.)
Debates about how to balance risk and benefit of a drug are nothing new to FDA. The final years of the Obama administration have seen more flexible regulation of certain drugs—for desperate cancer cases, for example.
In one of the most controversial episodes at FDA in recent years, Califf allowed the drug eteplirsen (Exondys 51) to come to market last year despite FDA staff protests that the key study, which gathered data from 12 patients, provided an inadequate level of clinical evidence. The drug, from Sarepta Therapeutics (NASDAQ: SRPT), is a treatment for a variant of the rare Duchenne muscular dystrophy. Patient advocates pushed hard for the approval, and the FDA’s top drug evaluator, Janet Woodcock, overruled her staff’s negative view of the drug. Califf supported Woodcock’s call and allowed the drug to come to market.
One of Woodcock’s deputies later slammed the decision, telling a group of rare disease advocates that the sponsor company’s efforts were “a rescue of a failed program.” (Two weeks ago, Sarepta’s CEO reported that insurers have not been eager to pay for the drug.)
Califf didn’t address the eteplirsen controversy this morning, but he acknowledged the power of patient groups. “If I had a disease with an ineffective advocacy group, I’d be worried right now,” Califf said, noting groups’ impact on research decisions and regulatory considerations.
He also warned, however, that patient groups, which often take funding from drug companies, can end up doing their funders’ bidding. “There’s a bad history of advocate groups funded by companies,” Califf said.
Public Citizen released a report last year showing that a majority of patient groups publicly opposing a Medicare B proposal to reduce drug costs—110 out of 147 total—received Big Pharma funding.
The omnibus 21st Century Cures Act, which Obama signed into law last month after overwhelming Congressional support, directed the FDA to use more patient input in its decisions. Critics like Public Citizen, Sen. Bernie Sanders (I-VT), and others considered the Act’s regulatory changes beneficial mainly for drug and device companies looking for less oversight of their products.
When asked if FDA is prepared to deal with the rapid incorporation of data and computation into health products, Califf said there’s a vast gray regulatory area between, for example, a defibrillator that uses “computation” to operate—“that needs to be a regulated device”—and measuring heart rate on your watch. “That’s a free for all,” Califf said.
In between the two, however, the agency should keep a light hand on regulation, Califf said, only stepping in when problems occur. “And I predict that we will have problems,” he said.
But in a nod perhaps to the agency’s finite resources—likely to shrink further under the Trump administration and Republican-led Congress—Califf also said that scientists working in cutting-edge areas, such as gene editing, need to share more data so that evaluation can, in some effect, be crowd-sourced. “Transparent sharing,” he said, will be important so “the ecosystem” can assess what’s accurate or not—“so that imposters and non-truth-tellers can get weeded out without FDA being an investigative unit.”