In the biomedical world, perhaps the biggest question looming over President Donald Trump’s upcoming pick for Food and Drug Administration commissioner is how drastically that person will help roll back FDA regulations.
Trump said at last week’s meeting in Washington D.C., with pharmaceutical executives that he wanted to slash FDA rules by 75 to 80 percent—“at a level no one has seen before”—while still promising “tremendous protection for the people.”
Taken at face value, those pledges would seem to benefit one group that has often fought red tape: patients and their advocates who feel regulatory strictures keep them from getting new, potentially life-saving drugs. They pressed hard for last year’s 21st Century Cures Act and its omnibus of looser regulations, guaranteed research funding, and mandates for the FDA to integrate patient feedback into its decisions.
Patient groups cheered when Barack Obama signed the Cures Act into law at the end of his presidency. Their growing impact on funding and regulatory decisions spurred this comment from former FDA commissioner Robert Califf at a conference just after his recent resignation: “If I had a disease with an ineffective advocacy group, I’d be worried right now.”
But as “patient-centric” gains traction as a Washington buzzword, many patient advocates say they are wary of what might be coming. “While this current climate of deregulation may seem popular, the potential harm and risk for patients is too great when considering our past and the important part the FDA plays in patient safety,” says Kimberly Haugstad, president and CEO of the nonprofit Hemophilia Federation of America. The mother of a boy with hemophilia, Haugstad cites thousands of hemophiliacs dying from HIV after receiving tainted blood three decades ago as “one of the largest regulatory failures in recent history.”
Adding to the misgivings are the known views, often stridently anti-FDA, of some of the potential commissioner choices who have emerged, matching a pattern of the new administration nominating critics of federal agencies to run those same agencies.
Barbara Handelin, CEO of nonprofit BioPontis Alliance for Rare Diseases in Raleigh, NC, says that an investor taking the helm—three of the names floated are active in private equity—“would once again be choosing the fox to guard the hen house.”
She says she wants to see a commissioner who can balance scientific rigor, encouragement of industry innovation, respect for drug firms’ business goals, and independence from industry at the same time. None of the current names meet those criteria, Handelin says.
An official nominee is likely to emerge soon, now that the Senate has confirmed Tom Price to run the Department of Health and Human Services. Price, who will oversee the FDA, the Medicare and Medicaid insurance programs, and other health-related agencies, won the seat despite strong Democrat objections over his healthcare stock trades as a member of Congress.
“RADICAL” AND “RAMP IT UP FASTER”
The rumored candidate whose views have caused the most concern is Jim O’Neill, a hedge fund manager and associate of Trump supporter Peter Thiel. O’Neill held a position in the George W. Bush administration at the HHS. When his name emerged in December, Bloomberg News found a recording of his appearance at a conference, during which he said the FDA should only evaluate drugs for safety. Whether they work or not—the standard of “efficacy,” in FDA-speak—should be determined once they are on the market, he said.
Hank Greely, the director of Stanford University’s Center for the Law and Biosciences, says that “radical” view of bringing drugs to market faster probably isn’t realistic—not the least of which because it would require Congress to change a key FDA statute. “It’s a nice libertarian talking point, but not many people—not insurers, not the drug industry, not doctors—really want it,” Greely says.
“It’s critically important to demonstrate effectiveness,” says Tim Coetzee, chief advocacy, services and research officer at the nonprofit National Multiple Sclerosis Society in New York. Many times an MS treatment has been “anecdotally beneficial,” Coetzee says, but getting to clinical trials didn’t change anyone’s health for the better.
As for a more moderate, “ramp it up faster” approach to drug evaluation, “it seems like it’s already faster,” says Greely. The perception of the FDA as a bottleneck … Next Page »