What's the worst that could happen if we weaken the Food and Drug Administration? We may have a chance to find out, since the Trump administration promises in their first 100 days to cut the red tape at the FDA in order to "speed the approval of life-saving medications." They may get a hand in doing so from two types of legislation. "Right to Try" laws have passed in thirty-one states and promise very early access to barely-tested drugs, and there is a federal version in the pipeline. And the 21st Century Cures Act is likely to pass in the Senate, which will hand the Trump administration a crucial bipartisan assist: It gives desperately needed funding to the National Institutes of Health with one hand while weakening the FDA's approval process with the other.
Only a monster would stop a dying person from life-saving treatment, but it's a myth that cures exist and the FDA just won't release them. Here's why you should be skeptical of anyone who suggests that what's standing between dying patients and a Wonka-esque paradise of cures is the FDA.
New Drugs Are Already Sped to Market.
The FDA already operates faster than European and Canadian regulatory agencies. That expedited approval process was a victory of AIDS activists like ACT UP (the AIDS Coalition to Unleash Power) which, among other actions, shut down the FDA on October 11, 1988 to protest the lack of access to experimental drugs. But they didn't want to weaken the organization.
"We didn't want the FDA to deregulate. We wanted them to change the way they did regulatory oversight," said Mark Harrington, a former member of ACT UP and co-founder of the Treatment Action Group, an HIV/AIDS think tank, which opposes the 21st Century Cures Act. Four years later, the FDA developed an accelerated approval program, which helped bring AIDS drugs known as protease inhibitors to the market and usher in the era of life-saving triple therapy.
Since 1992, the FDA has added three other options for expedited review: priority review, fast track, and breakthrough therapy. If anything, some critics say that the FDA should slow down, pointing to drugs that were granted approval only to be pulled off the shelves. One thing is clear, though: Trump's plan to speed approval is an attempt to solve a problem that doesn't exist.
Exceptions Are Already Made for the Terminally Ill.
The FDA also has a pathway for people who are terminally ill to try unapproved drugs if nothing else works. That's known as expanded access, and in 99.5 percent of cases, the FDA grants the request (between 2010 and 2015, 7048 people applied and only 35 were turned down).
But when the FDA gives the green light, it still doesn't mean that every person receives the drug. The problem is that pharmaceutical companies don't have to provide experimental drugs, no matter what the FDA says.
Trump's Changes Benefit Corporations—Not People.
One reason companies turn down expanded access requests is because they must report anything that goes wrong, which may jeopardize future approval or public trust in the drug. The federal Right to Try bill would let doctors and drug manufacturers wash their hands of liability and prevents the FDA from holding something like, say, a patient death against the company. In fact, the federal version would strip the FDA of its approval power in these cases, which is the not-so-hidden agenda. "They're not enshrining patient protections. They're enshrining corporate protections," said Gregg Gonsalves, a co-founder of TAG and co-director of the Yale Global Health Partnership.
This is dangerous territory because Right to Try laws, such as the one that Vice President-elect Mike Pence signed into law in Indiana, say patients should be able to access experimental drugs after Phase 1 testing only, when there's only been an initial evaluation of safety with a small group of volunteers. At that point, we don't know for sure if the drug is safe or effective or what the best dose is or how to take it. The most vulnerable should have some idea if the "cure" is worse than the disease.
Lowering Approval Standards Carries Risks.
The 21st Century Cures Act also proposes to lower the FDA's standards for drug approval. Instead of expensive Phase 3 two-to-ten year studies of large groups of people, the Act would let companies cut corners and save money by using anecdotal evidence and things like surrogate markers (which are easier though not entirely reliable ways to measure disease progression, such as blood pressure for cardiovascular disease, and are how most cancer drugs speed onto the market even if they don't mean longer lives).
"It's an industry wishlist," Gonsalves said. "We can use clinical experience to help guide FDA drug approvals, we can expand the use of surrogate markers widely beyond drugs for life-threatening diseases, we can base antibiotic approval on in-vitro data. This means that drugs that are unsafe and ineffective could be on the market in the next few years."
"What patients deserve are drugs that really work," said Joshua Sharfstein, former principal deputy commissioner of the FDA and currently an associate dean at the Johns Hopkins Bloomberg School of Public Health. "The best protection for patients is knowing whether things work or not."