For decades, patients desperate for live-saving treatments have had to deal with a slow and cumbersome federal approval process for pharmaceuticals. The Food and Drug Administration’s mandate stipulates that new drugs must be safe and effective in order to be granted agency approval, which in practice results in a multi-billion-dollar, years-long evaluation process. While this process has undoubtably prevented some dangerous and ineffective drugs from entering the marketplace, the FDA’s usual insistence of two clinical trials to demonstrate efficacy fails terminal patients who cannot wait for cutting-edge treatments.
Fortunately, the Promising Pathway Act (PPA) would fix this issue by creating a new pathway to early approval for game-changing medications. Lawmakers can save countless lives, but only if they’re willing to push for sensible regulatory flexibility.
The FDA’s insistence on multiple, “well-controlled” clinical trials to demonstrate efficacy is grounded in the search for statistical significance. The agency wants to be sure that preliminary results showing a drug’s effectiveness are not due to chance nor coincidence, but rather because of some identifiable chemical mechanism that works as intended. While this is an understandable regulatory goal, overly rigid statistical thresholds can pose problems for terminal patients. According to a 2019 analysis by the Massachusetts Institute of Technology and Boston College researchers, “conventional standards of statistical significance for approving drugs may be overly conservative for the most deadly diseases and overly aggressive for the mildest ones. Therefore, changing the ‘one size fits all’ statistical criteria for drug approval is likely to yield greater benefits to a greater portion of the population.”
To their credit, lawmakers and the FDA have been pushing for more expedited pathways in recent years to chip away at rigid and uniform approval procedures. One particularly promising development has been the “compassionate use” program, which allows patients with immediately threatening conditions and serious diseases with no other options to get access to not-yet-approved medications. The issue, though, is that this process requires the cooperation of drug companies, and manufacturers may be understandably reluctant to participate because patients can only be charged the “direct costs of making the drug available … such as manufacturing and shipping” without any margin for profit. And, just to recover the costs for these expenses, manufacturers need to go through an onerous bureaucratic process that includes having an independent certified public accountant review and approve calculations on which costs are truly “direct.”
These de-facto price controls set by the FDA mean that, even though the agency approves the vast majority of expanded use applications, manufacturers may have insufficient incentive to participate in the process. There are plenty of risks associated with giving patients unapproved products and the lack of profit hardly makes those risks worthwhile.
The PPA would help address this conundrum by allowing for an expedited approval process for medications that could help patients with devastating diseases. The proposed legislation would require the FDA to set up a “provisional approval” system in which manufacturers could sell their medications by submitting limited data up-front with the stipulation that sponsors would need to keep collecting and submitting data pending full approval by the agency. These “provisional” applications would be received on a rolling basis and the agency would have 90 days to examine submitted applications. Under this new system, promising treatments wouldn’t be stuck indefinitely in the black box of the FDA regulatory apparatus. And, unlike the “expanded access” program, drug companies would have every incentive to play ball and deliver cures to the most desperate patients.
The current FDA approval process fails far too many patients at far too great a cost. The PPA could go a long way toward saving lives and reestablishing trust in America’s food and drug regulator.
David Williams is the president of the Taxpayers Protection Alliance.
As much as this sounds like a great idea, and may be for a few drugs, the door it opens for the very clearly compromised FDA, and our very clearly compromised gov’t in general, to push new drugs on us with little evidence of usefulness and/or safety is simply not worth the risk… A permanent state of drug use from cradle to grave is what Big Pharm is pushing – and starting to get with their bought and paid for FDA, and CDC, and NIH, and … Gotta keep up profits!
It’s inconceivable (and incompetent) that this article fails to mention FDA’s Accelerated Approval Program, which was created “to allow for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint. A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval.” https://www.fda.gov/drugs/information-health-care-professionals-drugs/accelerated-approval-program