Operation Warp Speed, the Trump administration’s historic effort to deliver hundreds of millions of doses of a coronavirus vaccine by January, has demonstrated how quickly the Food and Drug Administration’s drug approval process can move in extreme circumstances.
Yet, even though it has, in some ways, validated longstanding criticisms of the FDA as overly conservative, the drive for a vaccine has not yet generated much enthusiasm for reform of the agency’s normal operations.
The pandemic has brought into stark relief that a longer approval process can cost lives. The FDA has retained all the normal standards for approval of a vaccine, including four phases of testing trials, but has dramatically expedited the process in an attempt to shorten the process from years to months.
It’s also true, though, that even in more normal times, lives are at stake in the approval process for drugs and medical devices.
One reason that the federal government has been able to speed up the COVID-19 vaccine approval effort so radically is the financial might that has been brought to bear on the problem. The federal government has spent $10 billion on Operation Warp Speed so far, resources that won’t be available in the development of future vaccines.
“As long as 200 companies across the globe are willing to invest tens of billions of dollars into the effort, sure, we could do it,” said Dr. Paul Offit, a pediatrician specializing in infectious diseases. “The government basically took the risk out of it for the industry.”
In recent years, the FDA has seen changes meant to speed approvals or allow for bypassing reviews when possible. In 2016, Congress passed the 21st Century Cures Act, which allows pharmaceutical companies, under limited conditions, to provide the FDA with data from studies other than randomized controlled trials. In 2018, Congress passed Right to Try, which allows patients with life-threatening conditions to try experimental drugs without FDA approval.
And, since 2017, the FDA has attempted to make its areas of expertise even more focused by expanding the number of clinical divisions in the Office of New Drugs from 19 to 27.
Yet, free market advocates think the agency could do much more.
“I think that the experience of COVID-19 will accelerate the FDA’s initiatives to understand and use 21st-century science going forward,” said Peter Pitts, president of the conservative Center for Medicine in the Public Interest and a former FDA associate commissioner for external relations. “That includes a more aggressive use of real-world data and real-world evidence.”
That will mean, according to Pitts, greater incorporation of data from more than just randomized controlled trials, considered the gold standard in medical research. He noted that the recent uproar over convalescent plasma as a treatment for COVID-19 centers around the fact that it was not studied using a randomized controlled trial. The study that was conducted examined over 35,000 patients. Pitts said that the FDA needs to give greater consideration to such studies in its analyses.
Other reforms proposed include reducing the scrutiny of digital diagnostic technology, or apps, that have minimal safety concerns and shortening the burdensome process that drug companies face scheduling conversations with the FDA on how to move drug development forward. Republican Sens. Ted Cruz of Texas and Mike Lee of Utah have introduced legislation, the RESULT Act, that would enable the FDA to speed up its approval process when a drug or device is approved by a similar agency in a “trustworthy” country.
“I think we are at an inflection point similar to where the FDA was in the early days of HIV/AIDS,” Pitts said. In 1988, the members of the activist group ACT UP protested the slow approval process for AIDS drugs by encircling the FDA building. It was a catalyst for reforms that enabled the FDA to expedite approval of drugs for life-threatening conditions.
Yet, there are few signs that there is momentum for administrative or legislative reforms along those lines.
“I’m not so sure [Operation War Speed] is going to mean very much,” said Joe Ross, a professor of medicine and public health at the Yale School of Medicine. “I think we are in a fairly unique situation, where lots of groups, industry and government, are trying to move things as quickly as possible.”
Thus far, there is little movement on Capitol Hill. Sen. Mike Braun, a Republican from Indiana, introduced the “Promising Pathway Act” back in June. The legislation would allow the FDA to give provisional approval for drugs that can treat life-threatening diseases or conditions.
A Congressional staffer who has worked on FDA reform told the Washington Examiner that the earliest opportunity to reform the FDA wouldn’t be until spring of 2021.
Pathways that speed up the FDA approval process will entail risks.
“In terms of the risk-benefit concerns, many of these pathways involve allowing drugs to come to market based upon lower levels of evidence and lower numbers of trials,” said Dr. Michael Carome, director of the Health Research Group at the liberal group Public Citizen and a former associate director for regulatory affairs at the Department of Health and Human Services. “We have the risk that large numbers of patients could be exposed to drugs for which the evidence ultimately shows that they are not safe and effective.”
Pitts, though, notes that reform could reduce the cost of getting drugs to market and, ultimately, the cost to consumers.
“The impact of the FDA on drug pricing isn’t on how long the review takes, it’s on what the process entails. As FDA embraces more tools of 21st-century regulatory science … the cost of development may not decrease, but the predictability of the results will increase,” he said. “The FDA can reduce the cost of overall research and development by helping drug developers to fail faster. For a single drug, a 10% improvement in predicting failure before clinical trials could save $100 million in development costs.”
