FDA Spurns Innovation—and Turns Its Back on Desperate Patients
The COVID-19 pandemic showed Americans how accelerating innovation in medicine can save countless lives. But now, it appears the federal government has once again turned its back on rapidly approved vaccines and therapeutics—and the consequences are dire for terminally ill patients, pre-term babies and their mothers, and all who are holding out hope for potentially life-saving treatments.
Consider how a promising new treatment offered a glimmer of hope to the 30,000 Americans with amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease—only for the federal government to snatch that hope away. Last month, the U.S. Food and Drug Administration (FDA) refused to even consider whether or not to approve a stem cell therapy that’s shown promise in treating this debilitating disease, which robs patients of their mobility, ability to communicate, and ultimately their lives. The FDA issued a refusal letter to Brainstorm Cell Therapeutics’ Biologics License Application for its “NurOwn” therapy—despite recent and growing data suggesting that the treatment may have a more significant clinical result in patients with less severe disease progression.
Meanwhile, the tiny handful of ALS patients who were fortunate enough to personally benefit from the treatment are now petitioning the FDA to hold an Advisory Committee meeting to review and discuss the treatment outcomes. It’s understandable that they want to shine a light on the FDA’s decision—after all, the FDA has a long and well-documented history of obscuring the process it uses to make life-or-death decisions. But these decisions should never be government secrets in the first place.
Countless infants and their mothers could also fall victim to the FDA’s latest tilt away from innovation. One in ten babies in the U.S. is born prematurely—a leading cause of infant mortality and disability. But the FDA is considering the withdrawal of its only approved treatment for mothers at high risk of pre-term labor. This fall, an FDA Advisory Committee even recommended pulling approval of the drug, Makena, and its generic equivalents. While the FDA is not bound to the recommendation and can also take other actions, such as revising the drug label to a more limited approved use for high-risk patients, it usually follows the panel’s recommendation.
Though there are legitimate questions about the drug’s overall effectiveness, studies suggest that the treatment may be effective in forestalling pre-term birth for high-risk patients, primarily African-American and Native American women who have previously given birth prematurely. Withdrawal would also limit the tools available to maternal-fetal specialists, many of whom continue to support the accessibility of this treatment.
Some who want Makena to be withdrawn maintain that the treatment could be obtained through compounding pharmacies, when necessary, though this is little more than a tacit admission that the treatment is, in fact, needed. But the ability to obtain treatments through compounding pharmacies can be difficult, especially since the FDA has actively undermined the availability of compound drugs for decades.
Cancer patients, who have much to gain from genetically tailored, individualized treatments, also have much to lose from the FDA’s restraint. Consider that the agency is poised to impose additional bureaucratic barriers that will make it harder for the most innovative companies to get the right treatment, to the right patient, at the right time. Dr. Richard Pazdur, who leads the agency’s oncology department, now wants to require that confirmatory trial studies for accelerated approvals be in place before accelerated approval is granted. That means smaller companies, which are responsible for most of today’s medical innovations, would face even higher financial burdens for getting their treatment through the time-intensive and costly approval process.
This new approach is already impacting companies, delaying the timeline within which a treatment might be available. That’s because many companies already count on the accelerated approval designation to fund the confirmatory studies. In fact, an estimated one-fifth (22 percent) of clinical trial failures in phase 3 occur due to a lack of funding.
The COVID-19 pandemic provided proof of the importance of removing the obstacles to innovation—from the rapid development of vaccines and therapeutics to the quick deployment of telehealth. But innovation isn't just a pandemic need: it must be a constant presence in our fight to put patients first, and it’s a necessary component of healthcare modernization.
Naomi Lopez is the Vice President for Healthcare Policy at the Goldwater Institute.
