After months of duplicative and confusing guidance from the Food and Drug Administration (FDA), patients finally have a glimmer of hope. The agency recently unveiled a new drug priority review program called the National Priority Voucher (NPV) system that will purportedly shorten the drug pre-approval process from one year to two months. On October 16, the FDA announced the first round of winners, including the medications Pergoveris for infertility, Teplizumab for Type 1 diabetes, and RMC-6236 for pancreatic cancer. This streamlined review is long overdue but is difficult to reconcile with the FDA’s recent rejection spree of promising drugs. Consumers deserve more than a patchwork approval system based on confusing criteria.
In announcing the first recipients of the NPV pilot program, FDA Commissioner Marty Makary claimed, “One of our core goals is to deliver more cures and meaningful treatments—especially ones that have an outsized impact on our most pressing national priorities.” This laudable sentiment is at odds with much of the agency’s recent decision-making. Drug approvals are down compared to previous quarters, and the agency is taking longer to evaluate marketing applications. Meanwhile, drug developers report inexplicable rejections from the FDA even after their products appear to be on track for approval.
In many cases, the rejected drugs have already been approved in other developed nations. The FDA has repeatedly refused to greenlight an ophthalmic version of the already-approved Avastin for wet age-related macular degeneration. The agency (wrongly) claims that there isn’t enough evidence that the medication works, a contention that even notoriously risk-averse European regulators reject. But what’s good enough for German and United Kingdom patients apparently isn’t good enough for their transatlantic cousins.
Ditto for a higher dose formulation of Spinraza for the treatment of spinal muscular atrophy. According to a September report in Pharmaceutical Technology, “The agency chose to decline the antisense oligonucleotide’s (ASO) approval due to insufficient technical information listed under the chemistry, manufacturing and controls (CMC) section of its supplemental new drug application.” In other words, paperwork took precedence over a medication proven to be safe and effective. Never mind that the purportedly missing information is “readily available,” and Japanese consumers have ready access to the high dose Spinraza regimen.
U.S. regulators similarly snubbed Ebvallo, used to fight a rare blood cancer that is fatal if left untreated. The FDA has been reluctant to even allow clinical trials for the medication, and not because of any safety or efficacy concerns. Rather, the agency is hung up on fixable deficiencies at third-party manufacturing sites. Somehow, this hasn’t been an issue for European patients, who have had access to the lifesaving medication since 2022.
According to the FDA’s former Center for Drug Evaluation and Research (CDER) Director Patrizia Cavazzoni, the agency’s approval standards are not getting stricter despite the uptick in manufacturing-related drug denials. Rather, the FDA is seeing “lesser quality in the facilities where these products are manufactured and we really need to work on this.” While Cavazzoni has a point that tainted medications are far-too-common facts of life, timely and targeted recalls are a far better alternative to barring an entire medication.
Imagine, for example, if the FDA completely withdrew approval for the diabetes drug metformin any time there was a manufacturing or distribution issue. While patients may be marginally safer from contaminants, they’d face a far tougher time keeping blood sugar levels in check. Life expectancy might even take a hit as a result of overly-strict policies. The FDA acknowledges this tradeoff and opts for a smarter and more tailored approach—for drugs that have already been approved.
The FDA should build on that approach by striving to approve more medications, especially ones already approved by peer nations. Handing out expedited review vouchers is better than nothing, but nothing beats real reform.
Ross Marchand is a senior fellow for the Taxpayers Protection Alliance.
