FDA Drug Rejections Have One Fatal Flaw
It’s notoriously difficult to get a medication approved through the Food and Drug Administration (FDA). In fact, bringing a new drug to market takes more than a decade and costs about $2 billion. Empirical analyses using the U.S. Burden of Disease Study support the idea that the FDA is far too cautious in approving life-saving medications. Unfortunately, the agency is doubling down on its rigid and risk-averse approach.
The FDA recently rejected a treatment for the rare hormonal disorder acromegaly despite ample evidence that the medication is well-tolerated and improves patients’ lives. Regulators dwelled not on safety nor efficacy, but rather on “[manufacturing] facility-related deficiencies.” This is just the latest instance in a worrying trend of game-changing drugs being delayed for months or even years because of one-off production problems with straightforward fixes. The FDA must embrace innovation and quit standing between patients and promising medications.
Patients suffering from acromegaly face constant swelling, fatigue, and high overall mortality. The already-approved medication octreotide offers some much-needed respite by suppressing and inhibiting excess growth hormone behind this deadly disease. The new medication CAM2029 (produced by Camurus) offers a longer acting and more effective version of octreotide, but is now delayed indefinitely because the FDA has qualms about how the drug is manufactured.
According to the FDA’s 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 would be a bit safer from probable carcinogens such as N-nitrosodimethylamine, 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.
When in doubt, the FDA could also look beyond its borders and see what its trusted international counterparts are doing. In January, the agency rejected a drug called zolbetuximab designed to treat gastric/gastroesophageal cancer. Similar to CAM2029, the issue was neither safety nor efficacy. According to clinical trial results, the medication improved median progression-free and overall cancer survival by 2-3 months compared to just chemotherapy. The drug was held up because of “unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility.”
These issues apparently weren’t deal-breakers for the FDA’s Japanese counterpart, who green-lit the drug in March. It took the FDA seven months after the Japanese approval to finally grant American patients access to the life-extending medication. In its October 18 press release announcing the approval, the FDA boasted that the application “was granted priority review, fast track, and orphan drug designation.” When the U.S. is still falling behind international approval times even after applying three different types of preferential treatment, there is clearly a need for reform.
If the FDA is not up to the task of fixing the drug approval process, then lawmakers need to step up. Congress can amend the Food, Drug, and Cosmetic Act to clarify that faulty manufacturing should not be the sole basis for drug rejections. Rather, the FDA should work with manufacturers on recalls of faulty batches if a drug otherwise meets the agency’s safety and efficacy requirements. It’s time for a tailored and flexible approach that improves patients’ lives while ensuring their safety.
Ross Marchand is a non-resident fellow for the Taxpayers Protection Alliance.
