Over the past few years, the FDA has unveiled a spate of new approval pathways in hopes of getting much-needed treatments on the market faster. But in the rush to accelerate drug development, the agency has wound up putting patients at risk, according to one congresswoman, fast-tracking unproven therapies despite serious safety concerns.
Rep. Rosa DeLauro, a Connecticut Democrat, fired off a letter to FDA Commissioner Margaret Hamburg chastising the agency for approving drugs based on Phase II data or pivotal trials with small enrollments.
As an example, DeLauro cites Johnson & Johnson's ($JNJ) Sirturo (bedaquiline), a drug-resistant tuberculosis treatment approved off of mid-stage data during the FDA's late-2012 frenzy of drug decisions. The drug was a beneficiary of the FDA's accelerated approval program, also receiving fast-track, priority review and orphan-drug designations, meaning it made it to the market despite the fact that patients who took it in combination with standard treatment were 5 times more likely to die than those taking older drugs alone, DeLauro wrote.
Any reduction in clinical trial rigor puts patients at risk, and "if the FDA permits smaller sample sizes, it would not be feasible to conduct subgroup analyses for safety and efficacy in women and men, in different racial and ethnic groups, or across age," DeLauro wrote. "... Clinical trial endpoints that prioritize clinically meaningful patient outcomes and methodological targets above surrogate markers of disease are the key to ensuring public health and drug safety."
But, in a blog post that doesn't address DeLauro but appeared the same day as her letter, Hamburg reiterates something the FDA has long held: Not all drug approvals are created equal. The agency prides itself on flexibility, working with sponsors to design trials that can efficiently and reliably determine safety and efficacy, the commissioner wrote.
"People with serious or life-threatening illnesses, particularly those who lack good alternatives, have told us repeatedly that they are willing to make some trade-offs in order to gain access," Hamburg wrote. "And, of course, 'thoroughness,' such as whether a clinical trial is large enough, is in the eyes of the beholder. There is no reason to expect drugs to be tested on similar numbers of patients, regardless of the disease."