Paper Calls for FDA Reforms on Transparency/Evidence
A white paper called “What Needs to Change at the FDA” says the agency’s evidence standards have become distorted, which have led to decisions that often favor industry interests over those of the public, and an “anecdotal ideology among FDA senior management that patients are willing to accept more uncertainty for the drugs they take.” The paper was prepared by a working group commissioned by PharmedOut, a Georgetown University Medical Center project that promotes evidence-based prescribing.
The paper takes issue with FDA transparency, particularly around the agency’s advisory committee process. “The use of invited speakers with ties to a sponsor should be minimized,” it says. “The FDA still grapples with the impact of emotional testimony by patient advocates who often have ties to the pharmaceutical industry and who speak at open public hearings, at workshops and at other events under a statutory requirement to ensure patients’ experiences are captured.”
FDA communications also need to “better emphasize the complexity of drug approval decisions, by pointing out the reservations that reviewers may have about a drug that they approve, or fail to approve, and the unanswered questions that remain at the time of approval,” the paper says. “Dissenting voices and varied views rooted in disciplinary perspectives are important in science and should be recorded and made public.”
Additionally, the paper says it is concerning that FDA’s emphasis shift from approving drugs based on rigorous evidence standards to moving drugs and devices to market rapidly is based on “preliminary evidence. Once in use, the theory is that ‘real world evidence (RWE)’ will adequately inform our understanding of the product.” It contends this assumption is “based on misconceptions about the rigor of both the upstream (pre-approval) and downstream (post-approval) processes and ignores the history of why randomized trials came to be the gold standard for evidence.”
The paper recommends that more decisions be based on head-to-head clinical trials with product comparisons rather than relying on placebo comparisons. “Necessary downstream changes include addressing the delays in post-market requirements and commitments, better systems of pharmacovigilance for both drugs and devices, moving responsibility for coordinating RWE from the FDA to a new coordinating agency within HHS, and material and staff resources to make these strengthened systems work,” it says.