Efthimios Parasidis
One week prior to the Supreme Court’s landmark ruling in the health care cases, Democrats and Republicans overwhelmingly voted in favor of health-related legislation (387-5 House; 96-1 Senate). Industry and HHS were quick to congratulate our elected officials on a triumphant bipartisan achievement, while the FDA enthusiastically welcomed its latest collaboration with Big Pharma. Lobby groups boasted of their ability to “craft” legislation with FDA and praised the “unprecedented level of public input” into the new law.
We should all be concerned.
The new law – PDUFA V – sets the fees that drug sponsors pay to have a product evaluated by the FDA. User fees comprise a hefty portion of the FDA’s budget for drug review – approximately 60%. Putting aside regulatory capture, the impact of user fees on the evaluation process, and FDA’s actions to suppress information that reveals how political or corporate favors lead to bad science within the agency, one of my primary concerns with PDUFA V relates to the expedited review process for new molecular entities (NMEs).
Industry has long lobbied for expedited review for NMEs since, quite simply, NMEs are an integral component of Big Pharma’s new drug pipeline. NMEs are molecules that have never been approved by the FDA, and thus carry unknown safety risks and limited information related to clinical benefits. The expedited pathway allows FDA to approve NMEs absent data that the product provides a clinical benefit to patients. The legislation was passed despite the well-known limitations of surrogate endpoints in the pre-market approval process and without addressing the fact that FDA’s post-market surveillance requirements are insufficiently enforced.
Many questions remain. Is Congress fueling the fire of uncertainty and increasing risk by expanding the ability of patients to receive treatment with products that contain risk-benefit profiles that lack meaningful information on clinical outcomes? Will the new law set the stage for innovations that reinvigorate patent portfolios and help industry shift away from “me-too” blockbusters? Will health outcomes improve? What impact on the cost and quality of care?
Through their votes and health care dollars, patients hold significant power to influence the relationship between lawmakers, FDA, and Big Pharma. Patients should use their authority to question, or refuse, medical products that lack meaningful data, and should demand that elected officials establish a legal framework that facilitates and incentivizes robust and accurate risk-benefit disclosures. A failure to do so may prove costly.