An analysis commissioned by Public Health Law Research has found that laws aimed at spurring development of new drugs have also led to unintended public health problems.
Aaron Kesselheim, M.D., J.D. of the Brigham and Women's Hospital in Boston, published a commentary, Using Market-Exclusivity Incentives to Promote Pharmaceutical Innovation, today in the New England Journal of Medicine.
The number of new drugs being approved by the FDA is at a low point, and in response, policymakers have suggested the strategy of providing longer patents or stronger market exclusivity for patent holders as an incentive for drug development. Kesselheim, reviews five notable occasions in the past where laws using this strategy have been passed that gave pharmaceutical companies incentives to invest in costly research and development. For example, the Orphan Drug Act, passed in 1983, offered enhanced market exclusivity for newly approved drugs that treat rare diseases.
Kesselheim’s analysis of these past cases finds a mix of both positive and negative outcomes, including some important public health risks. For example:
- The Orphan Drug Act was associated with an upswing in development of drugs for rare diseases, but was also applied to numerous drugs that were used beyond their rare disease indication in non-evidence-based ways.
- The Pediatric Exclusivity Extension (which provided 6 months of market exclusivity in exchange for new pediatric drug trials) encouraged manufacturers to study their drugs in children, an underserved population. But credible evidence has also emerged that the exclusivity extension has been earned for trials that were subpar and performed on drugs with little clinical applicability to children.
- The Prescription Drug User Fee Act (which created a system where drug companies paid user fees to the FDA to support review of their drugs) helped speed FDA regulatory review times, but the imposition of arbitrary accelerated deadlines have also been linked to approval by the FDA of drugs later found to have safety problems.
Any future legislative efforts that use market exclusivity to boost drug development should be crafted in a more precise manner to avoid misuse and subject to official cost-effectiveness analysis at regular intervals, says Kesselheim.