The Problem: The Child Health and Human Development Institute of the National Institutes of Health sought to increase the level of R&D in medicines for children. At the time, most medications had not yet been tested in children and as a result, children were treated “off Label.” It was imperative to improve pediatric pharmacologic research.
Our Engagement: We were engaged to develop a response to the lack of FDA-approved drugs to ensure better pharmaceutical care for children.
Our Actions: We identified and analyzed the variety of scientific, technical, economic, regulatory, legal and ethical reasons why children were not included in clinical studies. We also assessed all of the issues involved in pediatric research and reviewed the state of product development, prescribing, compliance issues and pediatric pharmaceutical care programs.
We inventoried the capacity of the Pediatric Pharmacology Research Units (PPRU) of the Child Health and Human Development, Maternal and Child Health Branch, identifying their extensive technical capabilities to do sophisticated pediatric clinical trials, comparing them to contract research organizations and making recommendations to accelerate research efforts.
Our analysis concluded with a recommendation to the FDA and the NIH that the agencies should support an “orphan drug” style legislative plan to grant market exclusivity for companies conducting clinical trials to determine the safety and efficacy of drugs in children. The agencies agreed and the Clinton Administration proposed the idea in its legislative proposal to Congress. In 1997, Congress passed the Food and Drug Administration Modernization Ac adding a new provision, Section 505A (21 U.S.C. 355a). That section provided six months of pediatric exclusivity would be added to previously earned marketing exclusivity and listed patent protection for certain drugs if the sponsoring company conducted studies requested by FDA.