The dean of Harvard Medical School, Jeffrey Flier, has an interesting piece in the Boston Globe about drug prices. He writes:
Procedures required by the FDA for development and approval of new products need to be thoroughly redesigned, including new approaches to adaptive design of clinical trials and use of biomarkers to provide earlier evidence of efficacy than available through mortality-based metrics. Thresholds to clear a drug through the FDA process should more readily consider both medical need and the severity of the disease being treated. Though many regulatory hurdles were established to guarantee safety, patients with otherwise untreatable diseases may be willing to accept an uncertain risk for the chance of benefit, and should be permitted to do so.
More important, many steps in the highly regulated development process, however originally justified, lack scientific rationale, drive up costs, and could be safely eliminated. One example: human small-scale tests of a new molecule must have the drug prepared by elaborate manufacturing procedures required for a commercial drug in full distribution, well beyond the scientific standards necessary to insure that a particular research batch of a drug is pure. This difference can literally cost millions of dollars for even the first testing, providing no significant benefit in safety. However straightforward, this reform has been difficult to achieve.
