Clinical Endpoints: What Do Payers And Regulators Want?

By Jack Modell, M.D. and David Shoemaker, Ph.D.

The requirement that new drugs show effectiveness is based on a 1962 amendment to the Federal Food, Drug, and Cosmetic Act. This law requires substantial evidence of effectiveness and specifies that this evidence must be derived from adequate and well-controlled clinical investigations. Clinical benefits that have supported drug approval have included important clinical outcomes – for example, increased survival or symptomatic improvement, but have also included effects on established surrogate endpoints – e.g., blood pressure, tumor markers, and serum cholesterol.

The question that naturally arises is how clinical endpoints should be chosen to optimize the chances for both regulatory
and reimbursement success. Fortunately, the answer is largely the same for both: