Accelerated Approval of Oncology Drugs: Can We Do Better?

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The modification of Food and Drug Administration (FDA) investigational new drug regulations to provide for “accelerated approval” of promising new drugs was put in place in 1992 ( 1 ), motivated largely by the emergence of HIV/AIDS. The increasing rigor of FDA review, beginning with the 1962 passage of the Kefauver–Harris amendments to the Federal Food, Drug and Cosmetic Act, and the resulting changes to the FDA regulations regarding evaluation of investigational drugs, were perceived as intolerable obstacles to making potentially life-saving therapy available in the most rapid way possible to those suffering from a deadly disease with very limited therapeutic options. Accelerated approval was to be based on positive results for surrogate endpoints that were considered likely predictors of clinical benefit, as typically measured for oncological drugs by an improvement in survival. The application of this less restrictive pathway to drug approval was quickly used in other medical areas; among the first eight drugs to receive accelerated approval, four were for HIV/AIDS indications, two were for cancer treatment (one was for Kaposi sarcoma, an AIDS-related malignancy), one was for multiple sclerosis, and one was for bacterial infection.