Surrogate Endpoints in Aids Drug Development: Current Status

During the past several years, significant efforts have been devoted to searching for surrogate endpoints to evaluate AIDS treatments. Efforts were made in the form of research sponsored by the government and trials conducted by the pharmaceutical industry. Rapidly changing treatment strategies, low incidences of clinical endpoints due to prophylactic drug usage for AIDS-defining illness, and poor patient compliance have made long-term large trials to evaluate the clinical benefits of AIDS drugs impossible. The latter has intensified the need for surrogate endpoints. This paper shares with readers activities that have taken place in the search process and what medical, statistical, regulatory, and patient advocacy groups as a whole have accomplished so far. Some results from the collaborative effort to validate CD4 count and HTV-1 viral load as surrogates for the clinical endpoints will be presented using data from AIDS clinical trials. The ultimate question this paper seeks to answer is: Are researchers likely to be misled in their pursuit of surrogate endpoints for AIDS trials?