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TLON..$.93 2008 review from Richard Pazdur on Endpoints for Assessing Drug Activity in Clinical Trials in the Oncologist. http://bit.ly/xcoDlQ. Key endpoints are discussed that are important to current & future trials.

"In rare diseases where randomized trials cannot be
performed because of very limited populations, the FDA
has approved indications on the basis of single-arm studies
supported by data from the published literature."


Endpoints for assessing drug activity in clinical trials.
http://www.ncbi.nlm.nih.gov/pubmed/18434634
Pazdur R.
SourceCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Rockville, Maryland 20825, USA. [e-mail]

Abstract
Overall survival remains the gold standard for the demonstration of clinical benefit. An improvement in overall survival is a direct clinical benefit to patients. An analysis of overall survival requires larger patient numbers and longer follow-up than other endpoints. Survival analysis may be confounded by subsequent therapies. Time to progression usually requires smaller clinical trials and may be more rapidly assessed than trials using overall survival as an endpoint. This endpoint is not confounded by subsequent therapies. Time to progression must use the same evaluation techniques and schedules for all therapies being evaluated. Blinding of trials or the use of an external blinded radiographic review committee is recommended in assessing time to progression. Unlike overall survival and time to progression, which must be evaluated in randomized trials, response rates can be accurately assessed using a single-arm trial. Stable disease is not included in a response rate determination and is optimally evaluated by assessing tumor progression in a randomized trial. Improvement in disease-related symptoms is considered clinical benefit and may be an appropriate endpoint for drug approval.