Desperate Regulation for Desperate Cancer Patients: The Unmet Needs of Accelerated Drug Approval

Abstract

As we explained in Chapter 1, the regular process of drug development (as occurred with the glitazones in Chapter 3) includes three sequential phases of clinical trials before government regulatory agencies consider granting marketing approval for new drugs. Specifically, under the regular drug regulatory process, the EMEA and the FDA have required at least two ‘pivotal’ phase III randomized controlled clinical trials (RCTs) to demonstrate new drug efficacy before marketing approval. Often that involves trials designed to provide evidence of direct efficacy of the drug on clinical symptoms or survival. However, as we saw in Chapter 3 regarding diabetes drugs, sometimes regular marketing approval is based on a new drug’s effectiveness on an ‘established’ surrogate measure of clinical efficacy, that is, a laboratory or physical measure, which is accepted by regulatory agencies to reasonably substitute for clinical efficacy. For example, as discussed in Chapter 3, in the case of diabetes drugs, regulatory agencies have long accepted (perhaps too readily and wrongly) that a drug’s capacity to control blood-glucose is a valid predictor, and hence surrogate marker, of clinical efficacy to reduce the micro- and macrovascular effects of diabetes.