Value-Added Decisions in Oncology

Am Soc Clin Oncol Educ Book. 2019 Jan:39:122-131. doi: 10.1200/EDBK_238831. Epub 2019 May 17.

Abstract

Registration of new anticancer drugs is decided too often not by their clinical value but by a p value. Approval is granted if the difference in an acceptable time-to-event outcome measure differs between experimental and control arms of a randomized controlled trial, such that the null hypothesis can be rejected based on a statistical test that meets the arbitrary criterion of p < .05. However, as stated by the American Statistical Association, a p value does not measure the size of an effect or the importance of a result; it does not provide a good measure of evidence related to a hypothesis, and policy decisions should not be made on the basis of whether a p value passes a specific threshold. Unfortunately, this statement is ignored by most journals, which emphasize p values in reporting results of clinical trials, and by regulatory agencies, such as the U.S. Food and Drug Administration and the European Medicines Agency; a significant p value is often a necessary and sufficient criterion for granting marketing approval. As a result, pharmaceutical companies often design large trials to increase the probability that a small difference in the primary outcome measure will be "significant." Moreover, the market price set for such drugs bears no relationship to the level of their benefit; drugs with small effects on outcome are sold at roughly the same price as "good drugs" that convey substantial benefit.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Clinical Decision-Making*
  • Clinical Trials as Topic
  • Delivery of Health Care
  • Disease Management
  • Humans
  • Medical Oncology* / methods
  • Multimodal Imaging
  • Neoplasm Staging
  • Neoplasms / diagnosis
  • Neoplasms / epidemiology
  • Neoplasms / mortality
  • Neoplasms / therapy
  • Public Health Surveillance

Substances

  • Antineoplastic Agents