Modernizing Eligibility Criteria for Molecularly Driven Trials

J Clin Oncol. 2015 Sep 1;33(25):2815-20. doi: 10.1200/JCO.2015.62.1854. Epub 2015 Jul 20.

Abstract

As more clinical trials of molecularly targeted agents evolve, the number of eligibility criteria seems to be increasing. The importance and utility of eligibility criteria must be considered in the context of the fundamental goal of a clinical trial: to understand the risks and benefits of a treatment in the intended-use patient population. Although eligibility criteria are necessary to define the population under study and conduct trials safely, excessive requirements may severely restrict the population available for study, and often, this population is not reflective of the general population for which the drug would be prescribed. The American Society of Clinical Oncology Cancer Research Committee, which comprises academic faculty, industry representatives, and patient advocates, evaluated this issue. Evaluation results were mixed. Most physicians agreed that excessive eligibility criterias slow study enrollment rates and prolong the duration of enrollment; however, this hypothesis was difficult to validate with the data examined. We propose the organization of a public workshop, with input from regulatory bodies and key stakeholders, with the goal of developing an algorithmic approach to determining eligibility criteria for individual study protocols, which may help guide future investigators and companies in streamlining eligibility criteria in the era of molecularly driven therapy.

MeSH terms

  • Algorithms
  • Antineoplastic Agents / pharmacology*
  • Biomarkers, Tumor* / genetics
  • Biomarkers, Tumor* / metabolism
  • Clinical Trials as Topic* / methods
  • Clinical Trials as Topic* / standards
  • Clinical Trials as Topic* / trends
  • Humans
  • Medical Oncology / trends*
  • Molecular Targeted Therapy*
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Neoplasms / metabolism
  • Patient Selection*
  • Treatment Outcome

Substances

  • Antineoplastic Agents
  • Biomarkers, Tumor