Health-Related Quality of Life Data in Cancer Clinical Trials for Drug Registration: The Value Beyond Reimbursement

JCO Clin Cancer Inform. 2021 Jan;5:112-124. doi: 10.1200/CCI.20.00100.


Purpose: A review of the literature was performed to evaluate how quality of life measures are collected, analyzed, and reported in cancer clinical trials intended to support drug registration.Health-related quality of life (HRQoL) data points are one of the patient-reported outcome (PRO) assessments used in clinical trials to evaluate the effects of treatments from the patient perspective. The use of PROs has gained focus in cancer clinical trials as more options become available for greater longevity of patients on treatment. Standardization of PRO data is evolving and involves unique challenges when used for assessing biologic and chemotherapeutic agents for the treatment of cancer.

Methods: In this study, a review of literature published between 2009 and 2019 was conducted using PubMed, COCHRANE Library, and Medline. The research focus was on the current guidance, implementation, and reporting as well as highlighting the issues, and recommendations for the inclusion of HRQoL end points in cancer clinical trials intended for use in drug registration.

Results: Although there exist many levels of guidance for HRQoL measures in cancer drug trials, challenges to operational implementation, the current inconsistent adherence to reporting standards, and the lack of consensus and understanding of analyses limit the value and potential of the resulting data collected.

Conclusion: The results of HRQoL data collected from cancer clinical trials can be difficult to interpret and apply to inform clinical decision making. Increased reporting and access to these data can provide opportunities for potential applications to improve translatability of HRQoL data collected in clinical trials into practice.

Publication types

  • Review

MeSH terms

  • Clinical Trials as Topic
  • Humans
  • Neoplasms*
  • Patient Outcome Assessment
  • Patient Reported Outcome Measures
  • Pharmaceutical Preparations*
  • Quality of Life


  • Pharmaceutical Preparations