This article is more than 5 years old. Information may no longer be current.
Patient-reported outcomes may not be appropriate primary, secondary endpoints in every trial
Click Here to Manage Email Alerts
Patient-reported outcomes (PROs) comprise measures that reflect patient perspectives and evaluations, including assessments of constructs such as quality of life, symptoms and experiences with care.
Considerable evidence demonstrates that PRO ratings from patients and observers, including clinicians, are not equivalent, and that the patient’s rating provides distinct information that can only come from the experience of undergoing cancer therapies.
PROs can play a crucial role in clinical trials. For example, PROs can provide information to distinguish between cancer therapies and define the standard of care. PROs also can identify the impact of therapies and guide supportive care and treatment modifications, or assure clinicians and decision-makers that an efficacious treatment does not have a negative impact on patient well-being.
Further, PROs can identify unanticipated symptoms and side effects that can be addressed in care or future research. PROs also have been shown to predict survival and may provide a basis for eligibility assessment and stratification that is more precise than traditional clinician-rated performance status criteria, such as the Karnofsky or ECOG scales.
Advances in questionnaire design — such as NIH’s Patient Reported Outcomes Measurement Information System (PROMIS) — and the emergence of electronic platforms for PRO data collection and monitoring have the potential to mitigate some longstanding challenges in PRO research, such as patient burden, missing data and the high cost of quality control.
All of these benefits, in an era when “patient-centered care” has become virtually a mantra, would seem to imply that the answer to the question of whether PROS should be required in clinical trials has to be “yes.”
However, even given all their positive contributions, PROs may not be appropriate primary or secondary endpoints in every trial. Only a small number of carefully selected endpoints can be measured in any given trial; if PRO outcomes would not provide information that affects trial interpretation, then including these measures may not be warranted. For example, for treatments where symptoms and side effects are well known and are not expected to differ across arms, PROs may not add value.
The Canadian Cancer Trials Group (CCTG) has long had a policy that states, “there should be a statement about the anticipated impact on quality of life with every proposed phase 3 clinical trial and whether quality-of-life measures will be incorporated in the protocol.” In practice, this policy has meant that PROs are carefully considered in trial design, and most CCTG phase 3 trials do include PROs.
Thoughtful discussion about the contributions of PROs and how they should be assessed in a given trial is preferable to a blanket requirement.
Reference:
Osoba D, et al. Value Health. 2007;10:S138-S145.
For more information:
Carolyn Gotay, PhD, is professor and Canadian Cancer Society chair in cancer primary prevention at University of British Columbia in Vancouver. She can be reached at carolyn.gotay@ubc.ca.
Disclosure: Gotay reports no relevant financial disclosures.