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What's Preventing Widespread Use of Patient-Reported Outcomes in Drug Development?

Jaime Rosenberg
Patient-reported outcomes (PROs) have the potential to enhance drug development, regulatory decisions, and shared decision making, and the benefits of using PROs are broadly agreed upon, but there are a variety of challenges facing stakeholders.
Traditionally, clinical end points such as progression-free survival and adverse events have served as clinically meaningful outcomes when guiding clinical and regulatory decision making. However, the US healthcare system continues to see a shift toward patient-centered care, where perspectives from patients and their caregivers play a more important role in these decisions. The hope is that patient-reported outcomes (PROs) will be integrated into clinical trial designs and, subsequently, help guide care and coverage decisions.

But despite the excitement behind these types of measures, integration has been slow, and PROs may not be considered in the beginning steps of the development process. There is also variability in PRO measure use and a lack of standard practice, preventing more widespread use of the measures.

“While there is a push to include PRO measures as end points in phase 3 clinical trials for drug approval, integration has been slow,” Somali Burgess, PhD, senior director, Xcenda, told The American Journal of Managed Care® (AJMC®).

Also commenting on the role of PROs in the drug development process, Theresa Mullin, PhD, associate director for strategic initiatives for the Center for Drug Evaluation and Research at the FDA, wrote in an email to AJMC®, “In the past, these types of outcomes measures have played an important role, but a more limited role and may not be considered until late in the drug development program. In addition, it was not uncommon for PRO measures to be developed with very little input or involvement of patients living with the disease of interest.”

PROs have the potential to enhance drug development, regulatory decisions, and shared decision making, and the benefits of using PROs are broadly agreed upon, but there are a variety of challenges facing stakeholders, explained Burgess. These include:
  • Validation of PRO measures requires a representative patient population, which may not be fully defined in the early stages of clinical development
  • Variability and subjectivity of patient experience and perception may make measuring a representative sample difficult
  • Too many questions or providing a survey too often can lead to response fatigue
  • Customized or disease-specific PRO measures may be more sensitive to the characteristics of a patient’s journey, but these measures often do not have the same level of validation of more global PRO measures.
In recent years, organizations nationwide have put forward tools and efforts to try and mitigate these challenges and facilitate widespread implementation of PRO measures. Last October, the Academy of Managed Care Pharmacy convened a forum1 of key stakeholders representing patients, payers, providers, government agencies, and pharmaceutical companies to discuss and identify solutions to the current challenges and barriers impeding further use of PROs.

In February, a study published in JAMA2 offered recommendations for factors that should be accounted for in clinical trial protocols in which PROs are a primary or key secondary outcome. The review identified 16 PRO-specific items relating to trial rationale, eligibility criteria, PRO instrument selection and measurement properties, and data collection plans.


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