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What's Preventing Widespread Use of Patient-Reported Outcomes in Drug Development?
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.
Despite efforts like these, major barriers still exist, most notably the absence of consistent standards and guidance documents for measuring and utilizing PROs. “For this information to be collected in a more consistent and reliable way, stakeholders need clear guidance on the methods to use and access to information on what measures have already used and accepted,” explained Mullin.
Seeking to fill this gap and incorporate PROs early in the development process, the FDA is creating a dialogue among stakeholders to identify appropriate measures and standards. Noting the agency's broad look at patient experiences, Millen explained that the FDA takes into acocunt various patient perspectives, rather than PROs alone.
“When considering a measurement approach, sponsors and others are encouraged to consider the type of clinical outcome assessment (COA) measure that might be most appropriate,” she said. These COAs can be obtained from a clinician, patient, or a nonclinician observer, or through a performance-based assessment, and include 4 types: PROs, clinical-reported outcome measures, observer-reported outcomes, and performance outcomes.
Today, the agency finished up a 2-day workshop of various stakeholders to address a series of 4 methodological patient-focused drug development documents3 that the agency is developing to provide guidance, in a stepwise fashion, for how stakeholders can collect and submit patient experiences and other relevant information from patients and caregivers. The goal is to facilitate the enhancement and use of systematic approaches to use collected patient and caregiver input to better inform both product development and regulatory decision making.
During the workshop, stakeholders weighed in on methods to identify what’s important to patients in regard to burden of disease, burden of treatment, and the benefits and risks in management of the patient’s disease; best practices for collecting information on what aspects of the disease symptoms, impact, and other issues are important to patients; measuring these symptoms, impacts, and other issues in a meaningful way; and selecting, developing, or modifying fit-for-purpose COAs to measure the patient experience in clinical trials.
Additionally, this summer, the FDA issued a Request for Information4 to inform planned future work to promote the development of publicly available standard core sets of COA measures for specific disease indications. These core sets would include measures, tools, and end points to assess a minimum list of impacts that matter most to patients and are likely to demonstrate change related to disease burden, treatment burden, and if applicable, physical function.
1. AMCP Partnership Forum: improving quality, value, and outcomes with patient-reported outcomes [published March 2018]. J Manag Care Spec Pharm. doi: 10.18553/jmcp.2018.17491.
2. Calvert M, Kyte D, Maercieca-Bebber, R, et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols [published February 6, 2018]. JAMA. doi: 10.1001/jama.2017.21903.
3. FDA patient-focused drug development guidance series for enhancing the incorporation of the patient’s voice in medical product development and regulatory decision making. FDA website. www.fda.gov/drugs/developmentapprovalprocess/ucm610279.htm. Published June 29, 2018. Accessed October 15, 2018.
4. FDA standard core clinical outcome assessments and endpoints. National Institutes of Health website. grants.nih.gov/grants/guide/notice-files/NOT-FD-18-014.html. Published July 31, 2018. Accessed October 15, 2018.