PCP22 - Core Outcome Sets in Clinical Research: What are They and How Can they Help Decision Makers?

Abstract

Variability in outcomes reported in clinical studies for the same condition is widespread. This limits our ability to consistently gather the most patient-relevant outcomes and assure their use by regulators and payers, hinders manufacturers from developing evidence most relevant to patient-centered assessments of value, hampers payers’ abilities to assess comparative effectiveness and value, and complicates the ability of reviewers to aggregate and analyze bodies of related evidence. Development and use of core outcome sets (COS) can address these challenges. A COS is an agreed-upon minimum set of outcomes that should be measured and reported in all clinical studies for related evaluations of a specific condition. Greater adoption of stakeholder-informed COS in clinical research can facilitate harmonization of relevant outcomes across multiple decision-making authorities. To identify ways to facilitate adoption of well-developed COS in clinical research. We convened an expert steering group and conducted key informant interviews with medical policy and research leaders able to speak to barriers and facilitators of COS adoption. With their input, we held an in-person multi-stakeholder meeting to agree on potential benefits of COS to decision-makers, discuss challenges to adoption, and brainstorm possible solutions. Stakeholders described challenges to decision-making due to inconsistent and inappropriate outcomes reported by researchers. The group agreed that promoting adoption of well-developed COS helps achieve more informed, patient-centered decision-making. Several challenges must first be addressed to make broad COS adoption more feasible and attractive, including: 1) lack of awareness of COS; 2) growing number of similar but siloed initiatives; 3) lack of incentives from key influencers in current healthcare environment; 4) trust in COS development methodology; and 5) complexity of outcomes measurement. Examples of potential solutions include: targeted COS awareness campaign, “mapping” the environment of relevant but siloed initiatives, and forming a network of stakeholder organizations.