Patient preference priorities: Questions to answer for patient-centric decision-making


Patient preference assessment is an increasingly popular approach to engaging patients in decisions across the medical product life cycle. But questions remain about how to incorporate scientifically valid preference measurements into decision-making.  Especially for decisions about medical treatment, including development, regulatory and reimbursement decisions. PREFER partners have identified questions related to the knowledge gaps that are most crucial to decision-making stakeholders. 

PREFER aims to strengthen patient-centric decision-making across the medical product life cycle. From pre-discovery to post-marketing. To get there, the project is developing evidence-based recommendations to fill knowledge gaps in the methodology of patient preference studies. So far, they have been mapping the field to outline research needs, and recently launched a series of clinical case studies where some of the most critical questions will be answered.

Important knowledge gaps identified by the project include the themes reliability and validity of preference study outcomes, generisability and transferability of results, and the impact of educational materials on the results. These themes, in turn, come with a series of questions to answer. 17 questions were identified as having the highest priority. For example, ‘how do different preference assessment techniques differ from one another?’ and ‘how do patient characteristics relate to patient preferences?.  

Currently, the PREFER project is working to answer some of these high-priority questions in a series of empirical case studies and simulation studies. Three of these case studies are being conducted in pre-specified patient groups: Rheumatoid Arthritis, Neuromuscular Disorders and Lung Cancer. Nine additional academic and industry led case studies are being conducted across a wide range of different disease and treatment contexts.

“With our clinical case studies, we hope to answer some of the questions that remain unanswered. But we are especially focusing on the issues that can only be resolved by a large public-private partnership like ours” says Ian Smith, one of the authors of a recent commentary published in The Patient.

But not all of the questions will be answered within the project. PREFER researchers are also encouraging others to help answer the questions that they can’t. The knowledge accumulated within the project will need to be complemented by additional work. By answering these questions together, we will be several steps closer to the patient-centric decision-making that stakeholders are after looking for.

By Anna Holm

Smith IP, DiSantostefano R, et al, Methodological Priorities for Patient Preferences Research: Stakeholder Input to the PREFER Public–Private Project, Patient, 15 March 2021

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Last modified: 2021-11-10