How can patient preferences be used and communicated in the regulatory evaluation of medicinal products? Findings and recommendations from IMI PREFER and call to action

• DOI: 10.3389/fphar.2023.1192770
• Publication Date: 2023-08-17T13:33:03Z
• Authors: Rosanne Janssens; Liese Barbier; Mireille Muller; Irina Cleemput; Isabelle Stoeckert; Chiara Whichello; Bennett Levitan; Tarek A. Hammad; Charis Girvalaki; Juan-Jose Ventura; Karin Schölin Bywall; Cathy Anne Pinto; Elise Schoefs; Eva G. Katz; Ulrik Kihlbom; Isabelle Huys
• Keywords: Regulatory Science; Marketing authorization; Regulatory focus theory

Objective: Patients have unique insights and are (in-)directly affected by each decision taken throughout the life cycle of medicinal products. Patient preference studies (PPS) assess what matters most to patients, how much, trade-offs patients willing make. IMI PREFER was a six-year European public-private partnership under Innovative Medicines Initiative that developed recommendations on use PPS in medical product decision-making, including regulatory evaluation This paper aims summarize findings from regarding i) applications evaluation, ii) when consult with regulators PPS, iii) reflect communication iv) barriers open questions for decision-making. Methods: performed six literature reviews, 143 interviews eight focus group discussions regulators, patient representatives, industry Health Technology Assessment bodies, payers, academics, clincians between October 2016 May 2022. Results: With respect applications, prior conduct clinical trials products, could inform regulators' understanding patients' unmet needs relevant endpoints during horizon scanning activities scientific advice. During marketing authorization application, inform: a) assessment whether meets an need, b) patient-relevant trial outcomes were studied, c) effect sizes acceptable trade-offs, d) identification key (un-)favorable effects uncertainties. consulting researchers should ideally early (e.g., advice) design research questions. Regarding external communication, be reflected report information Public Report Summary Product Characteristics). Barriers include lack cases demonstrated impact need (financial) incentives, guidance quality criteria implementing results Open concerning include: independent broad approach and/or product-specific one, who optimally financing, designing, conducting, coordinating (within outside trials) perform can best operationalized decisions. Conclusion: high potential needs, endpoints, benefits, risks matter their trade-offs. Regulatory guidelines, templates checklists, together incentives needed foster structural transparent submission More case conducted submitted enable discussion increase experience implementation evaluations.