TBD: Patient preferences in regulatory decision-making: Findings and recommendations from IMI PREFER
Postdoctoral Researcher, Patient Experience Data & Patient Preference Studies
KU Leuven Belgium
To summarize PREFER outcomes and recommendations regarding: i) the value and applications for patient preference studies (PPS) in regulatory evaluation, ii) how PPS could be reflected in regulatory documents and external communication, and iii) remaining questions for PPS use in regulatory context.
PREFER used a mixed-methods research consisting of literature reviews, individual interviews, and focus group discussions with patients, patient organisations, regulators, industry representatives, industry, HTA bodies and payers, and clinicians, in the EU and US, and between Oct 2016 and May 2022.
With respect to PPS’ value and application in regulatory evaluation, prior to clinical trials, PPS could inform regulators’ understanding of patients’ unmet needs and relevant endpoints (e.g., in horizon scanning activities and scientific advice). During the evaluation of a marketing authorisation application, PPS could inform: i) the assessment of whether a product meets an unmet need, ii) whether patient-relevant clinical trial endpoints and outcomes were studied, iii) the understanding?of patient-relevant effect sizes and acceptable trade-offs, and iv) the identification of key (un-)favorable effects and?uncertainties. In post-marketing assessments, PPS could inform re-assessment of product performance on patient-relevant endpoints and the regulatory decision about continued approval. With respect to external PPS communication, PPS could be reflected in the assessment report and product information (e.g., the European Public Assessment Report (EPAR) and the Summary of Product Characteristics (SmPC) in the EU). With respect to remaining questions for PPS, key questions include: i) designing/using product independent PPS vs industry PPS approach which is typically more product focused, whereas the PREFER, patient groups and others’ PPS are typically more broadly focused, ii) who should be financing, designing, conducting and coordinating PPS including data governance (industry, academia, patient organisations, multi-stakeholder efforts, public private partnerships), iii) uncertainties among stakeholders conducting PPS about the acceptance of PPS results by regulators, iv) when (within or outside clinical trials) to perform PPS, v) root causes for the regulators’ lack of trust in (or hesitance about) the field and methodological aspects such as regarding educational materials, attribute selection, and representativity of the PPS sample and vi) how PPS can be concretely applied/operationalized in this regard; aligning regulatory decisions with patients' preferences.
Findings from PREFER highlight the value of PPS in informing regulators and other stakeholders (patient organisations, medical product developers, HTA bodies) on the unmet needs, clinical trial endpoints and outcomes that matter most to patients, and the trade-offs patients are willing to make.?Insights and recommendations aim to foster targeted multi-stakeholder discussions, further research efforts following the PREFER projects’ trajectory and internationally accepted recommendations and guidelines (such as those by the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) respectively), joint horizon scanning activities, and (parallel) scientific advice on preference study use. Future efforts need to focus on building case examples for PPS design, use and communication in regulatory evaluation.