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Patient Preferences in benefit risk assessments during the drug life cycle - Sofia ref.: 115966

Periodic Reporting for period 5 - PREFER (Patient Preferences in benefit risk assessments during the drug life cycle - Sofia ref.: 115966)

Reporting period: 2020-10-01 to 2022-05-31

As of today, there is no generally accepted set of guidelines or recommendations on when and how to elicit patient preference information that is ready for medical decision making. As one of the most highly ranked requirements that was shared by decision makers in the IMI PREFER interviews was that preference studies should have a strong evidentiary standard so that they could be trusted to reflect preferences of the intended population. Patient preferences are currently not systematically considered for decision making to the full extend in drug development / patient access. By developing expert and evidence-based recommendations, PREFER aimed to guide industry, regulatory authorities, HTA bodies and reimbursement agencies on how patient preferences can be assessed and used to inform medical product decision making. This will ultimately strengthen patients’ voices and input into drug development throughout the entire medical product lifecycle.
The first phase of PREFER has been focused on the elicitation of stakeholders concerns and priorities, the identification of medical decision points across the medical products life cycle, the examination and prioritization of patient preference methods and methodological research questions. 33 preference exploration and elicitation methods were characterized and appraised. 56 instruments for measuring 29 psychological constructs were identified and assessed with regard to how they may explain patient’s preferences heterogeneity and psychological factors affecting preference formation. 33 publications so far provide reports of the project. In addition, PREFER has contributed at several international scientific conferences with organisations such as IAHPR, ISPOR, ECQI, HTAi, SMDM and DIA.

Based on discussions with the Scientific Advisory Group and the different stakeholder groups associated with PREFER (Regulators, HTA, Patients) a focused strategy identifying a selected number of methods and research questions that may lead to effective recommendations has been adopted. The second phase of PREFER aimed at evaluating preference methods and research questions in clinical context where there is a preference sensitive decision to be made, i.e. one where information about patient preferences may provide an added value to regulators, HTA and industry when making their decisions. Three core clinical case studies have provided results which informed the PREFER Recommendations. Data have been gathered on the use of Discrete Choice Experiments (DCE), Best-Worst Scaling, Threshold techniques, and Swing-Weighting with comparisons of a number of other methods for the elicitation of patient preferences. Four additional industry led case studies have presented interesting results both from explorative studies and quantitative based methods. Four additional case studies have been conducted from academic partners. 10 of these 11 case studies have delivered their final reports. One industry led case study will soon be finalised. One case study on eye-tracking could not be carried through due to the COVID-19 situation that has prevented meetings both with patients and health care professionals. These clinical case studies responded to a great number of the PREFER methodological research questions identified in the first phase of the project and results have been integrated in the PREFER recommendations and in the Qualification Opinion process that PREFER has initiated jointly with EMA and EUnetHTA.

While PREFER focussed on scientific questions, operational aspects are equally important for the credibility of patient preference studies. To that end, PREFER developed an operational best practice document that got informed by stakeholder needs and learnings from case studies. A set of training materials have been published to inform researchers on how to conduct patient preference studies.
Health preference studies have been performed both by the pharmaceutical industry and by academic institutions for different disease areas. However, this is the first time that an assessment of the methods used was made based on a comprehensive mapping of concerns and needs of all relevant stakeholders as well as on systematic and scoping reviews of the relevant literature. Of equal significance is the systematic and focused approach to evaluate the performance of these methods in real clinical cases. The EMA & EUnetHTA Qualification procedure was the first attempt within the field of health preference studies to set the standards for a preference study that is expected to bring added value to medical decision making and actually provide impact for patient concerns and needs.
A positive qualification opinion was provided by EMA (CHMP) on the Framework and Points to Consider for Methods Selection: https://www.ema.europa.eu/documents/regulatory-procedural-guideline/qualification-opinion-imi-prefer_en.pdf

The project has generated recommendations which provide scientifically based insights from six years of research on when and how to design and conduct a patient preference study. The PREFER recommendation are accessible under this link: https://doi.org/10.5281/zenodo.6470922

In addition a set of 11 templates for patient preference studies were set up by the project and are freely available here: https://doi.org/10.5281/zenodo.6400552

To train researchers and HTA and regulatory experts on patient preferences 16 webinars have been recorded and published. For most recordings, the links to the slides can be found in the description of the videos. Watch them here:
https://www.youtube.com/playlist?list=PLN-kgkHBHa25apivf7wIwqNv5HiVX_VCZ or https://doi.org/10.5281/zenodo.6397608
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