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

Periodic Reporting for period 3 - PREFER (Patient Preferences in benefit risk assessments during the drug life cycle - Sofia ref.: 115966)

Reporting period: 2018-10-01 to 2019-09-30

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 aims 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. Two scientific articles demonstrating the overall work of PREFER have been published and six publications so far provide reports of results during the first phase 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 stake holder 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 are underway and have provided results from the first set of explorative preference studies related to Rheumatoid Arthritis (in both UK and Germany), Neuromuscular disorders (in UK as well as gathering data from a large international network of clinical sites) and Lung Cancer (in Italy and Belgium). They will gather data on the use of Discrete Choice Experiments (DCE) as the prioritized methodology with comparisons of a number of other methods for the elicitation of patient preferences. Three additional industry led case studies have already presented interesting results both from explorative studies and quantitative based methods. Five additional case studies were selected after the same thorough review process from academic partners. Together, these clinical case studies will be able to respond to a great number of the PREFER methodological research questions identified in the first phase of the project and preparations are already in place for integrating the results in the final recommendations.

Based on recommendations by EMA and the Scientific Advisory Board, PREFER has decided to consider an EMA Methods Qualification Process that is conducted in the framework of scientific advice as a means to further focus and to maximize the impact of PREFER. Other stakeholders such as HTA bodies will be included through joint scientific advice. The steering committee agreed that Discrete Choice Experiments (DCE) will be used as the first method of choice for the qualification process and for the empirical and simulation case studies based on the rich set of evidence and experience that is readily available for this method. It is expected that several of the identified high priority methodological and non-methodological questions need to be addressed for a successful application for qualification. Providing clear guidance as to how DCE and other methods can be used in any appropriate development program this will provide the proof of concept that patient preference methods can be qualified as well as a blueprint for the qualification of further methods by other applicants. The qualification procedure will include Health and Technology Assessment concerns and represents in fact the first joint EMA & EUnetHTA qualification procedure, thus signifying still another important step towards attaining real impact of the PREFER project. A special Task Force was set up and a Briefing Book presenting the scientific
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 is 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 is also 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.
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