EMA/EUnetHTA Qualification of the IMI PREFER Patient Preference Framework and Points to Consider for Method Selection: Key Experiences, Outcomes, Value and Implications
Author(s)
Barbier L1, Muller M2, Cleemput I3, Strammiello V4, Dickinson S2, Kübler J5, Bullok K6, Levitan B7, Hauber B8, Steinbeisser C9, Stoeckert I10, Noel B6, Huys I1, Janssens R1
1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, VBR, Belgium, 2Novartis Pharma AG, Basel, Switzerland, 3Belgian Health Care Knowledge Centre (KCE), Brussels, Belgium, 4European Patients’ Forum, Brussels, Belgium, 5Quantitative Scientific Consulting, Marburg, Germany, 6Eli Lilly and Company, Indianapolis, IN, USA, 7Janssen Research & Development, LLC, Titusville, NJ, USA, 8Pfizer, New York, NY, USA, 9Collaborate, Munich, Germany, 10Bayer AG, Wuppertal, Germany
Presentation Documents
OBJECTIVES: To report (i) key experiences and (ii) outcomes of the EMA/EUnetHTA qualification process, and (iii) the value and implications of the qualification opinion itself on the IMI PREFER patient preference framework and points to consider for method selection.
METHODS: The PREFER framework, which provides detailed considerations on the design, conduct and use of patient preference studies (PPS), and points to consider for method selection, were assessed by EMA and EUnetHTA via a joint qualification procedure.
RESULTS: (i) With respect to key experiences, the EMA/EUnetHTA qualification procedure provided structured interaction between the PREFER consortium, regulators and HTA bodies on PPS design and use, thereby increasing mutual understanding on PPS design and application in regulatory and HTA evaluation. This also led to enhancements to the PREFER framework and expansion of methods included. (ii) With respect to outcomes, the process resulted in a positive qualification opinion from EMA in May 2022. EMA considered the framework comprehensive, useful to enable PPS planning and conduct, and valuable to support interactions between industry, regulators, HTA bodies/payers, academia, and patients. (iii) With respect to the value and implications of the positive qualification opinion itself, it underscores the value of PPS for (a) patients, by structuring their input into regulatory/HTA decision-making using methodologically robust approaches, (b) regulators and HTA assessors, by providing considerations on how to evaluate PPS, (c) PPS researchers, by offering a set of tools to design, conduct, and analyse state-of-the-art PPS, and (d) medical product developers, by providing advice on why, when and how to use PPS in medical product decision-making.
CONCLUSIONS: The EMA qualification opinion on IMI PREFER is a major step towards more structured conduct and evaluation of PPS in decision-making. Learnings from this qualification provide insight into the value and appropriate use of PPS in medical product development.
Conference/Value in Health Info
Value in Health, Volume 25, Issue 12S (December 2022)
Acceptance Code
P6
Topic
Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient Engagement, Stated Preference & Patient Satisfaction
Disease
no-additional-disease-conditions-specialized-treatment-areas