Author(s)
van Overbeeke E1, Janssens R1, Whichello C2, Schölin Bywall K3, Sharpe J4, Nikolenko N5, Phillips BS6, Guiddi P7, Mazzocco K7, Vergani L7, Marton G8, Cleemput I9, Simoens S1, Juhaeri J10, Levitan B11, Kübler J12, de Bekker-Grob EW2, Veldwijk J2, Huys I1
1University of Leuven, Leuven, Belgium, 2Erasmus University Rotterdam, Rotterdam, The Netherlands, 3Uppsala University, Uppsala, Sweden, 4Muscular Dystrophy UK, London, UK, 5Newcastle University, Newcastle Upon Tyne, UK, 6Pfizer Ltd, Surrey, UK, 7European Institute of Oncology, Milan, Italy, 8The University of Milan, Milan, Italy, 9Belgian Health Care Knowledge Centre (KCE), Brussels, Belgium, 10Sanofi, Bridgewater, NJ, USA, 11Janssen Research & Development, LLC, Titusville, NJ, USA, 12Quantitative Scientific Consulting, Marburg, Germany
OBJECTIVES: To investigate stakeholder perspectives on how patient preference studies (PPS) should be designed, conducted and used in assessments and decision making along the medical product lifecycle. METHODS: Focus group guides were set up and informed by a literature review and semi-structured interviews (n=142). Focus groups were conducted with patients from the UK, Sweden, Italy and Romania; and with European industry representatives, European regulators, US regulators and European/Canadian HTA representatives. Focus groups were analysed thematically using NVivo. RESULTS: Characteristics of medical products that influence treatment preferences according to patients, and thus to consider in the design of PPS, included benefits, side effects, price, quality, administration, packaging and storage. Patients highlighted factors influencing their participation in preference studies such as encouragement of family and the relationship with the recruiter. Across all focus groups it was found important that the PPS sponsor collaborates with other stakeholders including patients, patient organizations, regulators and HTA bodies to limit occurrence of bias, ensure comprehensibility of questions and that results are useful for decision makers. Participants indicated that currently, mostly qualitative input is collected by industry, regulators and HTA bodies and that use of quantitative methods is limited but might be useful for late stages of medical product development. Regulatory, HTA and industry participants agreed that the value of PPS in decision making depends on the level of uncertainty of the clinical evidence and profile of the medical product presented to decision makers. CONCLUSIONS: Stakeholders believed that PPS should be designed in collaboration with multiple healthcare stakeholders including patients. The value of PPS for decision making might depend on the match of the method (qualitative versus quantitative) to the lifecycle phase and on uncertainties relating to the clinical evidence and profile of the medical product.
Conference/Value in Health Info
2018-11, ISPOR Europe 2018, Barcelona, Spain
Value in Health, Vol. 21, S3 (October 2018)
Code
PRM199
Topic
Methodological & Statistical Research
Topic Subcategory
PRO & Related Methods
Disease
Multiple Diseases
