Use of Patient Preferences in Health Technology Assessment: Perspectives of Canadian, Belgian and German HTA Representatives

Abstract

Objective Patient preferences can be informative for health technology assessment (HTA) and payer decision making. However, applications may be different per country. The aim of this study therefore was to investigate HTA representatives' opinions on whether and how to incorporate patient preferences in HTA in their respective countries. Methods Three country-specific focus groups were conducted with three to seven HTA representatives from Germany, Belgium, and Canada. A predefined focus group guide was used that covered topics relating to how patient preferences can be used in HTA, namely HTA stage, weight, impact, and quality, as well as a case example of gene therapy. Transcripts were analyzed using NVivo 12 following thematic analysis. Results Across all HTA bodies, an interest in the use of patient preferences was observed for scientific advice and value assessments, but not through incorporation in quality-adjusted life-years and multi-criteria decision analysis. HTA representatives found it difficult to determine the weight patient preferences may receive in decision making, but thought it could have an impact on payer decision making if the study is of acceptable quality. Conclusions In the near future it may be impossible to achieve structural integration of patient preferences with other evidence in HTA (e.g., in cost-effectiveness analysis), but HTA bodies are willing to incorporate patient preferences in other HTA sections as supportive evidence. To allow for that use, future work should focus on meeting HTA and payer needs when conducting patient preference studies and on education of HTA and payer representatives regarding these studies.

How to integrate evidence from patient preference studies into health technology assessment: a critical review and recommendations

“…This is the more feasible of the PP use cases and easily acceptable to HTA agencies, as it does not attempt to analytically incorporate PP into an economic evaluation of an overall assessment of the value of a medical product (see below for more discussion). This is supported by a recent publication from staff at the NICE on how PP might support their decision making (7), by interviews with HTA agency staff from Germany, Canada, and Belgium (6), and by case studies of the use of PP by the Ontario HTA agency (11).…”

“…However, this acceptability is accompanied by concerns that the impact of this PP use case on reimbursement decisions will be limited without a more formal integration with evidence already considered by the agencies, such as economic analysis (6). This concern may, however, be mitigated by generating insights earlier in the product life cycle in order to inform the endpoints that will be included in the pivotal clinical trial (18).…”

“…Huls et al (8) identified thirty-seven research issues associated with the use of quantitative PP in HTA, including methodological, procedural, normative, practical, and conceptual issues. This is supported by reviews and interviews with stakeholders, which attributed poor integration of PP into HTA to a lack of accepted methodology and guidelines (5;6).…”

A Comparison of Caregiver and Patient Preferences for Treating Duchenne Muscular Dystrophy

“…Caregiver and patient input can inform patient-centered clinical trials, such as by informing the selection of relevant outcome measures [9][10][11]. Caregiver and patient preference information is also increasingly integrated into regulatory decision making in the USA and internationally [12][13][14]. The US Food and Drug Administration has endorsed the collection of patient experience data from both caregivers and patients, as well as from patient advocates [15].…”

Methodological Priorities for Patient Preferences Research: Stakeholder Input to the PREFER Public–Private Project

“…The interviews and focus groups were conducted with more than 140 stakeholders from seven European countries and the United States. These were used to identify the desires, expectations, concerns, and requirements of stakeholders about methodologies for patient-preference elicitation and their use in making well-informed decisions regarding medicinal products [22,[26][27][28][29]. Based on the findings of this early work within PREFER, a multistep approach was used to draft a research agenda for PREFER partners and other parties interested in patient preference information.…”