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.
Objectives:
Patient preference studies are increasingly used to inform decision-making during the medical product lifecycle but are rarely used to inform early stages of drug development. The ...primary aim of this study is to quantify treatment preferences of patients with neuromuscular disorders, which represent serious and debilitating conditions with limited or no treatment options available.
Methods:
This quantitative patient preferences study was designed as an online survey, with a cross-over design. This study will target two different diseases from the neuromuscular disorders disease group, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM). Despite having different physio-pathological pathways both DM1 and MM manifest in a clinically similar manner and may benefit from similar treatment options. The sample will be stratified into three subgroups: two patient groups differentiated by age of symptom onset and one caregivers group. Each subgroup will be randomly assigned to complete two of three different preference elicitation methods at two different time points: Q-methodology survey, discrete choice experiment, and best-worst scaling type 2, allowing cross-comparisons of the results across each study time within participants and within elicitation methods. Additional variables such as sociodemographic, clinical and health literacy will be collected to enable analysis of potential heterogeneity.
Ethics and Dissemination:
This study protocol has undergone ethical review and approval by the Newcastle University R&D Ethics Committee (Ref: 15169/2018). All participants will be invited to give electronic informed consent to take part in the study prior accessing the online survey. All electronic data will be anonymised prior analysis. This study is part of the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (IMI-PREFER) project, a public-private collaborative research project aiming to develop expert and evidence-based recommendations on how and when patient preferences can be assessed and used to inform medical product decision making.
•Regulators, industry, and HTA consider patient preference (PP) information important.•PP information is valuable in medical product decision-making.•PP information is not currently obligatory in the ...medical product lifecycle.•There are 15 decision points where PP information could be integrated.•Continued collaboration between stakeholders is needed for successful integration.
Patient preference (PP) information is not effectively integrated in decision-making throughout the medical product lifecycle (MPLC), despite having the potential to improve patients’ healthcare options. A first step requires an understanding of existing processes and decision-points to know how to incorporate PP information in order to improve patient-centric decision-making.
The aims were to: 1) identify the decision-making processes and decision-points throughout the MPLC for industry, regulatory authorities, and reimbursement/HTA, and 2) determine which decision-points can potentially include PP information.
A scoping literature review was conducted using five scientific databases. Semi-structured interviews were conducted with representatives from seven European countries and the US, including industry (n = 24), regulatory authorities (n = 23), reimbursement/HTA (n = 23). Finally, validation meetings with key stakeholders (n = 11) were conducted.
Six critical decision-points were identified for industry decision-making, three for regulatory decision-making, and six for reimbursement/HTA decision-making. Stakeholder groups agreed that PP information is not systematically integrated, either as obligatory information or pre-set criteria, but would benefit all the listed decision-points in the future.
Currently, PP information is not considered as obligatory information to submit for any of the MPLC decision-points. However, PP information is considered an important component by most stakeholders to inform future decision-making across the MPLC. The integration of PP information into 15 identified decision-points needs continued discussion and collaboration between stakeholders.
This study aimed to understand the importance of criteria describing methods (eg, duration, costs, validity, and outcomes) according to decision makers for each decision point in the medical product ...lifecycle (MPLC) and to determine the suitability of a discrete choice experiment, swing weighting, probabilistic threshold technique, and best-worst scale cases 1 and 2 at each decision point in the MPLC.
Applying multicriteria decision analysis, an online survey was sent to MPLC decision makers (ie, industry, regulatory, and health technology assessment representatives). They ranked and weighted 19 methods criteria from an existing performance matrix about their respective decisions across the MPLC. All criteria were given a relative weight based on the ranking and rating in the survey after which an overall suitability score was calculated for each preference elicitation method per decision point. Sensitivity analyses were conducted to reflect uncertainty in the performance matrix.
Fifty-nine industry, 29 regulatory, and 5 health technology assessment representatives completed the surveys. Overall, “estimating trade-offs between treatment characteristics” and “estimating weights for treatment characteristics” were highly important criteria throughout all MPLC decision points, whereas other criteria were most important only for specific MPLC stages. Swing weighting and probabilistic threshold technique received significantly higher suitability scores across decision points than other methods. Sensitivity analyses showed substantial impact of uncertainty in the performance matrix.
Although discrete choice experiment is the most applied preference elicitation method, other methods should also be considered to address the needs of decision makers. Development of evidence-based guidance documents for designing, conducting, and analyzing such methods could enhance their use.
•Given that there is no gold standard method for eliciting preferences, the selection of a patient preference method should be based on decision makers’ needs and performance of methods under consideration to meet those needs. Five commonly used preference elicitation methods were compared: discrete choice experiments, swing weighting, probabilistic threshold technique, and best-worst scale cases 1 and 2.•Whether a method estimates relative importance and trade-offs between treatment characteristics was considered important to decision makers across all medical product lifecycle (MPLC) decision points. Generally, obtaining qualitative information was more important during early lifecycle decisions, whereas internal and external validity and identifying preference heterogeneity were more important during later decision points. Methods performed relatively well on the criteria that were most important at the different decision points of the MPLC; nevertheless, the value of individual methods was sensitive to changes in the performance matrix.•Although discrete choice experiment is the most applied preference elicitation method, best-worst scale, swing weighting, and probabilistic threshold technique should also be considered to address the needs of decision makers throughout the MPLC as they comply with top-weighted methods criteria according to MPLC decision makers.
Introduction
It has become increasingly important to include patient preference information in decision-making processes for drug development. As neuromuscular disorders represent multisystem, ...debilitating, and progressive rare diseases with few treatment options, this study aimed to explore unmet health care needs and patient treatment preferences for two neuromuscular disorders, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM) to inform early stages of drug development.
Methods
Fifteen semi-structured interviews and five focus group discussions (FGDs) were held with DM1 and MM adult patients and caregivers. Topics discussed included (1) reasons for study participation; (2) disease signs/symptoms and their impact on daily lives; (3) top desired benefits; and (4) acceptability of risks and tolerance levels for a hypothetical new treatment. Data were analyzed following a thematic ‘code’ approach.
Results
A total of 52 participants representing a wide range of disease severities participated. ‘Muscle strength’ and ‘energy and endurance’ were the disease-related unmet needs most often mentioned. Additionally, improved ‘balance’, ‘cognition’ and ‘gut function’ were the top desired treatment benefits, while ‘damage to the liver, kidneys or eyes’ was the most concerning risk. Factors influencing their tolerance to risks related to previously having experienced the risk and differentiation between permanent and temporary risks. A few differences were elicited between patients and caregivers.
Conclusions
This qualitative study provided an open forum to elicit treatment-desired benefits and acceptable risks to be established by patients themselves. These findings can inform decisions for developing new treatments and the design of clinical trials for DM1 and MM.
PDF
