Opportunities and Challenges Incorporating Patient Preference Information in Health Technology Assessment Frameworks
Salah Ghabri, PhD, French National Authority for Health (Haute Autorité de santé, HAS), Saint-Denis, France; Axel Mühlbacher, PhD,
Hochschule Neubrandenburg, Germany
Why incorporate PPI in the HTA?
Incorporation of patient perspective is becoming essential at all the dimensions of decision making:
1. By consuming health technologies, patients not only gain benefits but also face adverse events/side effects. Therefore, insufficient knowledge about willingness to trade off perceived benefits versus risks might wrongly impact assessment of comparative-effectiveness and cost-effectiveness. Decision makers might not maximize patients’ value.
2. Patients and professional healthcare practitioners might have different treatment preferences. Therefore, the democratic aspect and transparency of HTA recommendations require the integration of patient preferences in treatment guidelines and disease managements manuals. If not considered, patients might not accept treatments, resulting in poor treatment adherence/compliance in real-life practice.
3. Statements regarding comparative effectiveness and economic evaluation require aggregated unidimensional benefit scores or indices. Patient preferences can be used to estimate these utility or benefit metrics.
"The main challenges related to the integration of patient preference information in health technology assessments are about the choice of method of analysis of patient preferences and the documentation of preference heterogeneity."
How can PPI be defined?
Decision makers have to adapt and incorporate PPI to support a paradigm shift towards patient-centered healthcare. The Institute of Medicine8 states that patient-centeredness aims at “providing care that is respectful of and responsive to individual patient preferences, needs, and values, and ensuring that patient values guide all clinical decisions.” Therefore, the primary task of the regulatory processes is to assess outcomes and measures to reduce uncertainty and appraise that these outcomes are meaningful not only to clinical and policy decision makers, but also to patients. More specifically, the content of PPI has been defined by the US Food and Drug Administration (FDA) as “qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions.”9
How best to incorporate PPI in HTA frameworks?
HTA is expected to be an interdisciplinary process, which gathers scientific, contextual, and historical types of evidence. Depending on the aim, quantitative and qualitative methods are or can be used in HTA. Patients ask for more participation in the HTA process to gain representation and to make better informed decisions. The desire to participate in the decision-making process results from a perceived lack of recognition by patients. Therefore, the consideration of PPI in the process of HTA is required.
Three crucial questions help to understand how patient preferences can be recognized in HTA frameworks:
1. What matters to patients? What decision criteria are maximizing treatment benefits?
2. How can outcomes and endpoints be weighted? How can evidence about the maximum acceptable risk be documented?
3. How can the net benefit be assessed related to the decision-relevant outcomes? What is the best way to aggregate effectiveness measures and assess heterogeneity and uncertainty surrounding these outcomes?
The first question covers the procedural dimension of PPI. The next 2 questions are together related to the methodological aspects related to the use of PPI in HTA.
Integration of patient preferences in chronic diseases (ie, rheumatoid arthritis) is a good example that answers these 3 questions and shows how patient preferences for rheumatoid arthritis with biologic disease-modifying anti-rheumatic drugs (DMARDs) might support HTA decision making.10.11 First, patients’ preferences contribute to inform decision makers on the patient-relevant decision criteria of DMARDs therapeutic classes (eg, route of administration, effectiveness, treatment-specific rare risks).
Second, elicitation of relative importance can be estimated using discrete choice experiments, which can be administrated to a representative sample of the population of interest.12,13 Health preference research can provide information on patients’ willingness to trade off benefits versus risks.
"The crucial challenge is how to integrate PPI into value assessment frameworks using systematic and standard scientific approaches."
Third, to support the related effectiveness or cost-effectiveness healthcare decision, the patients’ weights might be combined with those of other HTA stakeholders through a consistent, a transparent value assessment framework. The latter process should be optimized by analyzing the impact of the preference heterogeneity (identified by specific subgroups) on the outcome measures of the healthcare decision (eg, remission) or cost-effectiveness metrics (eg, incremental cost-utility ratio).
What are the key challenges related to the use of PPI in HTA and economic evaluation?
The main challenges related to the integration of PPI in HTA are mainly procedural and methodological. According to a recent systematic review,14 the most methodological issues are about the choice of method of analysis of patients’ preferences (narrative approach or patient’s elicitation) and the documentation of preference heterogeneity. A controversial question frequently discussed by HTA decision makers is how to use PPI beyond the quality of life-years given that patient preferences capture the differences in health states experienced by patients.7 In that regard, an example illustrating this issue is how HTA decision makers use PPI related to the patient preferences for rheumatoid arthritis with biologic DMARD.13
Conclusion
Despite conceptual progress in terms of the definition of PPI and the identification of its benefits in the HTA process, there is a lack of standard guidelines and a need for examples illustrating the impact of PPI on HTA decision making.
The crucial challenge is how to integrate PPI into value assessment frameworks using systematic and standard scientific approaches. Further research has to demonstrate how PPI can be incorporated into the existing guidelines and HTA value frameworks (eg, clinical, ethical, and economic HTA procedure) and the extent to which PPI can be considered as based evidence data.
References
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