Use of Patient Preference Studies in HTA Decision Making: A NICE Perspective

Abstract

Patient preference studies could provide valuable insights to a National Institute for Health and Care Excellence committee into the preferences patients have for different treatment options, especially if the study sample is representative of the broader patient population. We identify three main uses of patient preference studies along a technology's pathway from drug development to clinical use: in early clinical development to guide the selection of appropriate endpoints, to inform benefitrisk assessments carried out by regulators and to inform reimbursement decisions made by health technology assessment bodies. In the context of the National Institute for Health and Care Excellence's methods and processes, we do not see a role for quantitative patient preference data to be directly incorporated into health economic modelling. Rather, we see a role for patient preference studies to be submitted alongside other types of evidence. Examples where patient preference studies might have added value in health technology assessments include cases where two distinctly different treatment options are being compared, when patients have to decide between multiple treatment options, when technologies have important nonhealth benefits or when a treatment is indicated for a heterogenous population.

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

“…The focus groups showed two main applications for PP in HTA, confirming areas mentioned by the UK HTA body NICE [38,39]. First, a role in early scientific advice was suggested by Canadian and Belgian HTA representatives to justify unmet medical need and selection of clinical trial endpoints.…”

“…Secondly, participants overall agreed that PPS could be used as supportive information to complement clinical evidence during HTA. An assessment of clinical evidence was found necessary before PP could be considered, with PP only being considered if evidence is convincing, as also stated by NICE [39]. Participants from all focus groups wanted PPS to investigate attributes related to benefits, risks, and administration (Table 1).…”

“…According to Mott [26], PP can be incorporated into economic evaluations by using them within the calculation of the QALY. NICE recently stated that they do not want to use PP to value health states in CEA as this valuation needs to reflect societal values, rather than patient values, but that PP "could potentially provide additional support that would enable decision makers to make more informed recommendations in cases where the clinical trial data in isolation might not provide a clear demonstration on precisely what the value proposition is for patients" [38,39]. Results from our Belgian and Canadian focus groups confirm this statement; participants prefer to consider PP separately as supportive evidence.…”

“…Participants across all focus groups emphasized that the quality of PPS is crucial. Quality criteria coincided between countries-independence (relating to the initiator), robustness of the method, selection of attributes, information given to patients, and representativeness of the sample; representativeness and independence were also mentioned by NICE as important criteria [38,39]. To mitigate introduction of bias in the design of PPS by sponsors, Canadian participants suggested that HTA bodies could provide scientific advice on PPS design, confirming the need for collaboration with these stakeholders as previously identified by van Overbeeke et al [22], and in statements and recent activities by NICE [38,41].…”

The Importance of Collaboration in Pursuit of Patient-Centered Value Assessment

“…A common criticism of cost-effectiveness analyses, for example, is that the use of quality-adjusted life-years to capture the benefits of therapies does not adequately capture societal benefits or the relative importance of various outcomes to patients [4]. Some health technology assessment agencies and value assessment organizations (e.g., the Institute for Clinical and Economic Review in the USA and the National Institute for Health and Care Excellence in the United Kingdom) include qualitative data on patient experience as a supplement to quantitative value assessments, but patient-centered quantitative assessment remains an aspiration [5][6][7][8][9].…”