The Missing Stakeholder Group Why Patients Should

Authors: George A K van Voorn Pepijn Vemer Dominique Hamerlijnck Isaac Corro Ramos Geertruida J Teunissen Maiwenn Al Talitha L Feenstra

Publish Date: 2015/09/18

Volume: 14, Issue: 2, Pages: 129-133

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Abstract

Evaluations of healthcare interventions eg new drugs or other new treatment strategies commonly include a costeffectiveness analysis CEA that is based on the application of health economic HE models As end users patients are important stakeholders regarding the outcomes of CEAs yet their knowledge of HE model development and application or their involvement therein is absent This paper considers possible benefits and risks of patient involvement in HE model development and application for modellers and patients An exploratory review of the literature has been performed on stakeholderinvolved modelling in various disciplines In addition Dutch patient experts have been interviewed about their experience in and opinion about the application of HE models Patients have little to no knowledge of HE models and are seldom involved in HE model development and application Benefits of becoming involved would include a greater understanding and possible acceptance by patients of HE model application improved model validation and a more direct infusion of patient expertise Risks would include patient bias and increased costs of modelling Patient involvement in HE modelling seems to carry several benefits as well as risks We claim that the benefits may outweigh the risks and that patients should become involvedHealth technology assessment HTA has been used as a way to limit the trend of increasing healthcare expenditures in many European countries since the 1990s 1 2 Many countries use cost effectiveness as an important formal eg Austria Belgium the Netherlands and Sweden or informal eg France criterion for HTA information in their evaluation process 3 The clearest example is provided by the UK where official thresholds for cost effectiveness play a structured role 4Many costeffectiveness analyses CEAs use health economic HE models to obtain estimates of costs and benefits of new interventions Validation the evaluation of ‘whether a model is a proper and sufficient representation of the system it is intended to represent’ 5 is therefore emphasized by guidelines on good HE modelling practice 6 7 8 9 10 11 These guidelines mostly cover the scientific credibility of the models Experience from other fields like climate research nature policy assessments and water management shows that validation also entails other aspects related to fitness for purpose These are stakeholder perception of model salience model applicability within the context and legitimacy the inclusion of stakeholder concerns values and views in a proper way 12 Hence stakeholder involvement is considered to be a critical aspect of good modelling practice 12 13 14 15 16 17 18 19 20 21Patients are one group of stakeholders that is obviously important in terms of HTA decisions But while the validation of HE models has become a point of progression in HE literature the involvement of patients in modelling appears to remain underappreciated HE models play an implicit role by providing relevant information for the reimbursement decision process However they often are a ‘black box’ to patients and one can argue there may be a moral obligation to further open this ‘black box’ and involve patients in the development and application of HE models Moreover many patients have become experts who can provide valuable information for HE modellers Their involvement could improve the credibility salience and legitimacy of HE models The relevance of this issue is even higher as HE models are used in the early stages of HTA affecting the downstream drugevaluation processesIn this opinion paper we discuss benefits and risks of patient involvement in HE model development and application as well as possible ways of involving patients We conducted an exploratory literature search to gain insight into the issues at stake and arguments applied An extensive literature exists about stakeholder involvement in modelling but only few studies report on the current area of interest HE decision modelling In addition discussions were held with patient experts see the Sect 6 for more details In this paper double quotes “…” indicate arguments made by these patient experts where it has been attempted to retain as much as possible of the original meaning in the translation from Dutch to English

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