1 Introduction
Decision making in healthcare is often informed by economic evaluations, and—in many of those decisions—uncertainty exists concerning the effectiveness and costs of the technology under study [
1]. Value of information (VOI) is a tool that can be used to study the uncertainty associated with a coverage decision and its implications. VOI estimates the value of collecting additional data to reduce decision uncertainty and indicates the optimal design for additional research to obtain these data by combining the probability and monetary consequences of an incorrect decision [
2]. To assess the value of reducing all decision uncertainty, the expected value of perfect information (EVPI) can be calculated. The expected value of partial perfect information (EVPPI) is the EVPI focused on one parameter or a subset of parameters. More extensive VOI analyses can be used to examine the value of a certain sample size—the expected value of sample information (EVSI)—and to trade off this value against the costs of additional research by calculating the expected net benefit of sampling (ENBS) [
3]. Finally, methods like real options analysis (ROA) can assist in deciding whether to adopt a health technology for reimbursement now (with the risk of investing in a suboptimal therapy) or to wait for more evidence (with the risk of withholding from patients the optimal treatment: opportunity costs). This trade-off becomes particularly relevant when the decision to adopt a health technology is difficult to reverse due to high costs or other factors [
4].
In the Netherlands, a coverage with evidence development (CED) policy for expensive intramural drugs was implemented in the last decade. Intramural drugs are part of the treatment carried out by or under the responsibility of a medical specialist. This policy implied that intramural drugs with added therapeutic value and an annual budget impact exceeding €2.5 million should be reassessed after 4 years to decide upon their definitive adoption [
5]. During these 4 years, additional data are collected to inform the adoption decision, mainly through the use of registries. Within CED policy, companies were obliged to submit an initial assessment dossier to the National Health Care Institute in the Netherlands [Zorginstituut Nederland (ZIN)]. It is argued that VOI can provide a useful contribution in the process between initial assessment and re-assessment, e.g. deciding upon the value of applying the CED policy in the first place, and directing further research [
6].
Although VOI is described as best practice for handling decision uncertainty [
7], its application remains limited. Document analysis of five CED cases published between 2013 and 2014 revealed that no VOI analyses were performed in these five cases (see the “
Appendix”). These five cases represent all cases published by 2014 for the Netherlands, excluding those drugs that were used for multiple indications. If a drug was used for multiple indications, only the most recent case was included in the document analyses. Information concerning the perspectives of stakeholders on the use of VOI is lacking [
8]. The aim of this paper is to examine (1) the potential value of VOI within a CED policy, (2) the barriers and facilitators of using VOI and (3) the way forward with using VOI in the reimbursement of health technologies perceived by different stakeholders (policy makers, researchers and pharmaceutical companies) in the Netherlands.
4 Discussion
The current use of VOI in practice remains limited, although stakeholders agree that VOI potentially has value to inform research decisions. The participants of the focus groups perceived that the optimal research designs indicated by VOI may not be feasible in practice. Some practical challenges for using VOI could be identified, such as the long computational time and a lack of knowledge concerning VOI. Furthermore, not all uncertainties that should be regarded in health policy decision making are easily incorporated in VOI. Guidelines that clarify how VOI should be performed are needed. Moreover, a threshold ICER and clarity on how the VOI results will be used in decision making regarding adoption and additional research are needed to fully imbed VOI in current decision making.
VOI can support the adoption decision, because it goes beyond reflecting the probability of making a wrong decision by showing the consequences of such a decision [
7,
13]. In that respect, VOI can also be used in negotiations for risk-sharing agreements and price arrangements. This use of VOI appeared to be less obvious to the stakeholders, as some respondents questioned the added value of VOI compared with other uncertainty analyses like PSA. Other stakeholders stated that EVPI alone was regarded as insufficient. This does not imply that all VOI analyses are always necessary; to decide if further research is necessary, an EVPPI may be sufficient to prioritize groups of parameters for further research projects [
14,
15]. In general, more clarity is needed on which VOI analyses are to be performed in which situation.
The fact that randomized controlled trials are often unfeasible was perceived as a barrier to perform VOI. Particularly for less prevalent diseases, it may be desirable to initiate international research projects, and evidence from (ongoing) international studies could be included in the VOI analyses. This may enable more informative registries, especially for rare diseases. However, many authorities also require local information that is not easily obtained from international studies (e.g. local resource use or quality of life).
A CED policy decision should also take into consideration which research designs are practically and ethically feasible. Hence, VOI analyses should be performed for the study designs that are considered feasible for a specific case. In addition, the long computational time required to perform certain analyses was mentioned as a barrier to VOI. This point has also been raised by other researchers [
8,
14]. Participants described that VOI can be complex and that knowledge on how to perform and interpret VOI may be lacking. However, in the literature, new tools have been described that simplify VOI calculations and reduce computational challenges [
16‐
18]. The focus group with researchers did emphasize the importance of incorporating structural uncertainty to obtain robust and credible VOI assessments. Incorporating uncertainties like the uncertainty concerning the choice of comparators and the inclusion of specific events in the modelling process is important for decision-making purposes [
19,
20]. The policy makers stressed the importance of uncertainties unrelated to the cost-effectiveness assessment. These uncertainties (e.g. ethical dimensions of a decision) should be considered separately by decision makers and could be addressed more formally in a multi-criteria decision analysis [
21].
The focus groups results show that VOI is only relevant if it is embedded in the policy window of a jurisdiction regarding adoption decisions. The respondents stressed that decision makers should incorporate the VOI results in the decision-making process if these analyses are requested. VOI outcomes can guide further research, even though there is no fixed threshold ICER, if VOI does not vary within the range of plausible threshold ICERs. However, if VOI results are highly variable within a plausible range of threshold ICERs, a fixed threshold is needed to interpret the results of VOI and to use VOI in decision making [
22]. In the Netherlands, as well as in many other countries, this threshold has not been formally defined [
22,
23]. Different unofficial thresholds are described in the literature, e.g. a threshold of €20,000 per quality-adjusted life-year for preventive interventions [
24]. The focus group revealed that guidelines are needed that clarify the decision problems for which VOI should be performed, since the respondents agreed that VOI is not necessary in all cases. VOI could be useful if uncertainty concerning the cost effectiveness of expensive drugs or drugs with a high budget impact is substantial.
The focus groups were performed in the Netherlands; however, the insights gathered concerning the use of and way forward with VOI might be useful for other jurisdictions that apply a CED policy and a threshold ICER. However, by examining effectiveness only (e.g. using a threshold for minimum clinically relevant difference), VOI could also be used in countries that do not use cost-effectiveness information for coverage decisions (e.g. Germany and the USA) [
25].
To fully embed VOI in healthcare, a threshold ICER and guidelines that clarify how VOI should be used within research and reimbursement decisions are needed.
Compliance with Ethical Standards
This study was funded by the Netherlands Organization for Health Research and Development (Grant Number 1520020071). The views expressed in this article are those of the authors and should not be attributed to the authors’ employers.
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