شماره چه کسی ؟ نقش ارزیابی فناوری شخص ثالث به اطلاعات سلامت سیستم های تصمیم گیری در مورد سرمایه گذاری های فن آوری سلامت

Background There is an increasing number of health-care systems using economic evaluations to inform decisions about the reimbursement of health technologies. There are usually two separate elements of this process: assembling relevant evidence and undertaking analyses (technology assessment), and decision-making. In most systems, technology assessment is undertaken by the manufacturer of the technology. In a few, “third-party” assessment is used. Methods In the United Kingdom, the National Institute for Health and Clinical Excellence used a combination of third-party and manufacturer assessments between 1999 and 2005. After this point, a Single Technology Appraisal program (using manufacturer-based assessment) was instituted for some technologies. Here the role of third-party assessment is considered in this from of decision-making. The article reviews the requirements of economic evaluation to support decision-making, and considers the extent to which each type of assessment is likely to meet these requirements. It also attempts to address whether the two forms of assessment differ in their impact on decision-making using a comparison of the decisions made by National Institute for Health and Clinical Excellence (NICE) (under its multiple-technology appraisal system) and the Scottish Medicines Consortium (SMC), which relies on manufacturer assessment. Results The comparison is limited by the small number of technologies considered by both bodies. Nevertheless, it suggests that there are potentially important differences between the two bodies, with NICE generally placing more restrictions of the use of technologies.Conclusions The article concludes that there are potential advantages to third-party assessment, but its cost and timing may preclude its use for all new technologies. A hybrid arrangement is suggested where third-party assessment is used in particular circumstances.

Many health-care systems now require economic evaluations as a key input into a formal decision-making process about whether to reimburse/fund/cover new health technologies [1,2]. There are two distinct components to this use of economic evaluation. The first is the technology assessment process which, here, is understood as the process by which relevant evidence is identified, assembled, and synthesized to provide the basis for a clinical review and cost-effectiveness modeling. The second is decisionmaking, into which technology assessment is an important input. In most systems, the technology assessments are undertaken by the manufacturer, although they are usually critically reviewed by experts within the system or a third party. In a small subset of systems, however, there is a role for third-party organizations (usually academic groups) to undertake the assessments, usually in addition to assessments submitted by the relevant manufacturers. The use of such third-party assessments exists, for example, with National Institute for Health and Clinical Excellence (NICE) in the United Kingdom and the Medical Services Advisory Committee, which considers nonpharmaceutical technologies for the Australian health-care system. The role of third-party technology assessment in this form of decision-making has recently been brought to the fore following NICE’s decision to limit its use in their technology appraisal process. From its advent in 1999 until the end of 2005, NICE’s standard process involved a third-party academic group undertaking a technology assessment report (TAR) to input, together with manufacturer and professional evidence and analyses, into the Appraisal Committee’s deliberations. The TAR consists of a review of relevant clinical and economic evidence, a critical assessment of one or more manufacturer submissions (which includes the manufacturers’ own reviews and models), and, usually, the development of a cost-effectiveness model. These arrangements still exist for some technologies as part of NICE’s multiple-technology appraisal (MTA) process. Nevertheless, since 2006, many technologies (particularly newly licensed pharmaceuticals) enter a new single-technology appraisal (STA) process [3]. These arrangements are similar to those in many systems internationally in that the only reviews and analyses informing decision-making are undertaken by the manufacturer, although, as in some other systems, these are accompanied by critical review of the latter’s submission by a third-party evidence review group. This article considers the role of third-party technology assessments and their potential strengths and weaknesses. The aim is to inform the important policy question of whether healthcare systems should use third-party technology assessment when developing new decision-making processes. It can also inform the specific debate in the United Kingdom about the balance between the MTA and STA processes at NICE. A key issue to consider in addressing this question is the evidence regarding whether third-party assessment makes a difference to decisions—that is, are decision-making authorities more or less likely to support the use of a technology if they have access to a third-party assessment? Given the limited number of examples of third-party assessment internationally and the differences between decision-making processes in different systems, which hampers comparison, there is a dearth of such evidence. In time, some potentially valuable evidence will be provided by studying the decisions of the NICE Appraisal Committee and comparing those made under the MTA system with those from STAs. Nevertheless, such a comparison will inevitably be constrained by the fact that different technologies have been considered under the two sets of arrangements. Such a comparison is provided here for those technologies and indications considered by both organizations, and this updates and extends the comparison undertaken by Cairns [4]. To assess different assessment arrangements, the next part of the article describes the required features of economic evaluation to inform decision-making about health-care technologies and considers the possible strengths and weaknesses of third-party assessment for the achievement of these required features. The section called “Does Third-Party Assessment Make a Difference to Decisions” presents the comparison of NICE (MTA) and SMC decisions for a range of technologies. The last section provides a discussion that considers implications for health-care systems internationally.

There is evidence that some manufacturers may be overoptimistic in analyses of the cost-effectiveness of their products [7–9]. Nevertheless, the risks of inappropriate or misleading analyses in manufacturer assessments can be reduced using thorough critical reviews of submissions such as those adopted by many healthcare systems, including NICE (STA) and SMC. The success of careful review in identifying problems with manufacturer assessment can be increased in two ways. The first is for the system to produce detailed and prescriptive methods guidance that defines, as fully as possible, how it wants manufacturers to undertake economic evaluations, thus minimizing the room for judgment on the part of companies. The critical review of the manufacturer’s submission is then undertaken against this methods guidance. This would be supported by the decision-maker being as transparent as possible in describing its decisions and how they relate to the (lack of) evidence. Currently, however, most methods guidelines internationally are quite general and nonprescriptive [2], and few systems provide complete details of how their decisions are made. The second way to underpin the role of the review of company assessments is for decision-makers to encourage a relationship with the manufacturers that in the context of a particular piece of analysis, facilitate an iterative line of communication between the two about appropriate analytic methods. This article has argued that there may be situations when third-party assessment has some important advantages. The first is when an important element of the evidence base is under the control of a manufacturer but not the one undertaking the assessment. Here, it may be possible for the decision-maker to negotiate access to this evidence and to make it available to a third-party assessment team. Second, when the manufacturer is apparently unable to implement appropriate methods, this could be the case with a small manufacturer that does not have the appropriate expertise or necessary resources. Perhaps more often it will be when a manufacturer has failed to reflect all the uncertainties in their economic evaluation or to assess how costeffectiveness might vary between a fully specified set of patient subgroups. There are some important limitations to the comparison between the SMC and NICE (pre-STA) in the earlier section. First, relatively few comparisons of decisions were found (25, including 22 drugs and 18 indications), so the differences in decisions were also small in number. Hence, this sample may be too small to drive any firm conclusion about the comparability of the two approaches. Second, the timing of NICE and SMC guidance is often different, and a gap in published appraisal of more than 2 years was found for some products. Thus, new evidence might have been available during this gap of time and could partly explain some differences in the recommendations. Third, although NICE publishes its full guidance on the website, the SMC only provides a short version, and some details may not be available to the public. Finally, there may be some limits to the independence of decisions between SMC and NICE; in particular, in the nine cases where the SMC’s decision was taken after initial NICE guidance, the latter may have influenced the former, thus limiting the comparison. Despite these limitations, some cautious conclusions seem warranted. In particular, the comparison suggests a trend for the NICE guidance to be more restrictive and to give more information on patient subgroups for which a product is recommended. The example of the two organizations’ decisions about treatments for osteoporosis suggests that in terms of population net benefits, a failure to reflect heterogeneity can impose significant costs on health systems. The absolute value of these costs should be considered cautiously given the uncertainty in their estimation and the possibility of “indication creep” when actual use of a product in routine practice does not adhere to the initial guidance regarding the specific subgroups of patients for which it was recommended. Nevertheless, the analysis shows the broad scale of these potential costs, and this can be set against the costs of third-party assessment. Although there may be some advantages to third-party assessments, as discussed above, the resource cost of these arrangements cannot be ignored, particularly in smaller countries and in systems that use technology assessment to inform centralized decisions about all newly licensed pharmaceuticals. This might suggest that systems could consider some form of hybrid assessment arrangements. For many technologies, an adequate evidential and analytical basis for decisions could be provided by manufacturer assessment. This would be strengthened through high-quality review of submissions against clearly defined and prescriptive methods guidance. Nevertheless, there are likely to be situations when third-party assessment (in addition to analyses submitted by manufacturers) would be highly advantageous for reliable decision-making. One may exist when important sources of evidence are not available to a manufacturer, but could be accessed by a third-party group.Agood example of this would be when two or more new pharmaceuticals for a particular patient group are launched at a similar time and are comparators to each other in an assessment, but no single manufacturer has access to all manufacturers’ evidence. A third-party assessment (together with manufacturer submissions) may be the best approach in this context, although this would probably need to feed into a simultaneous decision about all the technologies. This ability to choose between manufacturer and third-party assessment in advance is a feature of the existing arrangements at NICE where technologies can either be channeled along the MTA or STA routes, although it should be noted that since the advent of STA, relatively few pharmaceuticals have been assessed using MTA. There may, however, be situations where information suggesting advantages for third-party assessment only emerges after a manufacturer assessment has taken place. For example, lack of access to important evidence sources by the manufacturer may only become clear at this stage. Perhaps more likely is a manufacturer assessment in which the decision-making body, informed by a critical review, believes there are methodological weaknesses that the manufacturer has been unable or unwilling to correct and that have important implications for the decision. These weaknesses may relate, for example, to the absence of a full quantification of uncertainty or analysis of heterogeneity, or the failure to incorporate appropriate comparators or relevant evidence. In such situations, the decision-maker may wish to commission a third-party assessment, particularly when the potential costs to the health system (in terms of health forgone) from an inappropriate decision are high (e.g., because the decision relates to a large patient population or where there are large sunk costs to be incurred in investing in a new technology). Under such arrangements, manufacturer assessment effectively becomes a screening process: when it is of good quality and the appropriate decision is relatively clear, it can suffice, but when there are major flaws that are likely directly to affect the decision and the costs of a wrong decision are high, proceeding to a third-party assessment may be appropriate.In conclusion, there are some important advantages to the use of third-party technology assessment to support decisionmaking, and there is some evidence that its availability may influence decisions. Nevertheless, there are serious questions about the practicality and efficiency of using such assessment for decisions regarding all new technologies. The use of a hybrid system where third-party assessment is used to support those decisions for which is it most crucial may offer a sensible way forward.