Devidas Menon

Canadian Guidelines for Economic Evaluation of Pharmaceuticals

SummaryIn 1994, p]Canada became the second country to release national guidelines for the economic evaluation of pharmaceuticals. The guidelines were developed over a period of 18 months through an elaborate process of broad consultation with a wide variety of relevant stakeholders. The intent of the guidelines is to provide guidance to doers and users of studies, by laying out the general ‘state of the art’ regarding methods, and by providing specific methodological advice on many matters. The aim is to improve the scientific quality and integrity of studies, and to enhance consistency and comparability across studies.This article presents the Canadian guidelines, both in summary and in detail. Because the techniques of economic evaluation are widely applicable beyond pharmaceuticals, the guidelines will be of interest to researchers and decision makers in all fields of healthcare. Because the methods are not country specific, the guidelines will be of interest to those in other countries as well as in Canada.

Funding the Unfundable Mechanisms for Managing Uncertainty in Decisions on the Introduction of New and Innovative Technologies into Healthcare Systems

As tensions between payers, responsible for ensuring prudent and principled use of scarce resources, and both providers and patients, who legitimately want access to technologies from which they could benefit, continue to mount, interest in approaches to managing the uncertainty surrounding the introduction of new health technologies has heightened.The purpose of this project was to compile an inventory of various types of ‘access with evidence development’ (AED) schemes, examining characteristics of the technologies to which they have been applied, the uncertainty they sought to address, the terms of arrangements of each scheme, and the policy outcomes. It also aimed to identify issues related to such schemes, including advantages and disadvantages from the perspectives of various stakeholder groups.A comprehensive search, review and appraisal of peer-reviewed and ‘grey’ literature were performed, followed by a facilitated workshop of academics and decision makers with expertise in AED schemes. Information was extracted and compiled in tabular form to identify patterns or trends. To enhance the validity of interpretations made, member checking was performed.Although the concept of AED is not new, evaluative data are sparse. Despite varying opinions on the ‘right’ answers to some of the questions raised, there appears to be consensus on a ‘way forward’ — development of methodological guidelines.All stakeholders seemed to share the view that AEDs offer the potential to facilitate patient access to promising new technologies and encourage innovation while ensuring effective use of scarce healthcare resources. There is no agreement on what constitutes ‘sufficient evidence’, and it depends on the specific uncertainty in question. There is agreement on the need for ‘best practice’ guidelines around the implementation and evaluation of AED schemes.This is the first attempt at a comprehensive analysis of methods that have been used to address uncertainty concerning a new drug or other technology. The analysis reveals that, although various approaches have been experimented with, many of them have not achieved the ostensible goal of the approach. This article outlines challenges related to AED schemes and issues that remain unresolved.

The Role of Economic Evidence in Canadian Oncology Reimbursement Decision-Making: To Lambda and Beyond

OBJECTIVE The overarching question addressed in this article is: what has been the impact of economic evidence to Canadian drug reimbursement decisions; within this, has an (explicit or implicit) threshold been identified for making such decisions; and is the impact or threshold different for oncology medications? METHODS Three sequential strategies were employed: a literature search, a review of publicly available Canadian reimbursement recommendations, and a one-day key informant roundtable, held with a purposive sample of 13 individuals from across Canada to gain information not readily accessible from the public domain. RESULTS Despite the formal requirement for structured economic evidence, the limited public information suggests that its uptake in the Canadian decision-making process has been tentative. Implicit economic thresholds have been published in Australia and the United Kingdom, but not in Canada. Based on reviews of reimbursement recommendations, thresholds specific to oncology medications may be higher than for nononcology medications, in Canada and elsewhere. Canadian reimbursement recommendations can appear inconsistent with respect to clinical evidence, economic evidence, and nonevidentiary factors, possibly because of a lack of transparency or context-sensitive interpretations. The key informant roundtable provided reasons for the inconsistent uptake of economic evidence: panelists were divided between those who found economic information useful and supportive to decision-making, and those who did not. Panelists generally agreed on the need for publicly defensible and ethical reimbursement restrictions. They suggested the following improvements: transparency of processes and decisions, dynamic formularies that can adapt with evolving treatment practices and clinical data, broader representation of expertise on review panels, greater use of ethics to resolve conflicts arising from different perspectives, and the development of an explicit Canadian weighting system for evidence and values. CONCLUSIONS Economic evidence has been tentatively incorporated in reimbursement decision-making in Canada. Public reasons for recommendation indicate that this evidence is used primarily with respect to the attractiveness of an incremental cost-effectiveness ratio. Oncology drugs seem to be adopted at the highest thresholds of acceptability. Yet, decision-makers expressed a need to move beyond lambda, rejecting the simplicity of the incremental cost-effectiveness ratio and considering alternative strategies to improve decision-making, including formal guidance for weighting both evidence and values.

Engaging the public in priority-setting for health technology assessment: findings from a citizens’ jury

Objectives  To assess the feasibility of using a citizens’ jury to elicit public values on health technologies and to develop criteria for setting priorities for health technology assessment (HTA).

Access with evidence development schemes: a framework for description and evaluation.

There is an inevitable tension between robust reimbursement processes and providing speedy access to new and novel technologies, given uncertainties about key pieces of evidence and subsequent concerns regarding their overall efficiency. The public perception of these treatments as ‘breakthrough’, combined with substantial clinical pressure, has led to healthcare payers looking for schemes that allow the new technology to be made available to (some) patients, while (at least partially) protecting the principles of their reimbursement decision-making processes. Current literature on these schemes is almost completely descriptive and provides little help in planning future schemes. We propose a framework for evaluating current schemes and informing the design of future schemes. We examine the value of the framework using the UK Multiple Sclerosis Risk-Sharing Scheme as a case study.

Diagnostic accuracy of level 3 portable sleep tests versus level 1 polysomnography for sleep-disordered breathing: a systematic review and meta-analysis

Background: Greater awareness of sleep-disordered breathing and rising obesity rates have fueled demand for sleep studies. Sleep testing using level 3 portable devices may expedite diagnosis and reduce the costs associated with level 1 in-laboratory polysomnography. We sought to assess the diagnostic accuracy of level 3 testing compared with level 1 testing and to identify the appropriate patient population for each test. Methods: We conducted a systematic review and meta-analysis of comparative studies of level 3 versus level 1 sleep tests in adults with suspected sleep-disordered breathing. We searched 3 research databases and grey literature sources for studies that reported on diagnostic accuracy parameters or disease management after diagnosis. Two reviewers screened the search results, selected potentially relevant studies and extracted data. We used a bivariate mixed-effects binary regression model to estimate summary diagnostic accuracy parameters. Results: We included 59 studies involving a total of 5026 evaluable patients (mostly patients suspected of having obstructive sleep apnea). Of these, 19 studies were included in the meta-analysis. The estimated area under the receiver operating characteristics curve was high, ranging between 0.85 and 0.99 across different levels of disease severity. Summary sensitivity ranged between 0.79 and 0.97, and summary specificity ranged between 0.60 and 0.93 across different apnea–hypopnea cut-offs. We saw no significant difference in the clinical management parameters between patients who underwent either test to receive their diagnosis. Interpretation: Level 3 portable devices showed good diagnostic performance compared with level 1 sleep tests in adult patients with a high pretest probability of moderate to severe obstructive sleep apnea and no unstable comorbidities. For patients suspected of having other types of sleep-disordered breathing or sleep disorders not related to breathing, level 1 testing remains the reference standard.

Transcutaneous Electrical Nerve Stimulation (TENS): A Technology Assessment

The scientific evidence for clinical effectiveness of transcutaneous electrical nerve stimulation (TENS) for treatment of acute, chronic, and labor and delivery pain is assessed in this paper, and it is concluded that there is little evidence for other than a limited use of TENS. The utilization of TENS in Canadian hospitals and payments for TENS services are addressed. Some practicalities regarding the use and assessment of health technologies are discussed.

Health Technology Funding Decision- Making Processes Around the World The Same, Yet Different

All healthcare systems routinely make resource allocation decisions that trade off potential health gains to different patient populations. However, when such trade-offs relate to the introduction of new, promising health technologies, perceived ‘winners’ and ‘losers’ are more apparent. In recent years, public scrutiny over such decisions has intensified, raising the need to better understand how they are currently made and how they might be improved. The objective of this paper is to critically review and compare current processes for making health technology funding decisions at the regional, state/provincial and national level in 20 countries.A comprehensive search for published, peer-reviewed and grey literature describing actual national, state/provincial and regional/institutional technology decision-making processes was conducted. Information was extracted by two independent reviewers and tabulated to facilitate qualitative comparative analyses. To identify strengths and weaknesses of processes identified, websites of corresponding organizations were searched for commissioned reviews/evaluations, which were subsequently analysed using standard qualitative methods.A total of 21 national, four provincial/state and six regional/institutional-level processes were found. Although information on each one varied, they could be grouped into four sequential categories: (i) identification of the decision problem; (ii) information inputs; (iii) elements of the decision-making process; and (iv) public accountability and decision implementation. While information requirements of all processes appeared substantial and decision-making factors comprehensive, the way in which they were utilized was often unclear, as were approaches used to incorporate social values or equity arguments into decisions.A comprehensive inventory of approaches to implementing the four main components of all technology funding decision-making processes was compiled, from which areas for future work or research aimed at improving the acceptability of decisions were identified. They include the explication of decision criteria and social values underpinning processes.

The revised Canadian Guidelines for the Economic Evaluation of Pharmaceuticals.

The first edition of the Guidelines for Economic Evaluation of Pharmaceuticals: Canada was published in November 1994. At that time, the Canadian Coordinating Office for Health Technology Assessment (CCOHTA) was assigned the task of maintaining and regularly updating the Canadian Guidelines.Since their introduction, a great deal of experience has been gained with the practical application of the guidelines. Their role has also evolved over time, from being a framework for pharmacoeconomic research to the point where a wide variety of decision-makers use economic evaluations based on the principles set out in the guidelines as a means of facilitating their formulary decisions. In addition, methodologies in certain areas (and the body of related research literature in general) have developed considerably over time. Given these changes in the science and the experience gained, CCOHTA convened a multi-disciplinary committee to address the need for revisions to the guidelines. The underlying principles of the review process were to keep the guidance nature of the document, to focus on the needs of ‘doers’ (so as to meet the information needs of ‘users’) and to provide information and advice in areas of controversy, with sound direction in areas of general agreement.The purpose of this review is three-fold: (i) to outline the process which lead to the revision of the Canadian Guidelines; (ii) to describe the major changes made to the second edition of this document; and (iii) to consider the ‘next steps’ as they relate to the impact of such guidelines and the measurement of outcomes related to economic assessments of pharmaceuticals in general.

Use of economic evaluation guidelines: 2 years' experience in Canada

Considerable effort has been expended in recent years in the development of methodology guidelines for economic evaluation of pharmaceutical products, driven in part by the desire to improve the rigour and quality of economic evaluations and to help decision making. Canada was one of the first countries to develop such guidelines and to encourage their use. This paper examines the extent to which the economic evaluations that were submitted to the Canadian Coordinating Office for Health Technology Assessment in the last two years adhered to Canadian guidelines. The analytic technique employed by twelve studies as well as the comparator used, the perspective taken, the outcome measure selected, the cost items that were taken into consideration and the extent of sensitivity analyses that were performed are reviewed in this paper. It can be concluded that although studies have been of variable quality, the majority of them were well presented, complete and transparent, due in part to the guidelines. Except for the perspective of the analysis, guidelines were, in many respects, adhered to and did not restrict investigators to specific methodologies or specific techniques. They were also instrumental in ensuring a minimum set of standards.