Tania Stafinski

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.

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.

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.

Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

BackgroundThe rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.ObjectivesThe aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources.MethodsA scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making.ResultsThe scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or ‘opportunity cost’-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework.ConclusionOur framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.

Effectiveness of photodynamic therapy for mammary and extra-mammary Paget's disease: a state of the science review

BackgroundPagets disease is a rare skin disorder occurring in the breast (mammary) or in the groin, genital, peri-anal and axillary regions (extra-mammary). Typical treatment involves surgical excision, which in the case of extra-mammary Pagets disease, can lead to significant morbidity. Photodynamic therapy (PDT) which uses a topical or intravenous photosensitizing agent that is activated by a light source to ablate abnormal tissue, offers a minimally invasive alternative. The purpose of this study was to assess the effectiveness of photodynamic therapy in the treatment of Pagets disease.MethodsFollowing Cochrane guidelines, a comprehensive systematic review of all clinical studies and reports examining the use of PDT for mammary and extra-mammary Pagets disease was conducted. Study quality was assessed using the Oxford Levels of Evidence Scale.Results21 retrospective and 2 prospective non-comparative studies were identified and included in the review: 9 case reports with 1-2 patients and 14 case series with 1-16 patients. These reports totalled 99 patients with 133 extra-mammary Pagets lesions and 3 patients (with 3 lesions) with mammary Pagets disease. Follow-up periods were typically one year or less, with 77/133 extra-mammary lesions exhibiting complete response to PDT. One recurrent mammary skin lesion and two mammary lesions treated concomitantly with surgery also exhibited complete responses.ConclusionsEvidence of the effectiveness of PDT for Pagets disease is promising, but limited. This may, in part, be explained by the rarity of the condition, making controlled comparative clinical trials challenging.

Endoscopic treatments for Barrett's esophagus: a systematic review of safety and effectiveness compared to esophagectomy

BackgroundRecently, several new endoscopic treatments have been used to treat patients with Barretts esophagus with high grade dysplasia. This systematic review aimed to determine the safety and effectiveness of these treatments compared with esophagectomy.MethodsA comprehensive literature search was undertaken to identify studies of endoscopic treatments for Barretts esophagus or early stage esophageal cancer. Information from the selected studies was extracted by two independent reviewers. Study quality was assessed and information was tabulated to identify trends or patterns. Results were pooled across studies for each outcome. Safety (occurrence of adverse events) and effectiveness (complete eradication of dysplasia) were compared across different treatments.ResultsThe 101 studies that met the selection criteria included 8 endoscopic techniques and esophagectomy; only 12 were comparative studies. The quality of evidence was generally low. Methods and outcomes were inconsistently reported. Protocols, outcomes measured, follow-up times and numbers of treatment sessions varied, making it difficult to calculate pooled estimates.The surgical mortality rate was 1.2%, compared to 0.04% in 2831 patients treated endoscopically (1 death). Adverse events were more severe and frequent with esophagectomy, and included anastomotic leaks (9.4%), wound infections (4.1%) and pulmonary complications (4.1%). Four patients (0.1%) treated endoscopically experienced bleeding requiring transfusions. The stricture rate with esophagectomy (5.3%) was lower than with porfimer sodium photodynamic therapy (18.5%), but higher than aminolevulinic acid (ALA) 60 mg/kg PDT (1.4%). Dysphagia and odynophagia varied in frequency across modalities, with the highest rates reported for multipolar electrocoagulation (MPEC). Photosensitivity, an adverse event that occurs only with photodynamic therapy, was experienced by 26.4% of patients who received porfimer sodium.Some radiofrequency ablation (RFA) or argon plasma coagulation (APC) studies (used in multiple sessions) reported rates of almost 100% for complete eradication of dysplasia. But the study methods and findings were not adequately described. The other studies of endoscopic treatments reported similarly high rates of complete eradication.ConclusionsEndoscopic treatments offer safe and effective alternatives to esophagectomy for patients with Barretts esophagus and high grade dysplasia. Unfortunately, shortcomings in the published studies make it impossible to determine the comparative effectiveness of each of the endoscopic treatments.

Health Technology Assessment in Canada: 20 Years Strong?

For over 40 years, Canada has had a publicly funded, national health-care system designed to ensure residents receive “reasonable access” to “medically necessary” health-care services, regardless of their ability to pay [1]. However, unlike many of its European counterparts, Canada’s system is a decentralized one, comprised of 13 separate provincial and territorial health insurance plans. Guided by common values (e.g., equity and solidarity) and responsible for meeting basic standards of coverage, these plans determine how best to organize, manage, and deliver health care within their jurisdictions. Decisions regarding which new technologies to include in the basket of publicly funded services, therefore, rest with individual provinces and territories, and the role of the federal government remains primarily limited to premarket approval and, in the case of patented pharmaceuticals, price regulation. It has, however, retained responsibility for providing services to limited populations, such as veterans, the military, first nations, and inmates. Canada’s history in health technology assessment (HTA), a field developed to support purchasing or coverage decisions, reflects the decentralized nature of the country’s health-care system. Its roots predominantly exist at the provincial level, with the establishment of the Conseil d’evaluation des technologies de la sante (CETS) (now called the Agence des technologies et des modes intervention en sante [AETMIS]) in Quebec 20 years ago [2]. At around the same time, a joint committee representing the federal, provincial, and territorial ministries of health identified HTA as one of its key priorities and announced the creation of a national, independent HTA body called the Canadian Coordinating Office of Health Technology Assessment (renamed the Canadian Agency for Drugs and Technologies in Health (CADTH) in 2006). Funded by the provincial, territorial, and federal governments, its mandate is to provide impartial, evidence-based information on the clinical and economic implications of drugs and other health technologies (including devices, procedures, and systems) to the 13 public insurance plans. Since then, HTA has played an increasingly important role in technology coverage policy in Canada. With the demand for assessments exceeding resources available to the national HTA agency and the types of requests broadening to include context specific questions framed from perspectives other than that of society, the past 20 years have seen the emergence of local HTA initiatives in hospitals, regional health authorities, and provinces across the country. Now more than ever, decision-makers at all levels of government are investing in “institutionalized” HTA, creating a landscape shaped by a combination of ongoing national and local efforts. In this article, the production and use of HTA in health-care decisions in Canada is described in Section I. Current issues in technology assessment are discussed in Section II. Section III represents our personal views on lessons learned from the HTA experience in Canada.

Principles of Design of Access with Evidence Development Approaches: A Consensus Statement from the Banff Summit

Healthcare payers are increasingly entering into innovative reimbursement agreements to manage the tension between funding new but expensive treatments, and obtaining value (measured in terms of clinical effectiveness, improved quality of care, health-related quality of life [HR-QOL], etc.) for money. These often commit substantial resources, and thus impose a significant opportunity cost on healthcare systems. Commentators and stakeholders are increasingly concerned that not all such approaches represent good value for money. However, there is little if any literature providing guidance on their design or evaluation. These reimbursement mechanisms have been given many names, such as ‘risk sharing’, ‘coverage with evidence development’, ‘field evaluations’ and ‘health impact guarantees’. We used the umbrella term ‘access with evidence development’ approaches (AEDs) to ensure discussions focussed upon the substantial issues common to all of these rather than their differences. The purpose of the Banff Summit (Alberta, Canada; 22–23 February 2009) was to bring together experts from around the world with direct experience of designing and implementing approaches to access with the aim of identifying principles to inform their design. The programme consisted of a presentation of findings from a review of the published literature and, drawing on that literature, a proposed checklist to describe and potentially evaluate such schemes. Participants from the US, the UK, Australia and Canadian provinces (British Columbia, Ontario and Alberta) gave presentations on their experience of AED approaches. Each talk was followed by an open discussion of the lessons that could be drawn from this experience. The final session considered whether it was possible to identify principles of good design in AED schemes. The Summit organizers were charged with producing a written statement of these principles for circulation to all participants. Each individual was then invited to sign up to the statement. We provide here the consensus statement from the summit. This is followed by the literature review completed in preparation for the meeting, a paper that outlines the descriptive framework (which was developed for the Summit) and an evaluation of an AED model implemented in the UK NHS using this framework. In addition, experts from the US and the UK write about the future development of AEDs in their respective healthcare systems and consider how adherence to the principles set out in the consensus statement would improve current and future AEDmodels.