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.

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.

Despite the immature state of stem cell medicine, patients are seeking and accessing putative stem cell therapies in an "early market" in which direct-to-consumer advertising via the internet likely plays an important role. We analyzed stem cell clinic websites and appraised the relevant published clinical evidence of stem cell therapies to address three questions about the direct-to-consumer portrayal of stem cell medicine in this early market: What sorts of therapies are being offered? How are they portrayed? Is there clinical evidence to support the use of these therapies? We found that the portrayal of stem cell medicine on provider websites is optimistic and unsubstantiated by peer-reviewed literature.

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). METHODS: Sixteen individuals were selected from 1600 randomly sampled residents of the Capital Health Region in Alberta, Canada. They participated in a 2 (1/2) day jury which comprised presentations by 'expert witnesses', who represented innovators, patients, health-care policy-makers and clinicians, as well as a series of small and large group priority-setting exercises based on actual examples of technologies that had recently been considered for assessment by local and national HTA bodies. The session was audio-taped, and transcripts were independently reviewed by two researchers using content analytical techniques in order to ensure that no important concepts expressed by individual jurors were missed during group development of the final list of priority-setting criteria. Jurors evaluated the process by completing self-administered, semi-structured questionnaires at the end of the session. Responses were analysed using qualitative methods. RESULTS: The jury identified 13 criteria, which they subsequently ranked in order of importance. The top two criteria included 'potential to benefit a number of people' and 'extends life with quality'. Based on feedback from questionnaires, jurors valued the opportunity to become engaged in such a process, and expressed interest in participating in future juries. CONCLUSIONS: Citizens' juries offer a feasible approach to involving the public in priority-setting for HTA. Furthermore, technologies that may benefit a number of people and improve quality of life appear to be of greatest importance to the public.

OBJECTIVE: In recent years, laser treatments have re-emerged as options for treating benign prostatic hyperplasia (BPH). This paper reviews the safety, efficacy, effectiveness and economics of photoselective vapourization of the prostate (PVP), compared with transurethral resection of the prostate (TURP). METHODS: We conducted a comprehensive literature search to identify 14 studies comparing 80 W PVP and TURP. We then reviewed and pooled these studies. RESULTS : We identified 12 adverse events or complications; rates were similar in both groups. No PVP patient required a blood transfusion; 5%-11% of TURP patients did. PVP patients had a significantly lower likelihood of clot retention. Collectively, the 14 studies led to the conclusion that PVP is a safe procedure. PVP is favourable to TURP regarding length of hospitalization and catheterization times. There were no meaningful differences in operation times, reoperation rates, peak flow rates, postresidual volumes or quality of life scores. Sexual function and prostate specific antigen value changes were similar. Four economic evaluations of PVP and TURP (from Canada, Switzerland, Australia and the United States) concluded that PVP is less costly per case than TURP. Cost differences are attributable to disposables, capital equipment and hospitalization. Based on our analysis of limited Canadian data, the cost of PVP is estimated to be about one-half that of TURP, even after accounting for start-up costs. CONCLUSION: The 80 W PVP laser appears to be a viable treatment for BPH. With the recent regulatory approval of the 120 W High Performance System (HPS), an additional option has become available. Therefore, it is anticipated that PVP will soon be considered part of a physician's repertoire for managing BPH.

INTRODUCTION: In recent years, heightened public awareness of new medical advances that offer improved therapeutic and diagnostic options coupled with increased fiscal pressure on health care systems to deliver both equitable and efficient care have magnified the need to examine carefully how and by whom health care priorities are set. OBJECTIVE: To assess processes for setting health care priorities in Alberta, Canada. METHODS: A demographically representative sample of senior management within Regional Health Authorities (RHAs) and specialized provincial boards was selected to participate in key informant interviews. The interviews, which were audio-taped and transcribed, comprised open-ended questions addressing priority-setting approaches employed and the extent to which the public was involved. Through a series of iterations, transcripts were analyzed using content analytic techniques. RESULTS: In general, priority-setting was found to involve four steps:(1) identification of health care needs,(2) allocation of resources,(3) communication of decisions to stakeholders, and (4) management of feedback from them. While approaches to accomplishing each step varied across RHAs and specialized provincial bodies, public involvement did not. In all cases, mechanisms for engaging them in priority-setting focused almost exclusively on the first step. From an "accountability for reasonableness" perspective, none of the organizations surveyed had established processes that met all four principles.

OBJECTIVES: This study sought to examine the safety, efficacy, and economic implications of the use of cerebral protection devices during carotid artery angioplasty and stenting (CAS) in high-risk patients with severe carotid artery disease (CAD). METHODS: A comprehensive search for peer- and non-peer-reviewed studies that compared carotid endarterectomy (CEA) or CAS without cerebral protection to CAS with cerebral protection and appeared in the English language literature between January 1990 and January 2005 was completed. Information from studies identified was extracted using a common data abstraction form and then critically appraised against published quality assessment criteria. RESULTS: Of the eight studies found, six provided information on technical or procedural success rates, with values ranging from 95.6 percent to 100 percent. Three of the four studies comparing groups of patients who received CAS with cerebral protection with those who received only CAS reported a non-statistically significantly higher 30-day incidence of death and stroke (major or minor) in the latter group. None of the three studies comparing CAS with cerebral protection to CEA demonstrated a statistically significant difference in the 30-day incidence of death, major stroke, or myocardial infarction between treatment groups. No economic analyses were found. CONCLUSIONS: In high-risk patients with severe CAD, the evidence suggests that CAS with cerebral protection may offer a safe and efficacious alternative to CEA, reducing the risk of embolic peri-procedural complications associated with CAS to acceptable levels.

"Evidence-informed" priority-setting in healthcare has become increasingly important in most health systems around the world. This paper presents the results of a two-part study of the role of academic health services research in healthcare priority-setting. First, a review of peer-reviewed literature was done to elicit the factors important to priority-setting. Second, a survey of authors of this literature was conducted to determine the value of relevant academic work to decision-making

OBJECTIVES: To review the status of universal newborn screening programs in Canada. METHODS: A brief questionnaire (seven questions) was circulated to one key individual in each province (n=10) and territory (n=3). These individuals were usually physicians or clinical biochemists closely involved in the diagnosis and treatment of genetic metabolic diseases. RESULTS: Universal newborn screening is under provincial jurisdiction. The number of diseases screened for varies and ranges from three to 28. Nine provinces/territories have a central computerized system for tracking initially positive cases. Only five provinces/territories have adequate personnel and resources for follow-up and treatment. Treatment costs are only partially covered in most jurisdictions. Only five provinces/territories have formal advisory committees with official mandates. Expensive, restricted access treatment products for adults with inherited metabolic diseases are only fully available in six provinces/territories. There is very limited access to these products in an additional four provinces/territories. To date, specific informed consent for newborn screening is not required in any province or territory. CONCLUSIONS: Canada is far behind the rest of the developed (and some 'emerging') countries of the world in the field of universal newborn screening. New strategies for advocating expanded screening, follow-up and (long-term) payment of treatment costs on behalf of the potentially affected infants and their families must be devised, and such initiatives should include participation from the new Public Health Agency of Canada.

BACKGROUND: As debate continues regarding pharmacare in Canada, little discussion has addressed appropriate drug plan coverage for vulnerable populations, such as children. The primary objective of this study was to determine the extent of medication coverage for children in publicly administered programs in each province across Canada. METHODS: Data were collected on provincial, territorial and federal government drug plans, and 2003 formulary updates were obtained. A simulation model was constructed to demonstrate costs to a low-income family with an asthmatic child in each province. Programs were compared descriptively. The extent of interprovincial variation in 2003 formulary approvals was summarized statistically. RESULTS: There was 39% variation between provinces with respect to 2003 formulary approvals (chi-square p < 0.0001) and 48% variation for 2003 paediatric-labelled products (chi-square p < 0.0001). Across Canada, only 8% of 2003 formulary approvals were indicated primarily for paediatric conditions. In the simulation model, costs were less than or equal to 3% of household income in provinces with plans for low-income families, catastrophic costs (Ontario) or for the population. Families who failed to qualify for low income plans or who resided in New Brunswick or Newfoundland faced costs up to 7% of household income. INTERPRETATION: With regard to pharmaceutical benefits for children, provincial drug programs vary considerably in terms of whom they cover, what drugs are covered and how much subscribers must pay out of pocket. Unlike seniors and social assistance recipients, the provinces do not agree on the importance of providing comprehensive coverage for all children. For many Canadian children, significant financial barriers exist to medication access.

Available evidence indicates that patients who have private sector drug insurance are immediately covered for all new medically necessary drugs certified by Health Canada, with few exceptions. The experience for patients who are dependent on public drug programmes is much worse. Evidence indicates that, on average across the provinces, far less than half of the drugs certified by Health Canada are finally approved by the provinces for public reimbursement. Publicly insured patients also wait up to 1 year longer than privately insured patients to get insurance coverage for new drugs. Evidence also shows that public drug coverage varies widely across provinces.