Objectives: Made available since 2002, transcatheter aortic valve implantation (TAVI) is a minimally invasive new intervention which can provide significant survival improvement to patients with aortic stenosis. However, TAVI is expensive and currently not reimbursed by many governments. Some governments and institutions have been conducting health technology assessments (HTAs) to inform their reimbursement decisions. The aim of the present study is to review HTAs that have relied on a cost-effectiveness analysis to inform reimbursement decisions of TAVI.

Methods: A systematic literature review was conducted among published literature as well as reports released by HTA agencies. Predetermined inclusion and exclusion criteria, following the Preferred Reporting System for Systematic Reviews and Meta-Analysis guidelines, were used to select relevant HTAs. The selected papers were assessed against the Consolidated Health Economic Evaluation Reporting Standards.

Results: HTAs on TAVI from three countries were available for this review: Canada, Belgium, and the United Kingdom. All three HTAs used the Placement of Aortic Transcatheter Valve (PARTNER) trial data with Markov models to estimate the incremental cost effectiveness ratio. The three HTAs recommended conditional reimbursement for TAVI for otherwise inoperable patients. The HTAs did not use clear methods to estimate the health-related utility which ultimately affected their cost-effectiveness results. The UK HTA showed the best value for money (US$20,416 per quality-adjusted life-year).

Conclusion: All studies found TAVI to be more costly and less effective for high-risk patients suitable for surgery, whereas TAVI was consistently found to be cost effective for otherwise inoperable patients.

Objectives: The objectives of this study were to identify and qualitatively describe, in a systematic literature review, published studies that collected prostate cancer patients’ health-related quality of life (HRQoL) estimates by using validated, generic instruments.

Methods: Systematic searches of the literature were made using the Medline, Cochrane Library, PsycINFO, and CINAHL electronic databases from 2002 to 2015.

Results: The search identified 2,171 references, of which 237 were obtained for full-text assessment; thirty-three of these articles were deemed relevant and included in the systematic review. An indirect valuation method was used in 73 percent (n = 24) of the studies. The most commonly used HRQoL instrument with an indirect valuation method was the EuroQol (EQ-5D; n = 21), and the second most common was the 15D (n = 5). A direct valuation method was used in 48 percent (n = 16) of the studies. Of these, the Visual Analogue Scale (VAS) was the most often used (n = 10), followed by the Time-Trade-Off (n = 6). HRQoL scores varied in localized and early stage disease between 0.63 and 0.91, and in advanced or metastatic disease stage between 0.50 and 0.87. There was also variance in the HRQoL instruments and study methods used, which explains the large variance in HRQoL scores between the various disease stages.

Conclusions: Although utility and quality-adjusted life-years gained are considered important measures of effectiveness in health care, the number of studies in which utilities of prostate cancer patients have been estimated using generic HRQoL instruments, based on either direct or indirect measurement of HRQoL, is fairly small.

Objectives: Long-term follow-up of the Caries Management System (CMS) protocol demonstrated that regular monitoring and noninvasive management of dental caries is effective in reducing the number of caries-related events over a 7-year period. This analysis complements the authors’ original economic evaluation of the CMS by re-evaluating the per-protocol cost-effectiveness of the CMS approach.

Methods: An individual patient-simulation Markov model was developed previously, based on 3-year randomized-controlled trial (RCT) data, to simulate the incidence and progression of dental caries, and resultant interventions, and to evaluate the lifetime cost-effectiveness of the CMS versus standard dental care from the Australian private dental practitioner perspective (in which the baseline age distribution was similar to that of the Australian population). The 4-year posttrial follow-up data are used to re-evaluate the long-term cost-effectiveness of the CMS in a more real-life setting.

Results: The reduction in caries risk was maintained among those practices within which the CMS protocols were adhered to. The per-protocol model appears to be reasonably accurate at predicting the risk of restorative events in the posttrial follow-up period. The per-protocol lifetime cost per restorative event avoided is AUD1,980 (USD1,409; 1 AUD = 0.71 USD).

Conclusions: The current analysis confirms that the CMS approach is both effective, when the protocols are adhered to appropriately, and cost-effective compared with standard care in the Australian private practice setting.

Introduction: Hospital-based health technology assessment (HB-HTA) has been introduced to help hospital management in decision making about the adoption of new health technologies (HTs). We reviewed the accuracy of the expected medical impact of HTs assessed at our hospital, as well as the acceptance of this process by clinicians.

Methods: For each HT adopted between 2002 and 2011, a semi-structured interview with the involved clinician was conducted, assessing (i) the perceived utility of the HB-HTA process, (ii) the accuracy of the new HT's expected medical impact as compared with observed patient data from the year 2012, and (iii) the compliance with the indications of the HB-HTA report.

Results: Over the 10-year period, forty HB-HTAs were carried out, of which thirty-four led to acceptance. Twenty-seven of the twenty-eight clinicians involved in these thirty-four HTs accepted the interview and 85 percent acknowledged the utility of the HB-HTA process. Five of the thirty-four HTs were no longer in use. For the twenty-nine remaining HTs, observed patients’ number was as expected in eight, higher in four, lower in fifteen, and not available in two cases. Available average length of stay was 61 percent longer than expected. Two HTs had a higher complication rate and three a lower success rate. Indications evolved in 55 percent of HTs after a few years (seven restrictions, six broadenings, and three other changes).

Conclusions: A HB-HTA process is useful to improve quality in decision making. Follow-up analysis should routinely be performed to adapt HB-HTA reports’ conclusions to practical experience and new scientific evidence.

Background: The adoption of new medical devices and diagnostics is often hampered by lack of published evidence which makes conventional health technology assessment (HTA) difficult. We now have 5 years’ experience of the Medical Technologies Advisory Committee of the National Institute for Health and Care Excellence (NICE) in the United Kingdom, addressing this problem. This committee assesses devices and diagnostics against claims of advantage, to produce guidance on adoption for the health service.

Methods: We have reflected on the practical, technical, and intellectual processes we have used in developing guidance for the health service.

Results: When scientific and clinical evidence is sparse, promise and plausibility play an increased part in decision-making. Drivers of promise include a clear design and mechanism of action, the possibility of radical improvement in care and/or resource use, and improving health outcomes for large numbers of patients. Plausibility relates to judgements about the whether the promise is likely to be delivered in a “real world” setting. Promise and plausibility need to be balanced against the amount of evidence available. We examine the influence they may have on decision-making compared with other factors such as risk and cost.

Conclusions: Decisions about adoption of new devices and diagnostics with little evidence are influenced by judgements of their promise and the plausibility of claims that they will provide benefits in a real-world setting. This kind of decision making needs to be transparent and this article explains how these influences can be balanced against the use of more familiar criteria.

Objectives: A patient reported outcome (PRO) is “any report of the status of a patient's health condition that comes directly from the patient without interpretation of the patient's response by a clinician or anyone else” (USFDA 2009). PROs are discussed widely, and many regard the patients’ perspective on treatment benefit as very valuable. Although many PROs have shown satisfactory measurement properties including reliability, validity, and responsiveness, there is great concern about risk of bias, that is, in clinical trials.

Methods: Differences in perspectives of PRO measurement in rare diseases are given arising from methodology, clinical, HTA, and patient advocacy views.

Results: PROs are playing an important role in dealing with treatment benefit especially in small sample size as occurring often in rare diseases. Challenges remain especially regarding lack of responsiveness of generic measures, limited capture of all patient relevant aspects, study design and high risk of bias.

Conclusions: PROs seem a valuable instrument to detect patient relevant aspects in rare diseases. They should be seen in addition to other approved assessment methods as randomized controlled trials but not as their substitute.

Objectives: External experts can be consulted at different stages of an HTA. When using vague information sources, it is particularly important to plan, analyze, and report the information processing in a standardized and transparent way. Our objective was to search and analyze recommendations regarding where and how to include expert data in HTA.

Methods: We performed a systematic database search and screened the Internet pages of seventy-seven HTA organizations for guidelines, recommendations, and methods papers that address the inclusion of experts in HTA. Relevant documents were downloaded, and information was extracted in a standard form. Results were merged in tables and narrative evidence synthesis.

Results: From twenty-two HTA organizations, we included forty-two documents that consider the use of expert opinion in HTA. Nearly all documents mention experts in the step of preparation of the evidence report. Six documents address their role for priority setting of topics, fifteen for scoping, twelve for the appraisal of evidence and results, another twelve documents mention experts when considering the dissemination of HTA results.

During the assessment step, experts are most often asked to amend the literature search or to provide expertise for special data analyses. Another issue for external experts is to appraise the HTA results and refer them back to a clinical and social context. Little is reported on methods of expert elicitation when their input substitutes study data.

Conclusions: Despite existing recommendations on the use of expert opinion in HTA, common standards for elicitation are scarce in HTA guidelines.

Objectives: We describe a new evidence-based method for screening and evaluating emerging medical technologies. Washington State agencies, under legislative direction, have granted authority to its agency Medical Directors and policy leaders to make coverage decisions on medical technologies using a “dossier” process. The dossier process is employed when technology advocates or manufacturers request Washington State healthcare purchasers to pay for new and emerging technologies. This offers the advocate an opportunity to submit scientific evidence and information classically associated with a more formal health technology assessment.

Methods: The submitted information is independently reviewed and summarized for Washington State's public healthcare purchasers allowing a more standardized coverage decision for all public purchasers in Washington State.

Results: This process has allowed Washington State to make twelve evidence-based coverage decisions at a fraction of the cost of classic technology assessment. To date, of twelve reviews over 6 years, one health technology was approved for coverage, ten were not covered and one did not require a coverage decision.

Conclusions: This evidence-based dossier process has yielded high-value coverage decisions of new and emerging medical technologies for public healthcare purchasers in Washington State.

Objectives: The aim of this study was to develop criteria for the prioritization of topics for health technology assessment (HTA) in the healthcare system of Kazakhstan.

Methods: Initial proposals for criteria were suggested through consultation with Ministry of Health (MoH) policy areas. These were refined through a workshop attended by HTA department staff, persons from medical universities and research institutes, and MoH policy makers. The workshop included discussion on methods used in international HTA practice. Opinions of participants on selection of criteria from those specified in a review of prioritization processes were used to define a list for inclusion in an instrument for routine use. A scoring system was established in later discussion.

Results: Selected criteria for HTA prioritization were burden of disease, availability of alternative technology, clinical effectiveness, economic efficiency, budget impact, and ethical, legal, and/or psychosocial aspects. For each criterion, a health technology under consideration is given a score from 3 (High) to 1 (Low). The total score determines whether the technology is of high to medium priority or of low priority. Determination of priorities for assessment, using the instrument, should be carried out by an expert group appointed by the MoH. The process was applied in 2014 to a selection of topics, and three health technologies were chosen for full assessments.

Conclusions: Criteria for prioritization have evolved with development of the HTA program in Kazakhstan. A method for HTA prioritization has been developed that is easy to apply, requires comparatively few resources, and is compatible with processes required by the MoH.

Objectives: The aim of this project was to identify the ten most important research questions for attention deficit/hyperactivity disorder (ADHD) treatment as identified by people with ADHD together with personnel involved in the treatment of ADHD in school, health, and correction services.

Methods: A working group consisting of consumers and personnel was established. The method for prioritization was primarily based on James Lind Alliance's guidebook, consisting of an interim priority setting exercise and a workshop.

Results: The top ten list includes the risk of drug dependency later in life when treated with methylphenidate as a child, teacher support, multimodal therapy, comparisons between atomoxetine and methylphenidate, methylphenidate treatment in substance abusers, parental support programmes, supported conversation, computer-aided working memory training, psychoeducative treatment, and melatonin.

Conclusions: We have shown that consumers and personnel can reach consensus on research priorities for treatments for ADHD. We encourage researchers and funders to consider the list for future studies.

Objectives: Treatment switching refers to the situation in a randomized controlled trial where patients switch from their randomly assigned treatment onto an alternative. Often, switching is from the control group onto the experimental treatment. In this instance, a standard intention-to-treat analysis does not identify the true comparative effectiveness of the treatments under investigation. We aim to describe statistical methods for adjusting for treatment switching in a comprehensible way for nonstatisticians, and to summarize views on these methods expressed by stakeholders at the 2014 Adelaide International Workshop on Treatment Switching in Clinical Trials.

Methods: We describe three statistical methods used to adjust for treatment switching: marginal structural models, two-stage adjustment, and rank preserving structural failure time models. We draw upon discussion heard at the Adelaide International Workshop to explore the views of stakeholders on the acceptability of these methods.

Results: Stakeholders noted that adjustment methods are based on assumptions, the validity of which may often be questionable. There was disagreement on the acceptability of adjustment methods, but consensus that when these are used, they should be justified rigorously. The utility of adjustment methods depends upon the decision being made and the processes used by the decision-maker.

Conclusions: Treatment switching makes estimating the true comparative effect of a new treatment challenging. However, many decision-makers have reservations with adjustment methods. These, and how they affect the utility of adjustment methods, require further exploration. Further technical work is required to develop adjustment methods to meet real world needs, to enhance their acceptability to decision-makers.

Objectives: Treatment switching occurs when patients in a randomized clinical trial switch from the treatment initially assigned to them to another treatment, typically from the control to experimental treatment. This study discusses the issues this raises and possible approaches to addressing them in trials of cancer drugs.

Methods: Stakeholders from around the world were invited to a 1.5-day Workshop in Adelaide, Australia. This study attempts to capture the key points from the discussion and the perspectives of the various stakeholder groups, but is not a formal consensus statement.

Results: Treatment switching raises challenging ethical issues with arguments for and against allowing it. It is increasingly common in cancer drug trials and presents challenges for the interpretation of results by regulators, clinicians, patients, and payers. Proposals are offered for good practice in the design, management, and analysis of trials and wider development programs for cancer drugs in which treatment switching has occurred or is likely to. Recommendations are also offered for further action to improve understanding of the importance and challenges of treatment switching and to promote agreement between key stakeholders on guidelines and other steps to address these challenges.

Conclusions: The handling of treatment switching in trials is of concern to all stakeholders. On the basis of the discussions at the Adelaide International Workshop, there would appear to be common ground on approaches to addressing treatment switching in cancer trials and scope for the development of formal guidelines to inform the work of regulators, payers, industry, trial designers and other stakeholders.

Objectives: Canada has witnessed expansion of the health technology assessment (HTA) infrastructure in the last 25 years. Local HTA entities at the hospital or regional level are emerging to assist decision makers in the acquisition, implementation, maintenance, and disinvestment of healthcare technologies. There is a need to facilitate collaboration and exchange of expertise and knowledge between these entities regarding the role of local HTA in Canada.

Methods: In November 2013, the pan-Canadian Collaborative hosted a symposium, Hospital/Regional HTA: Local Evidence-based Decisions for Health Care Sustainability, bringing together over 60 HTA producers, researchers, stakeholders, and manufacturers involved in local HTA across Canada. The objective was to showcase the diversity of local HTA in Canada, while highlighting common gaps to be addressed.

Results: The Symposium focused on current practices in local HTA in Canada to support informed decision making, and opportunities for information sharing and provide equal access to timely evidence-based information to decision makers. The main themes included assessment of evidence for local HTA, contextualization, stakeholder engagement in local HTA, knowledge translation and impact of recommendations, and challenges and opportunities for local HTA.

Conclusions: Local HTA in Canada complements HTAs conducted at the provincial and federal levels to improve the efficient and effective health service delivery in institutions or regions faced with limited resources. Some challenges faced by local HTA producers to influence hospital policies and clinical practice involve the engagement of healthcare professionals and potential lack of training and support necessary for the introduction of a new technology.

Objectives: This study was carried out to evaluate the opinions of stakeholders on their roles in health technology assessment (HTA) in Iran and to determine the barriers and facilitators existing in the organizations to help increase their involvement in the HTA program.

Methods: The study was conducted in two stages, semi-structured interviews, and “policy dialogue” with stakeholders. The data were analyzed through the framework approach.

Results: The interviews were held with ten stakeholder representatives from various organizations. In addition, Twenty-one representatives participated in the policy dialogue. Based on the findings, all the stakeholder organizations considered themselves as interest groups in all the stages of the HTA process; however, their tendencies and methods of involvement differed from one another. According to the participants, the most important issue to be considered in the context of HTA was that the structures, stages, and procedures of the HTA process must be made transparent.

Conclusions: Stakeholder involvement in the HTA program cannot readily take place. Various stakeholders have different interests, responsibilities, infrastructures, and barriers. If a program does not meet these considerations, its chances of succeeding will substantially decrease. Therefore, to prevent overlooking the needs and expectations of stakeholders from the HTA process, it is essential to create opportunities in which their thoughts and ideas are taken into account.