Objectives: Health technology assessment (HTA) has to innovate to best support changing health system environments and to help provide access to valuable innovation under fiscal constraint.

Methods: Issues associated with changing HTA paradigms were identified through scoping and explored through deliberation at a meeting of industry and HTA leaders.

Results: Five broad areas of change (engagement, scientific dialogue, research prioritization, adaptive approaches, and real world data) were identified. The meeting focused on two themes derived from these: re-thinking scientific dialogue and multi-stakeholder engagement, and re-thinking value, affordability, and access. Earlier and ongoing engagement to steer the innovation process and help achieve appropriate use across the technology lifecycle was perceived as important but would be resource intensive and would require priority setting. Patients need to be involved throughout, and particularly at the early stages. Further discussion is needed on the type of body best suited to convening the dialogue required. There was agreement that HTA must continue to assess value, but views differed on the role that HTA should play in assessing affordability and on appropriate responses to challenges around affordability. Enhanced horizon scanning could play an important role in preparing for significant future investments.

Conclusions: Early and ongoing multi-stakeholder engagement and revisiting approaches to valuing innovation are required. Questions remain as to the most appropriate role for HTA bodies. Changing HTA paradigms extend HTA's traditional remit of being responsive to decision-makers demands to being more proactive and considering whole system value.

The need to progress and innovate in health technology assessment (HTA) is a must in a continuously changing environment. The HTAi Policy Forum reflected on two specific areas for development where it was thought there was need for careful consideration and deliberation during the main annual meeting in February 2016. The study by Husereau et al. (1) in this journal presents the discussions resulting from this Forum. To further share the deliberations of the Forum and with a view to opening this debate to the wider HTA community, a panel session during the HTAi Annual Meeting in Tokyo was organized. Presentations at the panel included a summary of the HTAi Policy Forum discussions and perspectives from a patient, a representative of healthcare system provider, and a representative from an HTA organization and industry. This letter presents the issues raised in the panel session.

Objectives: Kids in Control OF Food (KICk-OFF) is a 5-day structured education program for 11- to 16-year-olds with type 1 diabetes mellitus (T1DM) who are using multiple daily insulin injections. This study evaluates the cost-effectiveness of the KICk-OFF education program compared with the usual care using data from the KICk-OFF trial.

Methods: The short-term within-trial analysis covers the 2-year postintervention period. Data on glycated hemoglobin (HbA1c), severe hypoglycemia, and diabetic ketoacidosis (DKA) were collected over a 2-year follow-up period. Sub-group analyses have been defined on the basis of baseline HbA1c being below 7.5 percent (58.5 mmol/mol) (low group), between 7.5 percent and 9.5 percent (80.3 mmol/mol) (medium group), and over 9.5 percent (high group). The long-term cost-effectiveness evaluation has been conducted by using The Sheffield Type 1 Diabetes Policy Model, which is a patient-level simulation model on T1DM. It includes long-term microvascular (retinopathy, neuropathy, and nephropathy) and macrovascular (myocardial infarction, stroke, revascularization, and angina) diabetes-related complications and acute adverse events (severe hypoglycemia and DKA).

Results: The most favorable within-trial scenario for the KICk-OFF arm led to an incremental cost-effectiveness ratio (ICER) of £23,688 (base year 2009) with a cost-effectiveness probability of 41.3 percent. Simulating the long-term complications using the full cohort data, the mean ICER for the base case was £28,813 (base year 2011) and the probability of the KICk-OFF intervention being cost-effective at £20,000/QALY threshold was 42.6 percent, with considerable variation due to treatment effect duration. For the high HbA1c sub-group, the KICk-OFF arm was “dominant” (meaning it provided better health gains at lower costs than usual care) over the usual care arm in each scenario considered.

Conclusions: For the whole study population, the cost-effectiveness of KICk-OFF depends on the assumption for treatment effect duration. For the high baseline HbA1c sub-group, KICk-OFF arm was estimated to be dominant over the usual care arm regardless of the assumption on the treatment effect duration.

Objectives: Curative hepatectomy and tumor thrombectomy for hepatocellular carcinoma with complicating biliary tumor thrombosis (HCC/BTT) is associated with high surgical morbidity and mortality. This retrospective study evaluated the effectiveness and safety of neoadjuvant transcatheter arterial chemoembolization (TACE) in HCC/BTT patients scheduled for curative resection.

Methods: Thirty consecutive patients with diagnosed HCC/BTT were hospitalized for neoadjuvant TACE and elective curative liver resection (group A; n=20) or curative liver resection alone (group B; n=10). The primary outcome measure was median survival.

Results: Group A had a significantly shorter overall operative time (160±25 versus 190±35 min; p < .01) and duration of inflow control (14.3±3.6 versus 25.1±5.1 min; p < .01) and significantly less intraoperative blood loss (150±35 versus 520±75 ml; p < .01) and transfusion (100±40 versus 375±55 ml; p < .01) as compared to group B. Among patients undergoing both thrombectomy and curative resection, the median survival of group A was significantly longer than that of group B (28.5 [9–54] versus 21.5 [6–39] months; p < .01); among those who received thrombectomy alone, the median survival of group A was also significantly longer than that of group B (12.8 [6–25] versus 4.5 [2–7] months; p < .01).

Conclusions: Neoadjuvant TACE significantly reduced the surgical risk of curative liver resection and significantly prolonged median survival in HCC patients with complicating BTT.

Objectives: We explore how broader aspects of a treatment's value and the impact of the condition on patients not captured by routine health technology assessment (HTA) methods using clinical and economic evidence, defined as “other considerations,” may influence HTA processes in different settings.

Methods: Countries included were England, Scotland, Sweden, and France. Data sources were the publicly available reports on HTA recommendations. Ten drugs with European Medicines Agency orphan designation and appraised in England were selected. Qualitative thematic analysis was used to systematically identify and code all “other considerations” based on a previously developed methodological framework, which also coded whether it was provided by stakeholders, and how it influenced the decision.

Results: A classification framework of scientific and social value judgments was developed and used throughout the study. A total of 125 “other considerations” were identified and grouped into ten subcategories based on the information provided. Eighteen to 100 percent of these, depending on the agency, were put forward as one of the main reasons for the final decision potentially contributing to accepting a higher incremental cost-effectiveness ratio or uncertain evidence. Some of these were nonquantified or nonelicited and pertained to the assessor's judgment. A taxonomy of these value judgments was created to be used in future cases. Results also contributed to better defining the determinants of social value and improving accountability for reasonableness.

Conclusions: The systematic identification of the scientific and social value judgments enables to better understanding the dimensions of value, which can be used to improve their transparency and consistent use across decisions and settings.

Objectives: Herpes zoster (HZ) is characterized by a painful skin rash. Its main complication is postherpetic neuralgia (PHN), pain persisting or occurring after the rash onset. HZ treatment aims to reduce acute pain, impede the onset complications, and disease progression. The aim of this study was to assess the cost-effectiveness of HZ vaccination compared with no vaccination strategy, within the Italian context.

Methods: The natural history of HZ and PHN was mapped through a Markov model with lifetime horizon. A population of patients aged between 60 and 79 years was hypothesized. Third party payer (Italian National Health Service, I-NHS) and societal perspectives were adopted. Data were derived from literature.

Results and Conclusions: The incremental cost-effectiveness ratio of the vaccination equaled EUR 11,943 per quality-adjusted life-year (QALY) under the I-NHS perspective and EUR 11,248 per QALY under the societal perspective. Considering a cost-effectiveness threshold of EUR 30,000/QALY, the multi-way sensitivity analysis showed that vaccination is cost-effective regardless of the perspective adopted, in 99 percent of simulations.

Objectives: The majority of patients in India access private sector providers for curative medical services. However, there is scanty information on the cost of treatment of critically ill patients in this setting. The study evaluates the cost and extent of financial subsidy required for patients admitted to an intensive care unit (ICU) in India.

Methods: Data on direct medical, direct nonmedical, and indirect cost were prospectively collected from critically ill patients admitted to a tertiary teaching hospital in India. Willingness-to-pay (WTP) amount was obtained from the next-of-kin following admission and the actual cost paid by the family at discharge was recorded.

Results: The main diagnoses (n = 499) were infection (26 percent) and poisoning (21 percent). The mean APACHE-II score was 13.9 (95 percent confidence interval [CI], 13.3–14.5); 86 percent were ventilated. ICU stay was 7.8 days (95 percent CI, 7.3–8.3). Hospital mortality was 27.9 percent. Direct medical cost accounted for 77 percent (US$ 2164) of the total treatment cost (US$ 2818). Indirect cost and direct nonmedical cost contributed to 19 percent (US$ 547.5) and 4 percent (US$ 106.5), respectively. Average total and daily ICU cost were US$ 1,897 and US$ 255, respectively. Although the family's WTP was 53 percent (US$ 1146; 95 percent CI, 1090–1204) of direct medical cost, their final contribution was 67.7 percent (US$ 1465; 95 percent CI, 1327–1604).

Conclusions: The cost of an ICU admission in our setting is US$ 2818. Although the family's contribution to expenses exceeded their initial WTP, a substantial subsidy (33 percent) is still required. Alternate financing strategies for the poor and optimization of ICU resources are urgently required.

Objectives: To review and compare current Health Technology Assessment (HTA) activities for medical devices (MDs) across European HTA institutions.

Methods: A comprehensive approach was adopted to identify institutions involved in HTA in European countries. We systematically searched institutional Web sites and other online sources by using a structured tool to extract information on the role and link to decision making, structure, scope, process, methodological approach, and available HTA reports for each included institution.

Results: Information was obtained from eighty-four institutions, forty-seven of which were analyzed. Fifty-four methodological documents from twenty-three agencies in eighteen countries were identified. Only five agencies had separate documents for the assessment of MDs. A few agencies made separate provisions for the assessment of MDs in their general methods. The amount of publicly available HTA reports on MDs varied by device category and agency remit.

Conclusions: Despite growing consensus on their importance and international initiatives, such as the EUnetHTA Core Model®, specific tools for the assessment of MDs are rarely developed and implemented at the national level. Separate additional signposts incorporated in existing general methods guides may be sufficient for the evaluation of MDs.

Objective: As health technology assessment (HTA) organizations in Canada and around the world seek to involve the public and patients in their activities, frameworks to guide decisions about whom to involve, through which mechanisms, and at what stages of the HTA process have been lacking. The aim of this study was to describe the development and outputs of a comprehensive framework for involving the public and patients in a government agency's HTA process.

Methods: The framework was informed by a synthesis of international practice and published literature, a dialogue with local, national and international stakeholders, and the deliberations of a government agency's public engagement subcommittee in Ontario, Canada.

Results: The practice and literature synthesis failed to identify a single, optimal approach to involving the public and patients in HTA. Choice of methods should be considered in the context of each HTA stage, goals for incorporating societal and/or patient perspectives into the process, and relevant societal and/or patient values at stake. The resulting framework is structured around four actionable elements: (i) guiding principles and goals for public and patient involvement (PPI) in HTA, (ii) the establishment of a common language to support PPI efforts, (iii) a flexible array of PPI approaches, and (iv) on-going evaluation of PPI to inform adjustments over time.

Conclusions: A public and patient involvement framework has been developed for implementation in a government agency’s HTA process. Core elements of this framework may apply to other organizations responsible for HTA and health system quality improvement.

Objectives: Rapid reviews are characterized as an accelerated evidence synthesis approach with no universally accepted methodology or definition. This modified Delphi consensus study aimed to develop a comprehensive set of defining characteristics for rapid reviews that may be used as a functional definition.

Methods: Expert panelists with knowledge in rapid reviews and evidence synthesis were identified. In the first round, panelists were asked to answer a seventeen-item survey addressing a variety of rapid review topics. Results led to the development of statements describing the characteristics of rapid reviews that were circulated to experts for agreement in a second survey round and further revised in a third round. Consensus was reached if ≥70 percent of experts agreed and there was stability in free-text comments.

Results: A panel of sixty-six experts participated. Consensus was reached on ten of eleven statements describing the characteristics of rapid reviews. According to the panel, rapid reviews aim to meet the requirements and timelines of a decision maker and should be conducted in less time than a systematic review. They use a variety of approaches to accelerate the evidence synthesis process, tailor the methods conventionally used to carry out systematic reviews, and use the most rigorous methods that the delivery time frame will allow.

Conclusions: This study achieved consensus on ten statements describing the defining characteristics of rapid reviews based on the opinion of a panel of knowledgeable experts. Areas of disagreement were also highlighted. Findings emphasize the role of the decision maker and stress the importance of transparent reporting.

Objectives: Rapid reviews can be conducted in a narrower time frame, as compared to systematic reviews, by featuring restrictions. To estimate the validity of the results, assessment of methodological quality is required. Our aim was to analyze the methodological restrictions of rapid reviews compared with systematic reviews using the AMSTAR checklist and assess its feasibility for rapid reviews.

Methods: A systematic search for literature on rapid reviews of surgical interventions was conducted in three databases: Medline, Embase, and the Cochrane library. Additionally, health technology assessment (HTA) databases were searched. We analyzed reviews using AMSTAR and additionally compared the results with those of an overview of reviews on the same topic.

Results: Items found more frequently in rapid reviews were search for gray literature (65 percent versus 33 percent), listing of excluded studies (59 percent versus 37 percent), and provision of study characteristics (77 percent versus 44 percent), whereas consideration of study quality in formulating conclusions, conduct of meta-analysis, and statement of conflicts of interest were less frequent. Median time between search and publication was 8 months, with a range between 1 and 27.

Conclusions: With some adjustments, AMSTAR can be used as a checklist for rapid reviews to describe methodological restrictions in comparison to systematic reviews and to roughly estimate the validity of the results. Strikingly, only 14.3 percent of rapid reviews were published within 3 months.

Background: Cardiovascular disease is one of the most common causes of death worldwide, with many individuals having experienced acute coronary syndrome (ACS). How patients with a history of ACS value aspects of their medical treatment have been evaluated rarely. The aim of this study was to determine patient priorities for long-term drug therapy after experiencing ACS.

Methods: To identify patient-relevant treatment characteristics, a systematic literature review and qualitative patient interviews were conducted. A questionnaire was developed to elicit patient's priorities for different characteristics of ACS treatment using Analytic Hierarchy Process (AHP). To evaluate the patient-relevant outcomes, the eigenvector method was applied.

Results: Six-hundred twenty-three patients participated in the computer-assisted personal interviews and were included in the final analysis. Patients showed a clear priority for the attribute “reduction of mortality risk” (weight: 0.402). The second most preferred attribute was the “prevention of a new myocardial infarction” (weight: 0.272), followed by “side effect: dyspnea” (weight: 0.165) and “side effect: bleeding” (weight: 0.117). The “frequency of intake” was the least important attribute (weight: 0.044).

Conclusion: In conclusion, this study shows that patients strongly value a reduction of the mortality risk in post-ACS treatment. Formal consideration of patient preferences and priorities can help to inform a patient-centered approach, clinical practice, development of future effective therapies, and health policy for decision makers that best represents the needs and goals of the patient.

Objectives: Health technology assessment (HTA) yields information that can be ideally used to address deficiencies in health systems and to create a wider understanding of the impact of different policy considerations around technology reimbursement and use. The structure of HTA programs varies across different jurisdictions according to decision-maker needs. Moreover, conducting HTA requires specialized skills. Effective decision making should include multiple criteria (medical, economic, technical, ethical, social, legal, and cultural) and requires multi-disciplinary teams of experts working together to produce these assessments. A workshop explored the multi-disciplinary skills and competencies required to build an effective and efficient HTA team, with a focus on low- and middle-income settings.

Methods: This proceeding summarizes main points from a workshop on capacity building, drawing on presentations and group discussions among attendees including different points of view.

Results and Conclusions: The workshop and thus this study would have benefited from a larger variety of stakeholders. Therefore, the conclusions arising from the workshop are not the opinion of a representative sample of HTA professionals. Nonetheless, organizations and speakers were carefully selected to provide a valuable approach to this theme. Thus, these proceedings highlight some of the gaps and needs in the education and training programs offered worldwide and calls for further investigation.

Background: Health technology assessment (HTA) examines the consequences of the application of health technologies and is aimed at better informing decision-makers. Over the past 30 years, different countries have implemented HTA organizations. Colombia established by law its own HTA agency (IETS) in 2011 which started operations in November 2012. The aim of this study was to assess the feasibility of conducting and using HTA to inform decision-making in this context. Through a qualitative approach, ten “drivers” emerged with the ability to help or hinder HTA development in this context: availability and quality of data, implementation strategy, cultural aspects, local capacity, financial support, policy/political support, globalization, stakeholder pressure, health system context, and usefulness perception.

Methods: Semi-structured interviews were conducted with key HTA researchers, after following rigorous transcription, and thematic content analysis, those aspects that may be barriers or facilitator for HTA development and use in Colombia were identified.

Results: Although HTA has become a tool to inform decision-making around the world, its use may vary according to setting. Determining those aspects which may enable or interfere with HTA development and use in Colombia may be useful for other countries when considering the establishment of HTA systems.

Conclusions: The conceptual transferability of concepts like “drivers” with caveats may be of interest for similar settings trying to incorporate HTA processes and institutions into systematic decision-making.

Objectives: In 2012, Colombia experienced an important institutional transformation after the establishment of the Health Technology Assessment Institute (IETS), the disbandment of the Regulatory Commission for Health and the reassignment of reimbursement decision-making powers to the Ministry of Health and Social Protection (MoHSP). These dynamic changes provided the opportunity to test Multi-Criteria Decision Analysis (MCDA) for systematic and more transparent resource-allocation decision-making.

Methods: During 2012 and 2013, the MCDA framework Evidence and Value: Impact on Decision Making (EVIDEM) was tested in Colombia. This consisted of a preparatory stage in which the investigators conducted literature searches and produced HTA reports for four interventions of interest, followed by a panel session with decision makers. This method was contrasted with a current approach used in Colombia for updating the publicly financed benefits package (POS), where narrative health technology assessment (HTA) reports are presented alongside comprehensive budget impact analyses (BIAs).

Results: Disease severity, size of population, and efficacy ranked at the top among fifteen preselected relevant criteria. MCDA estimates of technologies of interest ranged between 71 to 90 percent of maximum value. The ranking of technologies was sensitive to the methods used. Participants considered that a two-step approach including an MCDA template, complemented by a detailed BIA would be the best approach to assist decision-making in this context. Participants agreed that systematic priority setting should take place in Colombia.

Conclusions: This work may serve as the basis to the MoHSP on its interest of setting up a systematic and more transparent process for resource-allocation decision-making.

Objectives: The Medical Services Advisory Committee (MSAC) makes recommendations to the Australian Government for funding health technologies under the Medicare Benefits Schedule (MBS). Differences in public, clinical, commercial, and political opinions on health expenditure emphasize the importance of defensible funding decisions. We aimed to evaluate the quality of health technology assessment (HTA) reports over time and among health technologies assessed for MSAC.

Main Outcome Measures: A cohort study was performed of HTA reports prepared for MSAC between 1998 and 2013. We measured the quality of HTA reports using reporting guidelines proposed by the European Collaboration for Assessment of Health Interventions. Individual component scores across eleven domains were calculated, and summed for an overall aggregate score. We used linear regression to investigate any change in quality over time and among the types of technologies assessed.

Results: We included 110 HTA reports. The safety (80 percent), effectiveness (84 percent), economic (74 percent), and organizational (99 percent) domains were better reported than the psychological, social, and ethical considerations (34 percent). The basic (75 percent), methodological (62 percent), background (82 percent), contextual (46 percent), status quo (54 percent), and technical information (66 percent) that framed each assessment were inconsistently reported. On average, overall quality scores increased by 2 percent (p < 0.001) per year, from approximately 60 percent to 80 percent over the 15-year period, with no significant difference among surgical, diagnostic or other nonpharmaceutical health technologies (p = 0.22).

Conclusions: HTA reports prepared for MSAC are a key tool in allocating scarce health resources. The overall quality of these reports has improved, but the reporting of specific domains and subthemes therein could be better addressed.

As strong advocates and adopters of early cost-effectiveness modeling for test evaluation, we were excited to see the recent article “The Early Bird Catches the Worm: Early Cost-Effectiveness Analysis of New Medical Tests,” in which Buisman and colleagues present a succinct and generalizable framework for conducting this type of analysis. The authors have done an excellent job in identifying and summarizing the key components of this methodology, as well as highlighting fundamental differences between early analyses versus the traditional late-stage approach. As the number of medical tests coming to market continues to grow, there is an ever growing need to develop rigorous methodology in this area, and we commend the authors for this timely work.