Identification, Review, and Use of Health State Utilities in Cost-Effectiveness Models: An ISPOR Good Practices for Outcomes Research Task Force Report.

Cost-effectiveness models that present results in terms of cost per quality-adjusted life-year for health technologies are used to inform policy decisions in many parts of the world. Health state utilities (HSUs) are required to calculate the quality-adjusted life-years. Even when clinical studies assessing the effectiveness of health technologies collect data on HSUs to populate a cost-effectiveness model, which rarely happens, analysts typically need to identify at least some additional HSUs from alternative sources. When possible, HSUs are identified by a systematic review of the literature, but, again, this rarely happens. In 2014, ISPOR established a Good Practices for Outcome Research Task Force to address the use of HSUs in cost-effectiveness models. This task force report provides recommendations for researchers who identify, review, and synthesize HSUs for use in cost-effectiveness models; analysts who use the results in models; and reviewers who critically appraise the suitability and validity of the HSUs selected for use in models. The associated Minimum Reporting Standards of Systematic Review of Utilities for Cost-Effectiveness checklist created by the task force provides criteria to judge the appropriateness of the HSUs selected for use in cost-effectiveness models and is suitable for use in different international settings.

Adaptive Evolution in Rapid Assessments: A 25-Year Perspective.

OBJECTIVES: This article retrospectively examines the evolution of rapid assessments (RAs) produced by the Health Technology Assessment (HTA) Program at the Institute of Health Economics over its 25-year relationship with a single requester, the Alberta Health Ministry (AHM). METHODS: The number, types, and methodological attributes of RAs produced over the past 25 years were reviewed. The reasons for developmental changes in RA processes and products over time were charted to document the push-pull tension between AHM needs and the HTA Program's drive to meet those needs while responding to changing methodological benchmarks. RESULTS: The review demonstrated the dynamic relationship required for HTA researchers to meet requester needs while adhering to good HTA practice. The longstanding symbiotic relationship between the HTA Program and the AHM initially led to increased diversity in RA types, followed by controlled extinction of the less fit (useful) "transition species." Adaptations in RA methodology were mainly driven by changes in best practice standards, requester needs, the healthcare environment, and staff expertise and technology. CONCLUSIONS: RAs are a useful component of HTA programs. To remain relevant and useful, RAs need to evolve according to need within the constraints of HTA best practice.

Scientific Development of HTA-A Proposal by the Health Technology Assessment International Scientific Development and Capacity Building Committee.

OBJECTIVES: To report from the Scientific Development and Capacity Building Committee of Health Technology Assessment International (HTAi) on activities that are being undertaken within HTAi regarding the promotion of scientific rigor in the field of health technology assessment (HTA). METHODS: Retrieval of definitions of HTA that the SDCB committee considered reflective of the current practice of HTA, followed by a narrative synthesis of the core components of HTA. RESULTS: Several definitions of HTA have been provided, all sharing the notion that HTA is the formal, systematic, and transparent inquiry into the meaning and value, broadly defined, of health technologies, when used in specific patient populations.Many frameworks and tools have been developed for assessing the quality of specific tasks that may be conducted in the context of HTA. Collating such frameworks and tools is likely to be helpful in developing standards and in providing guidance as to how the scientific quality of HTA may be secured. Two current trends in HTA were noted: a stronger health systems focus, and the need to involve stakeholders throughout the HTA process. A wider systems' perspective requires that plausible alternative scenarios are being developed, and wide consultation of various stakeholders is a prerequisite to the development of such scenarios with data from various sources. CONCLUSIONS: Current trends in HTA will lead to different demands on the HTA expert. The task of this emerging policy professional would be not just to provide technical information for problem-solving, but also to combine it with a new function of facilitating public deliberation and learning.

Building Synergy between Regulatory and HTA Agencies beyond Processes and Procedures-Can We Effectively Align the Evidentiary Requirements? A Survey of Stakeholder Perceptions.

OBJECTIVES: To evaluate the current practice of companies and agencies to assess the changes made in aligning regulatory and health technology assessment (HTA) stakeholders; to identify areas of commonality of evidentiary requirements that could occur; and to identify strategic issues and trends of regulatory and HTA synergy. METHODS: Two separate questionnaires were developed to assess stakeholders' perceptions on regulatory and HTA alignment, one for pharmaceutical companies and the other for regulatory and HTA agencies. The responses were analyzed using descriptive statistics. RESULTS: Seven regulatory and 8 HTA agencies from Australia, Canada, and Europe and 19 international companies developing innovative medicine responded to the survey. This study provided a snapshot of the current regulatory and HTA landscape. Changes made over the past 5 years were reflected in three main areas: there is an increasing interaction between regulatory and HTA agencies; current conditional regulatory approvals are not always linked with flexible HTA approaches; and companies are more supportive of joint scientific advice. Four types of evidentiary requirements were identified as building blocks for better alignment: acceptable primary end points, inclusion of an active comparator, use of patient-reported outcomes, and choice and use of surrogate end point. CONCLUSIONS: The study showed that the gap between regulatory and HTA requirements has narrowed over the past 5 years. All respondents supported synergy between regulatory and HTA stakeholders, and the study provided several recommendations on how to further improve evidentiary alignment including the provision of joint scientific advice, which was rated as a key strategy by both agencies and companies.

UK medicines regulation: responding to current challenges.

The medicines regulatory environment is evolving rapidly in response to the changing environment. Advances in science and technology have led to a vast field of increasingly complicated pharmaceutical and medical device products; increasing globalization of the pharmaceutical industry, advances in digital technology and the internet, changing patient populations, and shifts in society also affect the regulatory environment. In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) regulates medicines, medical devices and blood products to protect and improve public health, and supports innovation through scientific research and development. It works closely with other bodies in a single medicines network across Europe and takes forward UK health priorities. This paper discusses the range of initiatives in the UK and across Europe to support innovation in medicines regulation. The MHRA leads a number of initiatives, such as the Innovation Office, which helps innovators to navigate the regulatory processes to progress their products or technologies; and simplification of the Clinical Trials Regulations and the Early Access to Medicines Scheme, to bring innovative medicines to patients faster. The Accelerated Access Review will identify reforms to accelerate access for National Health Service patients to innovative medicines and medical technologies. PRIME and Adaptive Pathways initiatives are joint endeavours within the European regulatory community. The MHRA runs spontaneous reporting schemes and works with INTERPOL to tackle counterfeiting and substandard products sold via the internet. The role of the regulator is changing rapidly, with new risk-proportionate, flexible approaches being introduced. International collaboration is a key element of the work of regulators, and is set to expand.

New trends and challenges in the European regulation of innovative medicines.

Regulators' marketing authorizations for innovative medicines are linked into a complex process with successive crucial decisions. Objectives and decision criteria of the stakeholders in this process, e.g. health technology assessment (HTA) bodies, payers, physicians and patients, vary and may result not only in different but even mutually exclusive requirements. Reacting to changes in scientific, economic and social demands, European regulatory agencies alter content and format of their assessment procedures and their communication. New diagnostic options (e.g. genotyping and biomarkers) and pharmaceutical innovations (e.g. targeted medicines, nanomedicines) are the scientific drivers of this development. Social drivers are the price and reimbursement decisions by HTA bodies and payers, prerequisites for most patients' access to innovative medicines. The European Medicines Agency's adaptive licensing concept and priority medicines scheme foster the early authorization of innovative medicines. HTA builds on regulators' assessment, with additional requirements and economic components. An intensified exchange between all stakeholders, e.g. in multilateral scientific advice procedures has been initiated. Diminishing the differences in the requirements of regulators and HTA bodies is in the best interest of both patients and the pharmaceutical industry, avoiding duplication of work and accelerating patients' access by early decisions on price and reimbursement.

Decision-Oriented Health Technology Assessment: One Step Forward in Supporting the Decision-Making Process in Hospitals.

OBJECTIVES: This article outlines the Decision-Oriented Health Technology Assessment: a new implementation of the European network for Health Technology Assessment Core Model, integrating the multicriteria decision-making analysis by using the analytic hierarchy process to introduce a standardized methodological approach as a valued and shared tool to support health care decision making within a hospital. METHODS: Following the Core Model as guidance (European network for Health Technology Assessment. HTA core model for medical and surgical interventions. Available from: http://www.eunethta.eu/outputs/hta-core-model-medical-and-surgical-interventions-10r. [Accessed May 27, 2014]), it is possible to apply the analytic hierarchy process to break down a problem into its constituent parts and identify priorities (i.e., assigning a weight to each part) in a hierarchical structure. Thus, it quantitatively compares the importance of multiple criteria in assessing health technologies and how the alternative technologies perform in satisfying these criteria. The verbal ratings are translated into a quantitative form by using the Saaty scale (Saaty TL. Decision making with the analytic hierarchy process. Int J Serv Sci 2008;1:83-98). An eigenvectors analysis is used for deriving the weights' systems (i.e., local and global weights' system) that reflect the importance assigned to the criteria and the priorities related to the performance of the alternative technologies. RESULTS: Compared with the Core Model, this methodological approach supplies a more timely as well as contextualized evidence for a specific technology, making it possible to obtain data that are more relevant and easier to interpret, and therefore more useful for decision makers to make investment choices with greater awareness. CONCLUSIONS: We reached the conclusion that although there may be scope for improvement, this implementation is a step forward toward the goal of building a "solid bridge" between the scientific evidence and the final decision maker's choice.

CHANGING FACE OF MEDICARE'S NATIONAL COVERAGE DETERMINATIONS FOR TECHNOLOGY.

OBJECTIVES: The Centers for Medicare and Medicaid Services (CMS) issues National Coverage Determinations (NCDs) for medical interventions expected to have a significant impact on Medicare, the health insurance program for US citizens aged 65 years and older and certain people with disabilities under the age of 65 years. The objective of this study was to evaluate NCDs issued from 1999 to 2013 to identify key trends, and to discuss implications for future CMS policy. METHODS: We used the Tufts Medical Center Medicare National Coverage Determination Database to examine characteristics of NCDs from 1999 through 2013. We examined various characteristics of NCDs, including: whether the intervention under review is used for prevention or treatment of disease, the type of intervention considered, evidence limitations cited by CMS, and coverage determination outcome. We evaluated longitudinal trends in categorical and continuous variables in the database, using Cochran-Armitage trend tests and linear regression, respectively. RESULTS: We found that NCDs increasingly focus on preventive care (p = 0.072), pertain to diagnostic imaging (p = 0.033), and evaluate health education/behavioral therapy interventions (p = 0.051). CMS increasingly cites the lack of relevant outcomes (p = 0.019) and the lack of applicability of study results to the Medicare population (p < 0.001) as evidence limitations. CMS less often restricts coverage to certain population subgroups in NCDs (p < 0.001), but increasingly applies coverage with evidence development policies (p < 0.001). CONCLUSIONS: Identified trends reflect broader changes in Medicare as CMS shifts its focus from treatment to prevention of disease, addresses potentially overutilized technologies, and attempts to issue flexible coverage policies.

Health Technology Assessment in the UK.

In this Review, we discuss the UK's Health Technology Assessment programme, which is 20 years old in 2013. We situate the programme in the context of the UK landscape for evidence-based medicine, including in relation to the National Institute for Health and Care Excellence and as guidance to the National Health Service. We identify features that might be of value to other health systems as they confront the challenges of rapid innovation and rising costs. We use examples of recent studies to show the strengths and weaknesses of the programme.

[The Health Technology Assessment Engine of the Academic Hospital of Udine: first appraisal]. / Il motore di ricerca Health Technology Assessment dell'Azienda Ospedaliero-Universitaria di Udine: prime valutazioni.

The Health Technology Assessment Engine (HTAE) of the Academic Hospital of Udine aggregates about one hundred of health technology assessment websites. It was born thanks to Google technology in 2008 and after about four years of testing it became public for everybody from the Homepage of the Italian Society of Health Technology Assessment (SIHTA). In this paper the first results obtained with this resource are reported. The role of the scientific librarian is examined not only as a support specialist in bibliographic search but also as a creative expert in managing new technologies for the community.

Novel challenges of multi-society investigator-initiated studies: a paradigm shift for technique and technology evaluation.

The introduction of innovative techniques and novel technologies into clinical practice is a challenge that confronts all aspects of healthcare delivery. Upheaval from shrinking research funding and declining healthcare reimbursements now forces patients, doctors, hospitals, payers, regulators, and even health systems into conflict as new therapies struggle to find a place in the therapeutic armamentarium. The escalating costs of healthcare force all parties to consider both the medical risks/benefits as well as the economic efficiency of proposed tools and therapies. We highlight these challenges by examining the process of initiating and conducting a "society-as-investigator" clinical trial to assess the safety of the natural orifice translumenal endoscopic surgery (NOTES) approach to cholecystectomy in the context of the issues that confront technology diffusion today.

Current status and future prospects for cultured limbal tissue transplants in Australia and New Zealand.

Cultured limbal tissue transplants have become widely used over the last decade as a treatment for limbal stem cell deficiency (LSCD). While the number of patients afflicted with LSCD in Australia and New Zealand is considered to be relatively low, the impact of this disease on quality of life is so severe that the potential efficacy of cultured transplants has necessitated investigation. We presently review the basic biology and experimental strategies associated with the use of cultured limbal tissue transplants in Australia and New Zealand. In doing so, we aim to encourage informed discussion on the issues required to advance the use of cultured limbal transplants in Australia and New Zealand. Moreover, we propose that a collaborative network could be established to maintain access to the technology in conjunction with a number of other existing and emerging treatments for eye diseases.

Making processes reliable: a validated pubmed search strategy for identifying new or emerging technologies.

BACKGROUND AND OBJECTIVES: Horizon scanning systems need to handle a wide range of sources to identify new or emerging health technologies. The objective of this study is to develop a validated Medline bibliographic search strategy (PubMed search engine) to systematically identify new or emerging health technologies. METHODS: The proposed Medline search strategy combines free text terms commonly used in article titles to denote innovation within index terms that make reference to the specific fields of interest. Efficacy was assessed by running the search over a period of 1 year (2009) and analyzing its retrieval performance (number and characteristics). For comparison purposes, all article abstracts published during 2009 in six preselected key research journals and eight high impact surgery journals were scanned. Sensitivity was defined as the proportion of relevant new or emerging technologies published in key journals that would be identified in the search strategy within the first 2 years of publication. RESULTS: The search yielded 6,228 abstracts of potentially new or emerging technologies. Of these, 459 were classified as new or emerging (383 truly new or emerging and 76 new indications). The scanning of 12,061 journal abstracts identified 35 relevant new or emerging technologies. Of these, twenty-nine were located within the Medline search strategy during the first 2 years of publication (sensitivity = 83 percent). CONCLUSIONS: The current search strategy, validated against key journals, has demonstrated to be effective for horizon scanning. Even though it can require adaptations depending on the scope of the horizon scanning system, it could serve to simplify and standardize scanning processes.

Trends in telemedicine assessment indicate neglect of key criteria for predicting success.

PURPOSE: This innovative analysis aims to quantify the use of evaluation criteria in telemedicine and to identify current trends in metric adoption. The focus is to determine the frequency of actual performance metric reporting in telemedicine evaluation, in contrast to systematic reviews where assessment of study quality is the goal. DESIGN/METHODOLOGY/APPROACH: Automated literature search identified telemedicine studies reporting quantitative performance metrics. Studies were classified by telemedicine class; store-and-forward (SAF), real-time consultation (RTC) and telecare (TC), and study stage. Studies were scanned for evaluation metric reporting, i.e. clinical outcomes, satisfaction, patient quality and cost measures. FINDINGS: Evaluation metric use was compared among telemedicine classes, and between pilot and routine use stages. Diagnostic accuracy was reported significantly more frequently in pilots for RTC and TC. Cost measures were more frequently reported in routine use for TC. Clinical effectiveness and hospital attendance were better reported in routine use for SAF. Comparison also revealed different evaluation strategies. In pilots, SAF favoured diagnostic accuracy, compared to RTC and TC. TC preferred clinical effectiveness evaluations and TC more frequently assessed patient satisfaction. Cost was only reported in less than 20 per cent of studies, but most frequently in RTC. Routine use led to increased reporting of all metrics, except diagnostic accuracy. Clinical effectiveness reporting increased significantly with routine use for RTC and SAF, but declined for TC. ORIGINALITY/VALUE: Clinical outcomes and patient satisfaction were reported frequently in telemedicine studies, but reporting of other performance metrics was rare. Understanding current trends in metric reporting will facilitate better design of future telemedicine evaluations.

Development of the Birmingham Rheumatoid Arthritis Model: past, present and future plans.

The Birmingham Rheumatoid Arthritis Model (BRAM) has been developed over a number of years to inform several appraisals of biologic drugs by the Technology Appraisals Committee of the UK National Institute for Health and Clinical Excellence. This article describes the processes used in the construction of the different versions of the BRAM.

Health technology assessment: what is it? Current status and perspectives in the field of electrophysiology.

Health technology assessment (HTA) is the multidisciplinary field of policy analysis that studies medical, social, ethical, and economic implications of the development, diffusion, and use of health technologies. Its worldwide diffusion needs to be understood in the context of evidence-based healthcare delivery policy, and it is strongly driven by the search for new cost-containment policies by the governments of universal healthcare systems. This article presents the three main pillars of HTA: evaluating comparative effectiveness, cost-effectiveness, and organizational impact. While comparative analysis is more familiar to cardiologists, cost-effectiveness and organizational studies are less widely known because they expand the perspective of the evaluation to institutional settings and society at large and require significant inter-disciplinary work. Sound economic and organizational studies that extend comparative effectiveness studies may facilitate dialogue between medical science and policymaking.

Guidance for future HTA applications to vaccines: the HPV lesson.

The HTA is a multidisciplinary approach which is increasingly applied worldwide in order to support decision-makers in the introduction of health technologies. The application of HTA to vaccines is quite recent and linked to the increasing number of vaccines available or in development, especially to those intended to non pediatric immunization. In this article a description of the HTA approach used in the evaluation of the bivalent HPV vaccine in Italy is provided; moreover the reasons for keeping HTA process "alive" and identifying new or pending Public Health issues are discussed. In fact, the project raised questions and challenges about the standardization and sharing of HTA methods in Italy. Recently the need of updating the results is starting to be seen as urgent due to the evolution of scientific knowledge, the availability of the first results after technology introduction and the observed differences in vaccination strategies among Italian regions. Moreover in these last two years after the completion of the HTA project a number of new issues have risen in the Italian context as immunization rates, regional vaccination strategies, processes for vaccine selection and health economic issues.

Health technology assessment in the era of personalized health care.

OBJECTIVES: This article examines the challenges for health technology assessment (HTA) in the light of new developments of personalized health care, focusing on European HTA perspectives. METHODS: Using the example of the Integrated Genome Research Network - Mutanom (IG Mutanom) project, with focus on personalized cancer diagnostics and treatment, we assess the scope of current HTA and examine it prospectively in the context of the translation of basic and clinical research into public health genomics and personalized health care. RESULTS: The approaches developed within the IG-Mutanom project are based on innovative technology potentially providing targeted therapies for cancer; making translation into clinical practice requires a novel course of action, however. New models of HTA are needed that can account for the unique types of evidence inherent to individualized targeted therapies. Using constructive health technology assessment (CTA) models is an option, but further suitable models should be developed. CONCLUSIONS: Integrative, systems biology-based approaches toward personalized medicine call for novel assessment methods. The translation of their highly innovative technologies into the practice of health care requires the development of new HTA concepts.

[Forecasting medical technologies--a global overview].

Forecasting new medical technologies is a crucial stage in the process of decision-making in health care systems on national, organizational, professional and personal levels. Knowing what is on the horizon is essential. It is a tool facilitating preparedness and planning for updating health care in the western world. The challenge is to identify new promising technologies at an early stage. This is due to the uncertainty in estimating developing trends and consequences (clinical, financial, political, legal, social and ethical). A balance must be found between the desire to adopt new emerging technologies and the necessity for accountability n basing decisions on efficient evidence. Scarce resources, pervading health systems everywhere, emphasize the need for this mechanism to justify and improve health system determinations. Planning for the future has expanded into new medical fields, thereby reinforcing the importance of national forecasting bodies. This article presents the basic terminology and principles of medical technology forecasting and reviews the agencies involved in early warning systems including Israel.