How innovation can be defined, evaluated and rewarded in health technology assessment

Abstract Background What constitutes innovation in health technologies can be defined and measured in a number of ways and it has been widely researched and published about. However, while many countries mention it as a criterion for pricing or reimbursement of health technologies, countries differ widely in how they define and operationalise it. Methods We performed a literature review, using a snowballing search. In this paper, we explore how innovation has been defined in the literature in relation to health technology assessment. We also describe how a selection of countries (England, France, Italy, Spain and Japan) take account of innovation in their health technology assessment frameworks and explore the key methodologies that can capture it as a dimension of value in a new health technology. We propose a way of coming to, and incorporating into health technology assessment systems, a definition of innovation for health technologies that is independent of other dimensions of value that they already account for in their systems, such as clinical benefit. We use Spain as an illustrative example of how innovation might be operationalised as a criterion for decision making in health technology assessment. Results The countries analysed here can be divided into 2 groups with respect to how they define innovation. France, Japan and Italy use features such as severity, unmet need and therapeutic added value as indicators of the degree of innovation of a health technology, while England, Spain consider the degree of innovation as a separate and additional criterion from others. In the case of Spain, a notion of innovation might be constructed around concepts of `step-change’, `convenience’, `strength of evidence base’ and `impact on future research & development’. Conclusions If innovation is to be used as operational criteria for adoption, pricing and reimbursement of health technologies, the concept must be clearly defined, and it ought to be independent from other value dimensions already captured in their health technology assessment systems.

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COMPREHENSIVE ASSESSMENT OF COMPLEX TECHNOLOGIES: INTEGRATING VARIOUS ASPECTS IN HEALTH TECHNOLOGY ASSESSMENT

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462317000678 ◽

2017 ◽

Vol 33 (5) ◽

pp. 570-576 ◽

Cited By ~ 1

Author(s):

Kristin Bakke Lysdahl ◽

Kati Mozygemba ◽

Jacob Burns ◽

Jan Benedikt Brönneke ◽

James B. Chilcott ◽

...

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Stakeholder Involvement ◽

Complex Interventions ◽

Health Technology ◽

Legal Aspects ◽

Added Value ◽

Health Technologies ◽

Stakeholder Perspectives ◽

Wide Range

Objectives: Despite recent development of health technology assessment (HTA) methods, there are still methodological gaps for the assessment of complex health technologies. The INTEGRATE-HTA guidance for effectiveness, economic, ethical, socio-cultural, and legal aspects, deals with challenges when assessing complex technologies, such as heterogeneous study designs, multiple stakeholder perspectives, and unpredictable outcomes. The objective of this article is to outline this guidance and describe the added value of integrating these assessment aspects.Methods: Different methods were used to develop the various parts of the guidance, but all draw on existing, published knowledge and were supported by stakeholder involvement. The guidance was modified after application in a case study and in response to feedback from internal and external reviewers.Results: The guidance consists of five parts, addressing five core aspects of HTA, all presenting stepwise approaches based on the assessment of complexity, context, and stakeholder involvement. The guidance on effectiveness, health economics and ethics aspects focus on helping users choose appropriate, or further develop, existing methods. The recommendations are based on existing methods’ applicability for dealing with problems arising with complex interventions. The guidance offers new frameworks to identify socio-cultural and legal issues, along with overviews of relevant methods and sources.Conclusions: The INTEGRATE-HTA guidance outlines a wide range of methods and facilitates appropriate choices among them. The guidance enables understanding of how complexity matters for HTA and brings together assessments from disciplines, such as epidemiology, economics, ethics, law, and social theory. This indicates relevance for a broad range of technologies.

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Health Technology Assessment in Health Economics

Handbook of Research on Information Technology Management and Clinical Data Administration in Healthcare ◽

10.4018/978-1-60566-356-2.ch005 ◽

2011 ◽

pp. 69-86

Author(s):

Steven Simoens

Keyword(s):

Health Economics ◽

Health Technology Assessment ◽

Technology Assessment ◽

Health Expenditure ◽

Health Technology ◽

Decision Makers ◽

Added Value ◽

Healthcare Sector ◽

Scarce Resources ◽

Health Technologies

This chapter introduces health technology assessment and health economics as tools for decision makers to allocate scarce resources in the healthcare sector. It argues that information about the safety, efficacy and effectiveness, organizational implications, social and ethical consequences, legal considerations, and health economic aspects of the application of a health technology needs to be taken into account with a view to informing decisions about the registration and reimbursement of a health technology. Also, the author hopes that understanding the methodology and use of health technology assessment and health economics will persuade the reader of the added value of such studies and promote the application of health technologies that support further health improvements, whilst containing health expenditure.

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Disinvestment Activities and Candidates in the Health Technology Assessment Community: An Online Survey

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462319000229 ◽

2019 ◽

Vol 35 (03) ◽

pp. 189-194

Author(s):

Julie Polisena ◽

Gerhard Trunk ◽

Iñaki Gutierrez-Ibarluzea ◽

Roberta Joppi

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Online Survey ◽

Health Technology ◽

Future Research ◽

International Network ◽

Health Technologies ◽

Inappropriate Use ◽

Services Delivery ◽

Survey Results

AbstractObjectivesAs healthcare decision makers continue to face challenges in health services delivery to their patients, disinvestment programs are being established for a sustainable healthcare system. This study aimed to collect data and information by means of a survey of disinvestment candidates and ongoing disinvestment projects in the health technology assessment (HTA) community.MethodsAn online survey was conducted to collect information on disinvestment candidates and activities from members of the Health Technology Assessment International Disinvestment & Early Awareness Interest Group, the EuroScan International Network and International Network of Agencies for Health Technology Assessment.ResultsAmong the 362 invitees, twenty-four unique responses were received, and almost 70 percent were involved in disinvestment initiatives. The disinvestment candidates identified represented a range of health technologies. Evidence or signaling of clinical ineffectiveness or inappropriate use typically led to the nomination of disinvestment candidates. Health technology assessments and reassessments were usually conducted to evaluate the technology in question, and decisions usually led to the limited use of the technology. Barriers to disinvestment decisions included the strength of interest and advocacy groups, insufficient data for assessments, a systematic decision process and political challenges, while obstacles to their implementation were clinicians’ reluctance and insufficient funding and incentives.ConclusionsThe survey results suggested that disinvestment activities are occurring in the HTA community, especially in the public sector. Future research can further investigate the processes and methods used to reach and implement disinvestment decisions from our survey respondents and explore to form closer ties between the HTA and clinical communities.

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Differences among formulary submission guidelines: Implications for health technology assessment

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462311000274 ◽

2011 ◽

Vol 27 (3) ◽

pp. 261-270 ◽

Cited By ~ 10

Author(s):

Josephine Mauskopf ◽

Jeffrey Walter ◽

Julie Birt ◽

Lee Bowman ◽

Catherine Copley-Merriman ◽

...

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Health Technology ◽

Evidence Base ◽

Health Technologies ◽

Health Value ◽

Literature Searches ◽

Bodies Of Evidence ◽

Study Designs ◽

Selection Of

Objectives: This article provides a detailed understanding of the differences in selected formulary submission guidelines supplied by various health technology assessment (HTA) agencies and indicates how these differences can impact the evidence base used to populate the HTA.Methods: Detailed summaries of the recommended methods for evidence generation, organized by topic areas relevant for clinical and economic data, for twelve countries in Europe, North America, and Australia where HTA processes are well developed were prepared. Using these summaries, we provide examples of the likely impact these differences in recommended methods could have on the evidence base used to evaluate new health technologies.Results: Areas where recommendations differed included methodologies for systematic literature reviews (e.g., preferred databases and study designs for inclusion); selection of appropriate comparators; guidance on critical appraisal and synthesis of clinical evidence; appropriate sources for health value measures, resource use, and cost data; and approaches to uncertainty analyses. Performing literature searches that capture all relevant studies and then creating subsets of the literature based on a listing of country-specific requirements could allow for direct comparison of the evidence bases associated with the different guidelines.Conclusions: If the formulary submission guidelines were followed as written, different (although overlapping) bodies of evidence likely would be generated for each country, which could contribute to disparate assessments and recommendations. This comparison of the formulary submission guidelines could contribute to an understanding of why clinical and reimbursement decisions vary across countries.

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Health Technology Assessment in Health Economics

Health Information Systems ◽

10.4018/978-1-60566-988-5.ch020 ◽

2011 ◽

pp. 297-314 ◽

Cited By ~ 3

Author(s):

Steven Simoens

Keyword(s):

Health Economics ◽

Health Technology Assessment ◽

Technology Assessment ◽

Health Expenditure ◽

Health Technology ◽

Decision Makers ◽

Added Value ◽

Healthcare Sector ◽

Scarce Resources ◽

Health Technologies

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PP228 Strategy For Including Information On The Research Priorities Of Patients And Experts In Health Technology Assessment Reports

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462321001379 ◽

2021 ◽

Vol 37 (S1) ◽

pp. 29-29

Author(s):

Beatriz León-Salas ◽

Ana Toledo-Chávarri ◽

Yolanda Ramallo-Fariña ◽

Claudia Morales-Manrique ◽

Francisco Rivas-Ruiz ◽

...

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

National Health System ◽

Research Priorities ◽

Health Technology ◽

Future Research ◽

List Type ◽

Research Needs ◽

Knowledge Gaps ◽

Health Technologies

IntroductionThe process of health technology assessment (HTA) is an opportunity to identify gaps in the existing knowledge on the technology assessed. In January 2020, the Evaluation Unit of the Canary Islands Health Service, belonging to the Spanish Network of Agencies for Assessing National Health System Technologies and Performance, started a structured strategy to include in its HTA reports information on research priorities for the assessed technologies from the perspective of patients, clinicians, and researchers. The aim of this poster is to explain the methodology behind this strategy.MethodsThe following three-step process was proposed: (i)Identify the knowledge gaps regarding the technology from systematic reviews on effectiveness, safety, and cost effectiveness, in addition to ethical, patient, social, legal, and organizational aspects analyzed for the HTA report;(ii)Search for specific studies on research needs regarding the technology on the websites of specialized initiatives such as the Core Outcome Measures in Effectiveness Trials (COMET) and the James Lind Alliance; and(iiii)If no needs are identified in the previous two steps, consult a group of clinicians or researchers and patients related to the technology being assessed with two online survey rounds using the Delphi method. The first round identifies the research needs and the second round prioritizes the identified needs.ResultsSince the methodology to identify future research needs during a HTA process was designed, it has been used in three HTA reports during 2020 with satisfactory results. Only one of the three reports required a Delphi study.ConclusionsThe proposed strategy provides a comprehensive list of knowledge gaps on health technologies that need to be addressed in the near future from the point of view of patients, clinicians, and researchers.

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Development of a checklist to guide equity considerations in health technology assessment

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462320002275 ◽

2021 ◽

Vol 37 ◽

Author(s):

Maria Benkhalti ◽

Manuel Espinoza ◽

Richard Cookson ◽

Vivian Welch ◽

Peter Tugwell ◽

...

Keyword(s):

Knowledge Translation ◽

Health Technology Assessment ◽

Priority Setting ◽

Technology Assessment ◽

Literature Search ◽

Health Inequities ◽

Health Technology ◽

Decision Makers ◽

Added Value ◽

Pilot Test

Abstract Objectives Health technology assessment (HTA) can impact health inequities by informing healthcare priority-setting decisions. This paper presents a novel checklist to guide HTA practitioners looking to include equity considerations in their work: the equity checklist for HTA (ECHTA). The list is pragmatically organized according to the generic HTA phases and can be consulted at each step. Methods A first set of items was based on the framework for equity in HTA developed by Culyer and Bombard. After rewording and reorganizing according to five HTA phases, they were complemented by elements emerging from a literature search. Consultations with method experts, decision makers, and stakeholders further refined the items. Further feedback was sought during a presentation of the tool at an international HTA conference. Lastly, the checklist was piloted through all five stages of an HTA. Results ECHTA proposes elements to be considered at each one of the five HTA phases: Scoping, Evaluation, Recommendations and Conclusions, Knowledge Translation and Implementation, and Reassessment. More than a simple checklist, the tool provides details and examples that guide the evaluators through an analysis in each phase. A pilot test is also presented, which demonstrates the ECHTA's usability and added value. Conclusions ECHTA provides guidance for HTA evaluators wishing to ensure that their conclusions do not contribute to inequalities in health. Several points to build upon the current checklist will be addressed by a working group of experts, and further feedback is welcome from evaluators who have used the tool.

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Estimating an EQ-5D-3L Value Set for Romania Using Time Trade-Off

International Journal of Environmental Research and Public Health ◽

10.3390/ijerph18147415 ◽

2021 ◽

Vol 18 (14) ◽

pp. 7415

Author(s):

Marian Sorin Paveliu ◽

Elena Olariu ◽

Raluca Caplescu ◽

Yemi Oluboyede ◽

Ileana-Gabriela Niculescu-Aron ◽

...

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Health Technology ◽

Assessment Process ◽

Computer Assisted ◽

Health State ◽

Health States ◽

Value Set ◽

Health Technologies ◽

Related Quality

Objective: To provide health-related quality of life (HRQoL) data to support health technology assessment (HTA) and reimbursement decisions in Romania, by developing a country-specific value set for the EQ-5D-3L questionnaire. Methods: We used the cTTO method to elicit health state values using a computer-assisted personal interviewing approach. Interviews were standardized following the most recent version of the EQ-VT protocol developed by the EuroQoL Foundation. Thirty EQ-5D-3L health states were randomly assigned to respondents in blocks of three. Econometric modeling was used to estimate values for all 243 states described by the EQ-5D-3L. Results: Data from 1556 non-institutionalized adults aged 18 years and older, selected from a national representative sample, were used to build the value set. All tested models were logically consistent; the final model chosen to generate the value set was an interval regression model. The predicted EQ-5D-3L values ranged from 0.969 to 0.399, and the relative importance of EQ-5D-3L dimensions was in the following order: mobility, pain/discomfort, self-care, anxiety/depression, and usual activities. Conclusions: These results can support reimbursement decisions and allow regional cross-country comparisons between health technologies. This study lays a stepping stone in the development of a health technology assessment process more driven by locally relevant data in Romania.

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Real-world evidence: perspectives on challenges, value, and alignment of regulatory and national health technology assessment data collection requirements

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462321000131 ◽

2021 ◽

Vol 37 ◽

Author(s):

Hannah Sievers ◽

Angelika Joos ◽

Mickaël Hiligsmann

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Real World ◽

Health Technology ◽

Practical Experience ◽

Added Value ◽

Real World Data ◽

Conflicting Demands ◽

Real World Evidence ◽

Semistructured Interviews

Abstract Objective This study aims to assess stakeholder perceptions on the challenges and value of real-world evidence (RWE) post approval, the differences in regulatory and health technology assessment (HTA) real-world data (RWD) collection requirements under the German regulation for more safety in drug supply (GSAV), and future alignment opportunities to create a complementary framework for postapproval RWE requirements. Methods Eleven semistructured interviews were conducted purposively with pharmaceutical industry experts, regulatory authorities, health technology assessment bodies (HTAbs), and academia. The interview questions focused on the role of RWE post approval, the added value and challenges of RWE, the most important requirements for RWD collection, experience with registries as a source of RWD, perceptions on the GSAV law, RWE requirements in other countries, and the differences between regulatory and HTA requirements and alignment opportunities. The interviews were recorded, transcribed, and translated for coding in Nvivo to summarize the findings. Results All experts agree that RWE could close evidence gaps by showing the actual value of medicines in patients under real-world conditions. However, experts acknowledged certain challenges such as: (i) heterogeneous perspectives and differences in outcome measures for RWE generation and (ii) missing practical experience with RWD collected through mandatory registries within the German benefit assessment due to an unclear implementation of the GSAV. Conclusions This study revealed that all stakeholder groups recognize the added value of RWE but experience conflicting demands for RWD collection. Harmonizing requirements can be achieved through common postlicensing evidence generation (PLEG) plans and joint scientific advice to address uncertainties regarding evidence needs and to optimize drug development.

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Determinants of Managed Entry Agreements in the context of Health Technology Assessment: a comparative analysis of oncology therapies in four countries

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462321000039 ◽

2021 ◽

Vol 37 ◽

Author(s):

Olina Efthymiadou ◽

Panos Kanavos

Keyword(s):

Decision Making ◽

Health Technology Assessment ◽

Technology Assessment ◽

Best Practice ◽

Health Technology ◽

Future Research ◽

Best Practice Guidelines ◽

Chi Squared ◽

Decision Making Factors ◽

Managed Entry Agreements

Abstract Background Managed Entry Agreements (MEAs) are increasingly used to address uncertainties arising in the Health Technology Assessment (HTA) process due to immature evidence of new, high-cost medicines on their real-world performance and cost-effectiveness. The literature remains inconclusive on the HTA decision-making factors that influence the utilization of MEAs. We aimed to assess if the uptake of MEAs differs between countries and if so, to understand which HTA decision-making criteria play a role in determining such differences. Methods All oncology medicines approved since 2009 in Australia, England, Scotland, and Sweden were studied. Four categories of variables were collected from publicly available HTA reports of the above drugs: (i) Social Value Judgments (SVJs), (ii) Clinical/Economic evidence submitted, (iii) Interpretation of this evidence, and (iv) Funding decision. Conditional/restricted decisions were coded as Listed With Conditions (LWC) other than an MEA or LWC including an MEA (LWCMEA). Cohen's κ-scores measured the inter-rater agreement of countries on their LWCMEA outcomes and Pearson's chi-squared tests explored the association between HTA variables and LWCMEA outcomes. Results A total of 74 drug-indication pairs were found resulting in n = 296 observations; 8 percent (n = 23) were LWC and 55 percent (n = 163) were LWCMEA. A poor-to-moderate agreement existed between countries (−.29 < κ < .33) on LWCMEA decisions. Cross-country differences within the LWCMEA sample were partly driven by economic uncertainties and largely driven by SVJs considered across agencies. Conclusions A set of HTA-related variables driving the uptake of MEAs across countries was identified. These findings can be useful in future research aimed at informing country-specific, “best-practice” guidelines for successful MEA implementation.

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