Using health technology assessment to assess the value of new medicines: results of a systematic review and expert consultation across eight European countries (original) (raw)
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Value in Health Regional Issues
To examine and compare the use of health technology assessment (HTA) for the reimbursement of new medicines in selected European Union member states with decades of experience in the use of HTA and in countries that have used it regularly since 2000. Methods: The selected countries were categorized into "earlier" adopters (group A: England, Germany, France, and Sweden) and more "recent" adopters (group B: Poland, Bulgaria, Hungary, and Romania). A systematic review of published literature was performed. The analysis and comparison of HTA procedures were done by using an analytical framework. Results: In all countries, the assessment criteria used include effectiveness, safety, relative effectiveness, and economic data. In group A countries, the main objectives are improving quality of care, ensuring equal access, and efficient use of resources. Group B countries have established HTA organizations with official guidelines but often seek the decisions of other developed countries. They place considerable emphasis on the budget impact of new therapies, and HTA is also used as a cost estimation tool for state budgets. Conclusions: HTA organizations have been developed dynamically not only in high-income countries but also in countries with limited resources. The experience and evolution of both can be used by countries that are in the dawn of creating an HTA organization.
Social science & medicine (1982), 2017
Escalating drug prices have catalysed the generation of numerous "value frameworks" with the aim of informing payers, clinicians and patients on the assessment and appraisal process of new medicines for the purpose of coverage and treatment selection decisions. Although this is an important step towards a more inclusive Value Based Assessment (VBA) approach, aspects of these frameworks are based on weak methodologies and could potentially result in misleading recommendations or decisions. In this paper, a Multiple Criteria Decision Analysis (MCDA) methodological process, based on Multi Attribute Value Theory (MAVT), is adopted for building a multi-criteria evaluation model. A five-stage model-building process is followed, using a top-down "value-focused thinking" approach, involving literature reviews and expert consultations. A generic value tree is structured capturing decision-makers' concerns for assessing the value of new medicines in the context of Heal...
Molecular oncology, 2014
This paper reviews the issues involved in undertaking HTA studies early in the development of new cancer therapies, and discusses the data and methods for estimating the cost-effectiveness of new diagnostics and treatments. The value for patients of new cancer therapies is based on access to the treatment and optimal use. Realising potential value depends on successful completion of a series of steps, from the initial economic evaluations based on clinical trial data, to the reimbursement decisions based on the evaluations and the implementation of these decisions in clinical practice. Considerable resources have been devoted to the study of the cost-effectiveness of new cancer drugs as a basis for decisions about payment and use. Such resources could be used much more effectively if industry and HTA agencies were to collaborate at an early stage in the development process. The traditional clinical trial approach of using progression-free survival and cross-overs has serious shortco...
Health Technology Assessment, Value-Based Decision Making, and Innovation
International Journal of Technology Assessment in Health Care, 2013
Background: Identifying treatments that offer value and value for money is becoming increasingly important, with interest in how health technology assessment (HTA) and decision makers can take appropriate account of what is of value to patients and to society, and in the relationship between innovation and assessments of value. Methods: This study summarizes points from an Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations, discussions among attendees, and background papers. Results and Conclusions: Various perspectives on value were considered; most place patient health at the core of value. Wider elements of value comprise other benefits for: patients; caregivers; the health and social care systems; and society. Most decision-making systems seek to take account of similar elements of value, although they are assessed and combined in different ways. Judgment in decisions remains important and cannot be replaced by mathematical approaches. There was discussion of the value of innovation and of the effects of value assessments on innovation. Discussion also included moving toward "progressive health system decision making," an ongoing process whereby evidence-based decisions on use would be made at various stages in the technology lifecycle. Five actions are identified: (i) development of a general framework for the definition and assessment of value; development by HTA/coverage bodies and regulators of (ii) disease-specific guidance and (iii) further joint scientific advice for industry on demonstrating value; (iv) development of a framework for progressive licensing, usage, and reimbursement; and (v) promoting work to better adapt HTA, coverage, and procurement approaches to medical devices.
Frontiers in Pharmacology
Health technology assessment (HTA) has traditionally relied on cost-effectiveness analysis (CEA) as a cornerstone of evaluation of new therapies, assessing the clinical validity and utility, the efficacy, and the cost-effectiveness of new interventions. The current format of cost-effectiveness analysis, however, does not allow for inclusion of more holistic aspects of health and, therefore, value elements for new technologies such as the impact on patients and society beyond its pure clinical and economic value. This study aimed to review the recent modelling attempts to expand the traditional cost-effectiveness analysis approach by incorporating additional elements of value in health technology assessment. A pragmatic literature review was conducted for articles published between 2012 and 2022 reporting cost-effectiveness analysis including value aspects beyond the clinical and cost-effectiveness estimates; searches identified 13 articles that were eligible for inclusion. These exp...
VP30 Research And Analysis Of European Health Technology Assessment Processes
International Journal of Technology Assessment in Health Care, 2017
INTRODUCTION: As part of the European Network for Health Technology Assessment (EUnetHTA) Joint Action 3, the National Institute for Health and Care Excellence (NICE) in collaboration with forty-nine Health Technology Assessment (HTA) agencies and payer organizations, is leading on research to gain a high level understanding of HTA processes across Europe. This will help to facilitate improved collaboration and use of EUnetHTA HTA reports and tools across member states and decrease the duplication of work. To analyze the similarities and differences in HTA processes and decision making on the reimbursement of pharmaceuticals and medical devices across Europe. METHODS: National agencies involved in the HTA and reimbursement processes shared data on HTA and decision-making processes. Data provided was extracted into an excel workbook including information relating to pharmaceuticals, medical devices, inpatient and outpatient care and assessments that inform reimbursement, pricing and ...
Value in Health, 2011
The user has requested enhancement of the downloaded file. dard OS extrapolation methods which fit 'traditional' parametric survival distributions to patient-level data, two different methods were explored in the modelling of OS beyond the trial duration (55 months) for the novel immunotherapy ipilimumab. In the first approach, the hazard rate from the Kaplan-Meier (KM) curve between 24 and 36 months (before reaching a plateau) was used to extend the curve. In the second approach, different parametric curves were fitted to the period of 18 months onwards. Akaike's Information Criterion (AIC) was used to determine the best fit curve. RESULTS: When compared to standard OS extrapolation methods, both methods exhibited a better visual fit to the data. Both approaches allow the hazard of the extrapolated tail to be based on a section of the KM curve that is more appropriate in describing the long-term survival of these patients. The hazard rate approach does not allow for a formal comparison with AIC, but allows extrapolation in line with the clinical interpretation. The 'parametric curves' approach allows for a statistically better fit with the patient level data using conventional AIC criteria. Both methods are in line with long-term observations of immunotherapy. CONCLUSION: For novel cancer therapies whose KM curves are not well described by standard survival distributions, other methods of extrapolation should be explored in conjunction with an understanding of the clinical rationale. In this case study, two alternatives are presented that describe the OS of immunotherapy patients in a more suitable way.
Frontiers in Drug Safety and Regulation
Health Technology Assessment (HTA) is a multidisciplinary process which supports policy “decision-making”, including those related to drug reimbursement and price negotiation based on comprehensive clinical and non-clinical evaluations. The discrepancies in HTA recommendations among European countries are related to the differences in health care systems and willingness to invest in health care, differences in assessment methodologies and appraisal practices, and variations in economic constraints, and may produce disparities in terms of patient access to medicines. The political discussion about the adoption of the European Regulation on HTA lasted for years due to concerns about its feasibility and its implications for national health care systems In this paper we highlighted the importance to harmonize HTA process in Europe and some critical issues emerged during the discussion among experts in the field carried out at the European Regulatory Conference.
Valorising and Creating Access to Innovative Medicines in the European Union
Frontiers in Pharmacology, 2011
This Perspective describes (a) the current situation, (b) challenges and initiatives, (c) and formulates recommendations to valorize and create access to innovative medicines in the EU. We are currently still far away from optimal assessment of value for money in the EU. On the one hand, valorizing innovative medicines involves a local appraisal by health technology assessment (HTA) bodies and competent authorities about the value for money, the budget impact, and the local medical need that can be filled with new medicines. Therefore, local priorities and national health care policy environments should be reflected in the processes and criteria used for assessing value for money and ultimately for reimbursement decisions. On the other hand, a pan-European assessment of both relative effectiveness and medical need (including general ethical and social considerations) should be envisaged in order to feed part of the data needed for the local decisions in an efficient way. This could be the task of the European Medicines Agency, HTA bodies, and competent authorities together.
International Journal of Technology Assessment in Health Care, 2014
Background: Many jurisdictions delivering health care, including Canada, have developed guidance for conducting economic evaluation, often in the service of larger health technology assessment (HTA) and reimbursement processes. Like any health intervention, personalized medical (PM) interventions have costs and consequences that must be considered by reimbursement authorities with limited resources. However, current approaches to economic evaluation to support decision making have been largely developed from population-based approaches to therapy-that is, evaluating the costs and consequences of single interventions across single populations. This raises the issue as to whether these methods, as they are or more refined, are adequate to address more targeted approaches to therapy, or whether a new paradigm for assessing value in PM is required. Objectives: We describe specific issues relevant to the economic evaluation of diagnostics-based PM and assess whether current guidance for economic evaluation is sufficient to support decision making for PM interventions. Methods: Issues were identified through literature review and informal interviews with national and international experts (n = 10) in these analyses. This article elaborates on findings and discussion at a workshop held in Ottawa, Canada, in January 2012. Results: Specific issues related to better guiding economic evaluation of personalized medicine interventions include: how study questions are developed, populations are characterized, comparators are defined, effectiveness is evaluated, outcomes are valued and how resources are measured. Diagnostics-based PM also highlights the need for analyses outside of economic evaluation to support decision making. Conclusions: The consensus of this group of experts is that the economic evaluation of diagnostics-based PM may not require a new paradigm. However, greater complexity means that existing approaches and tools may require improvement to undertake these more analyses.