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Improved Methods and Actionable Tools for Enhancing Health Technology Assessment

Deliverables

Training modules and manuals for HTA staff, reviewers, and modelers

this will produce enduser friendly manuals for use and develop training packages to equip endusers with the requisite lowlevel skills to undertake DICE modelling The training package will be pursued as an online publicly accessible package

A review of methods used to extrapolate RCT outcomes using Real World Data and Guidance relating to the matching of data from RCTs and Observational or Registry data banks

This deliverable will review methods used to extrapolate health benefits and treatment costs and will provide guidance on the matching of data from RCTs and Observational or registry data banks It will also outline the manner in which such matched data can form the basis for estimation of longterm outcomes such as average life expectancy

Concrete dissemination and communication plan

This report will provide a detailed plan for all the activities that will be undertaken by partners to communicate and disseminate the results of the research. It will determine the target groups for different results and the most adequate communication and dissemination modes: publications, face-to-face and electronic dissemination.

Algorithm for the estimation of the fiscal impact of new technologies

Algorithm for the estimation of the fiscal impact of new technologies The algorithm will be developed based on the results of the literature review and partner feedback collected through a survey

A toolbox on candidate cost-effective technologies for HTA producers and users

This deliverable will provide a specific methodological and policy guidance concerning the evaluation and implementation of candidate costeffective technologies outside incremental innovations It will include indications about suitable strategies for patient and public involvement reflecting stakeholder preferences and guidance on the use of these technologies according to regulatory environments and ethical principles in HTA

An analytical framework outlining the determinants of HTA recommendations across settings based on primary data collection and primary analysis of secondary data

An analytical framework will be developed outlining the determinants of HTA recommendations across countries based on primary and secondary data with the aim to provide insights on the relevant value concerns of decision makers beyond costs and clinical effects

Toolkit of instruments to identify the clinical variability and its impact on the use of health technology

Development of a toolkit which will include among others a checklist and a projection model aimed at analysing the different factors ie behaviour of healthcare professionals and organizational issues further to the technical characteristic of technology that contribute to clinical variability The toolkit will thus support local decisionmakers in identifying those actions which are most likely to enable an effective and appropriate use of health technologies

Guidelines for cost definitions and harmonization of cost accounting methodologies and methodology that determines the common basket

This deliverable provides a set of recommended definitions principles criteria and procedures for calculating health care costs for economic evaluation in the EU

Comparison of EQ-5D-5L value sets from different disease areas in 2 countries (Germany and Spain)

This report will provide a detailed document describing the differences between patients preference and general population preference regarding the health states described by the EQ5D5L instrument

Stakeholders’ perspective: a political economy approach:

This deliverable will analyse the obstacles and potential drivers in these areas using behavioural economics as well as ethical and legal analyses

Recommendations on how to analyse and interpret non-randomised studies

This document will recommend options for the analysis of nonrandomised studies and for each option how to assess the residual risk of bias The recommendations will be based on an empirical comparison of the performance of different analytical methods and will include pragmatic considerations relevant to HTA agencies such as an assessment of the data requirements for and the time taken to run the analytical methods

Value sets for the EQ-5D-Y in Germany, Slovenia and Spain and Comparison of EQ-5D-Y value sets across countries

This report will provide the countryspecific algorithms to calculate index values scores for the EQ5DY instrument in Germany Slovenia and Spain This includes regression coefficients and software In addition it will provide a detailed document describing the differences between adolescents preference for their own and general population preference for adolescent

Database (final version) with the initial core dataset of costs and the User Manual; Final version of the Guidelines; List of standard unit costs with country values.

This deliverable will produce an open database of unit health costs that will be linked to project website along with a manual for user instructions A first website version of the database will be presented in the M35 of the project An arrangement will be made with a HTA institution in order to ensure that the database is updated and maintained when this project comes to an end

case studies to quantify important different ways of incorporating the social perspective into economic analysis to inform European decisions.

By using a number of case studies this deliverable will quantify different ways of incorporating the social perspective into economic analysis It will focus on differences in methodological aspects such as costs included methods used to estimate costs resources used discount rate applied among others The analysis will be carried out across different European countries and will select a number of up to 5 interventions medicines and devices in therapeutic areas of interest

Validation of the DICE modelling technique against existing methods and Speeding up execution of the DICE Simulation macro

Validation of the DICE modelling technique against existing methods and Speeding up execution of the DICE Simulation macro This deliverable is two parts 1 a report on the Internal Validation of the DICE Simulation model against industry standard models It will consist of rebuilding existing models of standard type using DICE simulation and comparing ICER results Comparison will also focus on technical aspects including ease of model construction file size run time and ease of modification 2 an enhanced ready to use DICE Simulation Model It will be based around a number of technical fixes of the existing Beta prototype such as avoidance of repeated reading from and writing to worksheet the carrying out calculations in memory improving the placeholding of results in memory until simulation is complete incorporating faster calculation algorithms as well as creation of standard templates for commonly encountered modelling problems

State of art analysis in Europe with regard to chosen case studies

in this deliverable, a systematic retrieval and comparison will be carried out gathering HTA methodological and policy guidelines issued at regional and national levels across Europe, on HTA, budget impact evaluation, and patients’ public involvement (PPI). The comparison will hold on relevant aspects w.r.t. the aims of WP10 (those aspects to be impacted by considering adoption and implementation of candidate cost-effective technologies outside incremental innovations).

Results from designing, implementing and validating large scale Delphi processes and describing multi-criteria value models within a common value framework to evaluate new medicines

Following a combination of participatory processes with the engagement of key stakeholders Delphi panels and workshops a flexible methodological framework will be designed for enabling the structuring of multicriteria evaluation models for new medicines in HTA that can accommodate the views and preferences of a wide range of decisionmakers across health care systems

Toolkit including a list of relevant indicators to capture hospital performance variability and Toolkit to assess the transferability of evidence produced in other jurisdictions and decision-making levels

Evidence based indicators will be developed to measure the hospitals performance in terms of efficiency and health outcomes the schedule of assessment and the threshold value will also be defined A set of items describing the hospitals organization in terms of legal status size degree of specialization uptake of innovation and methods used for decision making upon health technologies will be identified The relation between the mentioned elements will be explored As a second section of the deliverable a focus on the assessment of health technologies will be done constructing a standard methodology to assess the transferability of evidence used to inform decision making

Results from applying the value framework to case studies in HTA agencies and Insights for resource allocation and policy-making

In collaboration with HTA agencies and through a combination of participatory processes with the engagement of relevant stakeholders Delphi panels and decision conferences the methodological framework will be tested in practice through the form of case studies as part of which contextspecific evaluation models will be structured for the assessment of alternative sets of medicines for particular disease indications Recommendations will be produced on the application of the new methods as tools for resource allocation in health care and more precisely on how they can inform decisionmaking around coverage and pricing of new medicines

Paper describing the meta-epidemiological comparison of effect sizes in the matched studies

This paper will report how we identified matched pairs of randomised and nonrandomised studies of the same intervention in comparable populations and our analysis of whether and to what extent the treatment effects obtained from the former study design differ from the latter

Evidence from pilot application from selected therapeutic areas

Evidence from pilot application from selected therapeutic areas These applications will be utilized to test the model and the algorithm produced

Revising the methodological aspects applied to the identification, measurement and valuation of social costs in economic evaluations.

This deliverable will provide a comprehensive view of the methodological advances in identification measurement and valuation of labour losses and informal care time and the inclusion of this type of resources in methodological guidelines and economic evaluations of health care interventions across countries

Core dataset of social costs

Core dataset of social costs and link this to the dataset proposed in WP2 which will contain all cost items with a manual on how to use them The database will be designed and developed in a way that allows its future updating and continuity after the end of the present project

A horizontal and vertical expose of lessons learned and policy recommendations

This deliverables will bring together the findings and novelties produced in each WP providing a concrete synthesis and analysis of the policy implications of the research results for HTA in the domains researched upon It will identify how the research results can be incorporated in the decisionmaking process and implemented at national supranational and international level and what implications are there for specific stakeholders

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Publications

Guidance on use and implementation of outcomes-based managed entry agreements for rare disease treatments

Author(s): Facey, K; Xoxi, E; Kent, E; Junghans, T; Whittal, A; Drummond, M; Nicod, E; Upadhyaya, S
Published in: 4, 2021
Publisher: IMPACT HTA
DOI: 10.5281/zenodo.5032658

List of standard unit costs

Author(s): Spacirova, Zuzana; Mochón, Leticia García; Rovira, Joan; Espín, Jaime
Published in: 4, 2021
Publisher: IMPACT HTA
DOI: 10.5281/zenodo.5044967

IMPACT-HTA WP10 - Country vignettes of appraisal processes for rare disease treatments

Author(s): Whittal, Amanda; Nicod, Elena; Facey, Karen; Drummond, Michael
Published in: 2, 2020
Publisher: IMPACT HTA
DOI: 10.5281/zenodo.5019195

Guidelines for cost definition and harmonization of cost accounting methodologies for economic evaluation

Author(s): Spacirova, Zuzana; Mochón, Leticia García; Rovira, Joan; Epstein, David; Espín, Jaime
Published in: 6, 2021
Publisher: IMPACT HTA
DOI: 10.5281/zenodo.5044847

HTA Appraisal Framework Suitable for Rare Disease Treatments

Author(s): Facey, K; Whittal, A; Drummond, M; Upadhyaya, S; Junghans, T; Nicod, E
Published in: 1, 2021
Publisher: IMPACT HTA
DOI: 10.5281/zenodo.5032615

How relevant is the societal perspective in economic evaluations? Evidence from five case studies

Author(s): Oliva-Moreno, Juan; Rodríguez-Sánchez, Beatriz; Peña-Longobardo, Luz María; Aranda-Reneo, Isaac; López-Bastida, Julio
Published in: 1, 2021
Publisher: IMPACT HTA
DOI: 10.5281/zenodo.5045123

Technical aspects of the European Healthcare and Social Cost Database

Author(s): Spacirova, Zuzana; Galera, Emilia Ramos; Espin, Jaime
Published in: 2, 2021
Publisher: IMPACT HTA
DOI: 10.5281/zenodo.5044869

Real-world evidence to support Payer/HTA decisions about highly innovative technologies in the EU—actions for stakeholders

Author(s): Karen M. Facey, Piia Rannanheimo, Laura Batchelor, Marine Borchardt, Jo de Cock
Published in: International Journal of Technology Assessment in Health Care, 36/4, 2020, Page(s) 459-468, ISSN 0266-4623
Publisher: Cambridge University Press
DOI: 10.1017/s026646232000063x

Cost transferability problems in economic evaluation as a framework for an European health care and social costs database

Author(s): Leticia García-Mochón, Joan Rovira Forns, Jaime Espin
Published in: Cost Effectiveness and Resource Allocation, 19/1, 2021, ISSN 1478-7547
Publisher: BioMed Central
DOI: 10.1186/s12962-021-00294-4

The estimation of health state utility values in rare diseases: overview of existing techniques

Author(s): Michela Meregaglia, Elena Nicod, Michael Drummond
Published in: International Journal of Technology Assessment in Health Care, 36/5, 2020, Page(s) 469-473, ISSN 0266-4623
Publisher: Cambridge University Press
DOI: 10.1017/s0266462320000665

‘Mapping’ Health State Utility Values from Non-preference-Based Measures: A Systematic Literature Review in Rare Diseases

Author(s): Michela Meregaglia, Amanda Whittal, Elena Nicod, Michael Drummond
Published in: PharmacoEconomics, 38/6, 2020, Page(s) 557-574, ISSN 1170-7690
Publisher: Adis International Ltd.
DOI: 10.1007/s40273-020-00897-4

How relevant are social costs in economic evaluations? The case of Alzheimer’s disease

Author(s): L. M. Peña-Longobardo, B. Rodríguez-Sánchez, J. Oliva-Moreno, I. Aranda-Reneo, J. López-Bastida
Published in: The European Journal of Health Economics, 20/8, 2019, Page(s) 1207-1236, ISSN 1618-7598
Publisher: Springer Verlag
DOI: 10.1007/s10198-019-01087-6

Can the Consideration of Societal Costs Change the Recommendation of Economic Evaluations in the Field of Rare Diseases? An Empirical Analysis

Author(s): Isaac Aranda-Reneo, Beatriz Rodríguez-Sánchez, Luz María Peña-Longobardo, Juan Oliva-Moreno, Julio López-Bastida
Published in: Value in Health, 24/3, 2021, Page(s) 431-442, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2020.10.014

Hospital contextual factors affecting the implementation of health technologies: a systematic review

Author(s): Adriano Grossi, Ilda Hoxhaj, Irene Gabutti, Maria Lucia Specchia, Americo Cicchetti, Stefania Boccia, Chiara de Waure
Published in: BMC Health Services Research, 21/1, 2021, ISSN 1472-6963
Publisher: BioMed Central
DOI: 10.1186/s12913-021-06423-2

Assessing the Effect of Including Social Costs in Economic Evaluations of Diabetes-Related Interventions: A Systematic Review

Author(s): Beatriz Rodriguez-Sanchez, Isaac Aranda-Reneo, Juan Oliva-Moreno, Julio Lopez-Bastida
Published in: ClinicoEconomics and Outcomes Research, Volume 13, 2021, Page(s) 307-334, ISSN 1178-6981
Publisher: Dove Medical Press Limited
DOI: 10.2147/ceor.s301589

EQ-5D-Y Value Set for Slovenia

Author(s): Valentina Prevolnik Rupel, Marko Ogorevc
Published in: PharmacoEconomics, 39/4, 2021, Page(s) 463-471, ISSN 1170-7690
Publisher: Adis International Ltd.
DOI: 10.1007/s40273-020-00994-4

Considering the societal perspective in economic evaluations: a systematic review in the case of depression

Author(s): Juliane Andrea Duevel, Lena Hasemann, Luz María Peña-Longobardo, Beatriz Rodríguez-Sánchez, Isaac Aranda-Reneo, Juan Oliva-Moreno, Julio López-Bastida, Wolfgang Greiner
Published in: Health Economics Review, 10/1, 2020, ISSN 2191-1991
Publisher: Springer
DOI: 10.1186/s13561-020-00288-7

Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

Author(s): Elena Nicod, Amanda Whittal, Michael Drummond, Karen Facey
Published in: Orphanet Journal of Rare Diseases, 15/1, 2020, ISSN 1750-1172
Publisher: BioMed Central
DOI: 10.1186/s13023-020-01462-0

Examining the impact of different country processes for appraising rare disease treatments: a case study analysis

Author(s): Amanda Whittal, Elena Nicod, Mike Drummond, Karen Facey
Published in: International Journal of Technology Assessment in Health Care, 37/1, 2021, ISSN 0266-4623
Publisher: Cambridge University Press
DOI: 10.1017/s0266462321000337

Using Discretely Integrated Condition Event Simulation To Construct Quantitative Benefit–Risk Models: The Example of Rotavirus Vaccination in France

Author(s): Hugo Arlegui, Gaëlle Nachbaur, Nicolas Praet, Bernard Bégaud, J. Jaime Caro
Published in: Clinical Therapeutics, 42/10, 2020, Page(s) 1983-1991.e2, ISSN 0149-2918
Publisher: Excerpta Medica, Inc.
DOI: 10.1016/j.clinthera.2020.08.013

Comparison of Adult and Adolescent Preferences Toward EQ-5D-Y-3L Health States

Author(s): Valentina Prevolnik Rupel, Juan M. Ramos-Goñi, Marko Ogorevc, Simone Kreimeier, Kristina Ludwig, Wolfgang Greiner
Published in: Value in Health, 2021, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2021.03.019

PNS26 Appropriate Indices of Inflation to Update Prices and Unit Costs of Healthcare Goods and Services for Use in Economic Evaluation

Author(s): J. Espin, D. Epstein, A. Gaggero, J. Rovira
Published in: Value in Health, 23, 2020, Page(s) S648, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2020.08.1470

The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

Author(s): Amanda Whittal, Michela Meregaglia, Elena Nicod
Published in: The Patient - Patient-Centered Outcomes Research, 2021, ISSN 1178-1653
Publisher: Adis International Ltd.
DOI: 10.1007/s40271-020-00493-w

EQ-5D-5L Slovenian population norms

Author(s): Valentina Prevolnik Rupel, Marko Ogorevc
Published in: Health and Quality of Life Outcomes, 18/1, 2020, ISSN 1477-7525
Publisher: BioMed Central
DOI: 10.1186/s12955-020-01584-w

Assessing hospital performance indicators. What dimensions? Evidence from an umbrella review

Author(s): Elettra Carini, Irene Gabutti, Emanuela Maria Frisicale, Andrea Di Pilla, Angelo Maria Pezzullo, Chiara de Waure, Americo Cicchetti, Stefania Boccia, Maria Lucia Specchia
Published in: BMC Health Services Research, 20/1, 2020, ISSN 1472-6963
Publisher: BioMed Central
DOI: 10.1186/s12913-020-05879-y

To What Extent Do Patient Preferences Differ From General Population Preferences?

Author(s): Kristina Ludwig, Juan M. Ramos-Goñi, Mark Oppe, Simone Kreimeier, Wolfgang Greiner
Published in: Value in Health, 2021, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2021.02.012

Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel

Author(s): Karen M. Facey, Jaime Espin, Emma Kent, Angèl Link, Elena Nicod, Aisling O’Leary, Entela Xoxi, Inneke van de Vijver, Anna Zaremba, Tatyana Benisheva, Andrius Vagoras, Sheela Upadhyaya
Published in: PharmacoEconomics, 2021, ISSN 1170-7690
Publisher: Adis International Ltd.
DOI: 10.1007/s40273-021-01050-5

Accounting for Unobservable Preference Heterogeneity and Evaluating Alternative Anchoring Approaches to Estimate Country-Specific EQ-5D-Y Value Sets: A Case Study Using Spanish Preference Data

Author(s): Ramos-Goñi JM, Oppe M, Estévez-Carrillo A, Rivero-Arias O, Wolfgang G, Kreimeier S, Ludwig K, Rupel V
Published in: Value in Health, 2021, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2021.10.013

Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art

Author(s): Mónica D. Oliveira, Inês Mataloto, Panos Kanavos
Published in: The European Journal of Health Economics, 2019, Page(s) 1-28, ISSN 1618-7598
Publisher: Springer Verlag
DOI: 10.1007/s10198-019-01052-3

Driving health technologies towards performance: a contextualisation within the hospital setting

Author(s): Gabutti I
Published in: International Journal of Technology, Policy and Management, 2020, ISSN 1468-4322
Publisher: Inderscience Publishers
DOI: 10.21203/rs.3.rs-109606/v1

A general framework for classifying costing methods for economic evaluation of health care

Author(s): Zuzana Špacírová, David Epstein, Leticia García-Mochón, Joan Rovira, Antonio Olry de Labry Lima, Jaime Espín
Published in: The European Journal of Health Economics, 21/4, 2020, Page(s) 529-542, ISSN 1618-7598
Publisher: Springer Verlag
DOI: 10.1007/s10198-019-01157-9

PND35 Social Costs in Multiple Sclerosis Economic Evaluations: Do Their Inclusion Change the Results and Conclusions? a Systematic Review

Author(s): J. Lopez-Bastida, A. Cicchetti, B. Rodríguez-Sánchez, S. Daugbjerg, L.M. Peña-Longobardo, I. Aranda-Reneo, J. Oliva-Moreno
Published in: Value in Health, 23, 2020, Page(s) S628-S629, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2020.08.1360

PMH14 Considering the Societal Perspective in Economic Evaluations: A Systematic Review in the Case of Depression-Related Interventions

Author(s): J. Lopez-Bastida, J.A. Düvel, L. Hasemann, L.M. Peña-Longobardo, B. Rodríguez-Sánchez, I. Aranda-Reneo, J. Oliva-Moreno, W. Greiner
Published in: Value in Health, 23, 2020, Page(s) S586, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2020.08.1092

PDB35 Assessing the Effect of Including Social Costs in Economic Evaluations: A Systematic Review of Diabetes-Related Interventions

Author(s): J. Lopez-Bastida, B. Rodríguez-Sánchez, I. Aranda-Reneo, J. Oliva-Moreno
Published in: Value in Health, 23, 2020, Page(s) S511-S512, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2020.08.632

PRO19 THE ROLE PLAYED BY SOCIAL COSTS IN ECONOMIC EVALUATIONS OF RARE DISEASE INTERVENTIONS

Author(s): J. Lopez Bastida, I. Aranda-Reneo, L.M. Peña-Longobardo, B. Rodríguez-Sánchez, J. Oliva-Moreno
Published in: Value in Health, 22, 2019, Page(s) S844, ISSN 1098-3015
Publisher: Blackwell Publishing Inc.
DOI: 10.1016/j.jval.2019.09.2350

Collaborative value modelling in widely participated decision support processes

Author(s): Carlos A. Bana e Costa, Ana C.L. Vieira, Mónica D. Oliveira, João Bana e Costa
Published in: EMC-ICDSST 2019 Proceedings – online version Euro mini international conference on decision support system technology, 2019, Page(s) 136-140
Publisher: P.S. Freitas, F. Dargam, R. Ribeiro, J.M. Moreno, J. Papathanasiou (editors)