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Development, testing, and implementation of a new procedure to assess the clinical added benefit of pharmaceuticals

Part of: Special Collection - Methods

Published online by Cambridge University Press:  12 July 2022

Veronika Dóczy Open the ORCID record for Veronika Dóczy [Opens in a new window] ,
Barbara Wernerné Sódar ,
Áron Hölgyesi ,
Gergő Merész  and
Péter Gaál
Veronika Dóczy*
Affiliation:
Department of Health Technology Assessment, National Institute of Pharmacy and Nutrition, Budapest, Hungary
Barbara Wernerné Sódar
Affiliation:
Department of Health Technology Assessment, National Institute of Pharmacy and Nutrition, Budapest, Hungary
Áron Hölgyesi
Affiliation:
Department of Health Technology Assessment, National Institute of Pharmacy and Nutrition, Budapest, Hungary Doctoral School of Molecular Medicine, Semmelweis University, Budapest, Hungary
Gergő Merész
Affiliation:
Department of Health Technology Assessment, National Institute of Pharmacy and Nutrition, Budapest, Hungary Doctoral School of Mental Health Sciences, Semmelweis University, Budapest, Hungary
Péter Gaál
Affiliation:
Faculty of Health and Public Administration, Health Services Management Training Centre, Semmelweis University, Budapest, Hungary Faculty of Technical and Human Sciences, Department of Applied Social Sciences, Sapientia Hungarian University of Transylvania, Targu Mures, Romania
*
* Author for correspondence: Veronika Dóczy, E-mail: doczy.veronika@ogyei.gov.hu
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Abstract

Objectives

The reimbursement process for innovative health technologies in Hungary lacks any formalized assessment of clinical added benefit (CAB). The aim of this research is to present the development, retrospective testing, and implementation of a local assessment framework for determining the CAB of cancer treatments at the Department of Health Technology Assessment of the National Institute of Pharmacy and Nutrition in Hungary.

Methods

The assessment framework was drafted after screening existing methods and a retrospective comparison of local reimbursement dossiers to that of German and French methods. The Magnitude of Clinical Benefit Scale of the European Society for Medical Oncology was chosen to rate the extent of CAB in oncology, as part of a conclusion complemented by the assessment of endpoint relevance and the quality of evidence. Several rounds of retrospective assessments have been conducted involving all clinical assessors, iterated with semistructured discussions to consolidate divergence between assessors. External stakeholders were consulted to provide feedback on the framework.

Results

Retrospective assessments resulted in average more than 75 percent concordance between assessors on each element of the conclusion. Input from ten stakeholders was also incorporated; stakeholders were generally supportive, and they mostly commented on the concept, the elements of the framework, and its implementation.

Conclusions

The procedure is suitable for routine use in the decision-making process to describe the CAB of antineoplastic technologies in Hungary. Further extension of the framework is required to cover more disease areas for structured and comparable conclusions on CAB of innovative health technologies.

Keywords

Technology assessmentHealth (MeSH Unique ID: D013673)Medical oncology (MeSH Unique ID: D008495)Comparative effectiveness research (MeSH Unique ID: D057186)Outcome assessmenthealth care (MeSH Unique ID: D017063)Decision makingorganizational (MeSH Unique ID: D003659)
Type
Method
Information
International Journal of Technology Assessment in Health Care , Volume 38 , Issue 1 , 2022 , e58
Copyright
© The Author(s), 2022. Published by Cambridge University Press

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Footnotes

The authors thank Professor Nathan Cherny and Mrs. Nicola Jane Latino and all the experts at ESMO-MCBS helpdesk for their quick responses and irreplaceable support, all stakeholders who provided feedback and thus improved the framework, Ms. Szilvia Szabó and all other clinical assessors of NIPN for their tireless help with the retrospective testing, the Department of Pharmacovigilance at NIPN for their useful contribution, and Ms. Antonia Bosworth Smith and Mr. Kenneth Paul White for their contribution to language editing.

References

Drummond, MF, Schwartz, JS, Jönsson, B, et al. (2008) Key principles for the improved conduct of health technology assessments for resource allocation decisions. Int J Technol Assess Health Care 24, 244–258; discussion 362–368.CrossRefGoogle ScholarPubMed
Akehurst, RL, Abadie, E, Renaudin, N, Sarkozy, F (2017) Variation in health technology assessment and reimbursement processes in Europe. Value Health 20, 67–76.Google ScholarPubMed
Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen. General Methods v6.1, 2022 [Internet] [accessed 8 Feb 2022]. Available at: https://www.iqwig.de/en/methods/methods-paper.3020.html.Google Scholar
Haute Autorité de santé. Transparency Committee Doctrine - Principles of medicinal products assessment and appraisal for reimbursement purposes [Internet] [accessed 8 Feb 2022]. Available at: https://www.has-sante.fr/upload/docs/application/pdf/2019-07/doctrine_de_la_commission_de_la_transparence_-_version_anglaise.pdf.Google Scholar
Kaló, Z, Gheorghe, A, Huic, M, Csanádi, M, Kristensen, FB (2016) HTA implementation roadmap in central and Eastern European Countries. Health Econ 25(Suppl 1), 179–192.CrossRefGoogle ScholarPubMed
Gulácsi, L, Rotar, AM, Niewada, M, et al. (2014) Health technology assessment in Poland, the Czech Republic, Hungary, Romania and Bulgaria. Eur J Health Econ 15(Suppl 1), S13–S25.CrossRefGoogle ScholarPubMed
Schünemann, HJ, Wiercioch, W, Etxeandia, I, et al. (2014) Guidelines 2.0: systematic development of a comprehensive checklist for a successful guideline enterprise. CMAJ 186, E123–E142.CrossRefGoogle ScholarPubMed
Boucaud-Maitre, D, Berdaï, D, Salvo, F (2021) Added therapeutic value of medicinal products for French and German health technology assessment organizations: a systematic comparison. Value Health 24, 346–352.CrossRefGoogle ScholarPubMed
EUR-Lex. Proposal for a REGULATION OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL on health technology assessment and amending Directive 2011/24/EU. COM/2018/051 final - 2018/018 (COD) [Internet] [accessed 7 Jan 2022]. Available at: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32021R2282.Google Scholar
Cherny, NI, Sullivan, R, Dafni, U, et al. (2015) A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann Oncol 26, 1547–1573. Erratum in Ann Oncol. 2017;28:2901–290CrossRefGoogle Scholar
EUnetHTA. Guideline - Endpoints used for relative effectiveness assessment: clinical endpoints [Internet] [accessed 3 Oct 2021]. Available at: https://www.eunethta.eu/wp-content/uploads/2018/02/WP7-SG3-GL-clin_endpoints_amend2015.pdf.Google Scholar
EUnetHTA. Guideline - Endpoints used in relative effectiveness assessment: surrogate endpoints [Internet] [accessed 3 Oct 2021]. Available at: https://www.eunethta.eu/wp-content/uploads/2018/03/surrogate_endpoints.pdf.Google Scholar
EMA. Clinical efficacy and safety guidelines [Internet] [accessed 3 Oct 2021]. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-guidelines/clinical-efficacy-safety-guidelines.Google Scholar
ESMO. ESMO Standard Operating Procedures (SOPs) for Clinical Practice Guidelines (CPGs) and ESMO Magnitude of Clinical Benefit (ESMO-MCBS) scores [Internet] [accessed 22 Nov 2021]. Available at: https://www.esmo.org/content/download/77789/1426712/file/ESMO-Clinical-Practice-Guidelines-Standard-Operating-Procedures.pdf.Google Scholar
Indirectness of evidence. In: Schünemann H, Brożek J, Guyatt G, Oxman A, editors, GRADE handbook for grading quality of evidence and strength of recommendations. Published online October 2013. [accessed 22 Nov 2021]. Available at: https://gdt.gradepro.org/app/handbook/handbook.html.Google Scholar
Gisev, N, Bell, JS, Chen, TF (2013) Interrater agreement and interrater reliability: key concepts, approaches, and applications. Res Social Adm Pharm 9, 330–338.Google ScholarPubMed
Augustovski, F, Alfie, V, Alcaraz, A, et al. (2021) A Value Framework for the Assessment of Diagnostic Technologies: A Proposal Based on a Targeted Systematic Review and a Multistakeholder Deliberative Process in Latin America. Value Health 24, 486–496.Google Scholar
Bae, G, Bae, S, Lee, D, et al. (2021) Value frameworks: adaptation of Korean versions of value frameworks for oncology. Int J Environ Res Public Health 18, 3139.CrossRefGoogle ScholarPubMed
Ezeife, DA, Dionne, F, Fares, AF, et al. (2020) Value assessment of oncology drugs using a weighted criterion-based approach. Cancer 126, 1530–1540.CrossRefGoogle ScholarPubMed
Janzic, U, Knez, L, Janzic, A, Cufer, T (2019) Time to access to novel anticancer drugs and the correlation with ESMO-Magnitude of Clinical Benefit Scale in Slovenia. Expert Rev Pharmacoecon Outcomes Res 19, 717–723.CrossRefGoogle ScholarPubMed
Kergall, P, Autin, E, Guillon, M, Clément, V (2021) Coverage and pricing recommendations of the French National Health Authority for innovative drugs: a retrospective analysis from 2014 to 2020. Value Health 24, 1784–1791.CrossRefGoogle ScholarPubMed
Gyawali, B, de Vries, EGE, Dafni, U, et al. (2021) Biases in study design, implementation, and data analysis that distort the appraisal of clinical benefit and ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) scoring. ESMO Open 6, 100117.CrossRefGoogle ScholarPubMed
Knapen, DG, Cherny, NI, Zygoura, P, et al. (2020) Lessons learnt from scoring adjuvant colon cancer trials and meta-analyses using the ESMO-magnitude of clinical benefit scale V.1.1. ESMO Open 5, e000681.CrossRefGoogle ScholarPubMed
Grössmann, N, Wolf, S, Rothschedl, E, Wild, C (2021) Twelve years of European cancer drug approval—A systematic investigation of the ‘magnitude of clinical benefit’. ESMO Open 6, 100166.CrossRefGoogle ScholarPubMed
Falcone, R, Lombardi, P, Filetti, M, et al. (2022) Oncologic drugs approval in Europe for solid tumors: overview of the last 6 years. Cancers (Basel) 14(4), 889.CrossRefGoogle ScholarPubMed
Merész, G, Dóczy, V, Hölgyesi, Á, Németh, G (2021) [A practical demonstration of critically assessing health economic analyses]. Az egészség-gazdaságtani elemzések kritikai értékelési gyakorlatának bemutatása. Lege Artis Medicinae 31, 519–529. Hungarian.CrossRefGoogle Scholar
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