Elena Nicod1, Lieven Annemans2, Anna Bucsics3, Anne Lee4, Sheela Upadhyaya5, Karen Facey6. 1. Centre for Research on Health and Social Care Management, Bocconi University, Via Roentgen 1, 20126 Milan, Italy; Department of Social Policy, London School of Economics and Political Science, Houghton Street, WC2A 2AE, London, United Kingdom. Electronic address: elena.nicod@unibocconi.it. 2. Department of Public Health, Ghent University, De Pintelaan 185, 4K3, 9000 Gent, Belgium. Electronic address: lieven.annemans@Ugent.be. 3. Department of Finance, University of Vienna, Oskar-Morgenstern-Platz 1 (6th floor), 1090 Vienna, Austria. Electronic address: anna.bucsics@univie.ac.at. 4. Scottish Medicines Consortium, Delta House (8th floor), 50 West Nile Street, G1 2NP Glasgow, Scotland, United Kingdom. Electronic address: annelee@nhs.net. 5. Centre for Health Technology Evaluation, National Institute for Health and Care Excellence, 10 Spring Gardens, SW1A 2BU London, United Kingdom. Electronic address: sheela.upadhyaya@nice.org.uk. 6. Usher Institute of Population Health Sciences and Informatics, University of Edinburgh, Nine Edinburgh BioQuarter, 9 Little France Road, EH16 4UX Edinburgh, Scotland, United Kingdom. Electronic address: k.facey@btinternet.com.
Abstract
BACKGROUND: Challenges commonly encountered in HTA of orphan medicinal products (OMPs) were identified in Advance-HTA. Since then, new initiatives have been developed to specifically address issues related to HTA of OMPs. OBJECTIVE AND METHODS: This study aimed to understand why these new HTA initiatives in England, Scotland and at European-level were established and whether they resolve the challenges of OMPs. The work of Advance-HTA was updated with a literature review and a conceptual framework of clinical, regulatory and economic challenges for OMPs was developed. The new HTA programmes were critiqued against the conceptual framework and outstanding challenges identified. RESULTS: The new programmes in England and Scotland recognise the challenges identified in demonstrating the value of ultra-OMPs (and OMPs) and that they require a different process to standard HTA approaches. Wider considerations of disease and treatment experiences from a multi-stakeholder standpoint are needed, combined with other measures to deal with uncertainty (e.g. managed entry agreements). While approaches to assessing this new view of value of OMPs, extending beyond cost/QALY frameworks, differ, their criteria are similar. These are complemented by a European initiative that fosters multi-stakeholder dialogue and consensus about value determinants throughout the life-cycle of an OMP. CONCLUSION: New HTA programmes specific to OMPs have been developed but questions remain about whether they sufficiently capture value and manage uncertainty in clinical practice.
BACKGROUND: Challenges commonly encountered in HTA of orphan medicinal products (OMPs) were identified in Advance-HTA. Since then, new initiatives have been developed to specifically address issues related to HTA of OMPs. OBJECTIVE AND METHODS: This study aimed to understand why these new HTA initiatives in England, Scotland and at European-level were established and whether they resolve the challenges of OMPs. The work of Advance-HTA was updated with a literature review and a conceptual framework of clinical, regulatory and economic challenges for OMPs was developed. The new HTA programmes were critiqued against the conceptual framework and outstanding challenges identified. RESULTS: The new programmes in England and Scotland recognise the challenges identified in demonstrating the value of ultra-OMPs (and OMPs) and that they require a different process to standard HTA approaches. Wider considerations of disease and treatment experiences from a multi-stakeholder standpoint are needed, combined with other measures to deal with uncertainty (e.g. managed entry agreements). While approaches to assessing this new view of value of OMPs, extending beyond cost/QALY frameworks, differ, their criteria are similar. These are complemented by a European initiative that fosters multi-stakeholder dialogue and consensus about value determinants throughout the life-cycle of an OMP. CONCLUSION: New HTA programmes specific to OMPs have been developed but questions remain about whether they sufficiently capture value and manage uncertainty in clinical practice.
Authors: Marcus V Simões; Fabio Fernandes; Fabiana G Marcondes-Braga; Philip Scheinberg; Edileide de Barros Correia; Luis Eduardo P Rohde; Fernando Bacal; Silvia Marinho Martins Alves; Sandrigo Mangini; Andréia Biolo; Luis Beck-da-Silva; Roberta Shcolnik Szor; Wilson Marques Junior; Acary Souza Bulle Oliveira; Márcia Waddington Cruz; Bruno Vaz Kerges Bueno; Ludhmila Abrahão Hajjar; Aurora Felice Castro Issa; Felix José Alvarez Ramires; Otavio Rizzi Coelho Filho; André Schmidt; Ibraim Masciarelli Francisco Pinto; Carlos Eduardo Rochitte; Marcelo Luiz Campos Vieira; Cláudio Tinoco Mesquita; Celso Dario Ramos; José Soares-Junior; Minna Moreira Dias Romano; Wilson Mathias Junior; Marcelo Iório Garcia Junior; Marcelo Westerlund Montera; Marcelo Dantas Tavares de Melo; Sandra Marques E Silva; Pedro Manoel Marques Garibaldi; Aristóteles Comte de Alencar Neto; Renato Delascio Lopes; Diane Xavier de Ávila; Denizar Viana; José Francisco Kerr Saraiva; Manoel Fernandes Canesin; Glaucia Maria Moraes de Oliveira; Evandro Tinoco Mesquita Journal: Arq Bras Cardiol Date: 2021-09 Impact factor: 2.000