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Volume 11
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10.1080/20016689.2023.2239557
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Journal of Market Access & Health Policy (JMAHP) is published by MDPI from Volume 12 Issue 1 (2024). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with Taylor & Francis.
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Background:
Meeting Report

Rule of Prevention: A Potential Framework to Evaluate Preventive Interventions for Rare Diseases

by
Eddie Gibson
1,
Daniel A. Ollendorf
2,
Steven Simoens
3,
David E Bloom
4,
Federico Martinón-Torres
5,6,7,
David Salisbury
8,
Johan Louis Severens
9,
Mondher Toumi
10,
Daniel Molnar
11,
Kinga Meszaros
11,*,
Woo-Yun Sohn
12 and
Najida Begum
13
1
Wickenstones Ltd., Abingdon, UK
2
Institute for Clinical Research and Health Policy Studies (ICRHPS), Center for the Evaluation of Value and Risk in Health (CEVR), Tufts Medical Center, Boston, MA, USA
3
Clinical Pharmacology and Pharmacotherapy, KU Leuven, Leuven, Belgium
4
Harvard T.H. Chan School of Public Health, Boston, MA, USA
5
Department of Pediatrics, Translational Pediatrics and Infectious Diseases, Pediatrics Department, Hospital Clínico Universitario de Santiago de Compostela, Santiago de Compostela, Spain
6
Genetics, Vaccines and Infections Research Group (GENVIP), Instituto de Investigación Sanitaria de Santiago, University of Santiago, Santiago de Compostela, Spain
7
Centro de Investigación Biomédica En Red de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain
8
Royal Institute of International Affairs, Chatham House, London, UK
9
Severens HTA Consultancy, Venray, The Netherlands
10
InovIntell, Île-de-France, France
11
GSK, Wavre, Belgium
12
GSK, Philadelphia, MD, USA
13
Freelance, C/O Gsk, Wavre, Belgium
 
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J. Mark. Access Health Policy 2023, 11(1), 2239557; https://doi.org/10.1080/20016689.2023.2239557
Submission received: 10 April 2023 / Revised: 19 June 2023 / Accepted: 18 July 2023 / Published: 10 August 2023

Abstract

Background: The benefits of preventive interventions lack comprehensive evaluation in standard health technology assessments (HTA), particularly for rare and transmissible diseases. Objective: To identify possible considerations for future HTA using analogies between the treatment and prevention of rare diseases. Study design: An Expert panel meeting assessed whether one HTA assessment framework can be applied to assess both rare disease treatments and preventive interventions. Experts also evaluated the range of value elements currently included in HTAs and their applicability to rare, transmissible, and/or preventable diseases. Results: A broad range of value should be considered when assessing rare, transmissible disease prevention. Although standard HTA can be applied to transmissible diseases, the risk of local outbreaks and the need for large-scale prevention programs suggest a modified assessment framework, capable of incorporating prevention-specific value elements in HTAs. A ‘Rule of Prevention’ framework was proposed to allow broader value considerations anchored to severity, equity, and prevention benefits in decision-making for preventive interventions for rare transmissible diseases. Conclusion: The proposed prevention framework introduces an explicit initial approach to consistently assess rare transmissible diseases, and to incorporate the broader value of preventive interventions compared with treatment.
Keywords: prevention; vaccination; assessment; value; rare disease prevention; vaccination; assessment; value; rare disease

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MDPI and ACS Style

Gibson, E.; Ollendorf, D.A.; Simoens, S.; Bloom, D.E.; Martinón-Torres, F.; Salisbury, D.; Severens, J.L.; Toumi, M.; Molnar, D.; Meszaros, K.; et al. Rule of Prevention: A Potential Framework to Evaluate Preventive Interventions for Rare Diseases. J. Mark. Access Health Policy 2023, 11, 2239557. https://doi.org/10.1080/20016689.2023.2239557

AMA Style

Gibson E, Ollendorf DA, Simoens S, Bloom DE, Martinón-Torres F, Salisbury D, Severens JL, Toumi M, Molnar D, Meszaros K, et al. Rule of Prevention: A Potential Framework to Evaluate Preventive Interventions for Rare Diseases. Journal of Market Access & Health Policy. 2023; 11(1):2239557. https://doi.org/10.1080/20016689.2023.2239557

Chicago/Turabian Style

Gibson, Eddie, Daniel A. Ollendorf, Steven Simoens, David E Bloom, Federico Martinón-Torres, David Salisbury, Johan Louis Severens, Mondher Toumi, Daniel Molnar, Kinga Meszaros, and et al. 2023. "Rule of Prevention: A Potential Framework to Evaluate Preventive Interventions for Rare Diseases" Journal of Market Access & Health Policy 11, no. 1: 2239557. https://doi.org/10.1080/20016689.2023.2239557

APA Style

Gibson, E., Ollendorf, D. A., Simoens, S., Bloom, D. E., Martinón-Torres, F., Salisbury, D., Severens, J. L., Toumi, M., Molnar, D., Meszaros, K., Sohn, W. -Y., & Begum, N. (2023). Rule of Prevention: A Potential Framework to Evaluate Preventive Interventions for Rare Diseases. Journal of Market Access & Health Policy, 11(1), 2239557. https://doi.org/10.1080/20016689.2023.2239557

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