Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases
ABSTRACTBackground: 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 prevent...
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-12-01
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| Series: | Journal of Market Access & Health Policy |
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| Online Access: | https://www.tandfonline.com/doi/10.1080/20016689.2023.2239557 |
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| author | Eddie Gibson Daniel A. Ollendorf Steven Simoens David E Bloom Federico Martinón-Torres David Salisbury Johan Louis Severens Mondher Toumi Daniel Molnar Kinga Meszaros Woo-Yun Sohn Najida Begum |
| author_facet | Eddie Gibson Daniel A. Ollendorf Steven Simoens David E Bloom Federico Martinón-Torres David Salisbury Johan Louis Severens Mondher Toumi Daniel Molnar Kinga Meszaros Woo-Yun Sohn Najida Begum |
| author_sort | Eddie Gibson |
| collection | DOAJ |
| description | ABSTRACTBackground: 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. |
| format | Article |
| id | doaj-art-4432939b134d41eb9d2da682a1d51f85 |
| institution | OA Journals |
| issn | 2001-6689 |
| language | English |
| publishDate | 2023-12-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Market Access & Health Policy |
| spelling | doaj-art-4432939b134d41eb9d2da682a1d51f852025-08-20T01:56:45ZengMDPI AGJournal of Market Access & Health Policy2001-66892023-12-0111110.1080/20016689.2023.2239557Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseasesEddie Gibson0Daniel A. Ollendorf1Steven Simoens2David E Bloom3Federico Martinón-Torres4David Salisbury5Johan Louis Severens6Mondher Toumi7Daniel Molnar8Kinga Meszaros9Woo-Yun Sohn10Najida Begum11Wickenstones Ltd, Abingdon, UKInstitute for Clinical Research and Health Policy Studies (ICRHPS), Center for the Evaluation of Value and Risk in Health (CEVR), Tufts Medical Center, Boston, MA, USAClinical Pharmacology and Pharmacotherapy, KU Leuven, Leuven, BelgiumHarvard T.H. Chan School of Public Health, Boston, MA, USADepartment of Pediatrics, Translational Pediatrics and Infectious Diseases, Pediatrics Department, Hospital Clínico Universitario de Santiago de Compostela, Santiago de Compostela, SpainRoyal Institute of International Affairs, Chatham House, London, UKSeverens HTA Consultancy, Venray, The NetherlandsInovIntell, Île-de-France, FranceGSK, Wavre, BelgiumGSK, Wavre, BelgiumGSK, Philadelphia, MD, USAFreelance, C/O Gsk, Wavre, BelgiumABSTRACTBackground: 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.https://www.tandfonline.com/doi/10.1080/20016689.2023.2239557Preventionvaccinationassessmentvaluerare disease |
| spellingShingle | Eddie Gibson Daniel A. Ollendorf Steven Simoens David E Bloom Federico Martinón-Torres David Salisbury Johan Louis Severens Mondher Toumi Daniel Molnar Kinga Meszaros Woo-Yun Sohn Najida Begum Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases Journal of Market Access & Health Policy Prevention vaccination assessment value rare disease |
| title | Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases |
| title_full | Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases |
| title_fullStr | Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases |
| title_full_unstemmed | Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases |
| title_short | Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases |
| title_sort | rule of prevention a potential framework to evaluate preventive interventions for rare diseases |
| topic | Prevention vaccination assessment value rare disease |
| url | https://www.tandfonline.com/doi/10.1080/20016689.2023.2239557 |
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