Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance
Background: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). Objectives: Review HTA bodies’ (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and...
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MDPI AG
2024-05-01
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| Series: | Journal of Market Access & Health Policy |
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| Online Access: | https://www.mdpi.com/2001-6689/12/2/9 |
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| author | Katia Thokagevistk Céline Coppo Laetitia Rey Amanda Carelli Veronica Díez Sarah Vaselenak Liana Oliveira Ajay Patel Emilia Sicari Teresa Ramos Susanne Schach Erika Schirghuber Alex Simpson Remy Choquet Katell Le Lay |
| author_facet | Katia Thokagevistk Céline Coppo Laetitia Rey Amanda Carelli Veronica Díez Sarah Vaselenak Liana Oliveira Ajay Patel Emilia Sicari Teresa Ramos Susanne Schach Erika Schirghuber Alex Simpson Remy Choquet Katell Le Lay |
| author_sort | Katia Thokagevistk |
| collection | DOAJ |
| description | Background: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). Objectives: Review HTA bodies’ (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and outline recommendations that may improve acceptance of RWE in efficacy/effectiveness assessments and appraisals processes. Methods: RWE requirements were summarized based on HTAbs’ guidelines. Acceptance by HTAbs was evaluated based on industry experience and case studies. Results: As of June 2022, RWE methodological guidelines were in place in three of the seven countries. HTAbs typically requested analyses based on local data sources, but the preferred study design and data sources differed. HTAbs had individual submission, assessment, and appraisal processes; some allowed early meetings for the protocol and/or results validation, though few involved external experts or medical societies to provide input to assessment and appraisal. The extent of submission, assessment, and appraisal requirements did not necessarily reflect the degree of acceptance. Conclusion: All the countries reviewed face common challenges regarding the use of RWE. Our proposals address the need to facilitate collaboration and communication with industry and regulatory agencies and the need for specific guidelines describing RWE design and criteria of acceptance throughout the assessment and appraisal processes. |
| format | Article |
| id | doaj-art-402fac064ee64a2797cde8a8b2c31ab4 |
| institution | OA Journals |
| issn | 2001-6689 |
| language | English |
| publishDate | 2024-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Market Access & Health Policy |
| spelling | doaj-art-402fac064ee64a2797cde8a8b2c31ab42025-08-20T02:16:23ZengMDPI AGJournal of Market Access & Health Policy2001-66892024-05-0112210511710.3390/jmahp12020009Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve AcceptanceKatia Thokagevistk0Céline Coppo1Laetitia Rey2Amanda Carelli3Veronica Díez4Sarah Vaselenak5Liana Oliveira6Ajay Patel7Emilia Sicari8Teresa Ramos9Susanne Schach10Erika Schirghuber11Alex Simpson12Remy Choquet13Katell Le Lay14Roche, 4 Cr de l’Île Seguin, 92100 Boulogne-Billancourt, FranceIQVIA, 17 bis Tsse. des Reflets, 92400 Courbevoie, FranceIQVIA, 17 bis Tsse. des Reflets, 92400 Courbevoie, FranceRoche, Rua Dr. Rubens Gomes Bueno, 691—Várzea de Baixo, São Paulo 04730-903, BrazilF. Hoffmann-La Roche Ltd., C/Ribera del Loira 50, 28042 Madrid, SpainRoche, 7070 Mississauga Rd, Mississauga, ON L5N 5M8, CanadaRoche, Rua Dr. Rubens Gomes Bueno, 691—Várzea de Baixo, São Paulo 04730-903, BrazilRoche Products Ltd., Hexagon Place, Shire Park, Falcon Way, Welwyn Garden City AL7 1TW, UKRoche SpA, Viale Gian Battista Stucchi, 110, 20900 Monza, ItalyF. Hoffmann-La Roche Ltd., C/Ribera del Loira 50, 28042 Madrid, SpainRoche, Emil-Barell-Straße 1, 79639 Grenzach-Wyhlen, GermanyF. Hoffmann-La Roche Ltd., Grenzacherstrasse 124, 4070 Basel, SwitzerlandF. Hoffmann-La Roche Ltd., Grenzacherstrasse 124, 4070 Basel, SwitzerlandRoche, 4 Cr de l’Île Seguin, 92100 Boulogne-Billancourt, FranceRoche, 4 Cr de l’Île Seguin, 92100 Boulogne-Billancourt, FranceBackground: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). Objectives: Review HTA bodies’ (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and outline recommendations that may improve acceptance of RWE in efficacy/effectiveness assessments and appraisals processes. Methods: RWE requirements were summarized based on HTAbs’ guidelines. Acceptance by HTAbs was evaluated based on industry experience and case studies. Results: As of June 2022, RWE methodological guidelines were in place in three of the seven countries. HTAbs typically requested analyses based on local data sources, but the preferred study design and data sources differed. HTAbs had individual submission, assessment, and appraisal processes; some allowed early meetings for the protocol and/or results validation, though few involved external experts or medical societies to provide input to assessment and appraisal. The extent of submission, assessment, and appraisal requirements did not necessarily reflect the degree of acceptance. Conclusion: All the countries reviewed face common challenges regarding the use of RWE. Our proposals address the need to facilitate collaboration and communication with industry and regulatory agencies and the need for specific guidelines describing RWE design and criteria of acceptance throughout the assessment and appraisal processes.https://www.mdpi.com/2001-6689/12/2/9real-world evidencereal-world datahealth technology assessmenthealth policiesguidelinesappraisal |
| spellingShingle | Katia Thokagevistk Céline Coppo Laetitia Rey Amanda Carelli Veronica Díez Sarah Vaselenak Liana Oliveira Ajay Patel Emilia Sicari Teresa Ramos Susanne Schach Erika Schirghuber Alex Simpson Remy Choquet Katell Le Lay Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance Journal of Market Access & Health Policy real-world evidence real-world data health technology assessment health policies guidelines appraisal |
| title | Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance |
| title_full | Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance |
| title_fullStr | Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance |
| title_full_unstemmed | Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance |
| title_short | Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance |
| title_sort | real world evidence to reinforce clinical trial evidence in health technology assessment a critical review of real world evidence requirements from seven countries and recommendations to improve acceptance |
| topic | real-world evidence real-world data health technology assessment health policies guidelines appraisal |
| url | https://www.mdpi.com/2001-6689/12/2/9 |
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