Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocol
Introduction Rare disease treatments (RDTs) promise considerable patient benefit but the evidence to demonstrate their value in health technology assessment (HTA) is often limited. HTA outcomes for RDTs vary across countries and there are differences in how uncertainty is dealt with by HTA agencies....
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BMJ Publishing Group
2025-02-01
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| Series: | BMJ Open |
| Online Access: | https://bmjopen.bmj.com/content/15/2/e089418.full |
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| author | Ellen Nolte John Cairns Lea Wiedmann |
| author_facet | Ellen Nolte John Cairns Lea Wiedmann |
| author_sort | Ellen Nolte |
| collection | DOAJ |
| description | Introduction Rare disease treatments (RDTs) promise considerable patient benefit but the evidence to demonstrate their value in health technology assessment (HTA) is often limited. HTA outcomes for RDTs vary across countries and there are differences in how uncertainty is dealt with by HTA agencies. Yet, there is limited comparative research assessing how different HTA agencies consider issues affecting evidence quality and uncertainty in RDT appraisals. This protocol describes a systematic and consistent approach for data extraction from RDT appraisal documents produced to inform decisions by HTA agencies. By documenting data extraction rules transparently, we ensure reproducibility and reliability of analyses of the extracted data.Methods and analysis We will select RDT appraisals issued by the National Institute for Health and Care Excellence (NICE) in England and the Federal Joint Committee (GBA) in Germany, using predefined inclusion criteria. We will extract data from appraisal documents in accordance with the rules set out in this protocol. We will analyse the extracted data to investigate how issues affecting evidence quality and uncertainty as documented in appraisals are considered, highlighting the similarities and differences between countries and identifying factors that are associated with HTA outcomes.Ethics and dissemination This study was approved by the Ethics Committee of the London School of Hygiene & Tropical Medicine (reference number 29156). Study results will be submitted for publication in peer-reviewed journals. |
| format | Article |
| id | doaj-art-63c4da45c0b7434fadcbf5501076a107 |
| institution | DOAJ |
| issn | 2044-6055 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | BMJ Publishing Group |
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| series | BMJ Open |
| spelling | doaj-art-63c4da45c0b7434fadcbf5501076a1072025-08-20T02:43:12ZengBMJ Publishing GroupBMJ Open2044-60552025-02-0115210.1136/bmjopen-2024-089418Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocolEllen Nolte0John Cairns1Lea Wiedmann2Department of Health Services Research and Policy, London School of Hygiene & Tropical Medicine, London, UKDepartment of Health Services Research and Policy, London School of Hygiene & Tropical Medicine, London, UKDepartment of Health Services Research and Policy, London School of Hygiene & Tropical Medicine, London, UKIntroduction Rare disease treatments (RDTs) promise considerable patient benefit but the evidence to demonstrate their value in health technology assessment (HTA) is often limited. HTA outcomes for RDTs vary across countries and there are differences in how uncertainty is dealt with by HTA agencies. Yet, there is limited comparative research assessing how different HTA agencies consider issues affecting evidence quality and uncertainty in RDT appraisals. This protocol describes a systematic and consistent approach for data extraction from RDT appraisal documents produced to inform decisions by HTA agencies. By documenting data extraction rules transparently, we ensure reproducibility and reliability of analyses of the extracted data.Methods and analysis We will select RDT appraisals issued by the National Institute for Health and Care Excellence (NICE) in England and the Federal Joint Committee (GBA) in Germany, using predefined inclusion criteria. We will extract data from appraisal documents in accordance with the rules set out in this protocol. We will analyse the extracted data to investigate how issues affecting evidence quality and uncertainty as documented in appraisals are considered, highlighting the similarities and differences between countries and identifying factors that are associated with HTA outcomes.Ethics and dissemination This study was approved by the Ethics Committee of the London School of Hygiene & Tropical Medicine (reference number 29156). Study results will be submitted for publication in peer-reviewed journals.https://bmjopen.bmj.com/content/15/2/e089418.full |
| spellingShingle | Ellen Nolte John Cairns Lea Wiedmann Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocol BMJ Open |
| title | Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocol |
| title_full | Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocol |
| title_fullStr | Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocol |
| title_full_unstemmed | Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocol |
| title_short | Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocol |
| title_sort | evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in england and germany a data extraction protocol |
| url | https://bmjopen.bmj.com/content/15/2/e089418.full |
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