Criteria to define rare diseases and orphan drugs: a systematic review protocol
Introduction Rare diseases (RDs) are often chronic and progressive life-threatening medical conditions that affect a low percentage of the population compared with other diseases. These conditions can be treated with medications known as orphan drugs (ODs). Unfortunately, there is no universal defin...
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| Format: | Article |
| Language: | English |
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BMJ Publishing Group
2022-07-01
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| Series: | BMJ Open |
| Online Access: | https://bmjopen.bmj.com/content/12/7/e062126.full |
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| author | Katie Kerr Ghada Mohammed Abozaid Amy McKnight Hussain A Al-Omar |
| author_facet | Katie Kerr Ghada Mohammed Abozaid Amy McKnight Hussain A Al-Omar |
| author_sort | Katie Kerr |
| collection | DOAJ |
| description | Introduction Rare diseases (RDs) are often chronic and progressive life-threatening medical conditions that affect a low percentage of the population compared with other diseases. These conditions can be treated with medications known as orphan drugs (ODs). Unfortunately, there is no universal definition of RDs or ODs. This systematic review (SR) will identify the quantitative and qualitative criteria and the underlying rationale used internationally to define RDs and ODs.Methods and analysis This protocol follows the conventions for the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (2015 guidelines). A SR will be conducted, including a search of the following databases: PubMed, MEDLINE, EMBASE, Scopus, Web of Science, GreyLit and OpenGrey. Eligible publications will be selected based on predetermined inclusion criteria. Extracted data will be analysed using thematic and content analyses of qualitative descriptors, whereas quantitative data will be analysed descriptively and reported in the form of frequencies and percentages.Ethics and dissemination No ethical approval is required since this SR focuses on the secondary analysis of data retrieved from the scientific literature. The outcomes of this SR will be published as part of a PhD thesis, presented at conferences, and published in peer-reviewed journals.PROSPERO registration number CRD42021252701. |
| format | Article |
| id | doaj-art-2c49fdb9848546c2929eded1da0e1278 |
| institution | Kabale University |
| issn | 2044-6055 |
| language | English |
| publishDate | 2022-07-01 |
| publisher | BMJ Publishing Group |
| record_format | Article |
| series | BMJ Open |
| spelling | doaj-art-2c49fdb9848546c2929eded1da0e12782025-01-31T16:45:12ZengBMJ Publishing GroupBMJ Open2044-60552022-07-0112710.1136/bmjopen-2022-062126Criteria to define rare diseases and orphan drugs: a systematic review protocolKatie Kerr0Ghada Mohammed Abozaid1Amy McKnight2Hussain A Al-Omar3Institute of Clinical Sciences B, Royal Victoria Hospital, Queen’s University Belfast School of Medicine, Dentistry and Biomedical Sciences,Centre for Public Health, Belfast, UKDepartment of Pharmacy Practice, Princess Nourah bint Abdulrahman University, Riyadh, Saudi Arabia1 Centre for Public Health, Queen`s University Belfast, Belfast, UKDeparment of Clinical Pharmacy, College of Pharmacy, King Saud University, Riyadh, Saudi ArabiaIntroduction Rare diseases (RDs) are often chronic and progressive life-threatening medical conditions that affect a low percentage of the population compared with other diseases. These conditions can be treated with medications known as orphan drugs (ODs). Unfortunately, there is no universal definition of RDs or ODs. This systematic review (SR) will identify the quantitative and qualitative criteria and the underlying rationale used internationally to define RDs and ODs.Methods and analysis This protocol follows the conventions for the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (2015 guidelines). A SR will be conducted, including a search of the following databases: PubMed, MEDLINE, EMBASE, Scopus, Web of Science, GreyLit and OpenGrey. Eligible publications will be selected based on predetermined inclusion criteria. Extracted data will be analysed using thematic and content analyses of qualitative descriptors, whereas quantitative data will be analysed descriptively and reported in the form of frequencies and percentages.Ethics and dissemination No ethical approval is required since this SR focuses on the secondary analysis of data retrieved from the scientific literature. The outcomes of this SR will be published as part of a PhD thesis, presented at conferences, and published in peer-reviewed journals.PROSPERO registration number CRD42021252701.https://bmjopen.bmj.com/content/12/7/e062126.full |
| spellingShingle | Katie Kerr Ghada Mohammed Abozaid Amy McKnight Hussain A Al-Omar Criteria to define rare diseases and orphan drugs: a systematic review protocol BMJ Open |
| title | Criteria to define rare diseases and orphan drugs: a systematic review protocol |
| title_full | Criteria to define rare diseases and orphan drugs: a systematic review protocol |
| title_fullStr | Criteria to define rare diseases and orphan drugs: a systematic review protocol |
| title_full_unstemmed | Criteria to define rare diseases and orphan drugs: a systematic review protocol |
| title_short | Criteria to define rare diseases and orphan drugs: a systematic review protocol |
| title_sort | criteria to define rare diseases and orphan drugs a systematic review protocol |
| url | https://bmjopen.bmj.com/content/12/7/e062126.full |
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