Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration
Background: Claims included in package inserts (PIs) for medicinal products approved by the US Food and Drug Administration (FDA) constitute the regulatory definition of drugs’ benefits and risks. Objective: We sought to assess the usage of patient-reported outcome (PRO) claims in a comprehensive se...
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| Format: | Article |
| Language: | English |
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MDPI AG
2018-01-01
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| Series: | Journal of Market Access & Health Policy |
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| Online Access: | http://dx.doi.org/10.1080/20016689.2018.1433426 |
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| _version_ | 1849765503178899456 |
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| author | Szymon Jarosławski Pascal Auquier Borislav Borissov Claude Dussart Mondher Toumi |
| author_facet | Szymon Jarosławski Pascal Auquier Borislav Borissov Claude Dussart Mondher Toumi |
| author_sort | Szymon Jarosławski |
| collection | DOAJ |
| description | Background: Claims included in package inserts (PIs) for medicinal products approved by the US Food and Drug Administration (FDA) constitute the regulatory definition of drugs’ benefits and risks. Objective: We sought to assess the usage of patient-reported outcome (PRO) claims in a comprehensive set of US FDA orphan drug approvals dated between 1/1/2012 and 31/12/2016, and characterize them. Study design: Orphan drug approval documentation was obtained from the US FDA website. Drug Package Inserts (PI) were analyzed to extract information on PRO-related language. Results: Among 178 drugs that met inclusion criteria, 16 (9%) products approved for 16 orphan indications contained PRO language in the Clinical Studies section of the PI. All PRO instruments concerned disease symptoms, and two also referred to patient functioning. The most common PRO instrument was a bleed-specific rating scale for four products approved for the treatment or prevention of bleeding episodes in patients with genetic bleeding disorders. Conclusions: There is a need to implement public incentives for academic development of PRO instruments for rare conditions and for regulatory policies that mandate the collection of PRO endpoints in pivotal trials of orphan drugs. |
| format | Article |
| id | doaj-art-ba71eac0958545c5962cf8a213f89b39 |
| institution | DOAJ |
| issn | 2001-6689 |
| language | English |
| publishDate | 2018-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Market Access & Health Policy |
| spelling | doaj-art-ba71eac0958545c5962cf8a213f89b392025-08-20T03:04:50ZengMDPI AGJournal of Market Access & Health Policy2001-66892018-01-016110.1080/20016689.2018.14334261433426Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administrationSzymon Jarosławski0Pascal Auquier1Borislav Borissov2Claude Dussart3Mondher Toumi4Aix – Marseille UniversityAix – Marseille UniversityPrescriptia LtdClaude Bernard UniversityAix – Marseille UniversityBackground: Claims included in package inserts (PIs) for medicinal products approved by the US Food and Drug Administration (FDA) constitute the regulatory definition of drugs’ benefits and risks. Objective: We sought to assess the usage of patient-reported outcome (PRO) claims in a comprehensive set of US FDA orphan drug approvals dated between 1/1/2012 and 31/12/2016, and characterize them. Study design: Orphan drug approval documentation was obtained from the US FDA website. Drug Package Inserts (PI) were analyzed to extract information on PRO-related language. Results: Among 178 drugs that met inclusion criteria, 16 (9%) products approved for 16 orphan indications contained PRO language in the Clinical Studies section of the PI. All PRO instruments concerned disease symptoms, and two also referred to patient functioning. The most common PRO instrument was a bleed-specific rating scale for four products approved for the treatment or prevention of bleeding episodes in patients with genetic bleeding disorders. Conclusions: There is a need to implement public incentives for academic development of PRO instruments for rare conditions and for regulatory policies that mandate the collection of PRO endpoints in pivotal trials of orphan drugs.http://dx.doi.org/10.1080/20016689.2018.1433426Patient-reported outcomesquality of liferare diseasesorphan drugslabelling |
| spellingShingle | Szymon Jarosławski Pascal Auquier Borislav Borissov Claude Dussart Mondher Toumi Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration Journal of Market Access & Health Policy Patient-reported outcomes quality of life rare diseases orphan drugs labelling |
| title | Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration |
| title_full | Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration |
| title_fullStr | Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration |
| title_full_unstemmed | Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration |
| title_short | Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration |
| title_sort | low rates of patient reported outcome claims for orphan drugs approved by the us food and drug administration |
| topic | Patient-reported outcomes quality of life rare diseases orphan drugs labelling |
| url | http://dx.doi.org/10.1080/20016689.2018.1433426 |
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