An approach to data collection in compassionate use/managed access
Compassionate Use (CU)/Managed Access programs provide access to locally unapproved medicines. As these programs become more global and involve a broader range of products, determining whether patients derive benefit from treatment could provide insights into therapeutic use in a real-word setting w...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2022-12-01
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| Series: | Frontiers in Pharmacology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fphar.2022.1095860/full |
| _version_ | 1811290721904230400 |
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| author | Séverine Sarp Ramona Reichenbach Paul Aliu |
| author_facet | Séverine Sarp Ramona Reichenbach Paul Aliu |
| author_sort | Séverine Sarp |
| collection | DOAJ |
| description | Compassionate Use (CU)/Managed Access programs provide access to locally unapproved medicines. As these programs become more global and involve a broader range of products, determining whether patients derive benefit from treatment could provide insights into therapeutic use in a real-word setting with diverse pools of patients. CU primary purpose is to provide treatment and it is not targeting research. However, it is increasingly considered as a source of real-world data. In the absence of a harmonized framework on CU data collection, Novartis developed a company-wide guidance to collect baseline patient data and prospective follow-up information at product resupply. Although this approach has recently been implemented and utilization of this data has been mainly internal to the company so far, the prospective collection of key efficacy parameters in patients receiving therapies via CU could potentially be used as a supportive set of information collected in a real-world setting to be submitted in addition to clinical trial data, if not as a main source of data for regulatory submission. |
| first_indexed | 2024-04-13T04:18:51Z |
| format | Article |
| id | doaj.art-94f719b17d4942d182470e8cec356a19 |
| institution | Directory Open Access Journal |
| issn | 1663-9812 |
| language | English |
| last_indexed | 2024-04-13T04:18:51Z |
| publishDate | 2022-12-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj.art-94f719b17d4942d182470e8cec356a192022-12-22T03:02:54ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122022-12-011310.3389/fphar.2022.10958601095860An approach to data collection in compassionate use/managed accessSéverine SarpRamona ReichenbachPaul AliuCompassionate Use (CU)/Managed Access programs provide access to locally unapproved medicines. As these programs become more global and involve a broader range of products, determining whether patients derive benefit from treatment could provide insights into therapeutic use in a real-word setting with diverse pools of patients. CU primary purpose is to provide treatment and it is not targeting research. However, it is increasingly considered as a source of real-world data. In the absence of a harmonized framework on CU data collection, Novartis developed a company-wide guidance to collect baseline patient data and prospective follow-up information at product resupply. Although this approach has recently been implemented and utilization of this data has been mainly internal to the company so far, the prospective collection of key efficacy parameters in patients receiving therapies via CU could potentially be used as a supportive set of information collected in a real-world setting to be submitted in addition to clinical trial data, if not as a main source of data for regulatory submission.https://www.frontiersin.org/articles/10.3389/fphar.2022.1095860/fullcompassionate use of drugspatient accessmanaged accessreal-world datapatient-centered carepatients’ benefits |
| spellingShingle | Séverine Sarp Ramona Reichenbach Paul Aliu An approach to data collection in compassionate use/managed access Frontiers in Pharmacology compassionate use of drugs patient access managed access real-world data patient-centered care patients’ benefits |
| title | An approach to data collection in compassionate use/managed access |
| title_full | An approach to data collection in compassionate use/managed access |
| title_fullStr | An approach to data collection in compassionate use/managed access |
| title_full_unstemmed | An approach to data collection in compassionate use/managed access |
| title_short | An approach to data collection in compassionate use/managed access |
| title_sort | approach to data collection in compassionate use managed access |
| topic | compassionate use of drugs patient access managed access real-world data patient-centered care patients’ benefits |
| url | https://www.frontiersin.org/articles/10.3389/fphar.2022.1095860/full |
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