The evolving role and utility of off-label drug use in multiple myeloma
The treatment landscape for multiple myeloma (MM) has dramatically changed over the last three decades, moving from no US Food and Drug Administration approvals and two active drug classes to over 19 drug approvals and at least eight different active classes. The advances seen in MM therapy have rel...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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Open Exploration Publishing Inc.
2021-08-01
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| Series: | Exploration of Targeted Anti-tumor Therapy |
| Subjects: | |
| Online Access: | https://www.explorationpub.com/Journals/etat/Article/100250 |
| _version_ | 1819130450964119552 |
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| author | James H Stoeckle Faith E Davies Louis Williams Eileen M Boyle Gareth J Morgan |
| author_facet | James H Stoeckle Faith E Davies Louis Williams Eileen M Boyle Gareth J Morgan |
| author_sort | James H Stoeckle |
| collection | DOAJ |
| description | The treatment landscape for multiple myeloma (MM) has dramatically changed over the last three decades, moving from no US Food and Drug Administration approvals and two active drug classes to over 19 drug approvals and at least eight different active classes. The advances seen in MM therapy have relied on both a structured approach to obtaining new labels and cautious off-label drug use. Although there are country and regional differences in drug approval processes, many of the basic principles behind off-label drug use in MM can be summarized into four main categories: 1) use of a therapy prior to the current approval regulations; 2) widespread use of a therapy following the release of promising clinical trial results but prior to drug approval; 3) use of a cheap therapy supported by clinical safety and efficacy data but without commercial backing; and 4) niche therapies for small well-defined patient populations where large clinical trials with sufficient power may be difficult to perform. This review takes a historical approach to discuss how off-label drug use has helped to shape the current treatment approach for MM. |
| first_indexed | 2024-12-22T08:59:49Z |
| format | Article |
| id | doaj.art-c58c94026e9649019d0f06e2f35dfc4f |
| institution | Directory Open Access Journal |
| issn | 2692-3114 |
| language | English |
| last_indexed | 2024-12-22T08:59:49Z |
| publishDate | 2021-08-01 |
| publisher | Open Exploration Publishing Inc. |
| record_format | Article |
| series | Exploration of Targeted Anti-tumor Therapy |
| spelling | doaj.art-c58c94026e9649019d0f06e2f35dfc4f2022-12-21T18:31:45ZengOpen Exploration Publishing Inc.Exploration of Targeted Anti-tumor Therapy2692-31142021-08-012410.37349/etat.2021.00050The evolving role and utility of off-label drug use in multiple myelomaJames H Stoeckle0https://orcid.org/0000-0003-3377-5120Faith E Davies1https://orcid.org/0000-0002-3971-2393Louis Williams2https://orcid.org/0000-0001-6680-4608Eileen M Boyle3https://orcid.org/0000-0001-8791-1744Gareth J Morgan4https://orcid.org/0000-0002-4271-6360Perlmutter Cancer Center, New York University Langone Health, New York, NY 10016, USAPerlmutter Cancer Center, New York University Langone Health, New York, NY 10016, USAPerlmutter Cancer Center, New York University Langone Health, New York, NY 10016, USAPerlmutter Cancer Center, New York University Langone Health, New York, NY 10016, USAPerlmutter Cancer Center, New York University Langone Health, New York, NY 10016, USAThe treatment landscape for multiple myeloma (MM) has dramatically changed over the last three decades, moving from no US Food and Drug Administration approvals and two active drug classes to over 19 drug approvals and at least eight different active classes. The advances seen in MM therapy have relied on both a structured approach to obtaining new labels and cautious off-label drug use. Although there are country and regional differences in drug approval processes, many of the basic principles behind off-label drug use in MM can be summarized into four main categories: 1) use of a therapy prior to the current approval regulations; 2) widespread use of a therapy following the release of promising clinical trial results but prior to drug approval; 3) use of a cheap therapy supported by clinical safety and efficacy data but without commercial backing; and 4) niche therapies for small well-defined patient populations where large clinical trials with sufficient power may be difficult to perform. This review takes a historical approach to discuss how off-label drug use has helped to shape the current treatment approach for MM.https://www.explorationpub.com/Journals/etat/Article/100250multiple myelomaoff-labeldrug developmentorphan drugsthalidomiderelapsed refractory |
| spellingShingle | James H Stoeckle Faith E Davies Louis Williams Eileen M Boyle Gareth J Morgan The evolving role and utility of off-label drug use in multiple myeloma Exploration of Targeted Anti-tumor Therapy multiple myeloma off-label drug development orphan drugs thalidomide relapsed refractory |
| title | The evolving role and utility of off-label drug use in multiple myeloma |
| title_full | The evolving role and utility of off-label drug use in multiple myeloma |
| title_fullStr | The evolving role and utility of off-label drug use in multiple myeloma |
| title_full_unstemmed | The evolving role and utility of off-label drug use in multiple myeloma |
| title_short | The evolving role and utility of off-label drug use in multiple myeloma |
| title_sort | evolving role and utility of off label drug use in multiple myeloma |
| topic | multiple myeloma off-label drug development orphan drugs thalidomide relapsed refractory |
| url | https://www.explorationpub.com/Journals/etat/Article/100250 |
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