Cargando…
Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021
IMPORTANCE: The US Food and Drug Administration (FDA) has 4 programs that can be used alone or in combination to expedite drug availability: Accelerated Approval, Breakthrough Therapy, Fast Track, and Priority Review. Drugs using these programs can include novel drugs, which do not contain a previou...
Autores principales: | , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Medical Association
2022
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9627417/ https://www.ncbi.nlm.nih.gov/pubmed/36318210 http://dx.doi.org/10.1001/jamanetworkopen.2022.39336 |
_version_ | 1784822964657586176 |
---|---|
author | Monge, Andrea N. Sigelman, Daniel W. Temple, Robert J. Chahal, Harinder Singh |
author_facet | Monge, Andrea N. Sigelman, Daniel W. Temple, Robert J. Chahal, Harinder Singh |
author_sort | Monge, Andrea N. |
collection | PubMed |
description | IMPORTANCE: The US Food and Drug Administration (FDA) has 4 programs that can be used alone or in combination to expedite drug availability: Accelerated Approval, Breakthrough Therapy, Fast Track, and Priority Review. Drugs using these programs can include novel drugs, which do not contain a previously FDA-approved active moiety, and orphan drugs, intended for diseases or conditions affecting fewer than 200 000 people; to date, no comprehensive evaluation of how these programs have been used in combination has been published. OBJECTIVE: To assess how often and in what combinations expedited programs are used in the development and review of approved novel biologics and small-molecule drugs, stratified by orphan drug status and indication. DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study evaluated all novel drugs that were FDA approved between January 1, 2008, and December 31, 2021. MAIN OUTCOMES AND MEASURES: The main outcome was the frequency with which expedited programs were used and in what combinations, stratified by orphan drug status and drug type (small molecule vs therapeutic biologic). The unit of analysis was the novel drug–indication pair because a drug can be approved for multiple indications, each of which may use a different expedited program or differ in orphan drug status. RESULTS: The study included 581 novel drug–indication pairs approved during the 14-year study period; 252 (43.4%) were orphan drugs, 139 (23.9%) were therapeutic biologics, and 442 (76.1%) were small-molecule drugs. Use of at least 1 expedited program increased from 11 of 26 drug-indication pairs (42.3%) in 2008 to 41 of 55 (74.5%) in 2021. Of the 363 approved drug-indication pairs using at least 1 expedited program, 225 (62.0%) were orphan drugs; at least 1 expedited program was used by 97 of the 139 approved biologic drugs (69.8%) and by 266 of the 442 approved small-molecule drugs (60.2%). Eighty-two of the 581 novel drug–indication pairs (14.1%) used the Accelerated Approval Program; of those, 65 (79.3%) were oncology drugs and 70 (85.4%) had an orphan designation. CONCLUSIONS AND RELEVANCE: The study showed that use of the FDA’s expedited programs to bring novel drugs to market in the US increased from 2008 to 2021. The findings suggest that this trend is likely to continue. |
format | Online Article Text |
id | pubmed-9627417 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | American Medical Association |
record_format | MEDLINE/PubMed |
spelling | pubmed-96274172022-11-28 Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021 Monge, Andrea N. Sigelman, Daniel W. Temple, Robert J. Chahal, Harinder Singh JAMA Netw Open Original Investigation IMPORTANCE: The US Food and Drug Administration (FDA) has 4 programs that can be used alone or in combination to expedite drug availability: Accelerated Approval, Breakthrough Therapy, Fast Track, and Priority Review. Drugs using these programs can include novel drugs, which do not contain a previously FDA-approved active moiety, and orphan drugs, intended for diseases or conditions affecting fewer than 200 000 people; to date, no comprehensive evaluation of how these programs have been used in combination has been published. OBJECTIVE: To assess how often and in what combinations expedited programs are used in the development and review of approved novel biologics and small-molecule drugs, stratified by orphan drug status and indication. DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study evaluated all novel drugs that were FDA approved between January 1, 2008, and December 31, 2021. MAIN OUTCOMES AND MEASURES: The main outcome was the frequency with which expedited programs were used and in what combinations, stratified by orphan drug status and drug type (small molecule vs therapeutic biologic). The unit of analysis was the novel drug–indication pair because a drug can be approved for multiple indications, each of which may use a different expedited program or differ in orphan drug status. RESULTS: The study included 581 novel drug–indication pairs approved during the 14-year study period; 252 (43.4%) were orphan drugs, 139 (23.9%) were therapeutic biologics, and 442 (76.1%) were small-molecule drugs. Use of at least 1 expedited program increased from 11 of 26 drug-indication pairs (42.3%) in 2008 to 41 of 55 (74.5%) in 2021. Of the 363 approved drug-indication pairs using at least 1 expedited program, 225 (62.0%) were orphan drugs; at least 1 expedited program was used by 97 of the 139 approved biologic drugs (69.8%) and by 266 of the 442 approved small-molecule drugs (60.2%). Eighty-two of the 581 novel drug–indication pairs (14.1%) used the Accelerated Approval Program; of those, 65 (79.3%) were oncology drugs and 70 (85.4%) had an orphan designation. CONCLUSIONS AND RELEVANCE: The study showed that use of the FDA’s expedited programs to bring novel drugs to market in the US increased from 2008 to 2021. The findings suggest that this trend is likely to continue. American Medical Association 2022-11-01 /pmc/articles/PMC9627417/ /pubmed/36318210 http://dx.doi.org/10.1001/jamanetworkopen.2022.39336 Text en Copyright 2022 Monge AN et al. JAMA Network Open. https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the terms of the CC-BY License. |
spellingShingle | Original Investigation Monge, Andrea N. Sigelman, Daniel W. Temple, Robert J. Chahal, Harinder Singh Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021 |
title | Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021 |
title_full | Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021 |
title_fullStr | Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021 |
title_full_unstemmed | Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021 |
title_short | Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021 |
title_sort | use of us food and drug administration expedited drug development and review programs by orphan and nonorphan novel drugs approved from 2008 to 2021 |
topic | Original Investigation |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9627417/ https://www.ncbi.nlm.nih.gov/pubmed/36318210 http://dx.doi.org/10.1001/jamanetworkopen.2022.39336 |
work_keys_str_mv | AT mongeandrean useofusfoodanddrugadministrationexpediteddrugdevelopmentandreviewprogramsbyorphanandnonorphannoveldrugsapprovedfrom2008to2021 AT sigelmandanielw useofusfoodanddrugadministrationexpediteddrugdevelopmentandreviewprogramsbyorphanandnonorphannoveldrugsapprovedfrom2008to2021 AT templerobertj useofusfoodanddrugadministrationexpediteddrugdevelopmentandreviewprogramsbyorphanandnonorphannoveldrugsapprovedfrom2008to2021 AT chahalharindersingh useofusfoodanddrugadministrationexpediteddrugdevelopmentandreviewprogramsbyorphanandnonorphannoveldrugsapprovedfrom2008to2021 |