Gene therapies development: slow progress and promising prospect
Background: In 1989, the concept of human gene therapies has emerged with the first approved human gene therapy trial of Rosenberg et al. Gene therapies are considered as promising therapies applicable to a broad range of diseases. Objective: The objective of this study was to review the descriptive...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2017-01-01
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| Series: | Journal of Market Access & Health Policy |
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| Online Access: | http://dx.doi.org/10.1080/20016689.2017.1265293 |
| _version_ | 1797227476426948608 |
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| author | Eve Hanna Cécile Rémuzat Pascal Auquier Mondher Toumi |
| author_facet | Eve Hanna Cécile Rémuzat Pascal Auquier Mondher Toumi |
| author_sort | Eve Hanna |
| collection | DOAJ |
| description | Background: In 1989, the concept of human gene therapies has emerged with the first approved human gene therapy trial of Rosenberg et al. Gene therapies are considered as promising therapies applicable to a broad range of diseases. Objective: The objective of this study was to review the descriptive data on gene therapy clinical trials conducted worldwide between 1989 and 2015, and to discuss potential success rates of these trials over time and anticipated market launch in the upcoming years. Methods: A publicly available database, ‘Gene Therapy Clinical Trials Worldwide’, was used to extract descriptive data on gene therapy clinical trials: (1) number of trials per year between 1989 and 2015; (2) countries; (3) diseases targeted by gene therapies; (4) vectors used for gene delivery; (5) trials status; (6) phases of development. Results: Between 1989 and 2015, 2,335 gene therapy clinical trials have been completed, were ongoing or approved (but not started) worldwide. The number of clinical trials did not increase steadily over time; it reached its highest peak in 2015 (163 trials). Almost 95% of the trials were in early phases of development and 72% were ongoing. The United States undertook 67% of gene therapy clinical trials. The majority of gene therapies clinical trials identified targeted cancer diseases. Conclusion: The first gene therapy was approved in the European Union in 2012, after two decades of dashed expectations. This approval boosted the investment in developing gene therapies. Regulators are creating a specific path for rapid access of those new therapies, providing hope for manufacturers, healthcare professionals, and patients. However, payers are increasingly scrutinizing the additional benefits of the new therapies. Major steps forward are expected in the field of gene therapies in the future. |
| first_indexed | 2024-04-24T14:41:24Z |
| format | Article |
| id | doaj.art-26d4515dbf384281a1a0c2dc1a230c65 |
| institution | Directory Open Access Journal |
| issn | 2001-6689 |
| language | English |
| last_indexed | 2024-04-24T14:41:24Z |
| publishDate | 2017-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Market Access & Health Policy |
| spelling | doaj.art-26d4515dbf384281a1a0c2dc1a230c652024-04-02T21:23:15ZengMDPI AGJournal of Market Access & Health Policy2001-66892017-01-015110.1080/20016689.2017.12652931265293Gene therapies development: slow progress and promising prospectEve Hanna0Cécile Rémuzat1Pascal Auquier2Mondher Toumi3Aix Marseille UniversityCreativ-ceuticalAix Marseille UniversityAix Marseille UniversityBackground: In 1989, the concept of human gene therapies has emerged with the first approved human gene therapy trial of Rosenberg et al. Gene therapies are considered as promising therapies applicable to a broad range of diseases. Objective: The objective of this study was to review the descriptive data on gene therapy clinical trials conducted worldwide between 1989 and 2015, and to discuss potential success rates of these trials over time and anticipated market launch in the upcoming years. Methods: A publicly available database, ‘Gene Therapy Clinical Trials Worldwide’, was used to extract descriptive data on gene therapy clinical trials: (1) number of trials per year between 1989 and 2015; (2) countries; (3) diseases targeted by gene therapies; (4) vectors used for gene delivery; (5) trials status; (6) phases of development. Results: Between 1989 and 2015, 2,335 gene therapy clinical trials have been completed, were ongoing or approved (but not started) worldwide. The number of clinical trials did not increase steadily over time; it reached its highest peak in 2015 (163 trials). Almost 95% of the trials were in early phases of development and 72% were ongoing. The United States undertook 67% of gene therapy clinical trials. The majority of gene therapies clinical trials identified targeted cancer diseases. Conclusion: The first gene therapy was approved in the European Union in 2012, after two decades of dashed expectations. This approval boosted the investment in developing gene therapies. Regulators are creating a specific path for rapid access of those new therapies, providing hope for manufacturers, healthcare professionals, and patients. However, payers are increasingly scrutinizing the additional benefits of the new therapies. Major steps forward are expected in the field of gene therapies in the future.http://dx.doi.org/10.1080/20016689.2017.1265293Gene therapyclinical trialsmarket access |
| spellingShingle | Eve Hanna Cécile Rémuzat Pascal Auquier Mondher Toumi Gene therapies development: slow progress and promising prospect Journal of Market Access & Health Policy Gene therapy clinical trials market access |
| title | Gene therapies development: slow progress and promising prospect |
| title_full | Gene therapies development: slow progress and promising prospect |
| title_fullStr | Gene therapies development: slow progress and promising prospect |
| title_full_unstemmed | Gene therapies development: slow progress and promising prospect |
| title_short | Gene therapies development: slow progress and promising prospect |
| title_sort | gene therapies development slow progress and promising prospect |
| topic | Gene therapy clinical trials market access |
| url | http://dx.doi.org/10.1080/20016689.2017.1265293 |
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