GENE THERAPIES DEVELOPMENT- SLOW PROGRESS AND PROMISING PROSPECT

Author(s)

Hanna E1, Rémuzat C2, Auquier P3, Toumi M1
1Aix-Marseille University, Marseille, France, 2Creativ-Ceutical, Paris, France, 3Faculté de Médecine, Laboratoire de Santé Publique, Aix-Marseille Université, Marseille, France

OBJECTIVES: 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. 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. CONCLUSIONS:  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. The potential budget impact may become the actual hurdle for gene therapies, leading to restricted access and lost opportunities for many patients.

Conference/Value in Health Info

2017-05, ISPOR 2017, Boston, MA, USA

Value in Health, Vol. 20, No. 5 (May 2017)

Code

PHP10

Topic

Health Service Delivery & Process of Care

Topic Subcategory

Treatment Patterns and Guidelines

Disease

Multiple Diseases

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