Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency
Bibliographic Data
| ID | 9104551 |
|---|---|
| Authors | Buthainah Ghanem (0000-0003-1682-4867, Department of Pharmaceutical Economics and Policy, Chapman University School of Pharmacy, Irvine, corresponding author), Enrique Seoane-Vazquez (0000-0002-2763-5685, Department of Pharmaceutical Economics and Policy, Chapman University School of Pharmacy, Irvine, corresponding author), Lawrence Brown (0000-0003-0673-2272, Department of Pharmaceutical Economics and Policy, Chapman University School of Pharmacy, Irvine), Lawrence M Brown (0000-0003-4532-7640, Chapman University, corresponding author), Rosa Rodriguez-Monguio (0000-0002-5526-9395, Department of Clinical Pharmacy) |
| Year | 2023 |
| Volume | 61 |
| Issue | 7 |
| Pages | 438-447 |
| Publication date | 2023-07-01 |
| Peer Reviewed | Yes |
| Open Access | No |
| Type | ARTICLE |
| Venue | Medical Care (JOURNAL) |
| Journal identifiers | ISSN: 0025-7079 • E-ISSN: 1537-1948 |
| Publisher | Ovid Technologies (Wolters Kluwer Health) (PUBLISHER) |
| DOI | 10.1097/mlr.0000000000001840 |
| PMID | 36884030 |
| OpenAlex | W4323533346 |
| Language | EN |
| Citations received | 1 |
| References cited | 21 |
BACKGROUND: Gene therapy, altering the genes inside human cells, has recently emerged as an alternative for preventing and treating disease. Concerns have been expressed about the clinical value and the high cost of gene therapies. OBJECTIVE: This study assessed the characteristics of the clinical trials, authorizations, and prices of gene therapies in the United States and the European Union. RESEARCH DESIGN: We collected regulatory information from the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and manufacturer-listed prices from the United States, UK, and Germany. Descriptive statistics and t tests were conducted in the study. RESULTS: As of January 1, 2022, the FDA and EMA authorized 8 and 10 gene therapies, respectively. The FDA and EMA granted orphan designation to all gene therapies except talimogene laherparepvec. Pivotal clinical trials were nonrandomized, open level, uncontrolled, phase I-III, and included a limited number of patients. Study primary outcomes were mainly surrogate endpoints without demonstration of direct patient benefit. The price of gene therapies at market entry ranged from $200,064 to $2,125,000 million. CONCLUSIONS: Gene therapy is used to treat incurable diseases that affect only a small number of patients (orphan diseases). Based on this, they are approved by the EMA and FDA with insufficient clinical evidence to ensure safety and efficacy, in addition to the high cost
Agency (philosophy) · Authorization · Bioinformatics · Business · Clinical research · Clinical trial · Disease · Drug · European union · Family medicine · Food and drug administration · Intensive care medicine · International trade · Marketing authorization · Orphan drug · Biomedical Ethics and Regulation · CAR-T cell therapy research · Internal Medicine · Medicine · Pharmacology · Virus-based gene therapy research
| Unique citing works | 1 |
|---|---|
| Citations per year | 0,33 |
| Citation span | 2023 - 2023 (1) |
| Citation velocity | historical |
| Highly cited | No |
| Citation types | Neutral: 1 |