A 25-Year Experience of US Food and Drug Administration Accelerated Approval of Malignant Hematology and Oncology Drugs and Biologics
| Autor: | Ann T. Farrell, Kim Ts, Jinzhong Liu, Julia A. Beaver, Paul G. Kluetz, Rajeshwari Sridhara, Patricia Keegan, Kirsten B. Goldberg, Lorraine Pelosof, Lynn J. Howie, Richard Pazdur, Gideon M. Blumenthal |
|---|---|
| Rok vydání: | 2018 |
| Předmět: |
Cancer Research
medicine.medical_specialty Databases Factual Endpoint Determination MEDLINE Antineoplastic Agents History 21st Century Confirmatory trial Food and drug administration 03 medical and health sciences 0302 clinical medicine Neoplasms Internal medicine Product Surveillance Postmarketing medicine Humans 030212 general & internal medicine Drug Approval Response rate (survey) Biological Products Clinical Trials as Topic Hematology United States Food and Drug Administration business.industry Surrogate endpoint Drugs Investigational History 20th Century Hematologic Diseases United States Hematologic Response Treatment Outcome Oncology 030220 oncology & carcinogenesis Accelerated approval business Biomarkers |
| Zdroj: | JAMA Oncology. 4:849 |
| ISSN: | 2374-2437 |
| Popis: | Importance Accelerated approval (AA) is a US Food and Drug Administration (FDA) expedited program intended to speed the approval of drugs and biologics that may demonstrate a meaningful advantage over available therapies for diseases that are serious or life-threatening. Observations This review describes all malignant hematology and oncology AAs from inception of the program on December 11, 1992, to May 31, 2017. During this period, the FDA granted AA to 64 malignant hematology and oncology products for 93 new indications. Of these AAs, 53 were for new molecular entities. Overall, the end point of response rate, including hematologic response rates, accounted for most AAs (81 [87%]), followed by time-to-event end points of progression-free survival or time to progression (8 [9%]) and disease-free survival or recurrence-free survival (4 [4%]). Single-arm trial designs provided the data for 67 (72%) of the initial AA indications. Of the 93 AAs, 51 (55%) have fulfilled their postmarketing requirement and verified benefit in a median of 3.4 years after their initial AA. Thirty-seven (40%) indications have not yet completed confirmatory trial(s) or verified benefit, and 5 indications receiving AA (5%) have been withdrawn from the market. Conclusions and Relevance The use of the AA program during the past 25 years has increased over time, and only a small portion of indications under the AA program fail to verify clinical benefit. For patients with serious or life-threatening oncologic diseases, AA brings products to the market years before confirmatory trials are typically completed. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|