scholarly journals Postmarketing safety of orphan drugs: a longitudinal analysis of the US Food and Drug Administration database between 1999 and 2018

2022 ◽  
Vol 17 (1) ◽  
Author(s):  
Min Fan ◽  
Adrienne Y. L. Chan ◽  
Vincent K. C. Yan ◽  
Xinning Tong ◽  
Lauren K. W. Lau ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Drug Administration ◽  
Drug Evaluation ◽  
Food And Drug Administration ◽  
Orphan Drugs ◽  
Orphan Drug Designation ◽  
Regulatory Body ◽  
Ensure Patient Safety ◽  
The Us

Abstract Background Information about the specific regulatory environment of orphan drugs is scarce and inconsistent. Uncertainties surrounding the postmarketing long-term safety of orphan drugs remain. This study aimed to evaluate the labelling changes of orphan drugs and to identify postmarketing safety-associated approval factors. Methods This retrospective cohort study includes all drugs with orphan drug designation approved by the Center for Drug Evaluation and Research of the US Food and Drug Administration between 1999 and 2018. Main outcomes are safety-related labelling changes up to 31 December 2019. We defined any safety-related labelling changes as postmarketing safety events (PMSE). Safety-related withdrawals, suspensions, and boxed warnings were further categorised as severe postmarketing safety events (SPSE). Outcome measurements include frequencies of PMSE, SPSE, and association between approval factors and the occurrence of safety events. Results Amongst the 214 drugs identified with orphan drug designation (25.7% biologics), 83.6% were approved through at least one expedited programme, and 29.4% were approved with boxed warnings. During a median follow-up of 6.74 years since approval, 69.2% and 14.5% of the analysed orphan drugs had PMSE and SPSE, respectively. Safety-related withdrawal (0%, 0/214), suspended marketing (0.46%, 1/214) and new boxed warnings are uncommon (3.7%, 8/214). The safety-related labelling changes were more frequent in the drugs approved with boxed warnings [Incidence rate ratio (IRR): 1.95 (1.02–3.73)] and approved for long-term use [IRR: 2.76 (1.52–5.00)]. Conclusions and Relevance In this long-term postmarketing analysis, approximately 70% of FDA-approved orphan drugs had safety-related labelling changes although severe safety events were rare. While maintaining early access to orphan drugs, the drug regulatory body has taken timely regulatory action with postmarketing surveillance to ensure patient safety.

scholarly journals Low rates of patient-reported outcome claims for orphan drugs approved by the us food and drug administration

2018 ◽  
Vol 6 (1) ◽  
pp. 1433426
Author(s):  
Szymon Jarosławski ◽  
Pascal Auquier ◽  
Borislav Borissov ◽  
Claude Dussart ◽  
Mondher Toumi
Keyword(s):  
Drug Administration ◽  
Food And Drug Administration ◽  
Orphan Drugs ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
The Us

Ultimate Fate of Oncology Drugs Approved by the US Food and Drug Administration Without a Randomized Trial

2009 ◽  
Vol 27 (36) ◽  
pp. 6243-6250 ◽  
Author(s):  
Apostolia-Maria Tsimberidou ◽  
Fadi Braiteh ◽  
David J. Stewart ◽  
Razelle Kurzrock
Keyword(s):  
Clinical Trials ◽  
Randomized Trial ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Objective Response ◽  
End Points ◽  
Median Response ◽  
Safety And Efficacy ◽  
The Us

Purpose To approve a new anticancer drug, the US Food and Drug Administration often requires randomized trials. However, several oncology drugs have been approved on the basis of objective end points without a randomized trial. We reviewed the long-term safety and efficacy of such agents. Methods We searched the Web site of the US Food and Drug Administration's Center for Drug Evaluation and Research and MEDLINE for initial applications of investigational anticancer drugs from 1973 through 2006. Results Overall, 68 oncology drugs, excluding hormone therapy and supportive care, were approved, including 31 without a randomized trial. For these 31 drugs, a median of two clinical trials (range, one to seven) and 79 patients (range, 40 to 413) were used per approval. Objective response was the most common end point used for approval; median response rate was 33% (range, 11% to 90%). Thirty drugs are still fully approved. United States marketing authorization for one drug, gefitinib (an epidermal growth factor receptor [EGFR] inhibitor), was rescinded after a randomized trial showed no survival improvement; however, this trial was performed in unselected patients, and it was subsequently demonstrated that patients with EGFR mutation are more likely to respond. Nineteen of the 31 drugs have additional uses (per National Comprehensive Cancer Network or National Cancer Institute Physician Data Query guidelines), and subsequent formal US Food and Drug Administration approvals were obtained for 11 of these (range, one to 18 new indications). No drug has demonstrated safety concerns. Conclusion Nonrandomized clinical trials with definitive end points can yield US Food and Drug Administration approvals, and these drugs have a reassuring record of long-term safety and efficacy.

scholarly journals 2020 FDA TIDES (Peptides and Oligonucleotides) Harvest

2021 ◽  
Vol 14 (2) ◽  
pp. 145
Author(s):  
Othman Al Musaimi ◽  
Danah Al Shaer ◽  
Fernando Albericio ◽  
Beatriz de la Torre
Keyword(s):  
Adverse Effects ◽  
Global Health ◽  
Drug Administration ◽  
Mode Of Action ◽  
Chemical Structure ◽  
Food And Drug Administration ◽  
Health Crisis ◽  
New Drug ◽  
Target Mode ◽  
The Us

2020 has been an extremely difficult and challenging year as a result of the coronavirus disease 2019 (COVID-19) pandemic and one in which most efforts have been channeled into tackling the global health crisis. The US Food and Drug Administration (FDA) has approved 53 new drug entities, six of which fall in the peptides and oligonucleotides (TIDES) category. The number of authorizations for these kinds of drugs has been similar to that of previous years, thereby reflecting the consolidation of the TIDES market. Here, the TIDES approved in 2020 are analyzed in terms of chemical structure, medical target, mode of action, and adverse effects.

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