Post-FDA Approval “Real-World” Safety Profile Between Different Steerable Sheaths During Catheter Ablation: A Food and Drug Administration MAUDE database study

Heart Rhythm ◽  
2022 ◽  
Author(s):  
Jakrin Kewcharoen ◽  
Kuldeep Shah ◽  
Rahul Bhardwaj ◽  
Tahmeed Contractor ◽  
Mohit K. Turagam ◽  
...  
Keyword(s):  
Catheter Ablation ◽  
Drug Administration ◽  
Real World ◽  
Safety Profile ◽  
Food And Drug Administration ◽  
Database Study ◽  
Fda Approval

Biosimilar epoetin for cancer and chemotherapy-induced anaemia in the US

2021 ◽  
Vol 10 (3) ◽  
pp. 122-122
Author(s):  
Charles L Bennett
Keyword(s):  
Advisory Committee ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Pharmacokinetics And Pharmacodynamics ◽  
Safety And Efficacy ◽  
Biological Drug ◽  
Drug Products ◽  
Fda Approval ◽  
The Us ◽  
Approval Letter

Biosimilars are biological drug products that are highly similar to reference products in analytic features, pharmacokinetics and pharmacodynamics, immunogenicity, safety and efficacy. Biosimilar epoetin received US Food and Drug Administration (FDA) approval in 2018 [1]. The manufacturer received an FDA non-approval letter in 2017, despite receiving a favourable review by the FDA’s Oncologic Drugs Advisory Committee (ODAC) and an FDA non-approval letter in 2015 for an earlier formulation.

Efficacy, Safety, and Regulatory Approval of Food and Drug Administration–Designated Breakthrough and Nonbreakthrough Cancer Medicines

2018 ◽  
Vol 36 (18) ◽  
pp. 1805-1812 ◽  
Author(s):  
Thomas J. Hwang ◽  
Jessica M. Franklin ◽  
Christopher T. Chen ◽  
Julie C. Lauffenburger ◽  
Bishal Gyawali ◽  
...  
Keyword(s):  
Adverse Events ◽  
Solid Tumors ◽  
Drug Administration ◽  
Mechanism Of Action ◽  
Food And Drug Administration ◽  
Progression Free Survival ◽  
Cancer Drugs ◽  
Serious Adverse Events ◽  
Hazard Ratios ◽  
Fda Approval

Purpose The breakthrough therapy program was established in 2012 to expedite the development and review of new medicines. We evaluated the times to approval, efficacy, and safety of breakthrough-designated versus non–breakthrough-designated cancer drugs approved by the US Food and Drug Administration (FDA). Methods We studied all new cancer drugs approved by the FDA between January 2012 and December 2017. Regulatory and therapeutic characteristics (time to FDA approval, pivotal trial efficacy end point, novelty of mechanism of action) were compared between breakthrough-designated and non–breakthrough-designated cancer drugs. Random-effects meta-regression was used to assess the association between breakthrough therapy designation and hazard ratios for progression-free survival (PFS), response rates (RRs) for solid tumors, serious adverse events, and deaths not attributed to disease progression. Results Between 2012 and 2017, the FDA approved 58 new cancer drugs, 25 (43%) of which received breakthrough therapy designation. The median time to first FDA approval was 5.2 years for breakthrough-designated drugs versus 7.1 years for non–breakthrough-designated drugs (difference, 1.9 years; P = .01). There were no statistically significant differences between breakthrough-designated and non–breakthrough-designated drugs in median PFS gains (8.6 v 4.0 months; P = .11), hazard ratios for PFS (0.43 v 0.51; P = .28), or RRs for solid tumors (37% v 39%; P = .74). Breakthrough therapy–designated drugs were not more likely to act via a novel mechanism of action (36% v 39%; P = 1.00). Rates of deaths (6% v 4%; P = .99) and serious adverse events (38% v 36%; P = 0.93) were also similar in breakthrough-designated and non–breakthrough-designated drugs. Conclusion Breakthrough-designated cancer drugs were associated with faster times to approval, but there was no evidence that these drugs provide improvements in safety or novelty; nor was there a statistically significant efficacy advantage when compared with non–breakthrough-designated drugs.

Real-world evidence for U.S. Food and Drug Administration-approved oncology products, 2015 to 2020.

2020 ◽  
Vol 38 (29_suppl) ◽  
pp. 280-280
Author(s):  
Bhakti Arondekar ◽  
Mei Sheng Duh ◽  
Rachel Bhak ◽  
Maral DerSarkissian ◽  
Lynn Huynh ◽  
...  
Keyword(s):  
Drug Administration ◽  
Real World ◽  
Food And Drug Administration ◽  
A Priori ◽  
New Drugs ◽  
Pivotal Trial ◽  
Real World Evidence ◽  
Data Source ◽  
Source Study ◽  
Clinical Trial Results

280 Background: Since the 21st Century Cures Act, there has been growing interest in using real-world evidence (RWE) to support regulatory filings for new drugs and indications. The goal of this study was to provide a comprehensive source of RWE use cases of U.S. Food and Drug Administration (FDA) approvals for oncology products. Methods: A systematic review of FDA new drug applications (NDAs) and biologics license applications (BLAs) from 1/2015-2/2020 for approved oncology products was performed. Data on the RWE study were extracted (data source, study design, statistical methods, results), and corresponding FDA comments were synthesized to identify patterns of the FDA’s review. Results: We identified 102 NDAs and BLAs, 8 (8%) of which included RWE, all post-Cures Act (see Table). RWE supporting avelumab, axicabtagene ciloleucel, and avapritinib were received positively and used by the FDA in their approval decision. RWE results were used to provide contextualization to the pivotal trial, rather than statistical comparison. Common data sources included Flatiron (38%) and chart reviews (38%). FDA critiques included lack of a priori study protocol, incomparability with the pivotal trial population and endpoints, and uncontrolled confounding. Conclusions: There have been few examples of RWE in oncology submissions, and most served to complement clinical trial results. To meet FDA standards, RWE studies should be clearly designed and discussed with the FDA and include robust methods to minimize bias. [Table: see text]

Abuse-deterrent Opioid Formulations

2018 ◽  
Vol 128 (5) ◽  
pp. 1015-1026 ◽  
Author(s):  
Ronald S. Litman ◽  
Olivia H. Pagán ◽  
Theodore J. Cicero
Keyword(s):  
Intravenous Injection ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Opioid Abuse ◽  
Marketing Approval ◽  
Opioid Overdose ◽  
Insufficient Evidence ◽  
Fda Approval ◽  
The U.S

Abstract Abuse-deterrent opioid formulations have been suggested as one way to decrease the abuse, addiction, and overdose of orally prescribed opioids. Ten oral opioid formulations have received abuse-deterrent labeling by the U.S. Food and Drug Administration (FDA). Their properties consist of physical and/or chemical means by which the pills resist manipulation and create a barrier to unintended administration, such as chewing, nasal snorting, smoking, and intravenous injection. In this review, we describe the mechanisms of abuse-deterrent technology, the types of premarketing studies required for FDA approval, the pharmacology of the currently approved abuse-deterrent opioid formulations, and the evidence for and against their influence on opioid abuse. We conclude that there is currently insufficient evidence to indicate that the availability of abuse-deterrent opioid formulations has altered the trajectory of opioid overdose and addiction; however, postmarketing studies are in their infancy, and novel deterrent formulations are continually being developed and submitted for marketing approval.

Unapproved Uses of Approved Drugs: The Physician, the Package Insert, and the Food and Drug Administration: Subject Review

PEDIATRICS ◽  
1996 ◽  
Vol 98 (1) ◽  
pp. 143-145 ◽  
Author(s):  
Keyword(s):  
Drug Administration ◽  
Food And Drug Administration ◽  
Age Groups ◽  
Package Insert ◽  
Age Group ◽  
Off Label Use ◽  
Off Label ◽  
Fda Approval ◽  
Approved Drugs

Physicians who prescribe a new drug that has not been approved for a specific indication or a specific age group frequently find themselves in a quandary. Physicians who prescribe "old," time-honored drugs usually do not consult the package insert or search for US Food and Drug Administration (FDA) approval. This statement was written to clarify the legal and informational status of the package insert and the role of the FDA in approving or not approving drugs for specific indications or specific age groups. The unapproved use of approved drugs, or so-called "off-label" use, is extremely prevalent among physicians who care for children. It is important that such use of compounds be brought up to date with current FDA policies and to emphasize the responsibility of the prescribing physician in the use of these compounds.

Impact of the Approval of Eculizumab by the Food and Drug Administration on the Frequency of Patients Screened for Paroxysmal Nocturnal Hemoglobinuria

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4377-4377
Author(s):  
Javier Munoz ◽  
Mohamad A. Younes ◽  
Karen Olarte-Merida ◽  
Philip Kuriakose
Keyword(s):  
Drug Administration ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Conflicts Of Interest ◽  
Food And Drug Administration ◽  
Thrombotic Event ◽  
Fda Approval ◽  
Number Of Patients ◽  
Study Population ◽  
Terminal Complement

Abstract Abstract 4377 Background: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired complement-mediated hemolytic disorder. Eculizumab, a monoclonal antibody designed to inhibit terminal complement activation, reduces intravascular hemolysis while leading to improvements in anemia, fatigue, and quality of life as well as reductions in blood transfusions and thrombosis. This medication was approved by the Food and Drug Administration (FDA) in March 16, 2007. The intent of our study was to assess if the number of patients screened for PNH has increased after the approval of eculizumab by the FDA. Methods: Retrospective single institution chart review over a period of 6 years from January 2004 to June 2010. Our study population comprised 174 patients screened for PNH by flow cytometry. Results: Between January 2004 and March 16, 2007 (39 months), a total of 68 cases were screened for PNH. On the other hand, between March 17, 2007 (date of FDA approval) and June 2010 (39 months), a total of 106 cases were screened for PNH with an increase in the frequency of testing by 55.8% (Figure 1). 1 out of 68 cases (1.5%) screened before March 16, 2007 were positive for PNH. 6 out of 106 cases (5.6%) screened after March 16, 2007 were positive for PNH (Table 1). 74 patients were male (42.5%) and 100 were female (57.5%). Average age at screening for PNH was 53.3 years. Screening was most commonly requested by Hematology (151 cases; 86.7%) followed by Internal medicine (5 cases), Hepatology (3 cases), Neurology (1 case) and Pulmonary (1 case). We found no information available in 13 cases regarding the location of the patient or the service that requested PNH screening. The clinical indications to screen for PNH were cytopenias (110 cases; 63.2%), thrombotic events (39 cases; 22.4%), or a combination of cytopenia plus a thrombotic event (9 cases; 5.2%). No indication for PNH screening was documented in 16 cases. Conclusion: Our hypothesis is that an approval of a new medication can increase both, the awareness of a disease among physicians, and the frequency of ordering its diagnostic tests. The number of patients screened for PNH sharply increased after the approval of eculizumab by the FDA, confirming our hypothesis. Disclosures: No relevant conflicts of interest to declare.

Brodalumab for Treatment-Resistant Psoriasis: Case Reports and Safety Update

2020 ◽  
Vol 5 (3) ◽  
pp. 82-85
Author(s):  
Nicholas D. Brownstone ◽  
Vidhatha Reddy ◽  
Quinn Thibodeaux ◽  
Stephanie Chan ◽  
Bridget Myers ◽  
...  
Keyword(s):  
Causal Relationship ◽  
Drug Administration ◽  
Safety Profile ◽  
Case Reports ◽  
Food And Drug Administration ◽  
Black Box ◽  
Interleukin 17 ◽  
Treatment Resistant ◽  
Effective Agent ◽  
Increased Risk

Introduction: Brodalumab is an interleukin-17 receptor blocker that is effective for the treatment of psoriasis. However, due to a Food and Drug Administration black box warning on depression and suicide, many providers are hesitant to use this agent despite its efficacy. Methods: We present the cases of 2 brothers seen at our clinic with treatment-resistant psoriasis who were both successfully treated with brodalumab, despite failing 6 other biologics. We also review the safety profile of brodalumab regarding the currently available evidence on the increased risk of suicidality or depression in patients treated with brodalumab. Results: Both patients achieved completely clear skin and maintained clearance on brodalumab. Discussion: Brodalumab appears to be an effective agent in severe treatment-resistant cases of psoriasis. In addition, a causal relationship between increased risk of suicidality or depression and brodalumab use has not been established.

Transcatheter Aortic Valve Replacement Program in the Post-Food and Drug Administration Approval Era: Early Outcomes at an Academic Medical Center

2015 ◽  
Vol 10 (1) ◽  
pp. 48-51 ◽  
Author(s):  
Spencer J. Melby ◽  
Kyle M. Bess ◽  
Kayla D. Isbell ◽  
Mark F. Sasse ◽  
Massoud A. Leesar ◽  
...  
Keyword(s):  
High Risk ◽  
Aortic Valve ◽  
Aortic Valve Replacement ◽  
Valve Replacement ◽  
Drug Administration ◽  
Real World ◽  
Transcatheter Aortic Valve Replacement ◽  
Food And Drug Administration ◽  
Transcatheter Aortic Valve ◽  
Very High

Objective Transcatheter aortic valve replacement (TAVR) has recently become a suitable alternative for treatment of symptomatic aortic stenosis in patients who are at very high risk for morbidity and mortality with conventional corrective surgery. In the fall of 2011, the Food and Drug Administration approved the use of TAVR, allowing for reimbursement at institutions outside of investigative trials. We report the initiation of a TAVR-based program at an academic tertiary care facility that did not participate in the Placement of Aortic Transcatheter Valves (PARTNER) 1 or PARTNER II trials. Methods A total of 160 patients were evaluated for TAVR from May 2012 through June 2013. Transcatheter aortic valve replacement was found to be appropriate for 50 (31%). Results In this experience, morbidity and mortality were similar to those reported for the PARTNER trial (30-day hospital mortality was 8%). A single case of presumed cerebrovascular accident was observed. These results demonstrate that the real-world application can be done with comparable results. Implementation of lessons learned in the trials allowed a very short learning curve and excellent results after only a limited number of patients. Use of computed tomography reconstructed images for operative planning, including fluoroscopic angle, facilitated minimal use of contrast in each case. Conclusions Transcatheter aortic valve replacement is a viable option for patients at very high risk for surgical intervention in the post-Food and Drug Administration approval era. Real-world results comparable with published outcomes from experienced centers involved in the PARTNER trial can be achieved.

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