Problems Confronting the Food and Drug Administration in Drug Evaluation

1966 ◽  
Vol 13 (3) ◽  
pp. 307-311
Author(s):  
Joseph F. Sadusk
Keyword(s):  
Drug Administration ◽  
Drug Evaluation ◽  
Food And Drug Administration

The U.S. Food and Drug Administration’s Complex Innovative Trial Design Pilot Meeting Program: Progress to date

2021 ◽  
pp. 174077452110505
Author(s):  
Dionne Price ◽  
John Scott
Keyword(s):  
Public Health ◽  
Drug Administration ◽  
Trial Design ◽  
Drug Evaluation ◽  
Food And Drug Administration ◽  
Medical Product ◽  
Global Public Health ◽  
Multiple Sources ◽  
Meeting Program ◽  
The U.S

Background The Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research of the U.S. Food and Drug Administration have been leaders in advancing science to protect and promote public health by ensuring that safe and effective drugs and biological products are available to those who need them. Recently, new therapeutic discoveries, increased understanding of disease mechanisms, the need for innovation to optimally use resources, and global public health crises have led to an evolving drug development landscape. As a result, the U.S. Food and Drug Administration and medical product developers are faced with unique challenges and opportunities. The U.S. Food and Drug Administration is proactively meeting the challenges of this evolving landscape through various efforts, including the Complex Innovative Trial Design Pilot Meeting Program. Our focus, here, will be on the pilot meeting program. Methods The U.S. Food and Drug Administration has defined a process to facilitate the implementation of the Complex Innovative Trial Design Pilot Meeting Program. The process is transparent and outlines the steps and timeline for submission, review, and meetings. Results Five submitted meeting requests have been selected for participation in the Complex Innovative Trial Design Pilot Meeting Program. Conclusion The pilot meeting program has been successful in further educating stakeholders on the potential uses of complex innovative designs in trials intended to provide substantial evidence of effectiveness. The selected submissions, thus far, have all utilized a Bayesian framework. The reasons for the use of Bayesian approaches may be due to the flexibility provided, the ability to incorporate multiple sources of evidence, and a desire to better understand the U.S. Food and Drug Administration perspective on such approaches. We are confident the pilot meeting program will have continued success and impact the collective goal of bringing safe and effective medical products to patients.

scholarly journals A Proposed Framework for Patient-Focused Policy at the U.S. Food and Drug Administration

Biomedicines ◽  
2019 ◽  
Vol 7 (3) ◽  
pp. 64 ◽  
Author(s):  
Carrie M. Kuehn
Keyword(s):  
Development Strategy ◽  
Drug Administration ◽  
Drug Evaluation ◽  
Food And Drug Administration ◽  
Medical Product ◽  
Specific Patient ◽  
Regulatory Decision ◽  
Regulatory Submissions ◽  
Guidance Documents ◽  
Product Strategies

Medical product sponsors are encouraged to include the patient perspective in their medical product development strategy to inform product design, augment regulatory submissions, argue for alternative clinical trial designs, or to support indications in specific patient populations. The goal is to create a patient-focused ecosystem that enables industry to integrate the patient voice throughout the medical product lifecycle. To this end, the U. S. Food and Drug Administration (FDA) has published several guidance documents to provide industry with the expectations and opportunities for conducting patient-focused activities. From an industry perspective, the Center for Devices and Radiologic Health (CDRH) and the Center for Drug Evaluation and Research (CDER)/Center for Biologics Evaluation and Research (CBER) patient-focused policies are complementary. The basic tenets promoted in all FDA patient-focused guidance could apply across therapeutic areas. However, there remain differences in these guidance documents across FDA centers, and there is no framework in place to provide industry with consistent recommendations. Without a coordinated patient-focused policy from the FDA, there is the potential for confusion and a lack of consistency among industry and regulatory decision-makers. The objective of this paper was to propose an alternative framework for patient-focused policy at the FDA, which recognizes the potential for different types of patient input to be used across therapeutic areas and medical product types. Further, these policies need to provide greater clarity on how patient input data is used, so that sponsors may navigate the opportunities to use patient input regardless of the FDA center under which their product is regulated. Creating consistent, coherent, and transparent FDA patient-focused policy will encourage sponsors to obtain patient input more often and with greater certainty of the value that these data may have to their medical product strategies.

scholarly journals Toxicity of Excipients—A Food and Drug Administration Perspective

2003 ◽  
Vol 22 (5) ◽  
pp. 377-380 ◽  
Author(s):  
Robert E. Osterberg ◽  
Norman A. See
Keyword(s):  
Drug Administration ◽  
Drug Evaluation ◽  
Food And Drug Administration ◽  
Safety Data ◽  
The United States ◽  
Repeat Dose ◽  
Guidance Document ◽  
Drug Products ◽  
Osmotic Diarrhea ◽  
Essential Components

Excipients are essential components of drug products. They are also potential toxicants. Examples of known excipient-induced toxicities include renal failure and death from diethylene glycol, osmotic diarrhea caused byingested mannitol, hypersensitivity reactions from lanolin, and cardiotoxicity induced by propylene glycol. Proposals to test or market new drug products in the United States should adequately address the safety of the proposed exposure to the excipients in those products. The specific safety data that may be needed will vary depending upon the clinical situation, including such factors as the duration, level, and route of exposure, but may include acute, repeat-dose, reproductive, and genetic toxicity data, carcinogenicity data, and specialized toxicology information, such as sensitization or local irritation data. Many guidances exist to aid in the development of pharmaceuticals, including the International Conference on Harmonization (ICH) documents and various Food and Drug Administration/Center for Drug Evaluation and Research (FDA/CDER) pharmacology and toxicology guidances. The FDA/CDER has recently adopted a new guidance for industry, “Nonclinical Studies for Development of Pharmaceutical Excipients,” which focuses on issues associated with development of safety databases that will support clinical use of excipients in drug products. The new guidance document is introduced and discussed in this article.

2020: A Record Year for Drug Approvals

2021 ◽  
Vol 36 (4) ◽  
pp. 174-175
Author(s):  
Nancy L. Losben
Keyword(s):  
United States ◽  
Drug Administration ◽  
Generic Drugs ◽  
Drug Evaluation ◽  
Food And Drug Administration ◽  
The United States ◽  
New Drugs ◽  
Novel Drugs ◽  
United States Food ◽  
States Food

In 2020 The United States Food and Drug Administration?s (FDA) Center for Drug Evaluation and Research (CDER) approved 53 novel drugs, five more than in 2019, but still an aggressive number when compared with 2015 when only 45 new drugs were released to the market. CDER, the largest department within the FDA, has robustly approved a rising number of generic drugs in the last several years, increasing their accessibility and reducing patient and payor costs.

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.

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