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Introduction
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow
With the most recent reauthorization, known as PDUFA VI, Congress and FDA created two new fee types: an application fee and an annual prescription drug program fee, which replaced the previously authorized supplemental application fee and annual establishment fee. The new fee types are assessed based on each “prescription drug product,” which is defined as an approved drug with specific strength or potency in its final dosage form that is dispensed only with a valid prescription. Because a single NDA or BLA can cover dozens of prescription drug products, PDUFA VI also limits prescription drug program fees to five fees (i.e., five prescription drug products) per NDA or BLA.
FDA Regulatory Acceptance of Bayesian Statistics
Published in Emmanuel Lesaffre, Gianluca Baio, Bruno Boulanger, Bayesian Methods in Pharmaceutical Research, 2020
More recently, the 21st Century Cures Act was passed by the U.S Congress and signed into law on December 13, 2016. While an earlier draft of the bill had mentioned Bayesian statistics explicitly, the final version in Section 3021 mentions “use of novel clinical trial designs for drugs and biological products” and in particular addresses “the use of complex adaptive and other novel trial designs” and “how sponsors may obtain feedback from the Secretary on technical issues related to modeling and simulations” (U.S. Congress, 2016). It also specified a public meeting and a draft guidance document on this topic. Regarding pharmaceutical drug user fees, in a commitment for the Pharmaceutical Drug Users Fee Act (PDUFA) VI “FDA agrees to develop staff capacity to facilitate appropriate use of complex adaptive, Bayesian and other novel clinical trial designs”. FDA also agreed to conduct a pilot program for “highly adaptive trial designs for which analytically derived properties (e.g. Type 1 error rate) may not be feasible, and simulations are necessary to determine trial operating characteristics”, to convene a public workshop to discuss complex adaptive trial designs and publish draft guidance on complex adaptive trial designs (Federation of American Scientists, 2017).
Pharmaceutical and Medical Device Product Liability Litigation
Published in Julie Dickinson, Anne Meyer, Karen J. Huff, Deborah A. Wipf, Elizabeth K. Zorn, Kathy G. Ferrell, Lisa Mancuso, Marjorie Berg Pugatch, Joanne Walker, Karen Wilkinson, Legal Nurse Consulting Principles and Practices, 2019
Vicki W. Garnett, Stacy Newsome
The Food and Drug Administration Modernization Act of 1997 (FDAMA) amended the FD&C Act to reform the regulation of food, drugs, and cosmetics. Important components of this act included the reauthorization of the Prescription Drug User Fee Act of 1992 (PDUFA), which sought to reduce the average time required for a drug review from 30 to 15 months. The FDA’s Center for Drug Evaluation and Research (CDER) was created to evaluate new drugs prior to sales and marketing by the pharmaceutical company. In 2005, an independent Drug Safety Oversight Board (DSOB) was created by the FDA to oversee management and communication of drug safety issues to physicians and patients (USFDA, n.d.-n).
Ophthalmic Drug Discovery in the United States over the past Two Decades
Published in Ophthalmic Epidemiology, 2021
This decrease in new molecular entities and biologics, in concordance with overall industry trends, demonstrates the significant challenges facing pharmaceutical discovery and approval. In 1992, the Prescription Drug User Fee Act (PDUFA) was passed which aimed to speed approval times in exchange for fees from drug manufacturers.10 An analysis of drug application approval times generally showed that although times are decreasing (with a low of 1.2 years from 2005 to 2009), the clinical development time, defined as the time from first filing a new investigational drug application to the submission of an NDA to the Food and Drug Administration, has been increasing since the 1990s.5 As a result, costs of clinical trials and drug development, in general, have been increasing, with some larger companies spending over 5 USD billion per drug approval.11 This industry maintains the highest percentage of research spending to revenues collected. Additionally, given the amount of time from preclinical phases to market (generally greater than 12 years), patent life during marketing of the drug is relatively short.
New and incremental FDA black box warnings from 2008 to 2015
Published in Expert Opinion on Drug Safety, 2018
Michael T. Solotke, Sanket S. Dhruva, Nicholas S. Downing, Nilay D. Shah, Joseph S. Ross
BBWs may be applied at the time of a drug’s regulatory approval or in the post-marketing period if safety issues emerge. Of novel therapeutics approved between 1996 and 2012, almost three quarters of BBWs were issued at the time of FDA approval, but over 40% of drugs receiving BBWs acquired the warnings in the post-marketing period [3]. Additionally, a recent study found that FDA took significant post-marketing safety actions for approximately one-third of novel therapeutics approved between 2001 and 2010, most commonly issuing a new BBW [4]. Further, the likelihood of drugs approved since the passage of the Prescription Drug User Fee Act (PDUFA) in 1992 receiving post-marketing BBWs or being withdrawn has increased compared to drugs approved before this period [5].
Omaveloxolone: an activator of Nrf2 for the treatment of Friedreich ataxia
Published in Expert Opinion on Investigational Drugs, 2023
Victoria Profeta, Kellie McIntyre, McKenzie Wells, Courtney Park, David R Lynch
Reata Pharmaceuticals initiated rolling submission on 31 March 2022, containing efficacy and safety data from the MOXIe Part one, two, and extension studies. After this announcement, the FDA approved the drug for a fast-track designation and orphan drug designation. If omav is approved, it will be the first approved treatment indicated for patients suffering with FRDA, a disease affecting approximately 5,000 people in the United States. On 26 May 2022, it was granted priority review of the application, with a current Prescription Drug User Fee Act (PDUFA) date of 28 February 2023. The most recent update states that the FDA will not be holding an advisory committee meeting regarding the NDA of omaveloxolone [100].