Regulatory histories of recently withdrawn ovarian cancer treatment indications of 3 PARP inhibitors in the US and Europe: lessons for the accelerated approval pathway [PDF]
Journal of Pharmaceutical Policy and PracticeBackground Withdrawals of drug indications may reveal potential inadequacies in the regulatory approval processes of new drugs. Understanding potential weaknesses of the regulatory approval process is paramount given the increasing use of expedited ...
Mahnum Shahzad +4 more
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The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases [PDF]
Orphanet Journal of Rare Diseases, 2011 Background Over 95% of rare diseases lack treatments despite many successful treatment studies in animal models. To improve access to treatments, the Accelerated Approval (AA) regulations were implemented allowing the use of surrogate endpoints to ...
Kakkis Emil D, Miyamoto Brigitta E
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JAMA Health Forum, 2022 This cohort study assesses the therapeutic value of drugs granted accelerated approval or conditional marketing authorization in the US and European Union from 2007 to 2021.
Vokinger KN +3 more
europepmc +2 more sources
Regulatory Disparities Between US Accelerated Approval and Japanese Authorization of Oncology Drugs: An Analysis of Evidence Quality [PDF]
Clinical and Translational ScienceWhile the accelerated approval (AA) program in the United States expedites the availability of drugs based on preliminary evidence to fulfill unmet medical needs, it has also raised significant concerns, including a lack of robust evidence of efficacy ...
Maho Iwata, Anju Murayama
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Clinical Benefit and Regulatory Outcomes of Cancer Drugs Receiving Accelerated Approval.
JAMAImportance The US Food and Drug Administration's (FDA) accelerated approval pathway allows approval of investigational drugs treating unmet medical needs based on changes to surrogate measures considered "reasonably likely" to predict clinical benefit ...
Liu ITT, Kesselheim AS, Cliff ERS.
europepmc +2 more sources
Regulatory Fate of Cancer Indications in the European Union After Accelerated Approval in the US.
JAMA OncolThis cohort study investigates the regulatory fate in the European Union for drugs that received accelerated approval for cancer indications in the US.
Petersen TS +4 more
europepmc +2 more sources
BMJ, 2021 Objectives To investigate the regulatory handling of cancer drugs that were granted accelerated approval by the US Food and Drug Administration (FDA) but failed to improve the primary endpoint in post-approval trials and to evaluate the extent to which ...
Gyawali B, Rome BN, Kesselheim AS.
europepmc +2 more sources
Reforming Reimbursement for the US Food and Drug Administration’s Accelerated Approval Program to Support State Medicaid Programs [PDF]
JAMA Health Forum, 2022 Importance The US Food and Drug Administration (FDA) has an accelerated approval program that has become the subject of scholarly attention and criticism, not only for the FDA's oversight of the program but also for its implications for payers ...
Rachel E. Sachs +2 more
openalex +2 more sources
JAMA Health Forum, 2021 This cross-sectional study identifies the number and class of drugs approved through the US Food and Drug Administration’s accelerated approval pathway and analyzes state Medicaid programs’ use and spending on these drugs from 2015 through 2019.
Sachs RE +3 more
europepmc +2 more sources
JAMA Intern Med, 2021 Key Points Question Are cancer drugs granted accelerated approval from the US Food and Drug Administration (FDA) recommended for funding through the National Health Service (NHS) in England?
Cherla A +4 more
europepmc +2 more sources