After the US Food and Drug Administration (FDA) solicited comments on an educational program for a new Empower Rare Disease Drug Developers (LEADER 3D) initiative in the Accelerating Rare disease Cures (ARC) program, nearly two dozen stakeholders offered feedback on what topics they believe are important for inclusion in educational materials on rare disease drug development.   FDA’s Center for Drug Evaluation and Research (CDER) asked for comments from rare disease st...

While the US Food and Drug Administration’s (FDA) latest draft guidance on improving the consistency of information in the dosage and administration section of prescription drug labeling is significantly larger than an earlier guidance issued in 2010, industry stakeholders said they wanted more information from the agency on how labeling of specific cases should be handled.   FDA’s draft guidance on improving development of the dosage and administration section of drug...

In the period between when three accelerated approval (AA) drugs received approval from the US Food and Drug Administration (FDA) for oncology indications and were later withdrawn due to negative confirmatory trials, more than one quarter of patients with breast, bladder, hepatocellular, gastric, or small cell lung cancer received an AA drug rather than standard of care, according to a research letter recently published in JAMA Oncology .   “Given the growth of withdr...

More than one-fifth of drugs recently approved by the US Food and Drug Administration (FDA) were not approved or recommended for public reimbursement in other countries, according to a recent study published in JAMA Internal Medicine .   “This cross-sectional study demonstrated that more than one-fifth of the new drugs approved by the FDA in 2017 through 2020 were either refused marketing authorization or not recommended for reimbursement in Australia, Canada, or the ...

About 10% of drugs approved by the US Food and Drug Administration (FDA) between 2018 and 2021 had pivotal trials with null findings, according to a research letter published in JAMA Internal Medicine .   “Our findings underscore the complexity of regulatory decision-making, as exemplified by evidence of effectiveness despite a null primary end point finding,” James L. Johnston, MD, of the department of medicine at Brigham and Women’s Hospital in Boston, and colleague...

Commenters on the US Food and Drug Administration’s (FDA) draft guidance outlining principles for in vivo or in vitro bioequivalence studies (BE) for investigational new drugs (INDs), new drug applications (NDAs), abbreviated new drug applications (ANDAs) indicated that they want the agency to elaborate on new topic areas introduced in the guidance.   The draft guidance, which replaces a 2001 guidance for industry published under the same name, added new informatio...

Clinical research in the US needs an overhaul to better optimize health outcomes in a way that matches advances in biomedical science, according to a recent perspective authored by US Food and Drug Administration Commissioner Robert M. Califf, published in Clinical Trials journal.   “Biomedical science and technology are undergoing a remarkable period of discovery and development, one driven in large part by the innovations of US scientists and engineers. Unfortunate...

The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) released a report on the 37 new drug therapies the Center approved in 2022, highlighting which had received expedited development and review status, first cycle approval, and met Prescription Drug User Fee Program Act (PDUFA) VII goal dates for acting on applications.   “We approved many drugs in 2022 for patients with few or no treatment options,” CDER Director Patrizia Cavazzon...

After several years with high numbers of novel drug approvals, the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved 37 new drug therapies in 2022.   CDER’s 37 new drug approvals in 2022 represents the lowest number of novel drugs approved by the Center since 2016 . The Center approved 50 novel drugs in 2021 , 53 novel drugs in 2020 , 48 drugs in 2019 and 59 drugs in 2018 . (RELATED: FDA approved more first-in-class...

The US Food and Drug Administration (FDA) has released new draft guidance on developing drugs for pulmonary tuberculosis.   FDA’s latest draft guidance is intended to update the agency’s 2013 draft guidance with the same name, which was never finalized. “Since the 2013 final guidance was issued, there have been improvements in nonclinical models and further interest in streamlined clinical development programs as well as consideration for combination regimens with trea...

All new antibiotics approved by the US Food and Drug Administration (FDA) used surrogate outcome measures, and about half had pivotal trials with a non-inferiority design, according to recent research published in BMJ Medicine .   Mayookha Mitra-Majumdar, MPH, of the Program On Regulation, Therapeutics, And Law (PORTAL) in the Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine at Brigham and Women's Hospital/Harvard Medical School in Boston...

The US Food and Drug Administration (FDA) has released a new guidance with immediate effect outlining its enforcement policy for preparation of certain beta-lactam oral antibiotic suspension products.   The agency said it has been asked to clarify how compounded versions of products from FDA-approved tablets and capsules currently in shortage can be prepared, as FDA’s insanitary conditions guidance states beta-lactam drugs are required to be processed with “complete ...