Expedited regulatory programs recognize the need for products targeting serious illnesses with unmet clinical need to proceed to market as soon as possible, without necessarily providing the amount of evidence required for regular market authorization. During the COVID-19 pandemic, there has been a race to develop treatments and vaccines, and the length of time required for regulatory considerations has been widely discussed. We seek to determine whether emerging regulator...

Members of the US Food and Drug Administration’s (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC) disagreed as to whether the agency should grant emergency use authorizations (EUAs) for COVID-19 vaccines for children at a 10 June meeting.   Some said that EUAs were necessary to get the pediatric population vaccinated quickly to achieve herd immunity as the country emerges from the pandemic and that these vaccines should be available soon as th...

Crushing workloads and statistical conundrums characterized the work of FDA’s staff as they raced to formulate guidelines and then evaluate vaccine candidates amid the pressure of the pandemic, attendees at a biostatistics conference heard on Wednesday.   More complex analysis awaits as real-world vaccine data roll in and FDA’s Center for Biologics Evaluation and Research (CBER) and sponsors both work toward full biologic license applications (BLAs) for the vaccines th...

The US Food and Drug Administration (FDA) recently released tables that can be included in submissions for new drug and biologic applications to provide information regarding the bioanalytical methods for pharmacokinetic assessments. “The templates in this guidance are applicable to bioanalytical procedures such as chromatographic assays (CCs) and ligand-binding assays (LBAs) that quantitatively determine the levels of drugs and their metabolites and therapeutic protein...

Beginning Monday, prospective applicants can submit pre-assigned number requests for four additional application types: new drug application (NDA), investigational new drug (IND) application, biologic license application (BLA) and drug master file (DMF), via the Center for Drug Evaluation and Research (CDER)’s NextGen Portal.   The pre-assigned number requests were previously made via email. But now the firms submitting such applications can use the portal, which is a ...

When a new drug application (NDA) or biologics license application (BLA) is deemed incomplete by the US Food and Drug Administration (FDA), the agency can decide not to review the application. So when can this occur? On Tuesday, the agency released draft guidance on what incompleteness means and when it can lead to a "refusal to file" decision. Explained simply: Refuse-to-file actions allow FDA to inform a sponsor as quickly as possible of deficiencies in an NDA or ...

As the year comes to a close, Patrick Frey, senior advisor to the director of the Office of New Drugs (OND) at the Center for Drug Evaluation and Research (CDER), looked back at FDA's performance over the final year of the fifth iteration of the Prescription Drug User Fee Act (PDUFA V) and what's to come in PDUFA VI. Speaking at the FDA/CMS Summit in Washington, D.C. on Tuesday, Frey said that before PDUFA, and during the first four PDUFA programs, FDA was approving ab...

As the number of chemistry, manufacturing and controls (CMC) postapproval manufacturing supplements continues to increase, the US Food and Drug Administration (FDA) on Tuesday released draft guidance offering recommendations for holders of biologics license applications (BLAs) on the types of minor changes to be documented in an annual report. "Under FDA regulations, postapproval changes in the product, production process, quality controls, equipment, facilities, o...

The US Food and Drug Administration's (FDA) Center for Biologics Research and Evaluation (CBER) has once again met or exceeded its performance goals for reviewing and acting on product applications and supplements, according to a report released Thursday from CBER Director Peter Marks. The report is Marks' first since he rose to head CBER last January, replacing former CBER Director Karen Midthun after her 22-year stint at the agency. As in previous years, CBER met or e...

The US Food and Drug Administration’s (FDA) Office of Pharmaceutical Quality (OPQ) on Tuesday released a new document outlining how supplements can be grouped together and submitted concurrently for the same chemistry, manufacturing and controls (CMC) changes to multiple approved new drug applications (NDAs), abbreviated new drug applications (ANDAs) and biological license applications (BLAs) submitted by the same applicant. The agency says the goal of its new policy is ...

A new independent analysis of the US Food and Drug Administration's (FDA) workload around biosimilars over the first three years shows how biosimilar development has slowly progressed as companies continue to seek more meetings with FDA on specific issues and targeted advice. As of 30 September 2015, the report says there are 57 programs participating in the biosimilar product development program (up from 33 in FY 2013 and 48 in FY 2014), and a total of seven Biologics L...

More than seven years since unveiling the draft document, the US Food and Drug Administration (FDA) on Wednesday finalized its guidance on how an Integrated Summary of Effectiveness (ISE) should be prepared for new drug applications (NDAs) and biologics license applications (BLAs). In terms of the changes between the draft and final versions, FDA has revised the definition of integrated analyses, clarified the components that constitute integrated analyses, defined poole...