The US Food and Drug Administration (FDA) has released a new final guidance document intended to assist sponsors in determining whether a research study involving human subjects may be conducted without first filing an investigational new drug (IND) application with US regulators. Background: The FMT Precedent In recent months, FDA has proven to be somewhat amicable to the idea of extending enforcement discretion-that is, expressing that it won't punish an entity-when h...

The US Food and Drug Administration (FDA) has announced the publication of a new guidance document intended to guide institutional review boards (IRBs), clinical investigators and sponsors of clinical trials on whether clinical sites are compliant with regulations and whether an investigational application must be submitted to FDA. Background Clinical research is at the heart of FDA's regulatory oversight capacity. Good review decisions rely on good clinical data-r...

The US Food and Drug Administration (FDA) has released a new draft guidance document intended to outline the considerations industry should take into account when designing early-phase clinical trials for cellular therapy (CT) and gene therapy (GT) products. Background The draft guidance, released on 2 July 2013 by FDA's Center for Biologics Evaluation and Research (CBER), is specifically targeted at investigational new drug applications (INDs), which are used by compan...

The US Food and Drug Administration (FDA) has found itself mired in - quite literally - a crappy situation. In February 2013, the agency announced that it would reach out to the public in an attempt to determine how it should go about regulating fecal microbiota for transplantation (FMT), a treatment that many doctors now say can be useful in eradicating C. Difficile . But after FDA proposed to regulate the products as biologics and require them to obtain investigational...

A new final guidance document released by the US Food and Drug Administration (FDA) seeks to clarify the scientific and regulatory factors that must be taken into consideration when developing two or more new investigational products for use as a combination therapy in patients. Background The guidance, Codevelopment of Two or More New Investigational Drugs for Use in Combination , is (as its name indicates) intended for drugs used in combination, but FDA is quick ...

Could anyone imagine a medical condition that would warrant administering a stool specimen from a donor? Such treatment would seemingly belie the "Above all, do no harm" axiom thought to be part of the Hippocratic oath. 1 It just so happens that such treatment first occurred more than 50 years ago, and continues today. In 1958, doctors in Denver, Colorado, administered donor feces by enema to patients with fulminant, life-threatening pseudomembranous enterocolitis, an ac...

What is an investigation drug worth? In a philosophical sense, an investigational drug may be priceless to a patient facing the prospect of certain death, offering them additional time, reduced suffering or a new chance at life. But for companies in the process of studying an investigational product, that drug has a very real cost. Now a new draft guidance published by the US Food and Drug Administration (FDA) wants to answer a basic question: Can a company charge a patie...

A new draft guidance document released by the US Food and Drug Administration (FDA) aims to clarify details regarding so-called "expanded access" to pharmaceutical therapies that are currently being evaluated under an approved investigational new drug application (IND). Background Under current FDA regulations, sponsors must obtain approval from FDA for their IND before beginning a clinical trial. That IND is essentially a license to be exempt from the Federal Food, Dr...

A new procedural document issued by the US Food and Drug Administration (FDA) instructs its staff on the best practices-known as Good Review Practices (GVPs)-used in the review of investigational new drug (IND) applications. INDs are used by pharmaceutical and biotechnology companies at the relative start of the new drug application (NDA) and biologics licensing application (BLA) process, and once approved allow sponsors to begin the clinical testing phase of product dev...

The US Food and Drug Administration (FDA) is soliciting certain members of the biopharmaceutical industry to participate in a new pilot program that will help it determine whether its eSubmitter Program (eSubmitter) helps in the development and submission of investigational new drug (IND) applications. Background FDA has been transitioning many programs to digital formats, such as the electronic common technical document (eCTD), in the hope that it will allow agency rev...

A long-awaited draft guidance requiring the submission of most pharmaceutical and biological product applications to be done using the electronic common technical document (eCTD) standard has been released by the US Food and Drug Administration (FDA). The 3 January 2012 release of the document, Draft Revision of Guidance for Industry on Providing Regulatory Submissions in Electronic Format-Certain Human Pharmaceutical Product Applications and Related Submissions Using ...

The US Food and Drug Administration (FDA) has release a new draft document aimed at providing guidance on the preclinical assessment of cellular, tissue and gene (CTG) therapy products. CTGs are essentially used to make therapeutic changes to a person's cells, tissue or genes by injecting a patient with altered DNA, cells or implanted with organs. The products include cellular therapies, gene therapies, therapeutic vaccines, xenotransplantation and other drug products, i...