FDA Considers Relaxing Clinical Testing Requirements for Some Biotherapeutic Products

Posted 31 March 2015 | By Alexander Gaffney, RAC 

FDA Considers Relaxing Clinical Testing Requirements for Some Biotherapeutic Products

The US Food and Drug Administration (FDA) is considering making it easier for companies to test certain "live biotherapeutic products" like probiotics in early-stage trials, the agency has announced.


The guidance document, Early Clinical Trials with Live Biotherapeutic Products: Chemistry, Manufacturing, and Control Information, was first released in draft form October 2010 by FDA's Center for Biologics Evaluation and Research (CBER) and later finalized in February 2012.

As written, the guidance focuses on the early stages of the investigational new drug (IND) process, better known as the clinical trials process. Early-stage clinical trials focus primarily on safety, including whether a product has been manufactured to acceptable standards.

The analysis of a product's safety and manufacturing data has always been somewhat complex, but the process is especially difficult for some products, and especially for those which contain or are comprised of live organisms like bacteria.

In its guidance, FDA defines live biotherapeutic products (LBPs) as a biological product that:

  1. contains live organisms, such as bacteria
  2. is applicable to the prevention, treatment, or cure of a disease or condition of human beings
  3. is not a vaccine

Excluded from FDA's definition are "filterable viruses, oncolytic bacteria, or products intended as gene therapy agents and, as a general matter, are not administered by injection."

When submitting an IND to FDA, companies are advised to include information about the name and strain of the LBP, the origin of the LBP, whether the LBP has been subject to any modifications, the method of its manufacture and if the LBP has any previous experience in human subjects.

A New Call for Comments

While FDA's guidance document received relatively few comments when it was published—just one, according to FDA records—the agency is now preparing to revisit parts of the guidance, and has asked the biotechnology industry for input.

"FDA is considering whether to revise the guidance to address when the label on the commercially available product(s) would be considered adequate to satisfy the requirement for CMC information under § 312.23," FDA explained in a Federal Register announcement on 31 March 2015.

In plain terms, FDA wants to know if an LBP product is eligible for relaxed regulatory oversight if it is already marketed as a dietary supplement like a probiotic, it said.

"For example, we are considering whether the label would be adequate to satisfy the CMC information when the following conditions are met," FDA said:

  1. the LBP product that is proposed for investigational use is a commercially available food or dietary supplement;
  2. the investigation does not involve a route of administration, dose, patient population, or other factor that significantly increases the risk (or decreases the acceptability of risk) associated with the use of the food or dietary supplement;
  3. the investigation is not intended to support a marketing application for a drug claim for the food or dietary supplement; and
  4. the investigation is conducted in compliance with the requirements for INDs (part 312), the requirements for review by an institutional review board (21 CFR part 56), and with the requirements for informed consent (21 CFR part 50). FDA is seeking public comment on this issue.

FDA will accept comments on the guidance until 29 May 2015.


Early Clinical Trials with Live Biotherapeutic Products: Chemistry, Manufacturing, and Control Information

Federal Register

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