REGULATORY

As a therapy at the forefront of a cutting-edge field, FMT follows a unique model of federal oversight. While FMT is considered an Investigational New Drug (IND), prevailing FDA guidance allows physicians to perform FMT using OpenBiome material to treat C. difficile infection not responding to standard therapies without filing an IND application.

For frequently asked regulatory questions, please see the FMT and the FDA section.

    • OpenBiome provides material to licensed medical doctors for use in a clinical setting. Material may be used for the treatment of C. difficile infection not responsive to standard therapies, or the treatment of other indications under an Investigational New Drug (IND) application. Our partners are most often gastroenterologists, infectious disease specialists, or internal medicine physicians.

      Today, over 700 hospitals across all 50 U.S. states have joined our clinical network. If you're a clinician interested in working with us for the first time, please send a completed Clinical Partner Registration Form to our Clinical Outreach Team at info@openbiome.org. The protocols for initiating a fecal transplantation program have varied significantly across our partners, and we would be more than happy to talk to you about what might work best for your facility.

      Full information about how to become a clinical partner is available on the Place an Order page. If you have any further questions about how to work with OpenBiome, please don't hesitate to write to us or call 617-575-2201, option 3.

    • The Food & Drug Administration (FDA) considers fecal microbiota for transplantation an investigational new drug (IND), a classification that typically requires physicians and scientists to file an IND application if they intend to use the treatment for clinical practice or research. However, FDA has issued guidance stating that FMT may be used to treat C. difficile infection not responsive to standard therapies outside of a clinical trial. The following are common questions about the regulation of FMT:

      Can physicians perform FMT without an IND application (outside of a clinical trial)?

      Yes, to treat patients with C. difficile infections not responsive to standard therapy. When the FDA announced in May 2013 that it would regulate fecal microbiota as an investigational drug, medical professional societies, physicians, and patients reacted with concern over how the policy would limit access to a therapy with 90% efficacy rates for this illness and no known short-term safety risks (Kassam et al., 2013).

      Responding to these concerns, FDA issued guidance in July 2013 stating that it would exercise “enforcement discretion” – that is, it would allow doctors to provide FMT (for patients with C. difficile infections not responding to standard therapies) without filing an IND application.

      What are the conditions for performing FMT without an IND application?

      Physicians may only use FMT without an IND application to treat patients with C. difficile not responsive to standard therapy. Physicians must also obtain adequate informed consent from the patient or a legal representative, that includes, at minimum, a statement that the use of FMT products to treat CDI is investigational, and a discussion of the therapy’s potential risks and alternative options.

      There are no FDA requirements about how to obtain stool for FMT. Physicians may use material from a donor identified by the patient or physician, or a from a universal stool bank. There are also no requirements dictating the route of administration for FMT (e.g. colonoscopy, naso-enteric delivery, oral capsule).

      Can physicians perform FMT for indications other than C. difficile?

      Physicians who would like to use FMT products for any other diseases must file an IND application with the FDA, but OpenBiome is well positioned to assist these investigations. We have worked closely with federal regulators, and our Biologics Master Files (BB-MF 15543, 17194, and 17195) provides the FDA with comprehensive insight into OpenBiome’s processes.

      Physicians who wish to conduct FMT under IND may reference the OpenBiome BB-MF. By doing so, physicians may use our robust quality and manufacturing protocols rather than needing to develop these components internally to support their IND applications. Please reach out to us at science@openbiome.org to learn more about how we can support your clinical trials.

      Do stool donors need to be "known" to the physician or patient?

      No: under the prevailing guidance the FDA does not require donors to be “known” to either the physician or patient.

      In March 2014, the FDA released draft guidance for public feedback only, concerning two proposed changes to the current policy: (1) that the donor be “known” to the patient or physician and (2) that all donor and stool screening be conducted under the supervision of the physician performing the FMT. Medical professional societies raised significant concerns about the proposal’s potential to compromise access and safety.

      I have seen a draft FDA guidance stating that physicians using stool from a stool bank must do so under IND. Is this the new policy?

      In March 2016, FDA released its second draft guidance to propose that enforcement discretion be narrowed so that physicians who obtain material from stool banks to treat CDI that is not responsive to stand therapy do so under IND. This document is a draft for discussion purposes, and does not alter current enforcement discretion policy.

      The prevailing FDA guidance, published in July 2013, states that physicians may perform FMT outside of an Investigational New Drug (IND) application to treat C. difficile infection (CDI) that is not responsive to standard therapy, so long as they obtain informed consent.

      OpenBiome continues to provide treatments to our clinical partners for recurrent CDI. No IND is necessary.

      In their draft, FDA requested feedback on how to implement this proposal so that it does not create undue burdens for physicians. We, along with medical societies, clinicians, and patients, responded to the guidance to call for an outcome that affords appropriate oversight of stool banking practices without placing undue burdens on physicians, healthcare facilities, and patients. OpenBiome's comment may be found here. You may review comments on the draft here.

      The FDA is reviewing all comments on the draft as it considers the finalization of guidance. We are glad to receive your questions or comments about this topic or about OpenBiome in general at info@openbiome.org.

      Under what conditions may physicians provide FMT in countries other than the U.S.?

      In many countries, as in the U.S., regulation governing the use of FMT is in nascent stages. If you are a physician from outside of the U.S. and would like to work with us to provide FMT for patients with recurrent C. difficile infection, or to support your clinical trial, please reach out at info@openbiome.org.

      Does the FDA have oversight of OpenBiome's work?

      OpenBiome's Biologics Master Files (BB-MF 15543, 17195, and 17194), registered with the FDA, provides regulators with comprehensive insight into OpenBiome’s processes. However, this is a voluntary move on our part. Regardless of their chosen source of stool, FMT practitioners should always closely evaluate the set of safety and quality controls used in donor and material screening, material processing, and storage and shipping.

      At OpenBiome, we are committed to ensuring rigorous standards for the quality and safety of our fecal microbiota samples, and to continue working closely with the FDA to expand safe access to FMT for our patients. Please reach out to us at info@openbiome.org if you have further questions.