Is FMT A Drug? Lance Shea, M.S., J.D. Washington Square, Suite 1100 1050 Connecticut Ave., NW Washington, DC, 20036 D 202-861-1648 LShea@bakerlaw.com
FMT Scenarios Intra-Office Bank Product
The Issue The FDA s decision to regulate fecal microbiota as a drug has both positive and negative consequences. Broadly speaking, there is a clear need for oversight of the use of fecal microbiota for transplantation, given the risks posed by stool-borne transmissible illnesses...
The Issue The uniformity and expertise that the FDA can offer the industry are important institutional advantages both for stool providers navigating their legal responsibilities and for physicians seeking guidance on how to best help their patients. Sachs 2015 at 398.
The Issue However, the FDA s decision has proven problematic... Not only does stool defy the typical scientific characterization that the FDA has long applied to small molecule and biologic drugs,...
The Issue but the potential do-it-yourself nature of the treatment poses particular concerns in the context of a regime involving periods of regulatory exclusivity. The goals of any regulation of FMT for the treatment of recurrent C. diffcile infection should be to optimize access to the therapy while maintaining strict screening and reporting standards and oversight. Id.
Road Map What is a drug? What is a biological product? What is exclusivity?
What Is A Drug? What products are regulated by the FDA as drugs?
Drug (21 U.S.C. 321(g)(1)) The FDCA defines drugs as articles... recognized in the official United States Pharmacopoeia, official Homoeopathic Pharmacopoeia of the United States, or official National Formulary, or any supplement to any of them; and intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals; and (other than food) intended to affect the structure or any function of the body of man or other animals; and intended for use as a component of any article specified [above].
Drug (21 U.S.C. 321(g)(1)) The FDCA defines drugs as articles... recognized in the official United States Pharmacopoeia, official Homoeopathic Pharmacopoeia of the United States, or official National Formulary, or any supplement to any of them; and intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals; and (other than food) intended to affect the structure or any function of the body of man or other animals; and intended for use as a component of any article specified [above].
Intended Use The FD&C Act defines drugs, in part, by their intended use... Some products... [can have] two intended uses... a shampoo is a cosmetic because its intended use is to cleanse the hair. An antidandruff treatment is a drug because its intended use is to treat dandruff. Consequently, an antidandruff shampoo is both a cosmetic and a drug.
Establishing Intended Use Claims stated on the product labeling, in advertising, on the Internet, or in other promotional materials. Consumer perception, which may be established through the product's reputation. Ingredients that cause a product to be considered a drug because they have a well-known (to the public and industry) therapeutic use.
New Drug (21 C.F.R. 320(h)) The newness of a drug may arise by reason (among other reasons) of: A new use of a substance of which the drug is composed A new use of a combination of approved drugs A change in the proportions of ingredients in an approved drug A new indication for use A change in dosage, or method or duration of administration or application
New... Active moiety means the molecule or ion... responsible for the physiological or pharmacological action of the drug substance. 21 C.F.R. 314.108(a). New chemical entity means a drug that contains no active moiety that has been approved by FDA in any other application submitted under section 505(b) of the act. 21 C.F.R. 314.108(a).
Prescription Drugs (21 U.S.C. 353(b)) A drug intended for use by man which... is not safe for use except under the supervision of a practitioner licensed by law to administer such drug; or... is limited by an approved application under section 355 of this title to use under the professional supervision of a practitioner licensed by law to administer such drug; shall be dispensed only (i) upon a written prescription... (ii) upon an oral prescription... reduced promptly to writing and filed by the pharmacist, or (iii) by refilling any such written or oral prescription...
Over-The-Counter Drugs 1951: Durham-Humphrey Amendment OTC drug characteristics: benefits clearly outweigh possible risks potential for misuse and abuse is low public can use them for self-diagnosed conditions can be labeled to help assure appropriate use by the average person healthcare practitioners are not needed for their safe and effective use
What Is A Biological Product
Biologic Product (42 U.S.C. 262(i)) The term 'biological product' means... a virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, protein [and others]... applicable to the prevention, treatment, or cure of a disease or condition of human beings.
What Is Exclusivity?
Exclusivity Exclusivity provides the holder of an approved new drug application limited protection from new competition in the marketplace for the innovation represented by its approved drug product. http://www.fda.gov/drugs/developmentappro valprocess/smallbusinessassistance/ucm06 9962.htm
Exclusivity - Drugs 7 years orphan drugs 5 years NCEs 3 years new indications, clinical exclusivity, new populations, new release formulations, new combination products, etc. +6 months pediatric indications +5 years QIDP
Exclusivity - Biologicals 12 years BLA +6 months pediatric indications 7 years orphan product 3 years Rx to OTC
For Discussion Definition fit Jurisdiction Practicalities of approval/licensure Impact of exclusivity
Is FMT A Drug? Lance Shea, M.S., J.D. Washington Square, Suite 1100 1050 Connecticut Ave., NW Washington, DC, 20036 D 202-861-1648 LShea@bakerlaw.com
Drug Approval Pathways
New Drug Approval Pathways Three pathways for marketing approval: 505(b)(1) New drug (RLD) 505(b)(2) Generic not identical to the RLD 505(j) Generic identical to the RLD
Three Drug Approval Pathways 505(b)(1): Application contains full reports of investigations of safety and effectiveness New drugs Brand drug a/k/a reference listed drug (RLD) RLD: The listed drug identified by FDA as the drug product upon which an applicant relies in seeking approval of its ANDA (21 C.F.R. 314.94(a)(3))
Three Drug Approval Pathways 505(b)(2): The later drug is not identical to the RLD and differences between them are not insubstantial. Information required for approval, to cover the differences, comes from studies not conducted by or for the RLD applicant. The identical features are covered by data in the RLD NDA, where the 505(b)(2) sponsor has obtained a right of reference.
Three Drug Approval Pathways 505(j): Application contains information to show that the proposed product is identical... In active ingredient, dosage form, strength, route of administration, labeling, quality, performance characteristics, and intended use, among other things... to a previously approved product.
Biologics Licensure Pathways
Biologics Price Competition and Innovation Act of 2009 Two types of biological product applications: 351(a) Original BLA 351(k) Biosimilar BLA
Dietary Supplement (21 U.S.C. 321(ff)) The FDCA defines dietary supplement as a product (other than tobacco) intended to supplement the diet that bears or contains one or more of the following dietary ingredients: a vitamin a mineral an herb or other botanical an amino acid A dietary substance for use by man to supplement the diet by increasing the total dietary intake...... and more
Dietary Supplement v. Food (http://www.fda.gov/aboutfda/transparency/basics/ucm194357.htm) Conventional foods are foods that are not dietary supplements. Dietary supplements must be labeled as such and must not be represented for use as a conventional food or as the sole item of a meal or the diet. One way to distinguish dietary supplements from conventional foods is by looking at the nutrition information on the label of the product. Conventional foods must have a Nutrition Facts panel on their labels, but dietary supplements must have a Supplement Facts panel.
Device (21 U.S.C. 321(h)) The FDCA defines device as an instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including any component, part, or accessory, which is recognized in the official National Formulary, or the United States Pharmacopeia, or any supplement to them, intended for use in the diagnosis of disease or other conditions, or in the cure, mitigation, treatment, or prevention of disease, in man or other animals, or intended to affect the structure or any function of the body of man or other animals, and which does not achieve its primary intended purposes through chemical action within or on the body of man or other animals and which is not dependent upon being metabolized for the achievement of its primary intended purposes.
Device v. Drug (http://www.fda.gov/medicaldevices/deviceregulationandguidance/overview/classifyyour Device/ucm051512.htm) Bracco Pharmaceuticals case If the primary intended use of the product is achieved through chemical action or by being metabolized by the body, the product is usually a drug.