Regulatory Pathway for Platform Technologies Quynh Hoang Neurotherapeutic and Neurodiagnostic Devices Branch Division of Ophthalmic, Neurological and Ear, Nose and Throat Devices Office of Device Evaluation Center for Devices and Radiological Health ASENT February 24, 2011

Disclaimer This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

Overview Regulatory definitions: drug, device, biological product, combination product Medical Device Platform Technology Examples OCP and Lead Center Medical Device Classifications and Corresponding Applications Medical Device Submissions for Platform Technology Examples Recent Development in CDRH

OCP

Drug Definition (A) articles recognized in the US Pharmacopoeia, Homeopathic Pharmacopoeia, or National Formulary; or (B) articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals; or (C) articles (other than food) intended to affect the structure or any function of the body of man or other animals. 21 USC 201(g)

Device Definition Instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including any component, part, or accessory, which is - (1) recognized in the official National Formulary, or the United States Pharmacopoeia, or any supplement to them, or (2) 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 (3) 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. 21 USC 201(h)

Biological Product Definition Virus Therapeutic Serum Toxin or Antitoxin Vaccine Blood, Blood Component or Derivative Allergenic Product Protein (except any chemically synthesized polypeptide) Analogous Product (e.g., human tissues) Arsphenamine or its derivatives applicable to the prevention, treatment, or cure of diseases or injuries of man 42 USC 262 (as amended 2010)

Combination Product Definition Combination of Biological Product & Device Biological Product & Drug Device & Drug Biological Product & Device & Drug NOT of drug&drug, device&device or biologic&biologic Combination from being: Physically or chemically combined Co-packaged in a kit Separate, cross-labeled products 21 CFR 3.2(e)

Medical Device Platform Technology Examples New indication for a marketed device New component for a marketed device to support new indication Combining a marketed device with a marketed drug and/or biological product

Medical Device Platform Technology Examples New indication for a marketed device New component for a marketed device to support new indication Combining a marketed device with a marketed drug and/or biological product Device Device Combination Product !

Office of Combination Product (OCP) OCP determines lead Center

OCP’s algorithm Primary mode of action 2. If unable to determine most important therapeutic action with reasonable certainty, consider: Consistency: is there an agency component that regulates other combination products presenting similar questions of S & E with regard to combination product as a whole? Safety and Effectiveness: which agency component has the most expertise related to most significant S&E questions presented by combination product?

Medical Device Platform Technology Examples New indication for a marketed device New component for a marketed device to support new indication Combining a marketed device with a marketed drug and/or biological product Device Device CDRH-Lead

Device Regulation is Risk-based Medical Device Classes: Class I General Controls Most exempt from premarket submission Class II Special Controls Premarket Notification [510(k)] Class III Premarket Approval Premarket Approval Application [PMA] Additional Classification: De Novo Device "types" that have never been marketed in the U.S., but whose safety profile and technology are now reasonably well understood Humanitarian Device Exemption (HDE) Devices for orphan diseases intended to benefit patients in diagnosis and/or treatment of disease or condition affecting or manifested in fewer than 4,000 patients per year in the US.

Investigational Device Exemptions (IDE) application An FDA approved IDE allows a significant risk investigational device to be used in a clinical study in the U.S. in order to collect safety and effectiveness data 21 CFR 812

Medical Device Platform Technology Examples New indication for a marketed device New component for a marketed device to support new indication Combining a marketed device with a marketed drug and/or biological product What was the marketing pathway for the platform technology?

Platform Technology was 510(k) “cleared” or De Novo “classified” Change to Indication: Does the change to the indication alter the intended therapeutic/diagnostic/etc. effect (or impact safety or effectiveness)? Change to Technology: Does the change raise new types of safety or effectiveness questions? No to both  510(k) Yes to either  De Novo, PMA, or HUD/HDE AND supporting clinical (IDE) data for a significant risk device

Platform Technology was PMA “approved” Original PMA Panel-Track PMA Supplement, or PMA Supplement (with IDE data to support new indication or claim)

Platform Technology was HDE “approved” Request a Humanitarian Use Device (HUD) Designation from the Office of Orphan Products Development for the new device. If HUD is granted, HDE (with clinical to support new indication). If HUD is not granted, 510(k) or PMA.

Marketing Applications 510(k) – substantial equivalence to a predicate De Novo After a not-substantially-equivalent 510(k) determination Low risk, Available data and Special controls PMA – reasonable assurance of safety and reasonable assurance of effectiveness HDE – reasonable assurance of safety and probable benefit

Some Recent Developments in CDRH CDRH Preliminary Internal Evaluations of the 510(k) review process CDRH Innovation Pathway Streamline the De Novo process www.fda.gov (search highlighted terms)

FDA Contacts OCP: combination@fda.gov or 301-796-8930 Orphans Product: 301-796-8660 Medical Device Pre-market Process-Programs Operations Staff: Robert.Gatling@fda.hhs.gov or 301-796-6560 Neuro Device Pre-market Questions: Quynh.Hoang@fda.hhs.gov or 301-796-6610 CDRH Information Resources: search term “CDRH Learn” or “Device Advice” from Google or on www.fda.gov

Thank You!