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

Center for Food Safety & Applied Nutrition (CFSAN) Center for Tobacco Products (CTP) Center for Veterinary Medicine (CVM) Center for Biologics Evaluation & Research (CBER) Center for Devices & Radiological Health (CDRH)

FDA

OCP Center for Drug Evaluation and Research (CDER) National Center for Toxicology Research (NCTR)

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

metabolized for the achievement of its primary intended purposes.

and which is not dependent upon being

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

Device

• New component for a marketed device to support new indication • Combining a marketed device with a marketed drug and/or biological product

Device Combination Product !

Office of Combination Product (OCP)

OCP determines lead Center

OCP’s algorithm

1. 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

Device

• New component for a marketed device to support new indication • Combining a marketed device with a marketed drug and/or biological product

Device CDRH-Lead

Device Regulation is Risk-based

Medical Device Classes: Additional Classification:

De Novo Class I

General Controls Most exempt from premarket submission Device "types" that have never been marketed in the U.S., but whose safety profile and technology are now reasonably well understood

Class II

General Controls Special Controls

Premarket Notification [510(k)] Class III

Premarket Approval

Premarket Approval Application [PMA] 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

What was the marketing pathway for the platform technology?

• Combining a marketed device with a marketed drug and/or biological product

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  Yes to either 510(k)  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. a. If HUD is granted, HDE (with clinical to support new indication).

b. 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: [email protected] or 301-796-8930 • Orphans Product • Medical Device Pre-market Process-Programs Operations Staff: : 301-796-8660 [email protected] or 301-796-6560 • Neuro Device Pre-market Questions: [email protected] or 301-796-6610 CDRH Information Resources or on www.fda.gov

: search term “CDRH Learn” or “Device Advice” from Google

Thank You!