Regulatory Pathway for Platform Technologies

Quynh HoangNeurotherapeutic and Neurodiagnostic Devices BranchDivision of Ophthalmic, Neurological and Ear, Nose and

Throat DevicesOffice 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 Tobacco Products

(CTP)

Center forBiologics

Evaluation &Research(CBER)

Center for Devices &

Radiological Health(CDRH)

Center for DrugEvaluation and

Research(CDER)

National Center for Toxicology

Research(NCTR)

Center for VeterinaryMedicine

(CVM)

Center for Food Safety & Applied Nutrition(CFSAN)

FDA

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 derivativesapplicable to the prevention, treatment, or cure of diseases or

injuries of man42 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&biologicCombination 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

CombinationProduct !

Device

Office of Combination Product (OCP)

OCP determines lead Center

OCP’s algorithm

1. Primary mode of action2. 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

CDRH-Lead

Device

Device Regulation is Risk-based

Medical Device Classes:

Class I General ControlsMost exempt from premarket submission

Class II General ControlsSpecial ControlsPremarket 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.

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: 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!