1. 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

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

3. 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

4. OCP

5. 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)

6. 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)

7. 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)

8. 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)

9. 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

10. 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 !

11. Office of Combination Product (OCP)
OCP determines lead Center

12. 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?

13. 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

14. 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.

15. 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

16. 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?

17. 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

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

19. 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.

20. 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

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

22. FDA Contacts OCP: combination@fda.gov or 301-796-8930
Orphans Product:
Medical Device Pre-market Process-Programs Operations Staff: or
Neuro Device Pre-market Questions: or
CDRH Information Resources: search term “CDRH Learn” or “Device Advice” from Google or on

23. Thank You!