CBER Regulation of Devices for Cell Therapy

Transcription

1 CBER Regulation of Devices for Cell Therapy Richard D. McFarland, Ph.D., M.D. Associate Director for Policy Office of Cellular, Tissue and Gene Therapies Center for Biologics Evaluation and Research Food and Drug Administration

2 Scope of Talk Introduction to Device Regulation Devices and Cell Therapy Recent Guidances

3 Device Regulations Investigational Device Exemption (IDE) 21 CFR 812 Premarket Notification (510(k)) 21 CFR 807 (Subpart E) Premarket Approval (PMA) 21 CFR 814 (Subparts A-E) Humanitarian Device Exemption(HDE) 21 CFR 814 (Subpart H) Quality System Regulations (QSR) 21 CFR 820 GMPs for devices

4 Device Regulation Determined by 3 Levels of Classification Class I minimal potential for harm General controls sufficient to ensure safety & effectiveness. Must register and list (forms 2891 & 2892) and follow QSRs. Class II General controls insufficient. Special controls needed to ensure safety and effectiveness. Usually require 510(k). Special controls can include clinical studies under IDE

5 Class III Devices General and Special Controls insufficient to assure safety & effectiveness. Such devices: Support or sustain human life, Are of substantial importance in preventing impairment of human health, or Present a potential, unreasonable risk of illness or injury. Generally regulated through IDE followed by PMA.

6 IDEs 21 CFR 812 IDEs are approved or disapproved. For cost recovery, sponsor must submit request. Required to submit informed consent materials. 30 day clock for original submissions AND supplements. IDEs have supplements, INDs have amendments. Sponsor may withdraw No inactivation.

7 510(k)s (21 CFR 807) Filed by sponsor to notify FDA that they intend to market a device that is substantially equivalent (SE) to a predicate device. 510(k) required when: Introducing a device to the market for the first time Change in intended use for a marketed device Making significant modification to a marketed device.

8 Substantial Equivalence (SE) Can claim SE to: Preamendment device A device already found by FDA to be SE A reclassified device Can NOT use a PMA device as a predicate. Intended Use: What the sponsor labels the product to be used for Indication for Use: Use in a population of individuals.

9 Substantial Equivalence (Cont.) SE based on intended use and technological features. If not the same intended use, it is NSE. If same intended use AND same tech. Features, then SE. If same intended use and different tech characteristics, still SE if does not raise new questions of safety and effectiveness and is as safe and effective as the predicate. Performance data can be submitted.

10 Recent Guidances Guidance for Industry: Class II Special Controls Guidance Document: Cord Blood Processing System and Storage Container (1/31/2007) Office of Cellular Tissue and Gene Therapies Draft Guidance for Industry: Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage (7/6/2007) Draft Guidance Office of Cellular Tissue and Gene Therapies

11 Recent Guidances Devices Used to Process Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Somatic cell therapy Jurisdictional Update Office of Combination Products Cell Selection Devices for Point of Care Production of Minimally Manipulated Autologous Peripheral Blood Stem Cells (PBSCs) Gene therapy Draft Guidance Office of Cellular Tissue and Gene Therapies

12 Devices Used to Process Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Somatic Cell Therapy Reason for Jurisdictional Update Requests for Designation (RFDs) for devices intended to process human cells, tissues, or cellular and tissuebased products (HCT/Ps) Frequent request has been Lead Center Assignment

13 Devices Used to Process Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Somatic Cell Therapy CBER Devices Devices intended to process HCT/Ps ex vivo to create a therapeutic article Intended therapeutic effect is mediated by the biologic output of the device CBER should be consulted for further information about the regulatory pathway for such products, including information on the review and regulation of the HCT/P

14 Devices Used to Process Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Somatic Cell Therapy CBER Devices Cell sorters used at the point of care to isolate and/or concentrate autologous stem cells or hematopoietic progenitor cells for in vivo use Devices that process autologous blood or tissue to produce a new HCT/P (e.g., a tissue engineered, live cell construct) at the point of care Autologous Direct re-administration

15 Devices Used to Process Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Somatic Cell Therapy CDRH Devices Cell sorters designed to isolate or concentrate a specific cell population have been when the sorted cells are intended only for in vitro diagnostic use. The labeling limits them to in vitro diagnostic use Output material is not evaluated for suitability for readministration

16 Cell Selection Devices for Point of Care Production of Minimally Manipulated Autologous PBSCs Published: July 24, 2007 Comment Period Closes: October 24, 2007 Scope for this guidance Title reflects scope Five specific factors must be met for guidance Regulation of cellular products that fall outside the scope is unaffected by the guidance

17 Cell Selection Devices for Point of Care Production of Minimally Manipulated Autologous PBSCs The Five Factors The cells are autologous and are intended for use for a specific clinical indication The cells are minimally manipulated The device is solely responsible for the production of the autologous cells The cells are used within a short period of time The device and selected cells are only used at the point of care

18 Cell Selection Devices for Point of Care Production of Minimally Manipulated Autologous PBSCs Immediate Consequences of the Five Factors FDA would NOT require an individual clinical site using a cell selection device approved for this use to: Register and list as tissue establishment Comply with 21 CFR 1271 Submit an IND or BLA for the cellular product The cells such HCT/Ps WOULD be covered under same surgical procedure exception 21 CFR (b) The cell selection devices would be subject to all applicable premarketing and postmarketing provisions of the act as well as all applicable FDA regulations for devices.

19 Resources References for the Regulatory Process for the Office of Cellular, Tissue and Gene Therapies (OCTGT) CBER Product Approvals: Richard McFarland Ph.D., M.D. Associate Director for Policy Office of Cellular, Tissue and Gene Therapies

20 Upcoming FDA Public Workshops Processing of Orthopedic, Cardiovascular, and Skin Allografts October 11-12, 2007 NIH, Bethesda, MD In Vitro Analyses of Cell/Scaffold Medical Products December 6-7, 2007 NTSB Conference Center, Washington, DC