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Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use: Guidance for Industry and Food and Drug Administration Staff

FinalCenter for Biologics Evaluation and Research Center for Devices and Radiological Health07/20/2020
Minimal ManipulationHomologous Use

Description

We, FDA, are providing you, human cells, tissues, and cellular and tissue-based product (HCT/P) manufacturers, healthcare providers, and FDA staff, with our current thinking on the criteria under Title 21 of the Code of Federal Regulations (CFR) Part 1271, specifically the 21 CFR 1271.10(a)(1) criterion of minimal manipulation and the 21 CFR 1271.10(a)(2) criterion of homologous use.

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

7
HCT/Ps

Human cells, tissues, or cellular or tissue-based products defined in 21 CFR Part 1271

Human Cells, Tissues, and Cellular and Tissue-Based Products

HCT/Ps are the primary products covered by this guidance.; Guidance regarding the reduction of HBV transmission risk in HCT/Ps.; Products for which HBV transmission risk recommendations are provided

Combination Product

Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.

HCT/P

Human cells, tissues, and cellular and tissue-based products regulated as devices; Human cells, tissues, and cellular and tissue-based products regulated as medical devices; Human Cell, Tissue or Cellular or Tissue-Based Product

Structural Tissue

Tissue that provides physical support, reconstruction, repair, or replacement; Tissues that physically support, serve as a barrier, conduit, connect, cover, or cushion

Nonstructural Tissues

Cells or tissues regulated based on biological characteristics

Cellular/Nonstructural Tissue

Tissues that serve metabolic or other biochemical roles

Stakeholders

3
Manufacturer

Entity responsible for submitting NDINs

Healthcare Provider

User role with specific access privileges

Healthcare Providers

Users of HCT/Ps in patients

Regulatory Context

Regulatory Activities

6
IND

Investigational New Drug submissions

Request for Designation

Process to obtain a formal classification determination

New Drug Application

Rule does not apply to products marketed under an NDA

Premarket approval

PMA application for high-risk devices

BLA

Biologics License Application

Premarket Review

Consistency and predictability in the premarket review process

Document Types

1
Labeling

Cybersecurity information should be included in device labeling

Attributes

7
Systemic Effect

Criterion for determining regulatory pathway

potency

Key search term and scientific factor for evaluation.

purity

Key tests for vaccine purity should be validated

Multipotency

Relevant biological characteristic of stem cells

Tensile strength

Original relevant characteristic of ligament

Metabolic Activity

Primary function dependency for living cells

Original relevant characteristics

Properties of tissue in the donor that contribute to its function

Technical Details

Substances

10
Adipose Tissue

Specific tissue type discussed regarding regulatory considerations; Considered a structural tissue providing cushioning and support; Structural tissue providing cushioning and support; Tissue used for cushioning, support, and energy storage

Platelet rich plasma

Blood product excluded from HCT/P definition

Hematopoietic stem/progenitor cells

Cells that serve metabolic or biochemical roles; HCT/P used for replenishing the lymphohematopoietic system

Cartilage allograft

Structural tissue used for load-bearing functions

Decellularized dermis

Example of a structural HCT/P

Crystalloids

Infused fluids that can contribute to plasma dilution.; infusion volume considered in plasma dilution algorithm

Amniotic membrane

Structural tissue serving as a barrier; Tissue used for barrier, protection, or covering functions

Bone

HCT/P type requiring AFB culture

Skin

Structural tissue serving as a protective covering

Demineralized bone matrix

HCT/P product with specific regulatory classification factors

Processes

8
Manufacture

The process for which all components must be listed

Minimal Manipulation

Regulatory threshold for HCT/P classification

Enzymatic digestion

Processing of adipose tissue to isolate cellular components

Cell selection

Processing method for mobilized peripheral blood apheresis products

Cryopreservation

If cryopreservation poses a risk, potency assays should be performed after this step.

Processing

Activity that triggers the requirement for facility registration.

Decellularization

Removal of cells from structural tissue

Lyophilization

Physical modification that does not result in chemical alteration.; A process that does not typically chemically alter an ingredient

Clinical Concepts

5
Dura mater defects

Clinical condition where pericardium may be used as a wound covering

Cerebral palsy

Condition where cord blood infusion is considered non-homologous

Myocardial infarction

For studies in which the outcome or outcomes of interest (e.g., myocardial infarction or stroke) include fatal outcomes.

Homologous use

Repair, reconstruction, replacement, or supplementation of a recipient’s cells or tissues with an HCT/P that performs the same basic function

Myeloablative treatment

Treatment resulting in the need for hematopoietic stem cell replacement

Identified Hazards

Hazards

1
Communicable Disease

Prevention of introduction, transmission, and spread

Related CFR Sections (3)

Enforcement Impact

Deficiencies cited in Warning Letters referencing the same regulations

Quality control unit failed to exercise its responsibility
101
Failure to conduct at least one test to verify the identity of each component
95
Failure to establish adequate written procedures for production and process control
88
Failure to thoroughly investigate any unexplained discrepancy or failure of a batch
56
Failure to have appropriate laboratory determination of satisfactory conformance to final specifications
56
Failure to establish an adequate quality control unit
49
Failure to test samples of each component for identity and conformity
47
Failure to establish laboratory controls
36
Failure to thoroughly investigate any unexplained discrepancy
28
Failure to test samples of each component for identity
25

Related Warning Letters (10)

Related MFDS Guidelines

Korean regulatory guidelines covering similar topics

See Also (8)