Description
This draft guidance describes considerations regarding a comparative clinical study or studies with efficacy endpoints (a comparative efficacy study or CES) intended to support a demonstration that a proposed therapeutic protein product is biosimilar to a reference product for the purpose of submitting a marketing application under the Public Health Service Act (PHS Act).
Key Topics
Terms and concepts identified from this document
Scope & Applicability
Product Classes
4The biological product to which a biosimilar is compared
Focus of the guidance for determining when a CES may inform biosimilarity
Biosimilar product determined to be interchangeable with the reference product
Biological product shown to be highly similar to an FDA-licensed reference product; Biological product demonstrated to be highly similar to a reference product
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Regulatory Activities
3BLA for biological products
Application seeking licensure of a biological product as biosimilar or interchangeable
Biologics license application submitted under section 351(k) of the PHS Act
Attributes
1Terms in which there must be no clinically meaningful differences; Standards required to be demonstrated for biological product licensure
Technical Details
Substances
1Highly purified proteins that can be structurally characterized
Testing Methods
4Clinical study to detect differences in efficacy between biosimilar and reference products
Comparative studies used to support biosimilarity
Used to determine if there are clinically meaningful differences
Data derived from these studies are required for a 351(k) BLA
Processes
2Assessment used to detect differences between two products at a structural and functional level
Clinical study with efficacy endpoints to support a demonstration of biosimilarity
Clinical Concepts
3Study of the biochemical and physiological effects of drugs
Study of how the body interacts with administered substances
Assessment of the immune response to the biological product
Identified Hazards
Hazards
1Assessment of toxicity required in a 351(k) BLA
Related MFDS Guidelines
Korean regulatory guidelines covering similar topics