Description
This guidance has two purposes: 1) to make it clear that an effect on symptoms or physical function, without a favorable effect on survival or risk of hospitalization, can be a basis for approving drugs to treat heart failure; and 2) to provide recommendations to sponsors on the need to assess mortality effects of drugs under development to treat heart failure.
Key Topics
Terms and concepts identified from this document
Scope & Applicability
Product Classes
1Specific population with unique safety considerations
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Regulatory Activities
2eligible for one or more of FDA's expedited programs
Regulatory milestone for heart failure drugs
Attributes
3Endpoint for approving drugs
Endpoint for approving drugs
Primary endpoint for efficacy studies
Technical Details
Substances
10Biomarker used for prognostic enrichment
Specific drug with established safety profile
Mineralocorticoid receptor antagonists
Angiotensin II receptor blockers with established safety
Approved drug for heart failure
Used to reduce signs and symptoms
MRAs
Pharmacologic class for heart failure; Drug class used in heart failure treatment
ARBs
Pharmacologic class for heart failure; Class of drugs with well-established safety profiles
Testing Methods
1Endpoint influenced by expectation bias requiring blinding
Clinical Concepts
10Measure of morbidity and clinical outcome
Therapeutic area standard
Closely related population to heart failure
include estimates of acute morbidity and mortality; Deaths included in the public health impact evaluation.
Patient population classification
Patient population classification
HFpEF
HFrEF
Discussed briefly in the guidance
Primary focus of the guidance
Standards & References
Specifications
2Biomarker used to characterize risk
Acceptable efficacy endpoint for heart failure
ICH References (2)
Addendum: Clinical Investigation of Medicinal Products in the Pediatric Population
Clinical Investigation of Medicinal Products in the Pediatric Population.
Related MFDS Guidelines
Korean regulatory guidelines covering similar topics
See Also (8)
- Advanced Manufacturing Technologies Designation Program
- Direct-to-Consumer Prescription Drug Advertisements: Presentation of the Major Statement in a Clear, Conspicuous, and Neutral Manner in Advertisements in Television and Radio Format Final Rule Questions and Answers
- Q4B Annex 8: Sterility Test General Chapter
- Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products: Draft Guidance for Industry
- Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products: Guidance for Industry
- Failure to Respond to an ANDA Complete Response Letter Within the Regulatory Timeframe Guidance for Industry: Guidance for Industry
- CMC Postapproval Manufacturing Changes for Specified Biological Products To Be Documented in Annual Reports
- Best Practices in Developing Proprietary Names for Human Nonprescription Drug Products; Draft Guidance for Industry: Draft Guidance for Industry