Description
This document is intended to provide guidance to applicants planning to file new drug applications (NDAs), biologics license applications (BLAs), or applications for supplemental indications on the evidence to be provided to demonstrate effectiveness.
Scope & Applicability
Product Classes
4Product category subject to PDUFA user fees.
Not addressed in this guidance
Requires analytical comparability per ICH Q5E
each active substance should be described
Stakeholders
4Entity submitting development data and knowledge; Entity performing the work process for change
responsible for justifying omission of studies
Entity responsible for submitting NDINs
Target population for drug development and disease definition
Regulatory Context
Attributes
5well-understood relationship between blood concentration and response
consistency across key patient subsets addresses concerns about generalizability
Evaluation used to determine if expanded access is appropriate
Measurement of potency for biological products
Field B.2.2.2 describing the physical form of the VMP
Identified Hazards
Hazards
1Risk factor in single-center efficacy results
Related CFR Sections (3)
- 21CFR600.3§ 600.3 Definitions.
As used in this subchapter:Read full regulation →
- 21CFR314.126§ 314.126 Adequate and well-controlled studies.
(a) The purpose of conducting clinical investigations of a drug is to distinguish the effect of a drug from other influences, such as spontaneous change in the course of the disease, placebo effect, or biased observation. The characteristics described in paragraph (b) of this section have been develRead full regulation →
- 21CFR201.57§ 201.57 Specific requirements on content and format of labeling for human prescription drug and biological products described in § 201.56(b)(1) .
The requirements in this section apply only to prescription drug products described in § 201.56(b)(1) and must be implemented according to the schedule specified in § 201.56(c) , except for the requirement in paragraph (c)(18) of this section to reprint any FDA-approved patient labeling at the end oRead full regulation →
See Also (8)
- Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations: Draft Guidance for Industry (Status: Draft)
- Guidance for Industry for the Evaluation of Combination Vaccines for Preventable Diseases: Production, Testing and Clinical Studies: Guidance for Industry (Status: Final)
- Clinical Data Needed to Support the Licensure of Seasonal Inactivated Influenza Vaccines: Guidance for Industry (Status: Final)
- Clinical Data Needed to Support the Licensure of Pandemic Influenza Vaccines: Guidance for Industry (Status: Final)
- Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products (Status: Draft)
- Human Gene Therapy Products Incorporating Human Genome Editing: Guidance for Industry (Status: Final)
- Potency Assurance for Cellular and Gene Therapy Products (Status: Draft)
- Rare Diseases: Considerations for the Development of Drugs and Biological Products (Status: Final)