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. This guidance complements and expands on the 1998 guidance entitled Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products (the 1998 guidance).
Scope & Applicability
Product Classes
4Regulated under section 351(i) of the PHS Act; Virus, therapeutic serum, toxin, vaccine, or protein applicable to prevention or treatment; Alternative regulation category for products meeting device definition
Requires analytical comparability per ICH Q5E
scope of the guidance document
Defined in section 201(g) of the FD&C Act; Articles intended for use in diagnosis, cure, mitigation, treatment, or prevention of disease; Product classification based on chemical action or specific intended uses like altering pH
Stakeholders
3Entity responsible for submitting applications under section 524B
individuals who conclude if a drug has the intended effect
Entity submitting development data and knowledge; Entity performing the work process for change
Regulatory Context
Attributes
4Measurement of potency for biological products
Legal standard for drug approval under the FD&C Act
typical criterion for concluding a trial is positive
The need for substantiation of experimental results through multiple trials.
Identified Hazards
Hazards
2Reversal of signs via antidote as evidence of effectiveness
Agents causing conditions that trigger the applicability of the Animal Rule.
Related CFR Sections (9)
- 21CFR601.90§ 601.90 Scope.
This subpart applies to certain biological products that have been studied for their safety and efficacy in ameliorating or preventing serious or life-threatening conditions caused by exposure to lethal or permanently disabling toxic biological, chemical, radiological, or nuclear substances. This suRead full regulation →
- 21CFR314.600§ 314.600 Scope.
This subpart applies to certain new drug products that have been studied for their safety and efficacy in ameliorating or preventing serious or life-threatening conditions caused by exposure to lethal or permanently disabling toxic biological, chemical, radiological, or nuclear substances. This subpRead full regulation →
- 21CFR314.105§ 314.105 Approval of an NDA and an ANDA.
(a) FDA will approve an NDA and send the applicant an approval letter if none of the reasons in § 314.125 for refusing to approve the NDA applies. FDA will issue a tentative approval letter if an NDA otherwise meets the requirements for approval under the Federal Food, Drug, and Cosmetic Act, but caRead full regulation →
- 21CFR312.80§ 312.80 Purpose.
The purpose of this section is to establish procedures designed to expedite the development, evaluation, and marketing of new therapies intended to treat persons with life-threatening and severely-debilitating illnesses, especially where no satisfactory alternative therapy exists. As stated § 314.10Read 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 →
- 21CFR312.81§ 312.81 Scope.
This section applies to new drug and biological products that are being studied for their safety and effectiveness in treating life-threatening or severely-debilitating diseases.Read full regulation →
- 21CFR312.3§ 312.3 Definitions and interpretations.
(a) The definitions and interpretations of terms contained in section 201 of the Act apply to those terms when used in this part:Read full regulation →
- 21CFR600.3§ 600.3 Definitions.
As used in this subchapter:Read full regulation →
- 21CFR312.300§ 312.300 General.
(a) Scope. This subpart contains the requirements for the use of investigational new drugs and approved drugs where availability is limited by a risk evaluation and mitigation strategy (REMS) when the primary purpose is to diagnose, monitor, or treat a patient's disease or condition. The aim of thisRead full regulation →
Related Warning Letters (1)
- 2020-08-19
Unapproved and Misbranded Product Related to Coronavirus Disease 2019 (COVID-19)
Predictive Biotech
See Also (8)
- Neglected Tropical Diseases of the Developing World: Developing Drugs for Treatment or Prevention (Status: Final)
- Product Development Under the Animal Rule (Status: Final)
- Emergency Use Authorization of Medical Products and Related Authorities: Guidance for Industry and Other Stakeholders (Status: Final)
- Antibacterial Therapies for Patients With an Unmet Medical Need for the Treatment of Serious Bacterial Diseases (Status: Final)
- Mpox: Development of Drugs and Biological Products; Guidance for Industry (Status: Draft)
- Acute Radiation Syndrome: Developing Drugs for Prevention and Treatment (Status: Draft)
- Guidance for Industry: Acute Bacterial Otitis Media: Developing Drugs for Treatment (Status: Final)
- Acute Bacterial Sinusitis — Developing Antimicrobial Drugs for Treatment (Status: Final)