Description
The 21st Century Cures Act (Cures Act), signed into law on December 13, 2016, was intended to accelerate medical product development and bring innovations and advances faster and more efficiently to the patients who need them. Among other provisions, the Cures Act added section 505F to the Federal Food, Drug, and Cosmetic Act (FD&C Act). In response to the requirements in section 505F, FDA created a framework for a Real-World Evidence (RWE) Program to evaluate the potential use of RWE to help support the approval of a new indication for a drug already approved under section 505(c) of the FD&C Act or to help support or satisfy postapproval study requirements. In the context of this program, this guidance provides considerations for sponsors proposing to design a new registryor use an existing registry to support regulatory decision-making about a drug’s effectiveness or safety. This guidance does not provide recommendations on choice of study design or statistical methods used to analyze data from registries.
Key Topics
Terms and concepts identified from this document
Scope & Applicability
Product Classes
2Defined 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
Requires analytical comparability per ICH Q5E
Stakeholders
3Responsible for submission and communication oversight; Safeguard the rights, safety and well-being of trial participants; Reviewing trial conduct and records
Entity responsible for submitting applications under section 524B
Governs top dose in clinical studies
Regulatory Context
Regulatory Activities
5RWE may help support or satisfy these requirements
available for FDA to review during sponsor inspections
Mechanism for applicants to discuss total nitrosamine limits with FDA.
The regulatory activity where inclusion of specific populations is discussed; The primary vehicle for generating robust clinical data for these populations.; Trials including pregnant participants for non-obstetric or obstetric indications; Inclusion of breastfeeding women in clinical trials; Research studies where breastfeeding women may be included.; The context in which these outcome parameters are collected.
Alternate strategy for data collection
Document Types
7Administrative data used as a source of RWD.
An established data dictionary, which is available for those who intend to use the registry data
Source of RWD for evaluation of safety or effectiveness.
System where patients using the drug are enrolled; A surveillance strategy to evaluate the incidence and severity of risk.
Process by which a subject voluntarily confirms his or her willingness to participate in a particular trial; Integral feature of the ethical conduct of a trial
Document for protocol execution; The sponsor should develop a statistical analysis plan that is consistent with the trial protocol; Deviations from this plan must be justified
Defines the standard of veterinary practice and limits for anesthetic regimens
Attributes
5Attributes of a registry that support collection of relevant and reliable data
Considerations for cybersecurity when devices connect to other systems
relevance includes the availability of data for key study variables
reliability includes accuracy, completeness, and traceability
Responsibility for the integrity of the trial data
Technical Details
Substances
1Used to increase development efficiency
Processes
1Validation of the electronic systems used to collect registry data
Clinical Concepts
1Characterizing disease progression using registries
Standards & References
External Standards
2Licensing of biological products
Legislation that amended the FD&C Act regarding patient experience data
Specifications
2Number of subjects needed to achieve pre-specified power
Planning interventional study endpoints using registry data
Related CFR Sections (3)
- 21CFR314.50§ 314.50 Content and format of an NDA.
NDAs and supplements to approved NDAs are required to be submitted in the form and contain the information, as appropriate for the particular submission, required under this section. Three copies of the NDA are required: An archival copy, a review copy, and a field copy. An NDA for a new chemical enRead full regulation →
- 21CFR601.2§ 601.2 Applications for biologics licenses; procedures for filing.
(a) General. To obtain a biologics license under section 351 of the Public Health Service Act for any biological product, the manufacturer shall submit an application to the Director, Center for Biologics Evaluation and Research or the Director, Center for Drug Evaluation and Research (see mailing aRead full regulation →
- 21CFR312.58§ 312.58 Inspection of sponsor's records and reports.
(a) FDA inspection. A sponsor shall upon request from any properly authorized officer or employee of the Food and Drug Administration, at reasonable times, permit such officer or employee to have access to and copy and verify any records and reports relating to a clinical investigation conducted undRead full regulation →
Enforcement Impact
Deficiencies cited in Warning Letters referencing the same regulations
Related Warning Letters (3)
Related MFDS Guidelines
Korean regulatory guidelines covering similar topics
See Also (8)
- COVID-19: Developing Drugs and Biological Products for Treatment or Prevention: Guidance for Industry (Status: Final)
- Chapter 1 - General (Status: Final)
- Using Electronic Means to Distribute Certain Product Information: Guidance for Industry (Status: Final)
- CPG Sec. 400.210, Radiofrequency Identification Feasibility Studies and Pilot Programs for Drugs (Status: Final)
- Guidance for Industry: Questions and Answers on Juice HACCP Regulation (2003) (Status: Final)
- Guidance for Industry: Juice Hazard Analysis Critical Control Point Hazards and Controls Guidance, First Edition (Status: Final)
- Small Entity Compliance Guide: Current Good Manufacturing Practice in Manufacturing, Packaging, Labeling, or Holding Operations for Dietary Supplements (Status: Final)
- Final In Vivo Bioavailability-Bioequivalence Studies- Analytical (Status: Final)