Description
This guidance provides recommendations to industry about the use of placebos and blinding in randomized controlled clinical trials in development programs for drug or biological products to treat hematologic malignancies and oncologic diseases. This guidance does not address the statistical analyses that can be considered when data are unblinded in these trials.
Key Topics
Terms and concepts identified from this document
Scope & Applicability
Product Classes
4placebo is given as monotherapy
Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.
Regulated 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
RTRT and CTD sections apply to drug products
Stakeholders
2facilitate discussions between a prospective subject and an investigator; Responsible for conducting research and providing informed consent
Entity responsible for submitting applications under section 524B
Regulatory Context
Regulatory Activities
1development programs for drug or biological products
Document Types
3Basic statistical principle for clinical trials
Submitted to an existing active IND
Document required for patient participation; document describing risks, benefits, and procedures to the patient
Technical Details
Substances
8primary adverse event prophylaxis
primary adverse event prophylaxis
primary adverse event prophylaxis
treatment for adverse events
treatment for adverse events
Positive control substance used in the assay; positive control used with S9 metabolic activation
Circulating hormones produced by the adrenal gland.; Used as rescue therapy for adrenal insufficiency
Accountability procedures for the investigational product including placebo; used as a reference in a clinical trial
Processes
3setting where justification for trial design is important
trials where standard of care is surveillance
trial design where placebo-controlled study may be useful
Clinical Concepts
7Reason a patient may be unable to complete a PRO measure; Treatment discontinuation due to AE.; Reason for treatment discontinuation or PRO non-completion.
maintenance of blinding at the time of disease progression
determination of disease recurrence for DFS
prevent infusion reaction
SAEs collected during interventional clinical trials
Treatment of oncologic diseases
bone marrow biopsies in patients with certain malignancies
Identified Hazards
Hazards
1concern in open-label comparative trials
Standards & References
External Standards
1Ethical principles for medical research involving human subjects
ICH References (1)
Choice of Control Group in Clinical Trials.
Related MFDS Guidelines
Korean regulatory guidelines covering similar topics
See Also (8)
- E11A Pediatric Extrapolation
- M15 General Principles for Model-Informed Drug Development
- Clinical Pharmacology Data to Support a Demonstration of Biosimilarity to a Reference Product
- Q6A Specifications: Test Procedures and Acceptance Criteria for New Drug Substances and New Drug Products: Chemical Substances
- Rare Diseases: Considerations for the Development of Drugs and Biological Products
- Q3C Impurities: Residual Solvents_2011
- Providing Regulatory Submissions in Electronic Format --Content of the Risk Evaluation and Mitigation Strategies Document Using Structured Product Labeling: Guidance for Industry
- Q4B Annex 5: Disintegration Test General Chapter