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Major Depressive Disorder: Developing Drugs for Treatment

DraftCenter for Drug Evaluation and Research06/21/2018

Description

The purpose of this guidance is to assist sponsors in the clinical development of drugs for the monotherapeutic, combination, and adjunctive treatment of major depressive disorder (MDD). Specifically, this guidance addresses the FDA’s current thinking regarding the overall development program and clinical trial designs for antidepressant drug products. This draft guidance is intended to serve as a focus for continued discussions among the Division of Psychiatry Products (the Division), pharmaceutical sponsors, the academic community, and the public.

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

2
Antidepressant drug products

The class of products for which development programs are described.

Rapid-acting antidepressant drugs

clinical trial design issues and regulatory considerations may differ from previously approved drugs

Stakeholders

7
Division of Psychiatry Products

The specific division within CDER preparing the guidance.

Sponsor

Entity responsible for submitting applications under section 524B

pediatric patients

relevant age groups to study in drug development

children

Defined as persons who have not attained the legal age for consent; Patient population from whom assent may be required for biopsies

adolescents

relevant age groups to be adolescents (ages 13 through 17)

neonates

Subjects less than 28 days old

Geriatric patients

Subpopulation with potentially different metabolism

Regulatory Context

Regulatory Activities

4
guidance meeting

Sponsors seeking to include a biomarker should request a guidance meeting

Postmarketing

Phase 4 safety considerations mentioned in the table of contents.

Pre-investigational new drug application meeting

Sponsors encouraged to consult FDA early

Randomized withdrawal trial

FDA typically requests a postmarketing commitment to conduct this

Document Types

2
Statistical analysis plan

include the overall strategy for handling different intercurrent events... in the statistical analysis plan

Protocols

Protocols for adaptive trials should include pre-specified criteria

Attributes

1
Noninferiority margin

Justification essential for trials using a noninferiority design

Technical Details

Substances

3
Selective serotonin-reuptake inhibitors

A conventional class of antidepressants mentioned.

Serotonin-norepinephrine reuptake inhibitors

A conventional class of antidepressants mentioned.

N-methyl-d-aspartate receptor antagonists

found to cause Olney lesions

Testing Methods

5
Histopathology evaluation

thorough evaluation of at least seven slices of the brain

Hamilton Depression Rating Scale

accepted as primary endpoint in phase 3 studies

Montgomery Asberg Depression Rating Scale

accepted as primary endpoint in phase 3 studies

Children's Depression Rating Scale

accepted as primary endpoint in phase 3 studies

juvenile animal studies

Supplementary juvenile animal studies may be needed

Processes

1
Exposure-response modeling

to integrate information obtained in early phase clinical trials

Clinical Concepts

10
Major Depressive Disorder

The primary disease state addressed by this guidance.; diagnosis should be confirmed via a semi-structured interview; low prevalence of MDD in this age range

Treatment-resistant depression

A specific clinical condition and potential indication.; patients who have not responded to more than one prior antidepressant

Olney lesions

vacuoles that may precede the onset of permanent injury in the form of neuronal cell death

renal insufficiency

patients with renal insufficiency should be included in trials

cardiac disease

Co-morbid condition in the general population

chronic pain

patients with chronic pain should be included in trials

hepatic impairment

patients with hepatic impairment should be included in trials

human immunodeficiency virus

patients with human immunodeficiency virus should not be excluded

hepatitis C

patients with hepatitis C should not be excluded

substance abuse

Patients with a history of substance abuse should also be considered

Standards & References

External Standards

1
Diagnostic and Statistical Manual of Mental Disorders

Used to define symptoms of MDD.

ICH References (4)

ICH E9

Statistical Principles for Clinical Trials; Discourages deterministic procedures due to high risk of bias; Notes that the use of Bayesian methods in clinical trials may be considered.

ICH E10

Choice of Control Group in Clinical Trials.

ICH M3(R2)

Guidance on when particular studies can be abbreviated or deferred for life-threatening diseases

ICH E1A

The Extent of Population Exposure to Assess Clinical Safety: For Drugs Intended for Long-term Treatment of Non-Life-Threatening Conditions

Related MFDS Guidelines

Korean regulatory guidelines covering similar topics

See Also (8)