Compassionate Use

How Compassionate Use Works

Eligibility Requirements

Criterion	Requirement
Serious condition	Life-threatening or severely debilitating disease
No alternatives	No comparable approved therapy available
Clinical trial access	Unable to enroll in ongoing trials
Benefit-risk	Potential benefit justifies potential risks
No compromise	Won’t interfere with drug development

Types of Expanded Access

Type	Description	Application
Individual patient	Single patient request	Emergency or non-emergency
Intermediate-size	Small group of patients	When no trial is available
Treatment protocol	Widespread use	Near approval, demonstrated efficacy

The Application Process

Individual Patient Access

1. Physician initiates request - Determines patient meets criteria
2. Manufacturer agreement - Company must agree to provide drug
3. FDA authorization - Submit Form 3926 (simplified) or full IND
4. IRB review - Institutional review board approval (can be concurrent)
5. Treatment begins - With ongoing safety monitoring

Timeline Expectations

Request Type	FDA Response Time
Emergency	Within 24 hours (often same day)
Non-emergency individual	30 days (often faster)
Intermediate/treatment	Standard IND timeline

When Compassionate Use Applies

Appropriate Situations

• Terminal illness with no approved options
• Serious condition failing all standard treatments
• Rare disease with no approved therapies
• Patient ineligible for clinical trials (age, comorbidities, location)

Not Appropriate For

• Preference for unapproved over approved treatment
• Cost avoidance for expensive approved drugs
• Enhancement or optimization purposes
• Conditions with adequate approved alternatives

Relevance to Peptide Therapies

Current Landscape

Most peptide therapies in the metabolic space have approved options available, making compassionate use uncommon. However, expanded access may apply for:

Scenario	Example
Novel mechanisms	Investigational peptides in development
Refractory cases	Patients failing all approved GLP-1 agents
Rare conditions	Lipodystrophy, specific genetic disorders
Pediatric patients	When adult-only approval exists

Historical Context

Before GLP-1 agonists gained obesity indications:

• Compassionate use was not typically available (condition not “serious” by FDA standard)
• Off-label prescribing was the access route
• Obesity’s recognition as serious disease changed regulatory landscape

Stakeholder Responsibilities

Physician Responsibilities

Duty	Description
Determine eligibility	Assess patient meets criteria
Obtain informed consent	Explain investigational nature, risks
Submit application	Complete FDA forms with documentation
Monitor patient	Track and report safety data
Report outcomes	Submit required adverse event reports

Manufacturer Responsibilities

Duty	Description
Respond to requests	Timely decision on providing drug
Supply drug	Provide at cost or free (varies)
Provide information	Share available safety data with physician
Support FDA submission	Authorize regulatory filing

FDA Role

Function	Description
Review applications	Assess benefit-risk for specific patient
Authorize access	Grant permission to use investigational product
Ensure safety	Require monitoring and reporting
Protect trials	Ensure expanded access doesn’t compromise development

Cost Considerations

Who Pays

Cost Component	Typical Responsibility
Drug itself	Often provided free by manufacturer
Administration	Patient/insurance
Monitoring/tests	Patient/insurance
Complications	Patient/insurance

Insurance Coverage

• Variable coverage for compassionate use
• May cover medical care but not investigational product
• Prior authorization typically required
• Some states mandate coverage considerations

Ethical Considerations

Balancing Interests

Interest	Consideration
Individual patient	Deserves access to potentially beneficial treatment
Clinical trials	Need enrollment for drug development
Future patients	Benefit from rigorous trial completion
Fair access	Should not favor connected or wealthy patients

Right to Try Laws

Federal and state “Right to Try” laws provide alternative pathway:

• Bypasses FDA for terminal patients
• Requires drug passed Phase 1
• More limited than FDA expanded access
• Less safety reporting required

Frequently Asked Questions

How often does FDA approve compassionate use requests?

FDA authorizes over 99% of compassionate use requests. Denials are rare and typically involve safety concerns or situations where clinical trial enrollment is possible.

Can any physician request compassionate use?

Yes, any licensed physician can submit an expanded access request. However, they must be willing to take responsibility for the patient’s care, monitoring, and required reporting.

Will compassionate use delay drug approval?

Generally no. FDA structured the program to minimize impact on clinical development. However, extensive safety signals from expanded access could affect approval decisions.

What if a company refuses to provide their drug?

Companies are not required to provide drugs for compassionate use. Reasons for refusal include limited supply, ongoing trials needing enrollment, or manufacturing constraints. FDA cannot compel companies to participate.