ensure that patients with serious or life-threatening conditions, who lack therapeutic alternatives, can access investigational drugs outside traditional clinical trials, in compliance with ICH-GCP, WHO, FDA, EMA, CDSCO, and other global regulatory authority requirements.

Scope

This SOP applies to sponsors, investigators, regulatory affairs personnel, and clinical research teams involved in Expanded Access/Compassionate Use submissions. It includes dossier preparation, submission to regulatory authorities and ethics committees, monitoring of patient safety, and documentation requirements.

Responsibilities

• Principal Investigator (PI): Identifies eligible patients and initiates requests for compassionate use.
• Regulatory Affairs Manager: Prepares and submits applications to regulatory agencies and ethics committees.
• Pharmacovigilance Officer: Monitors patient safety and reports adverse events.
• Quality Assurance Officer: Ensures compliance of submissions with applicable regulations.
• Head of Clinical Research: Reviews and approves applications before submission.

Accountability

The Head of Regulatory Affairs is accountable for ensuring compliance with all regulatory requirements for compassionate use submissions. Failure to comply may result in rejection of applications or regulatory sanctions.

Procedure

1. Patient Eligibility Assessment
PI evaluates patient condition to determine eligibility for compassionate use.
Patient must have a serious or life-threatening condition with no satisfactory alternatives.
Obtain informed consent from the patient or legal guardian.

2. Preparation of Submission Dossier
Prepare a comprehensive dossier including patient history, treatment rationale, Investigator’s Brochure, and product safety data.
Include compassionate use protocol, informed consent form, and risk mitigation strategies.
Compile cover letter, application forms, and regulatory checklists.

3. Ethics Committee/IRB Submission
Submit dossier to Ethics Committee (EC) or Institutional Review Board (IRB) for review and approval.
Ensure minutes of approval are documented and archived.

4. Regulatory Authority Submission
Submit application to national regulatory authority (e.g., FDA Expanded Access IND, EMA Compassionate Use Program, CDSCO expanded access submission).
Obtain written authorization prior to product dispensing.

5. Product Supply and Dispensing
Investigational product must be dispensed under controlled conditions.
Maintain product accountability logs and reconcile supply.

6. Safety Monitoring and Reporting
Monitor patients closely for adverse events.
Report SAEs and SUSARs to regulatory authorities within defined timelines.
Document safety follow-up in communication logs.

7. Documentation and Archiving
Maintain all compassionate use documentation in the Trial Master File (TMF).
Retain records for minimum of 5 years post completion.

Abbreviations

• SOP: Standard Operating Procedure
• PI: Principal Investigator
• EC: Ethics Committee
• IRB: Institutional Review Board
• SAE: Serious Adverse Event
• SUSAR: Suspected Unexpected Serious Adverse Reaction
• TMF: Trial Master File
• IND: Investigational New Drug

Documents

1. Compassionate Use Application Form (Annexure-1)
2. Patient Eligibility and Consent Form (Annexure-2)
3. Compassionate Use Communication Log (Annexure-3)

References

• ICH E6(R2) Good Clinical Practice
• US FDA Expanded Access IND Guidance
• EMA Compassionate Use Program Guidelines
• WHO Good Clinical Practices
• CDSCO Clinical Trial Rules, 2019

Version: 1.0

Approval Section

Prepared By	Rajesh Kumar, Regulatory Affairs Specialist
Checked By	Sunita Reddy, QA Officer
Approved By	Dr. Anil Sharma, Head of Clinical Research

Annexures

Annexure-1: Compassionate Use Application Form

Date	Patient ID	Product	Reason for Request	Initiated By
01/09/2025	CU-001	Drug-X	No approved alternative	Dr. Meera Joshi

Annexure-2: Patient Eligibility and Consent Form

Patient ID	Eligibility Criteria	Consent Obtained	Date	Investigator
CU-001	Meets inclusion criteria	Yes	02/09/2025	Dr. Meera Joshi

Annexure-3: Compassionate Use Communication Log

Date	Authority	Query	Response	Responsible Person
05/09/2025	FDA	Provide updated safety profile	Submitted latest IB	Rajesh Kumar

Revision History

Revision Date	Revision No.	Revision Details	Reason for Revision	Approved By
26/08/2025	00	Initial version	New SOP creation	Head, Clinical Research

For more SOPs visit: Pharma SOP