Compassionate Use

• Compassionate Use provides a pathway for patients with severe conditions to access unapproved treatments.
• It is typically considered when patients have exhausted all other treatment options and cannot participate in clinical trials.
• The process involves a request from the treating physician, agreement from the drug manufacturer, and regulatory approval.
• The primary goal is to offer potential benefit to patients while minimizing risks and not hindering ongoing clinical development.

What is Compassionate Use?

Compassionate use definition refers to a regulatory pathway allowing patients with serious or immediately life-threatening diseases to access investigational medical products outside of clinical trials. This option is for those who have exhausted all comparable or satisfactory alternative therapies and cannot participate in ongoing clinical research. It balances urgent patient needs with maintaining clinical trial integrity and patient safety, overseen by regulatory bodies like the U.S. Food and Drug Administration (FDA).

How Compassionate Use Programs Function

The functioning of compassionate use programs is a multi-stakeholder process, typically initiated by a patient’s treating physician. If a patient meets the criteria, the physician requests the investigational product from the pharmaceutical company. Upon company agreement, the physician seeks approval from the relevant regulatory authority, such as the FDA. This review ensures potential benefits outweigh risks and does not hinder ongoing clinical trials.

Key steps in the process often include:

• Physician Request: The treating physician identifies a patient who meets the medical criteria and has exhausted other options.
• Company Agreement: The pharmaceutical company evaluates the request, considering factors like drug supply, potential risks, and the impact on clinical trials.
• Regulatory Submission: The physician submits an expanded access application (e.g., an Investigational New Drug (IND) application for individual patient expanded access) to the regulatory agency.
• Institutional Review Board (IRB) Approval: An independent ethics committee reviews the treatment plan to protect the patient’s rights and welfare.

Compassionate use is distinct from clinical trials, which are designed for systematic data collection on safety and efficacy. While compassionate use focuses on individual patient treatment, limited safety data may still be collected to monitor patient progress and adverse events.

Eligibility and Application for Compassionate Use

Eligibility for compassionate use is stringent. The primary compassionate use program requirements dictate that patients must have a serious or immediately life-threatening disease with no comparable or satisfactory alternative therapies. Crucially, patients must also be unable to participate in a clinical trial for the investigational drug due to exclusion criteria, lack of trial access, or other barriers.

The application process is physician-driven and involves several critical steps:

1. Confirm the patient’s severe or life-threatening condition and exhaustion of standard treatments.
2. Determine that the potential benefits of the investigational drug outweigh its risks for the individual.
3. Obtain informed consent from the patient, explaining the experimental nature and potential uncertainties.
4. Secure agreement from the drug manufacturer and submit a comprehensive application to the regulatory authority.

Regulatory authorities review each application case-by-case, considering the patient’s urgent need, available scientific evidence, and impact on the drug’s development. This rigorous process ensures compassionate use remains a carefully managed, last-resort option to protect patient welfare.