Compassionate Use & Expanded Access: A Parent’s Guide

What Is Compassionate Use / Expanded Access?

Compassionate Use (officially called Expanded Access by the FDA) is a pathway that allows people with serious or life-threatening diseases to access an investigational drug — one that has not yet been approved by the FDA — outside of a clinical trial, when no comparable or satisfactory approved treatment exists.

The FDA now prefers the term “Expanded Access,” but the terms are used interchangeably. For rare diseases like KCNQ2-associated Developmental and Epileptic Encephalopathy (KCNQ2-DEE), where only a handful of experimental drugs may exist, and clinical trials have strict eligibility criteria, this pathway may be a family’s only remaining option.

Key Definitions

IND — Investigational New Drug

An IND is an application submitted to the FDA that, if approved, allows a drug that has not yet been approved to be used in humans. Before any human testing of a new drug can begin in the U.S., the company (or researcher) must file an IND with the FDA. It is essentially the FDA’s legal permission for the drug to “cross state lines” and be administered to people. An IND application contains:

• The drug’s chemical composition and manufacturing information
• Animal study data showing it is reasonably safe to test in humans
• Clinical protocols (how the drug will be given, to whom, and how safety will be monitored)
• Investigator information

sIND — Single Patient Investigational New Drug

An sIND (also called a Single Patient IND, Individual Expanded Access IND, or Compassionate Use IND) is a special, individualized IND filed for one specific person. Rather than enrolling in a clinical trial with restrictive eligibility criteria, a physician submits a request to the FDA on behalf of an individual who needs the drug for treatment. The FDA reviews it and, if approved, grants permission for that individual only to receive the investigational drug under the supervision of a doctor.

A sIND:

• Is generally limited to a single course of therapy for a specified timeframe
• Requires FDA approval prior to each use (unless it is an emergency)
• Can be extended if the FDA expressly authorizes ongoing or chronic therapy
• Is not considered formal clinical research, but still requires oversight

The Full Process: Step by Step

STEP 1 — The Parent/Family: Recognize the Need & Do Initial Research

• Confirm with your child’s physician that all currently approved treatments have been tried or are not appropriate
• Research whether any investigational drugs are in clinical trials for your child’s condition (search ClinicalTrials.gov)
• Connect with disease-specific foundations (e.g., KCNQ2 Cure Alliance) — they often track which companies have relevant drugs in development and whether expanded access has been granted before
• Confirm your child cannot enroll in any existing clinical trial (due to age, geography, disease severity, or other eligibility barriers)

Important: Only a licensed treating physician, not a parent, can formally submit a compassionate use request on a patient’s behalf. However, parents can and should advocate, research, and initiate conversations.

STEP 2 — The Physician: Evaluate and Agree to Be the Sponsor-Investigator

The treating physician (typically a specialist like a neurologist or epileptologist for KCNQ2-related disorders) must:

• Confirm the person has a serious or immediately life-threatening condition
• Confirm no comparable or satisfactory alternative therapy exists
• Agree to take on the legal responsibilities of the Sponsor-Investigator, meaning they will oversee the treatment, monitor for adverse events, and report to the FDA
• Assess whether the potential benefits justify the known and unknown risks

Not all physicians are willing or able to take on these responsibilities. A candid conversation between the parent and physician is the essential first step.

STEP 3 — Contact the Drug Manufacturer 

This is often the hardest step and the biggest potential roadblock.

The physician (with the family’s support and advocacy) must contact the company that manufactures the investigational drug. Without the company’s agreement to supply the drug, the process cannot move forward.

How to find the right contact:

• Review the Reagan-Udall Foundation’s “Expanded Access Navigator“ — a directory of company contacts and compassionate use programs
• Check ClinicalTrials.gov to find the sponsor of relevant trials
• Contact the disease foundation directly — they may have existing relationships with relevant contacts

What you are asking the company:

• Whether the drug is available for compassionate use
• Whether your patient meets any eligibility criteria they have established
• Whether they will provide the drug (and at what cost, if any)
• Whether they will authorize the physician to submit a single patient IND to the FDA

Why companies may deny the request:

• The patient doesn’t meet their criteria
• The company cannot manufacture enough supply
• They have concerns about safety data interfering with their ongoing clinical trials
• Regulatory or liability concerns

STEP 4 — The Physician Submits to the FDA

Once the company agrees, the physician submits a Single Patient IND Request to the appropriate FDA review division (e.g., the Division of Neurology Products for epilepsy drugs). The submission includes:

1. Statement at the top: “Request for Single Patient IND for Compassionate Use”
2. Brief Clinical History: diagnosis, disease status, prior therapies tried, response to prior therapy, and rationale for requesting this drug
3. Proposed Treatment Plan: dose, route of administration, planned duration, monitoring procedures, and modifications for toxicity
4. Informed Consent Statement: confirming that informed consent will be obtained
5. Physician CV: demonstrating expertise to oversee treatment
6. Letter of Authorization (LOA) from the drug manufacturer authorizing the physician to cross-reference their existing IND
7. The IND number of the company’s existing IND for the drug

The FDA submission and IRB review (see Step 5) are typically submitted simultaneously to save time.

FDA Review Timeline:

• The FDA has 30 days to respond to a non-emergency single patient IND
• For immediately life-threatening emergencies, the FDA can provide verbal authorization within hours, with a follow-up written IND submission within 15 days
• Approximately 99% of expanded access applications are approved by the FDA; ~75% are approved within just a few days

STEP 5 — IRB Review at the Hospital

Even though a single-patient IND is not formal clinical research, it still requires oversight by the hospital’s Institutional Review Board (IRB) — the ethics committee that reviews any use of investigational treatments in human subjects.

What the IRB reviews:

• The treatment protocol (dose, duration, monitoring plan)
• The informed consent document
• The physician’s qualifications
• The risk/benefit assessment

For non-emergency use: IRB approval must be obtained before treatment begins. Submission to the FDA and IRB typically happens simultaneously.

For emergency use: Treatment can begin, but the IRB must be notified within 5 working days.

What the hospital’s research/clinical trials office does:

• Many major hospitals have a research office (sometimes called an Office of Sponsored Research or Human Research Protection Office) that manages the administrative submission process via electronic systems (such as PittPRO at Pitt and eIRB at Penn).
• They help the physician prepare and file the correct documentation
• Some IRBs (like WCG, a specialized IRB) offer free, expedited expanded access review for patients and providers

STEP 6 — Informed Consent

Once the FDA and IRB approvals are in place (or simultaneously, depending on the hospital), the family must provide written informed consent, acknowledging:

• The drug is investigational and has not been proven safe and effective for this specific use
• All known potential risks and benefits
• That participation is voluntary
• What monitoring will be required during treatment

STEP 7 — Treatment Begins

The drug manufacturer ships the drug to the hospital or pharmacy. The physician administers or prescribes it in accordance with the approved treatment plan.

During treatment, the physician must:

• Monitor the patient for adverse events
• Report any serious adverse events to the FDA and the drug manufacturer
• Submit annual IND reports to the FDA if treatment continues long-term
• Comply with all conditions set by the FDA in their approval

Who Pays for What?

This is one of the most frustrating and confusing aspects of compassionate use.

The Drug Itself

• Most pharmaceutical and biotech companies provide the investigational drug for free under expanded access — this is the current norm
• Companies are permitted to charge patients for the drug’s manufacturing cost, shipping, and in some cases, data collection/monitoring costs, but must submit their pricing justification to the FDA before doing so
• In rare pediatric cases (like KCNQ2-related disorders), drug manufacturers typically provide the drug at no charge

Associated Medical Costs (Clinic Visits, Lab Tests, Imaging, Monitoring)

• Insurance (private or Medicaid/CHIP) may or may not cover associated treatment costs — most private insurers do not cover investigational treatments
• The hospital or clinic may absorb some costs
• The drug manufacturer  may cover some monitoring costs (varies by company and agreement)
• Patient/family may be responsible for some out-of-pocket costs
• It is often unclear upfront exactly who will cover these costs — families should ask explicitly before proceeding

Right to Try Act (Alternative Pathway)

The 2018 Right to Try Act is a separate, parallel pathway authorized by Congress that bypasses FDA review and allows patients and their physicians to work directly with the drug manufacturer. It requires:

• That the person’s condition be considered life-threatening
• The drug has completed Phase 1 clinical trials
• The patient has exhausted approved treatments
• The patient is not able to participate in clinical trials
• The manufacturer agrees to supply the drug

However, Right to Try also bypasses the FDA’s oversight and patient protections, and many companies still prefer the Expanded Access route for liability reasons. More than 40 states also have their own Right to Try laws, which are also worth investigating.

Summary Table: Key Players and Their Roles

Sources

Here are all the sources used to research and build the guide, organized by category:

FDA (Official Government Sources)

• FDA Expanded Access — Information for Patients
• FDA Expanded Access — Information for Physicians
• FDA — Physician Request for a Single Patient IND (Compassionate or Emergency Use)

Reagan-Udall Foundation (Expanded Access Navigator)

• Navigator homepage (company directory + physician tools)
• Reagan-Udall Foundation main site

Hospital / IRB Guidance

• University of Pittsburgh HRPO — Single Patient Expanded Access
• Penn IRB — Treatment Use of Investigational Agent
• Cottage Health — Guidelines for Compassionate Use of Investigational Agents (PDF)
• OSF HealthCare — FDA Expanded Access Program
• WCG IRB (free expedited expanded access review)

Patient-Facing Resources

• NYU Langone CUPA — Compassionate Use FAQ (also available in Spanish)
• American Cancer Society — Compassionate Use explainer
• Early Access Care — Compassionate Use and Expanded Access Programs
• MIB Agents — Compassionate Use of Investigational Drugs

Scientific / Regulatory Literature

• NIH/PMC — Efficacy and Safety of Compassionate Use for Rare Diseases (scoping review 1991–2022)
• NIH/PMC — Expanded Access vs. Right to Try
• Wikipedia — Expanded Access (overview with history)
• Facet Life Sciences — Expanded Access / Compassionate Use: FDA Guidelines & Key Facts
• Clinical Trial Vanguard — Navigating Compassionate Use

KCNQ2-Specific Sources

• KCNQ2 Cure Alliance — Expanded Access of Kv7.2 Channel Activators
• ClinicalTrials.gov — Xenon XEN496 (EPIK trial for KCNQ2-DEE)
• Epilepsy Foundation Australia — KCNQ2 overview

Guide compiled June 2026