Navigating care
The short answer on asking for an experimental drug
Through your doctor, with a short clinical brief, starting with the company's Medical Affairs team. The FDA lets almost every single-patient request go forward. The hard part is getting the company to agree to supply the drug. Make it easy for them to say yes, stay polite and persistent, and pursue other options at the same time.
What to know about asking for an experimental drug
- The FDA is rarely the obstacle. It says it allows over 99% of single-patient expanded access requests to proceed.
- The company decides. No pathway requires a company to provide its drug.
- Your doctor files the request, using FDA Form 3926, once the company agrees.
- Preparation beats pressure. A one-page clinical brief and a doctor-to-doctor call do more than an emotional appeal.
- Expect a long shot, and run this alongside other options rather than instead of them.
What are the three main pathways to an experimental cancer drug?
Expanded access, also called compassionate use. The FDA describes it as a potential pathway for a patient with a serious or immediately life-threatening condition to use an investigational drug outside a clinical trial, when there's no comparable alternative and joining a trial isn't possible. For a single patient, the treating physician submits FDA Form 3926. In an emergency, the request can be made by phone. Otherwise, treatment can start 30 days after the FDA receives it, or sooner if the FDA says so. An ethics board (IRB) approval and informed consent are required before treatment.
The FDA states that it allows over 99% of single-patient requests to proceed. A 2017 Government Accountability Office review found the same: 99% of requests allowed from 2012 to 2015, with emergency requests usually answered in under a day.
Right to Try. A 2018 federal law lets eligible patients ask a company for certain investigational drugs without FDA review of the request. According to the FDA, the patient must have a life-threatening condition, have exhausted approved options and be unable to join a trial, and the drug must have completed a Phase 1 trial and still be in active development. The FDA is clear that the law does not require a company to provide its drug.
Joining a trial, including new arms or expansion groups. This is often easier for a company because it uses infrastructure it already runs.
Across all three, the bottleneck is usually the company's willingness to supply.
How do you prepare before asking a drug company for an experimental drug?
Before any email or post, Future of Cancer Care Today suggests having these in place:
A doctor who has agreed to file the request on Form 3926.
A one-page clinical brief: diagnosis, prior treatments, current status and the specific scientific reason this drug makes sense.
Confirmation that no trial of the drug is open to you, or a clear reason a trial isn't feasible.
A check of the company's expanded access policy on the Reagan-Udall Foundation's Expanded Access Navigator. Many smaller companies haven't posted one, so no policy doesn't mean no program.
The right first contact. Medical Affairs is usually a better place to start than the CEO.
How do you contact a drug company about compassionate use?
Lead with the clinical brief, not the story. A short cover note and a one-page brief carry more weight than a long emotional letter, and they're easy to forward internally.
Write to Medical Affairs and the Chief Medical Officer. Copy the CEO; don't start there.
Use the company's own science. Frame the request as fitting the drug's intended patients, not as an exception to them.
Ask your doctor to call the company's medical director. The guide calls a doctor-to-doctor call one of the most reliably effective steps. It can move a case further in fifteen minutes than weeks of email.
For doctors new to this, the FDA's Project Facilitate is a call center that helps oncology providers submit single-patient expanded access requests.
What can you do when a drug company says no to compassionate use?
A published "no" isn't always final, and the wording matters. "We don't currently have a program" may still allow case-by-case requests. "Not at this stage of development" can change after major trial results or as approval nears. "Trials only" is firmer but not always absolute. "Under no circumstances" is the rare true dead end.
What has sometimes changed a no, according to the guide: a doctor's call that raises a clinical angle the company hadn't seen, offering to pay for the drug where that's allowed, an endorsement from a recognized expert, timing the request to a trial milestone, and access through international partners. What doesn't: social pressure alone, repeating the same request, threats, or asking the FDA to step in. The FDA can authorize use, but it can't make a company supply a drug.
What can you do if a compassionate use request stalls?
After two or three weeks without a real response, the guide suggests a steady, not escalating, approach: a short, factual LinkedIn post that names the drug and the scientific rationale without accusation; respectful engagement on the company's own posts; an endorsement from your doctor or an expert; contacting the lead investigator of the closest trial; and asking about access through partners in Australia, the EU, the UK or Israel. Press coverage comes last, only after direct outreach is exhausted.
What tends to backfire: petitions against the company, personal attacks, threats, going to the press first and fast-escalating campaigns. Escalation tends to bring in lawyers and communications teams instead of doctors.
Questions about experimental drugs to talk through with your oncologist
- Is there an investigational drug that fits my cancer's biology?
- Is any trial of it open to me, or likely to open soon?
- Would you file a single-patient expanded access request on Form 3926?
- Would you call the company's medical director yourself?
- Can you help me write a one-page clinical brief?
- What other options should we pursue at the same time?
From the Toolkit
- Questions about treatment and clinical trials Printable
- Guides for complicated care Toolkit
- Building your care team Guide
Sources
Future of Cancer Care Today, Sijbrandij Foundation, Reaching Drug Companies for Investigational Therapies: A guide for patients and families (2026)
U.S. Food and Drug Administration, Expanded Access
U.S. Food and Drug Administration, Physicians: How to Request Single Patient Expanded Access
U.S. Food and Drug Administration, Right to Try
U.S. Food and Drug Administration, Project Facilitate
U.S. Government Accountability Office, Investigational New Drugs: FDA Has Taken Steps to Improve the Expanded Access Program (GAO-17-564, 2017)
Reagan-Udall Foundation, Expanded Access Navigator
Babette is an independent media and education company, not a medical provider. This article explains common experiences and options; it is not advice for your situation. All decisions about your care belong to you and your doctor.
