What Is Compassionate Use?
Compassionate use, officially called expanded access, lets patients with serious or life-threatening conditions try investigational drugs that haven't received FDA approval. It's a pathway for patients who've exhausted standard treatments and can't join a clinical trial.
The FDA created this program because they recognize that some patients can't wait years for a drug to complete the approval process. But there's something crucial to understand upfront: while the FDA usually says yes to these requests, getting the drug company to agree is often the bigger challenge.
Who Can Request Compassionate Use?
You might be eligible if you meet three key criteria: you have a serious or life-threatening condition, no comparable treatment options exist, and you can't participate in a clinical trial. Your doctor must believe the potential benefits outweigh the risks.
But here's the reality check: having a qualifying condition doesn't guarantee access. Drug companies have limited supplies of experimental medications, and they might need to reserve their supply for clinical trials that could help get the drug approved for everyone.
The Request Process
Your doctor starts the process by contacting the drug company. If the company agrees, your doctor submits a formal request to the FDA. The FDA typically responds within days, sometimes within hours for emergency cases.
A few things you should know about timing and costs:
- Insurance rarely covers experimental treatments
- Some companies provide the drug for free, others charge
- The company review process can take weeks or months
- You'll need a doctor willing to handle extensive paperwork
Right to Try: An Alternative Path
The Right to Try Act of 2018 created another way to access experimental treatments. Unlike compassionate use, Right to Try bypasses FDA review. But there's a catch: the drug must have completed Phase 1 trials, and companies still have the right to say no.
Right to Try hasn't led to widespread access so far. Most patients still use the traditional compassionate use pathway, partly because it offers more protections and support from the FDA.
Finding a Doctor Who Knows the System
Here's something that makes a real difference: having a doctor who has handled compassionate use requests before. They know which companies are more likely to approve requests, what documentation strengthens an application, and how to handle the process efficiently.
If your doctor hasn't done this before, consider asking for a referral to someone who has. Major cancer centers often have more experience with these requests than community hospitals.
Getting Help
The process can feel difficult, but you don't have to figure it out alone. Patient advocacy organizations can help you understand your options. The FDA's Division of Drug Information answers questions about expanded access. And groups like Wondrlink Foundation provide free support to help you understand the process.
Remember: a 'no' from one company doesn't mean you're out of options. There might be other experimental treatments, clinical trials, or compassionate use programs that could help. Keep asking questions and seeking information.
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