The Right to Try Act is one way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain investigational treatment options. FDA’s role in implementing the Right to Try Act is limited to receiving and posting certain information submitted to the agency.
For patients with serious or immediately life-threatening diseases or conditions, the FDA remains committed to enhancing access to promising investigational medicines for those unable to access investigational medical products through clinical trials. This is the mission of our expanded access program. The agency is dedicated to these purposes, and it has been for more than three decades.
The Right to Try Act permits/allows eligible patients to have access to eligible investigational drugs.
A patient who is eligible for Right to Try is a patient who has:
- Been diagnosed with a life-threatening disease or condition
- Exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug (this must be certified by a physician who is in good standing with their licensing organization or board and who will not be compensated directly by the manufacturer for certifying)
- Provided, or their legally authorized representative has provided, written informed consent regarding the eligible investigational drug to the treating physician
An eligible investigational drug is an investigational drug:
- For which a Phase 1 clinical trial has been completed
- That has not been approved or licensed by the FDA for any use
- For which an application has been filed with the FDA or is under investigation in a clinical trial that is intended to form the primary basis of a claim of effectiveness in support of FDA approval and is the subject of an active investigational new drug application submitted to the FDA
- Whose active development or production is ongoing, and that has not been discontinued by the manufacturer or placed on clinical hold by the FDA
If you are interested in Right to Try, you should discuss this pathway with your doctor. Companies who develop and make drugs and biologics can provide information about whether their product is considered an eligible investigational drug and whether they are able to provide the product under the Right to Try Act. Ultimately, companies developing drugs for life-threatening diseases or conditions are responsible for determining whether to make their products available to patients who qualify for access under the law.
1. If I’m a patient or a physician and I have a question about the Right to Try Act, who do I ask?
A: FDA recommends that patients first consult with their physician and that physicians consult with the sponsor of the investigational drug or biological product. The sponsor is in the best position to provide information about whether the drug or biological product meets the criteria to be considered an eligible investigational drug for use under the Right to Try Act.
2. If I identify a drug that I would like to use under the Right to Try Act, how do I know if it is an “eligible investigational drug”?
A: FDA recommends that physicians consult with the sponsor of the investigational drug or biological product. The sponsor is in the best position to provide information about whether the drug or biological product meets the criteria to be considered an eligible investigational drug for use under the Right to Try Act.
3. Does FDA review or approve the Right to Try Act requests?
A: FDA does not review or approve requests for Right to Try Act use. FDA’s role is limited to receipt and posting of certain information submitted under the Right to Try Act.
4. What does FDA do with the annual summaries submitted under the Right to Try Act?
A: FDA will receive annual summaries from manufacturers or sponsors on use of an eligible investigational drug under the Right to Try Act. FDA will post a consolidated annual summary report of Right to Try Act use.
5. Does an IRB review and/or approve Right to Try Act requests?
A: Individual Right to Try Act requests do not require IRB review or approval; however, eligible investigational drugs under the Right to Try Act must meet certain criteria.
6. Is written informed consent required for the Right to Try Act?
A: Yes, a physician is responsible for getting written informed consent from the eligible patient or their legally authorized representative.
7. Does the Right to Try Act obligate a sponsor to provide an eligible investigational drug to an eligible patient?
A: No, the Right to Try Act does not require a sponsor provide an eligible investigational drug to an eligible patient.
8. If the IND for a drug is on clinical hold, is the drug an eligible investigational drug for use under the Right to Try Act?
A: No, a drug under IND clinical hold is not an eligible investigational drug and a sponsor cannot provide the drug for use under the Right to Try Act.
9. I am a sponsor or manufacturer who has provided an eligible investigational drug under the Right to Try Act. What are my annual reporting responsibilities?
A: The FDA has published a final rule that specifies the deadline and content for submission of an annual report of eligible investigational drugs supplied under the Right to Try Act. Mandatory reporting includes:
- The name of the eligible investigational drug
- The IND (Investigational New Drug) number
- The number of doses supplied
- The number of patients treated
- The disease or condition that the drug is intended to treat; and
- Information on serious adverse events
10. I am a sponsor or manufacturer who has provided an eligible investigational drug under the Right to Try Act. How should I submit my annual report?
Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Expanded access may be appropriate when all the following apply:
- Patient has a serious or immediate life-threatening disease or condition.
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- Patient enrollment in a clinical trial is not possible.
- Potential patient benefit justifies the potential risks of treatment.
- Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
Investigational drugs, biologics or medical devices have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.
Information for Patients, Physicians and Industry
Learn about expanded access, including information about the expanded access process, what FDA considers, and what costs may be involved.
Learn about expanded access, including information about the different types of expanded access, how to submit expanded access requests, and reporting requirements.
Learn about expanded access, including information about posting your expanded access policy, how to submit expanded access requests, and reporting requirements.
Learn about how to complete and submit forms needed for each type of expanded access request.