The FDA published a final rule, Annual Summary Reporting Requirements Under the Right to Try Act, which specifies the deadline and content for annual reports by sponsors or manufacturers who provide an eligible investigational drug under the Right to Try Act. Manufacturers or sponsors who have participated in the Right to Try program should submit annual reports to RTTAnnualReports@fda.hhs.gov. Sponsors may use the Right to Try annual reporting form.
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:
An eligible investigational drug is an investigational drug:
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:
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?
A: A Right to Try annual reporting form is available. Submit annual reports to RTTAnnualReports@fda.hhs.gov by March 31 annually as detailed in the final rule.