Right to Try

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. 

• Now, a manufacturer or sponsor of a CDER-regulated drug or biological product provided under the Right to Try Act  should submit their annual summary through the CDER NextGen Portal (the Portal). Manufacturers or sponsors should continue to use Form FDA 5023, which should be uploaded as part of their submission to the Portal. Manufacturers or sponsors will have to register and sign into the Portal before submitting the Right to Try submission. The CDER NextGen Portal Registration Instructions Video and FDA Alternate Submission for Right to Try Quick Reference Guide will be accessible after registering and signing into the Portal. 
• For CBER-regulated biological products, manufacturers and sponsors should continue to submit annual summaries using Form FDA 5023 to RTTAnnualReports@fda.hhs.gov.
  

FDA Right to Try Fact Sheet

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, 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 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); and
• 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 FDA for any use;
• For which an application has been filed with 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 (IND) submitted to FDA; and
• Whose active development or production is ongoing, and that has not been discontinued by the manufacturer or placed on clinical hold by FDA.

If you are interested in Right to Try, you should discuss this pathway with your doctor. Companies who develop and make drugs and biological products 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.

Right to Try FAQs

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 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 receives annual summaries from manufacturers or sponsors on use of an eligible investigational drug under the Right to Try Act. FDA posts 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 to 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: 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:

1. The name of the eligible investigational drug;
2. The IND number(s);
3. The number of doses supplied;
4. The number of patients treated;
5. The disease or condition that the drug is intended to treat; and
6. 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?

A: Manufacturers or sponsors of a CDER-regulated drug or biological  product provided under the Right to Try Act should  submit their annual report through the CDER NextGen Portal (the Portal). Manufacturers or sponsors should continue to use Form FDA 5023, which should be uploaded to the Portal. For CBER-regulated biological products, manufacturers and sponsors should continue to submit annual reports using Form FDA 5023 to RTTAnnualReports@fda.hhs.gov. Reports for both CDER and CBER-regulated products are due annually by March 31, as detailed in the final rule. 

 

Resources

• Annual Summary Reporting Requirements Under the Right to Try Act
• Right to Try annual reporting form (Form FDA 5023)
• Expanded access program
• CDER NextGen Portal
• CDER NextGen Portal Registration Instructions Video
• CDER NextGen Portal - FDA Alternate Submission for Right to Try Quick Reference Guide (Please register and sign into the Portal to access the Quick Reference Guide.)
• CDER NextGen Portal Frequently Asked Questions (FAQs)

Contact

• FDA is available to answer questions regarding the Right to Try Act. Patients with specific Right to Try requests should consult their physician.
• For investigational drugs, contact druginfo@fda.hhs.gov or 301-796-3400.
• For investigational biological products, contact industry.biologics@fda.hhs.gov or 240-402-8020 or 800-835-4709.
• For annual reporting requirements, contact RTTannualreports@fda.hhs.gov.
• For general questions, or if you are unsure, contact patientaffairs@fda.hhs.gov or 301-796-8460.