Pharmaceutical companies that have provided investigational drugs to patients under the Right to Try law should be prepared to comply with the U.S. Food and Drug Administration’s (FDA’s) reporting requirements by March 31, 2023.
These requirements, which the FDA adopted in a final rule published on September 14, 2022 (the “Final Rule”),[1] implement certain of the provisions added by the Right to Try law to the Federal Food, Drug, and Cosmetic Act (the “FD&C Act”).
What Is the Right to Try Law?
The Right to Try law, which was enacted on May 30, 2018,[2] amended the FD&C Act to create a new section 561B (21 U.S.C. 360bbb-0a). The Right to Try law established a new pathway—as an alternative to FDA’s Expanded Access program—for patients to request and manufacturers or sponsors to choose to provide access to certain unapproved, investigational drugs, including biological products, when the patient has been diagnosed with a life-threatening disease or condition and, as certified by a physician, has exhausted approved treatment options and is unable to participate in a clinical trial involving the investigational drug. Importantly, neither the Right to Try pathway nor Expanded Access requires manufacturers to make their investigational drugs available to patients. However, unlike Expanded Access, the Right to Try pathway does not require (i) the parties to obtain the FDA’s approval or (ii) oversight by an institutional review board.
Per section 561B of the FD&C Act, the FDA was required to specify, through regulation, the deadline for submissions of an annual summary of investigational drugs supplied by a manufacturer or sponsor under the Right to Try law.[3] The Final Rule specified that deadline and provided additional information on the necessary contents of the annual summary.
Who Is Required to File the Annual Summary Report?
A manufacturer or sponsor that has provided its investigational drug under the Right to Try pathway is required to submit to the FDA an annual summary of such use. The FDA defines the term “manufacturer or sponsor” as the person who (i) is responsible for, and initiates a clinical investigation of, the investigational drug and otherwise meets the definition of a “sponsor” under 21 C.F.R. § 312.3;[4] (ii) has submitted an application for the investigational drug under section 505(b) of the FD&C Act; or (iii) is producing the investigational drug that is being provided, excluding, however, contract manufacturers.[5] The FDA has clarified that the annual reporting obligation does not apply to the certifying physician or the physician treating a patient under the Right to Try law, and at this point in time, the FDA is not requiring any information about the treating physician as part of the annual summary report.[6]
What Information Is Required in the Annual Summary Report?
According to the Final Rule, the annual summary report should contain six specific elements:[7] (1) the name of the investigational drug; (2) the investigational new drug (IND) number;[8] (3) the total number of doses supplied; (4) the total number of patients treated; (5) the disease(s) or condition(s) that the drug is intended to treat; and (6) information on serious adverse events, including the outcomes.[9] In addition to the six required elements, additional information (e.g., the time between doses received if multiple doses were supplied to a patient, demographic information about patients, and other data about patients receiving the investigational drug) may be reported as optional information.
How Is This First Annual Reporting Period Unique?
Because this is the first year since 2018 that manufacturers and sponsors have been required to file the annual summary report, the report due on March 31, 2023, must include a list of all the investigational drugs supplied by the manufacturer or sponsor under the Right to Try law from May 30, 2018, to December 31, 2022.[10] Manufacturers and sponsors should ensure that they have gathered and organized the required information for this three-and-a-half-year period.
For 2024 and subsequent years, manufacturers and sponsors will be required to submit an annual summary report for the investigational drugs supplied under the Right to Try law from January 1 to December 31 of that year, by March 31 of the following year.
How Can Manufacturers and Sponsors Submit the Annual Summary Reports?
Manufacturers and sponsors should attach a completed version of Form FDA 5023 to an email and send it to the following email address: RTTAnnualReports@fda.hhs.gov.
Where Can Manufacturers and Sponsors Find More Information?
The FDA has published on its “Right to Try” web page a set of FAQs.
To date, there has been little public information regarding the use of the Right to Try pathway or patients who have received investigational drugs through this pathway. However, both the Final Rule and the implementation of the annual reporting requirement are expected to provide insight into the Right to Try law’s reach and use thus far.
Epstein Becker Green is available to assist with any questions related to the annual reporting requirement under the Right to Try Act.
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This Insight was authored by Melissa D’Arcy, Marylana Saadeh Helou, and Emily Chi Fogler. For additional information about the issues discussed in this Insight, or if you have any other questions or concerns regarding the Right to Try law or the FDA’s Expanded Access program, please contact one of the authors or the Epstein Becker Green Health Care and Life Sciences attorney who regularly handles your legal matters.
Endnotes
[1] Annual Summary Reporting Requirements Under the Right to Try Act, 87 Fed. Reg. 56,269 (Sept. 14, 2022).
[2] Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (Pub. L. 115-176).
[3] Section 561B(d)(1).
[4] 21 C.F.R. § 300.200(a)(5)(i).
[5] 21 C.F.R. § 300.200(a)(5)(iii). See also, the Final Rule at 56,272-56,273.
[6] Id. at 56,273.
[7] Form FDA 5023 contains six separate columns for the required elements, but there are only five items listed in the annual summary requirements under 21 C.F.R. § 300.200(c). The name of the investigational drug and the applicable IND number are combined under 21 C.F.R. § 300.200(c)(1), but they appear in separate columns in Form FDA 5023.
[8] Sponsors may have separate annual reporting requirements under 21 C.F.R. § 312.33 relating to an IND. The FDA confirmed that sponsors may include information about their Right to Try activities in the IND annual report. However, even if a sponsor reports information about their Right to Try activities in the IND annual report with appropriate labeling and in a separate section, the sponsor must still submit the Right to Try annual summary report as well. See the Final Rule at 56,271.
[9] 21 C.F.R. § 300.200(c).
[10] 21 C.F.R. § 300.200(b)(2).
