FDA proposes updates to expanded access request form to collect racial, ethnicity data

A proposed update to the form used by the FDA to facilitate access to its Expanded Access program would make several changes, among them the optional collection of race and ethnicity data for patients seeking investigational access to products under the program.

Regulatory Background

• Clinical trials used to assess a pharmaceutical product’s safety and efficacy have eligibility criteria for participation. These criteria may exclude patients in certain age groups, persons with specific conditions or patients who are seriously or terminally ill. This is done to protect patients from products that have not yet been proven to be safe and also ensures that an effect caused by an investigational drug is attributable to the product and isn’t caused by the health condition of the patient.
• Unfortunately, these criteria often exclude seriously ill patients who are desperately seeking additional treatment options. Because of the severity of their health conditions, such patients often don’t have the luxury of waiting until a drug is ultimately approved by the FDA. To address this challenge, the FDA’s Expanded Access program (also called “Compassionate Use”) permits patients with serious or life-threatening illnesses to request access to investigational drugs through participating companies.
• Expanded Access is only available for drugs under an active Investigational New Drug application (used to initiate clinical trials). In addition, several other criteria must be met for a drug and patient to be eligible. The patient must have a “serious” or “life-threatening” disease or condition, respectively defined  as “a disease or condition associated with morbidity that has substantial impact on day-to-day functioning,” and a disease where “there is reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment.”
• There are three types of Expanded Access for drugs and biologics: Individual Patient Investigational New Drug (IND) Applications, intended for use with single patients; Intermediate-size Patient Population INDs, allowing investigational drugs to be used for “a patient population smaller than that typical of a treatment IND or treatment protocol”; and Treatment INDs, in which the FDA “may permit an investigational drug to be used for widespread treatment use.” The FDA may also receive Individual Patient INDs on an emergency basis.
• In addition, existing trials may also be amended using a “protocol” amendment, which happens more frequently for larger changes (such as for treatment protocols or intermediate size patient population protocols) but infrequently for individual patients.
• Even if the FDA is willing to grant a request for Expanded Access, companies are not obligated to provide access to their drug under this program. A company may not wish to grant access to its drug if, for example, it doesn’t believe it has sufficient supply of the drug to accommodate both its ongoing clinical trial and additional patients. It may also not wish to incur the added cost of making the drug available, or may have concerns about the safety of the drug in a specific patient.
• The FDA has long allowed the vast majority of requests it receives for expanded access to proceed. In the last 11 years, FDA has on average allowed more than 99% of annual requests.

What’s New

• To obtain Expanded Access for an individual, physicians generally must submit FDA Form FDA 3926, otherwise known as “Individual Patient Expanded Access Investigational New Drug Application.” (They may also file an Investigational New Drug Application, though that process is generally more complex.) The form is relatively short – just three pages in length, of which just two are substantive – and generally basic. It asks for information about the type of submission, the patient, a brief clinical history of the patient, information about the treatment, and the contents of the submission.
• Now the FDA says it wants to change Form FDA 3926 to collect some additional information that might prove helpful. In a Federal Register notice today the FDA said it is proposing to add an optional “race and ethnicity” item to the clinical information field of the form. At present, FDA only asks for the patient’s age, gender, and weight.
• Other changes: FDA also wants to re-order the form to have the physician information be the first piece of information, and include a field to facilitate a “request for withdrawal.”
• Another change is to make some “technological enhancements” to an electronic version of the form. “Currently, certain fields become grayed out if not required for the submission type selected,” FDA explained.
• The additional information could allow the FDA to better understand the types of patients benefiting from its Expanded Access program. Because the form requires an engaged, knowledgeable physician to advocate for the patient, it’s possible that persons benefiting from the program are disproportionately of certain racial or ethnic groups. While submission of the information is optional, the information could be helpful for the FDA to help design other related programs – for example, a form to benefit non-English speaking patients and physicians. Even the Reagan-Udall Foundation, which runs an Expanded Access Navigator program, does not have resources available in any language other than English.

To contact the author of this piece, please email Alec Gaffney ( [email protected]).
To contact the editor of this piece, please email Kari Oakes ( [email protected])

Key Documents and Dates

• Agency Information Collection Activities; Proposals, Submissions, and Approvals: Expanded Access to Investigational Drugs for Treatment Use