EA Legislation: 21st Century Cures and Clinical Trials Registration
Submitting a request for single-patient expanded access requires the involvement of physicians, patients, the Food and Drug Administration (FDA), Institutional Review Boards (IRBs) and pharmaceutical companies. It’s crucial to be aware of and acknowledge the role and perspective of each party during an expanded access request.
Two recent policies have led to changes related to expanded access that affect not only pharmaceutical companies, but physicians and patients, too.
21st Century Cures Act
The 21st Century Cures Act, passed in December 2016, has brought sweeping reforms to expedite the discovery, development, and delivery of new treatments and cures. That law also includes new requirements that pharmaceutical companies publicly provide their policies and procedures for processing requests for expanded access (EA). While companies are not required to offer EA, they are required to provide the following information:
- Contact information for the developer, manufacturer, or distributor
- Procedures for making the EA request
- Anticipated time it will take to acknowledge receipt of a request
- General criteria used to evaluate the request
- A hyperlink or reference to the clinical trial record in ClinicalTrials.gov containing information about EA for their drugs that may be available through the EA request process
It is important to reiterate that while the posting of policies does not serve as a guarantee of access to any specific investigational treatment by any individual patient, transparent and readily accessible policies are extremely helpful to physicians and patients so they can make important medical decisions in a timely manner.
It is also important to understand that companies can post general policies rather than policies specific to each of their investigational treatments. Physicians and patients can locate some of these policies in the EA Navigator Company Directory or on a manufacturer’s website. (Please note that companies voluntarily participate in the Navigator Directory so it may not be comprehensive.)
Before requesting single-patient EA, a physician should review a company’s procedures for EA as they may change or be updated at any time. Pharmaceutical companies generally work with the physician to process a request for EA.
For more information on how to find specific investigational treatments, visit our ClinicalTrials.gov User Guide.
The “Final Rule” on Clinical Trials Registration and Results Information Submission
The National Institutes of Health (NIH) issued a new policy called the “Final Rule” on Clinical Trials Registration and Results Information Submission in September 2016 that clarifies clinical trials registration requirements that apply to Phase 2-4 trials of FDA-regulated drugs and biologics manufactured or exported from the U.S. for study. One provision of the “Final Rule” on Clinical Trials Registration and Results Information Submission requires pharmaceutical companies to include information about the availability of EA by product and the type of EA, including single-patient, when registering an investigational drug clinical trial on ClinicalTrials.gov. As part of the EA record, a hyperlink to the clinical trial record and drug under investigation will be identified clearly. These records must be entered and updated within 30 days of initiating or changing a clinical trial. Additionally, results from applicable clinical trials must be posted in a timely manner on ClinicalTrials.gov.
ClinicalTrials.gov previously included a field for Expanded Access Programs, but not specifically for single-patient EA. Now, this rule requires direct action from pharmaceutical companies to list the availability of single-patient expanded access. These changes help to make this valuable information readily available to physicians, patients, researchers, and other stakeholders. This information is useful for physicians and their patients as they search for ongoing trials and results of past trials, make decisions on which treatments to pursue, and find all of their treatment options.