Patients with serious or life-threatening conditions may find themselves with no treatment options once approved therapies are exhausted. Faced with this situation, these patients and their physicians may consider the use of an investigational medicine, such as by enrolling in a clinical trial. If no suitable research trial is available for enrollment, access to investigational drugs may be provided through expanded access.
But the process for navigating expanded access requests can be difficult for patients, physicians, and sponsors alike. On Tuesday, a panel of longstanding leaders of the space discussed the current environment for Expanded Access to investigational drugs, at “Modern Solutions for Pre-Approval Expanded Access.” Jess Rabourn, Managing Director of WideTrial (www.WideTrial.com), raised the possibility that a system could be created in which multiple EA programs could be operated and managed simultaneously. WideTrial is the first organization to serve as a third-party platform for sponsoring and conducting well-designed Expanded Access (EA) programs under license by originating therapeutics manufacturers. They offer centralized expertise and advisory services to drug companies, clinical research centers, and disease foundations. It also offers direct support by way of multi-party collaborations and sponsorship of large-group Expanded Access trials.
Kay Holcombe, Senior Vice President for Science Policy at BIO, encouraged the audience to take advantage of an important new tool to facilitate expanded access from patients and physicians.
The Reagan-Udall Foundation Expanded Access Navigator provides a one-step shop for information and assistance to doctors and patients seeking access to investigational medicines. The Navigator answers questions like “What clinical trials are available? What is expanded access? How can I talk with companies about potentially promising investigational medicines for my disease?”
The Navigator, available at http://navigator.reaganudall.org, also includes information specific to questions doctors may have about their responsibilities when administering an unapproved drug, and their FDA and IRB requirements. Patient-focused information includes defining clinical trials and expanded access, as well as a tutorial on using clinicaltrials.gov, the NIH sponsored website for clinical trials.
The Navigator company directory links users to company expanded access policies, including the company contact point for more information.
Expanded Access policies now required by law – is your company compliant?
Several participants also noted that companies must now publicly post their expanded access policies under a new requirement contained in the 21st Century cures Act, with the goal of ensuring that patients and physicians can easily find information on requesting access to investigational drugs. Companies may fulfill this requirement by posting on their own website, or by submitting their information to the Reagan-Udall Navigator at http://navigator.reaganudall.org/expanded-access-company-directory-submission-form.