FDA Advances Drug Development Innovation by Establishing ISTAND as Permanent Qualification Program
By: ShaAvhrée Buckman-Garner, M.D., Ph.D., Director of Office of Translational Sciences, Center for Drug Evaluation and Research (CDER), and Mary Thanh Hai, M.D., Acting Director of Office of New Drugs, CDER
The U.S. Food and Drug Administration recognizes that innovating drug development is an important way to bring new treatments to patients and consumers expeditiously. For this reason, the FDA is excited to announce that our Innovative Science and Technology Approaches for New Drugs (ISTAND) pilot program has transitioned to a permanent drug development tool (DDT) Qualification Program. ISTAND will continue to support innovative, science-driven approaches that improve drug development and regulatory decision-making, ultimately helping to make therapies available to address patients’ unmet needs.
In November 2020, the FDA launched ISTAND to provide a pathway to “qualify” novel DDTs that did not fit within the agency’s existing qualification programs. DDTs are methods, materials, or measures that may help with drug development and may include biomarkers like biological molecules that can signal a condition or disease, or clinical outcome assessments (COAs), which include measurements of a patient’s feelings, ability to function, and survival.
“Qualified” DDTs have a proven, specific use, and can be incorporated in any drug development program for a particular purpose, known as a “context of use.” One example is a biomarker that can identify patients with a certain disease for clinical trial enrollment. By helping with enrollment, this biomarker may allow investigators to complete the drug trial faster and bring the treatment to market, assuming it meets the FDA’s rigorous safety and effectiveness standards.
In its pilot phase, ISTAND helped facilitate the integration of advanced tools, such as organ-on-a-chip models (small devices that mimic the structure and function of a human organ), artificial intelligence (AI)-enabled tools, digital health technologies (e.g., a fitness tracker), and complex in vitro models into regulatory science. Since its inception, ISTAND has accepted eight submissions: three AI-based tools, two tools that assess preclinical safety without using animals, two novel methods involving tissues, and one novel statistical approach. The FDA is now providing feedback on the submissions. The FDA has a database of the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research DDT Qualification programs, including ISTAND, searchable by stage of submission, qualification status, disease, and therapeutic area.
Based on this high number of acceptances, ISTAND will transition to a permanent program. It will remain focused on tools that do not fall within the FDA’s existing qualification pathways, making ISTAND a valuable driver of cutting-edge approaches in drug development. ISTAND will keep offering engagement with tool developers, access to expert regulatory feedback from FDA reviewers, and potential qualification of novel tools for broad use across drug development programs and interested parties—with the ultimate goal of bringing FDA-approved medications to patients.
ISTAND’s activities complement other agency initiatives to expediate drug development, such as our efforts to reduce, replace, and refine the use of animal testing, as described in the FDA’s recently published Roadmap. In addition, we have programs such as the Rare Disease Endpoint Advancement Pilot Program, Model-Informed Drug Development, Complex Innovative Trial Design, and the CDER Center for Clinical Trial Innovation. While these efforts are different in scope, they all support using innovation to further the FDA’s mission of bringing safe, effective treatments to patients and consumers.
For more information about ISTAND or to submit a proposal, visit the ISTAND webpage or contact ISTAND at ISTAND@fda.hhs.gov.
