Innovative Trial Design Work Highlighted in CDER Report
A new CDER Office of New Drugs (OND) annual report highlights some of its work with sponsors to advance innovative clinical trial designs. “These innovations enabled the launch of new drug development programs, the inclusion of previously under-represented patient populations in clinical trials that might otherwise have been impracticable, the use of real-world data to inform new trial designs and support the approval of treatments, guidance on clinical trials for transdermal delivery of drugs, and guidance on consumer behavior studies to expand the nonprescription drug market, to name a few,” the OND report says.
For example, CDER advised sponsors on innovative trial designs to develop therapies for serious diseases like systemic lupus erythematosus (SLE), an autoimmune disease in which the immune system attacks its own tissues, causing widespread inflammation and tissue damage in the affected organs. It “encouraged drug development by actively advising sponsors on developing both master protocols and platform clinical trials for SLE,” it says. “Master protocol trials have multiple sub-studies that may have different objectives and evaluate one or more drugs or diseases. Platform trials are clinical trials with a single master protocol that evaluates multiple treatments simultaneously. These trial designs are innovative because they can streamline clinical trial operations, enable early decision-making about promising therapeutic candidates for further development, and reduce drug development time and cost.”
The report also addresses innovations in using data to develop new trial designs and treatments, such as data collected through electronic health records, mobile health technology, and other electronic data-capture technology. “This type of data can be used with new trial designs and can streamline and improve the efficiency and outcomes of clinical studies,” it says. For example, observational data led to the first FDA approval of an immunosuppressant drug to prevent lung transplant rejection — Astellas Pharma’s Prograf (tacrolimus). Additionally, new data uses improved screening for risk factors for sudden cardiac death in the young (SCDY), the report notes. A pilot study was launched in response to the lack of accurate and reliable screening methods to identify underlying risks for SCDY. The pilot study “collects high-quality, real-world data obtained by lay public groups, then applies an iterative approach to improve data quality, and enables researchers to establish best practices to prevent SCDY,” it says.
On the drug safety side, OND is helping develop an International Council for Harmonization guideline, “ICH E19: A Selective Approach to Safety Data Collection in Specific Late-Stage Pre-Approval or Post-Approval Clinical Trials.” The document is intended to advise industry organizations on collecting human data in clinical trials. “Once finalized, ICH E19 will significantly impact global drug development by facilitating large-scale efficacy and safety clinical trials with many participants and long-term follow-up,” the report says. “Such clinical trials have proven very important in evaluating medicines, improving their appropriate use, and supporting public health outcomes.”