Clinical trials are critical to the discovery and development of innovative medicines that enable patients to live longer, healthier lives. As the biopharmaceutical industry works to identify, test and bring new medicines to patients, clinical trials are instrumental in determining the safety and efficacy of a potential new treatment.
While clinical trials are long and complex, innovative biopharmaceutical companies are leveraging the latest scientific and technical advances to streamline the process and create efficiencies. The use of novel trial designs and real-world evidence, for example, is helping to drive these advances, and recently passed legislation is helping to accelerate the uptake of these tools.
As the pace of science continues to evolve, the drug development process – from discovery to approval – remains long and costly. Developing a new medicine takes an average of 10 or more years, and the clinical trial component alone takes nearly six to seven years. Improving clinical trial design and efficiency thus presents an important challenge that deserves attention from all stakeholders.
I recently had a chance to discuss these issues at a public workshop on Evaluating Inclusion and Exclusion Criteria in Clinical Trials organized by the Duke Margolis Center for Health Policy. I believe enhancing use of real-world evidence represents a significant opportunity for clinical trial innovation. Real-world evidence can come from a variety of sources, including observational studies, electronic health records, payer administrative claims or patient registries. These data equip manufacturers with enhanced information to accelerate and advise the drug development process.
The good news is the U.S. Food and Drug Administration (FDA) continues to modernize its regulatory processes for reviewing new medicines and strengthening clinical trials. Bipartisan legislation, such as the Prescription Drug User Fee Act (PDUFA) VI and the 21st Century Cures Act, are helping to create greater efficiencies in the clinical trials process. In particular, these two laws have charged FDA with establishing processes to facilitate the use of novel clinical trial designs, which are critical to ensure clinical trials are as efficient and up-to-date as possible.
Patient participation in the drug development process is also vital to ensuring optimal treatments. Clinical trials are the primary means for involving patients in the drug development process, and trial sponsors place great importance on respecting and protecting the safety of research participants.
Ultimately, by investing PDUFA VI resources into advancing novel clinical trial approaches, we can establish a more competitive, innovative and efficient framework for developing life-saving medicines for a range of unmet medical needs.
Richard Moscicki, M.D. Dr. Moscicki came to PhRMA in 2017 after serving as the Deputy Center Director for Science Operations for the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) since 2013. While at FDA, Dr. Moscicki brought executive direction of Center operations and leadership in overseeing the development, implementation, and direction of CDER’s programs. Previous positions include serving as Chief Medical Officer at Genzyme Corporation from 1992 to 2011, where he was responsible for worldwide global regulatory and pharmacovigilance matters, as well as all aspects of clinical research and medical affairs for the company. He served as the senior vice president and head of Clinical Development at Sanofi-Genzyme from 2011-2013.