Advancing Adaptive Design and Personalized Medicine in CNS Trials: Clinical, Regulatory and Statistical Viewpoints
July 23, 2009 10:07 AM Central Time
SAN DIEGO–(BUSINESS WIRE)–Ever-increasing costs of developing medicines over the past decade necessitate drug development methods that allow for faster, more efficient decision making based on informed utilization of emerging, cumulative data to mitigate this worrisome trend. One approach to this challenge is the application of adaptive trial designs to the drug development process. Pharmaceutical companies, and regulatory agencies around the world, are increasingly interested in the appropriate utilization of adaptive design methods. Though these methods are being used more extensively in certain therapeutic areas, such as oncology and cardiovascular diseases, the application in disorders of the central nervous system is less apparent.
Realizing the need to examine and discuss the application of adaptive design for more efficient conduct of CNS trials, the International Society for CNS Clinical Trials and Methodology (ISCTM) will gather key stakeholders from Academia, Industry and Regulatory agencies, 5-6 October 2009, in San Diego. Co-Chairs Donald Berry, Ph.D., Andrew C. Leon, Ph.D., with a team of experts (Conference Presenter Profiles) will present sessions ranging from an introduction to concepts of adaptation to how trials with pre-specified design modification might expedite the regulatory approval process for specific indications. Target areas for adaptation to be considered include modification of inclusion/exclusion criteria, sample size, number of treatment cells, and treatment duration. There will be a focus on methods to control the rate of false positive results and other safeguards, such as blinding of investigators and comparison of results pre and post modifications. Updates on guidance from regulators in the US, Europe and Japan will also be presented. Interactive CNS case study practical application sessions will highlight logistics and pragmatics of the application of adaptive design. (Detailed Agenda)
“This important conference, sponsored by the ISCTM, brings together some of the foremost experts on adaptive design methodologies, and, along with clinicians and scientists from academia, industry and government, will help to advance the application of these innovative strategies to CNS drug development.” -Andrew C. Leon, PhD, Professor of Biostatistics in Psychiatry and Professor of Public Health/Weill Cornell Medical College.
Second day of conference will address the opportunities and challenges of advancing personalized medicine for disorders of the central nervous system. The sequencing of the human genome has informed more tailored approaches to certain diseases. However, there are significant hurdles which must be overcome to realize its full potential. This session will show how clinical trials can be adapted to help realize the value of personalized medicine for CNS conditions. Selection of viable indications, study questions, study populations, endpoints and optimal designs will be covered. Approaches to statistical and regulatory challenges will be addressed by international experts in the field. Examples of work that is already being developed according to this new paradigm will be presented.
In addition the October meeting of ISCTM will host workshops on specific methodological challenges in the areas of negative symptoms of schizophrenia, suicidal thinking and behavior, placebo response and conducting large clinical trials with specific participatory workshops led by leading trialists in each of these areas.
The International Society for CNS Clinical Trials and Methodology (ISCTM) is a multi-disciplinary, independent organization devoted to addressing strategic clinical, regulatory, and methodological challenges that arise in the development and clinical use of CNS therapeutic agents through partnership with industry, academia, governmental and non-governmental agencies and the public.
Contacts
ISCTM
Carlotta McKee, 615-383-7688
Executive Director
isctm.org