Track 1: Optimizing Clinical Trials
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THURSDAY, SEPTEMBER 24
11:30-12:00 Safety and Efficacy Considerations for Biomarkers in Retrospective Analysis of Completed Clinical Trials
Robert L. Becker, Jr., M.D., Ph.D., Chief Medical Officer, Office of In Vitro Diagnostic Device Evaluation and Safety, Center for Devices and Radiological Health, U.S. Food and Drug Administration
Personalized medicine ties safe and efficacious use of drugs to performance of diagnostic tests. For biomarkers recognized late in drug development, re-examining cases and samples collected in completed trials is attractive for speedy clinical validation of drug/marker combinations. This approach presents risks for bias that need to be controlled. The level of evidence needed for safety considerations may vary from the level needed for efficacy claims. In any event, reliable performance characteristics for the diagnostic test are essential, and best assured with thorough regulatory review of the test.
12:00-12:30 Translational Medicine Strategies in Musculoskeletal Diseases: On a Quest to Improve Predictability
Salvatore Alesci, M.D., Ph.D., Director, Discovery Translational Medicine, Wyeth Research
The development of innovative drugs that address unmet medical needs in Musculoskeletal Disorders is often halted by the heterogeneity of the patient populations, and in particular lack of biomarkers predictive of disease progression and likelihood of response/resistance to treatment. This presentation will provide an overview of translational medicine strategies and research implemented to address these issues and advance drug development in this area.
12:30-12:45 A Universal Predictor of Drug Response
Steen Knudsen, Ph.D., Chief Science Officer, Medical Prognosis Institute
A universal predictor of drug response is presented. It is based on measuring the gene expression in cell lines treated with the drug. Response predictors for more than 50 drugs have been developed and several of these predictors have been tested clinically. Clinical results from AML and other diseases will be shown, as well comparisons of predicted and observed response rates in clinical trials of drugs in development.
12:45-2:00 Lunch on your own
2:00-2:30 Use of Biomarkers and Translational Science to Accelerate and Improve Oncology Drug Development: Opportunities and Roadblocks
J. Carl Barrett, Ph.D., Vice President and Global Head, Oncology Biomarkers and Imaging, Oncology Translational Medicine, Novartis Institutes of BioMedical Sciences, Inc.
The steps in oncology drug development in patients include: optimizing dose-schedule, predicting patients that will respond, detecting tumor responses rapidly for proof-of-concept trials, using surrogate endpoints for disease monitoring, assuring safety of drug therapy, and developing rational-based combination therapies. Biomarkers are pivotal in meeting each of these challenges. A general strategy for using biomarkers in oncology drug development will be presented and includes: having a systematic biomarker plan for each new agent that is consistent, science-based and focused using common standards for assays and data; building a biomarker tool kit with analytically and clinically validated biomarker assays; building on clinical experience (positive and negative) and execution excellence involving a team effort (physicians, clinical staff, biomarker experts and data management) and building a strong partnership between Novartis and its clinical investigators.
2:30-3:00 Enabling Personalized Medicine through Application of Biomarkers in Clinical Development
Nicholas C. Dracopoli, Ph.D., Vice President, Biomarkers, Centocor Research & Development, Johnson & Johnson
The observer effect describes the changes that the act of observation will make on the phenomenon being observed and has many applications in the physical and experimental sciences. In drug development, if we consider biomarkers as the observer and the clinical trial as the phenomenon, we can ask how the process of analyzing biomarkers impacts the clinical trial process. It is clear that the simple act of collecting biopsies, let alone completing complex bioanalytical studies of these samples, impacts the ability to run clinical trials quickly and economically. Consequently, it is necessary to demonstrate that the value derived from the observation exceeds the cost to the phenomenon. This presentation will discuss how different types of biomarkers can be used during the drug development process to increase probability of success in the successive stages of drug discovery and development, and support decisions for further investment in subsequent development phases. Several examples of biomarker applications to confi rm mechanism of action, explore PK/PD interactions and to derive predictive markers in ongoing drug development programs will be described.
3:00-4:00 Networking Refreshment Break with Poster and Exhibit Viewing
(Shared Session with Targeted Therapy meeting)
Chairperson's Opening Remarks
Bruce Quinn, M.D., Ph.D., Senior Health Policy Specialist, Foley Hoag; former California Medical Director, NHIC
4:00-4:30 The Potential Impact of Recently Approved and Emerging Molecular Diagnostics in Drug-Diagnostics Co-Development
Francis Kalush, Ph.D., Network Leader, Diagnostics and Personalized Medicine, Office of the Center Director, Center for Devices and Radiological Health, U.S. Food and Drug Administration
The FDA under its Critical Path Initiative is leading several efforts to streamline regulatory pathways in Personalized Medicine. An overview of the strategies and impact of recently and emerging molecular diagnostic biomarkers in companion drug-diagnostics will be discussed.
4:30-5:00 Biomarkers - Driven Drug and Diagnostic Co-Development in Oncology: Current Trends and Future Approaches
Miro Venturi, Ph.D., Senior Biomarker & Experimental Medicine Leader, Roche Pharma Development, Roche Diagnostics GmbH
Nowadays a combination of novel and established molecular tools with biomarker analysis embedded on most of the trials in clinical development are offering huge opportunities to embark as early as possible in co-development of the drug and its associated diagnostic test. As the approach evolves and refines, both the private and the public sector build the mission to cooperate together to make personalized healthcare a reality. We will review some of the modern approaches to develop oncology companion diagnostics, critically shed light into the necessary analytical and clinical steps and provide some thinking for the future geared to improve the lives of cancer patients.
5:00-5:30 Challenges of Integrating Targeted Biomarker Tests into Clinical Practice
Walter Carney, Ph.D., Head, Oncogene Science, Diagnostics, Siemens Healthcare Dx
For HER-2/neu Positive breast cancer patients, the availability of HER-2 targeted therapies is becoming increasingly important. The challenge that exists is to select the patients who will benefit most from these therapies, as well as correctly identify who is eligible and who is not eligible for HER-2/neu targeted therapies. Unfortunately, not all HER-2/neu tests are equivalent, thus leading to uncertainty in the HER-2 status of many patients. Therefore it is critical that the HER-2 status be accurately determined which is not always the case leading to some patients not having access to these valuable new therapies.
5:30 Close of Day