CONFERENCE SERIES: Clinical Trials & Translational Medicine
Recorded at: ADAPT Congress
Digital Course: Personalized Medicine
Cambridge Healthtech Institute’s ADAPT 2009: Accelerating Development & Advancing Personalized Therapy gathered 450+ delegates from industry, academia and government to address the need for more efficient clinical development, overcome challenges in biomarker implementation, and further develop the potential of personalized medicine. Out of over 90 presentations, ten were selected to be recorded and produced into the Personalized Medicine digital course, providing a comprehensive overview of all angles of personalized medicine implementation from thought-leaders at big pharmaceutical and diagnostic companies, academia, FDA, NIST, insurance provider, law firm and the Personalized Medicine Coalition. The Personalized Medicine digital course offers a convenient multi-media format, allowing you to learn from the experts from the comfort of your own office and on your own schedule.
This digital course was recorded at:
ADAPT 2009 Congress: Accelerating Development & Advancing Personalized Therapy
September 22-25, 2009, Washington, D.C.
About this Product:
Over 380 Slides
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The FDA’s Role in Improving Drug Development
Douglas C. Throckmorton, M.D., Deputy Director, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
There are many pressures on the medical products endeavor, including the need for more timely and efficient development to support their marketing, and then assessment after marketing to support their best uses. There are many stakeholders with important responsibilities in the healthcare system. As regulators, the FDA has a clear role in responding to these pressures, one that includes both providing a clear path to efficient development as well as a critical role in supporting innovation and collaboration.
Oncology Clinical Trial Design: Opportunities for Rational (and Irrational) Incorporation of Biomarkers to Achieve the Goal of Individualized Therapy
Mark J. Ratain, M.D., Leon O. Jacobson Professor of Medicine; Chairman, Committee on Clinical Pharmacology and Pharmacogenomics; Associate Director, Clinical Sciences, Cancer Research Center, The University of Chicago
Biomarkers are often hailed as a panacea to reduce attrition rates of oncology drug development, thereby decreasing drug development costs. To date, the incorporation of predictive biomarkers has had mixed results, including important successes (e.g., HER2 testing for trastuzumab) and failures (e.g., EGFR testing for cetuximab). Pharmacodynamic biomarkers are similarly of theoretical value to accelerate decision-making, but in practice have had limited utility due to lack of technical validation and misconceptions of the value of a biomarker of unknown clinical importance. Novel trial designs incorporating predictive and pharmacodynamic biomarkers will be discussed.
Disease Biology as a Precompetitive Space: Emerging Opportunities for Distributed Contributors to Jointly Evolve Disease Models
Stephen H. Friend, M.D., Ph.D., President, Sage Bionetworks
Significant advances in generating probabilistic casual models as pioneered by Eric Schadt and colleagues at Rosetta Inpharmatics over the last five years have afforded an opportunity to share data not as linear files but as they reflect onto predictive models of disease. Examples will be shown that highlight the power of such models in metabolic and oncologic diseases. Emphasis will be placed on how classical target and pathway representations of disease miss capturing the essential biology needed to generate biomarkers and to develop drugs. Also to be discussed are the new organizational structures being built at the private/public interface that will allow investigators to modify and evolve each other’s models of biology. We posit that when biological data can be reflected onto evolving disease models that clinicians and scientists in academia and industry will be able to enter into connected projects that can evolve the resolving power of disease models in ways natural to chemists but alien to most biologists.
The Future of Personalized Medicine and How Standards can Play a Role in Innovation of New Technologies
Michael D. Amos, Ph.D., Biosciences Advisor, Director’s Office, Chemical Science and Technology Laboratory, National Institute of Standards & Technology Measurement; Ex-Officio Member, Secretary’s Advisory Committee on Genetics Health and Society (SACGHS), Department of Health and Human Services
New measurement technologies can play an important role in expanding the current vision of personalized medicine from mostly encompassing pharmacogenomics and electronic health records to one involving early detection and prevention of the chronic diseases (cancer, diabetes, cardiovascular and other diseases) that cause massive pain and suffering and represent more than 80% of U.S. health care spending. New multiplex measurement tools are making it possible to, for the first time, analyze the complex biomolecular network systems and gain a better understanding of the molecular pathology of diseased cells. DNA microarray, IVD-MIA products are reaching market and the nucleic acid-based signatures they can discern appear to possess greater diagnostic and prognostic value than single measurements alone. The same will probably also be true for multiplex proteome analysis. However, because these technologies are considerably more complex, their utility in the clinic will require entirely new and innovative approaches to standards to enable their further development and deployment.
How Pharmacy Benefit Management Enables the Translation of Personalized Medicine Approaches into Clinical Practice
Felix W. Frueh, Ph.D., Vice President, Research & Development, Personalized Medicine, Medco Health Solutions, Inc.
The translation of personalized medicine into clinical practice requires access to patients at the time of clinical therapeutic decision making. Physicians and pharmacists are ideally positioned to implement this translation, while pharmacy benefit managers (PBMs) are a "hub" for broadly and consistently providing pertinent information to physicians, pharmacists, as well to patients themselves. Medco, the nation's largest PBM, has created several commercial and research programs in personalized medicine that already today provide access to genetic testing for warfarin and tamoxifen for >6 million lives and investigate the clinical effectiveness of a variety of new markers for optimizing drug therapy, respectively.
Personalized Medicine: The Changing Landscape of Healthcare
Edward Abrahams, Ph.D., Executive Director, Personalized Medicine Coalition
This presentation will explore the case for, the status of, and the barriers to the development and implementation of personalized medicine from discovery to delivery.
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.
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 confirm mechanism of action, explore PK/PD interactions and to derive predictive markers in ongoing drug development programs will be described.
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.
Personalized Diagnostics: The Struggle for Position
Bruce Quinn, M.D., Ph.D., Senior Health Policy Specialist, Foley Hoag; Former California Medical Director, NHIC
Multiplex and other complex diagnostics apply cutting-edge molecular biology techniques to clinical challenges in patient care. As technology advances, the technology becomes reliable enough for clinical use and the per-patient costs of the technology become practical. However, the tests are similar to pharmaceuticals in that the cost of goods sold may be small in proportion to the development risks and the costs of clinical trials to validate the test. Therefore, while the value-based market price may be fully commeasurate with the test's clinical worth, a substantial market price is also required just to recover the development costs. Otherwise, the tests will have a negative total value for the test developer regardless of the size of their clinical value to the public. The problem is, historically, the prices of laboratory tests are very small compared to their clinical value. For example, the troponin test saves lives by diagnosing heart attacks, but the market price of the test is under $20 dollars. This lecture discusses the opportunities and challenges that face multiplex diagnostics in the marketplace.