Implementing Personalized Medicine
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Wednesday, September 19
» Opening Plenary Session
2:00-3:00 Main Conference Registration
3:00-3:10 Welcoming Remarks from Conference Director
Julia Boguslavsky, Executive Director, Conferences, Cambridge Healthtech Institute
3:10-3:15 Chairperson’s Opening Remarks
3:15-3:45 Rules, Tools and Data Pools: The Critical Path's Recipe for Speeding Drug Development
Carolyn Compton, M.D., Ph.D., President & CEO, Critical Path Institute
Both regulatory agencies and the medical products industry recognize the need for new biomarkers and methods to speed the development and delivery of effective, safe medicines to patients. However, developing consensus on the utility of new biomarkers and establishing a process for putting them into routine practice in medical product development and regulatory decision making is a complex undertaking. In 2009 and 2010, respectively, the European Medicines Agency and the FDA released guidance that describes a voluntary pathway for "qualification" of novel drug development tools or methods. Determining the relative advantages/disadvantages and evidentiary standards required for regulatory "qualification," applying a novel biomarker on a specific drug development program, or submitting an application for a diagnostic device are challenging tasks. The Critical Path Institute (C-Path) acts as a trusted third party to lead six pre-competitive global consortia that, with the cooperation of the FDA, develop and qualify molecular and imaging biomarkers as well as patient-reported outcomes instruments for specific contexts of use in medical product development. Several C-Path consortia use Clinical Data Interchange Standards Consortium disease area data standards in constructing clinical trial databases from which disease progression models and virtual clinical trial simulations are developed. Specific examples from these consortia will be utilized to illustrate the power of pre-competitive collaboration in the generation and qualification of drug development tools.
3:45-4:15 Technology Transformation at FDA: Driving Efficiency and Unleashing Innovation
Eric D. Perakslis, Ph.D., Chief Information Officer and Chief Scientist, Informatics, U.S. Food and Drug Administration
The U.S. Food and Drug Administration has recently taken significant steps to modernize and improve its information technology and informatics capabilities. The resulting infrastructure, architecture, innovation pathways and data sharing initiatives are intended to ease regulatory burden on innovators while maintaining the highest quality standards. In this talk, Eric Perakslis, Ph.D., the Chief Information Officer and Chief Scientist for Informatics at FDA, will discuss these initiatives in detail and will provide success examples, recent progress and future directions.
4:15-4:45 Turning an Active Compound into a Personalized Medicine: Do Biomarkers Help or Hinder?
Geert Kolvenbag, M.D., Ph.D., Global Product Vice President, AstraZeneca
The co-development of drug and biomarker has several inherent risk and challenges. In addition the expectations and demands of the oncology community have increased over the last years. These challenges will be illustrated by a case study of a very active compound destined for a fast development program as a personalized medicine, but running into scientific, diagnostic and development challenges. This experience creates questions for development of drugs and diagnostics today and in the future.
4:45-5:15 Strategies to De-Risk Drug Development Utilizing Biomarkers in Early Clinical Trials
Scott Kennedy, Ph.D., Global Head, Biomarker Development, Novartis Institutes for BioMedical Research, Inc.
Other than recent examples in oncology, the emerging field of personalized medicine has yet to live up to its promise of enabling more rapid, efficient drug development and providing customized therapies to patients. This presentation will exemplify and discuss how leveraging biomarkers which inform a biological understanding of targeted and disease pathways can increase the success of early clinical development. These biologically relevant markers can also enable the identification and treatment of parallel disease populations and serve as stratification or response markers for later stage development. This presentation will also discuss several of the challenges that this paradigm presents.
5:15-5:45 Implementing a Personalized Medicine Strategy: Is There Light at the End of the Tunnel?
Jeremy Barton, M.D., Vice President, Head of Oncology Clinical Research, Pfizer
Personalized medicine, the practice of tailoring treatment to individual characteristics of the patient, has emerged as a major force in oncology in the last decade with the expectation of significant improvement in safety and efficacy of therapeutics. The ultimate success of this paradigm is dependent on a variety of factors. The choice of drug target, selection of patients by companion diagnostic, regulatory authority acceptance of novel trial designs which best serve this strategy, improved health care information technology, insurance coverage and reimbursement are just a few of the important variables in the equation. This presentation will provide an overview of the current situation and discuss factors which will impact future progress in the field.
5:45-6:45 Welcome Reception in the Exhibit Hall with Poster Viewing
Thursday, September 20
7:30-8:15 am Morning Coffee or Sponsored Breakfast Presentation (Opportunity Available)
Contact Ilana Quigley at email@example.com or 781-972-5457
8:30-8:55 Co-Development of Vemurafenib and the cobas® BRAF Mutation Test
Walter H. Koch, Ph.D., Vice President and Head of Global Research, Roche Molecular Diagnostics
Categorization of cancers based on oncogenic genomic alterations has identified numerous new kinase drug targets. Development of targeted kinase inhibitor therapies alongside companion diagnostics that will identify patients most likely to receive benefit represents a new model for drug development, and portends increased success rates for therapeutics, with decreased development times and associated costs. The co-development of vemurafenib (ZelborafTM) and the cobas® 4800 BRAF V600 mutation test will be presented from the diagnostics perspective. This focused and integrated process resulted in the first personalized medicine for the treatment of metastatic melanoma less than five years after the Investigational New Drug Application was filed.
8:55-9:20 Integrating a Companion Diagnostic into the Clinical Development of Crizotinib
Ian Taylor, Ph.D., Senior Director, Translational Oncology, Pfizer
Fully integrating a diagnostic test into the development of crizotinib (XalkoriTM) for treatment of NSCLC patients presented technical, clinical and regulatory challenges as well as opportunities to conduct smaller clinical trials with a greater chance of those trials being successful by appropriately identifying a patient population expected to benefit from crizotinib treatment. This presentation will describe the novel approaches as well as close collaboration between Pfizer, Abbott, CDER, and CDRH that resulted in the simultaneous submission and approval of a new drug-diagnostics combination.
9:20-9:45 Creating a Framework for Personalized Medicine in CF: The Kalydeco® Experience
Preston W. Campbell, III, M.D., Executive Vice President for Medical Affairs, Cystic Fibrosis Foundation
The Therapeutics Development Program of the Cystic Fibrosis Program will be highlighted as a model of non-profit-industry collaboration. Kalydeco was discovered and developed through this program and was recently approved for the treatment of persons with CF that carry at least one copy of the CFTR gene mutation G551D, less than 5% of the US CF population. Physical-chemical studies of CFTR mutation phenotypes suggest that there are patients carrying many other, but less prevalent, CFTR mutations that may also benefit from Kalydeco therapy. As an advocacy organization, our challenge is to support processes by which every patient that has a reasonable chance of benefiting from a therapy be given the opportunity to do so. Our approach will be discussed.
9:45-10:45 Coffee Break in the Exhibit Hall with Poster Viewing
10:45 am-11:00 Rapid Discovery and Development of Multivariate Tests for Personalized MedicinePaul Beresford Ph.D., Vice President Business Development & Strategic Marketing, BiodesixBiodesix’ unbiased, rather than hypothesis-driven approach to biomarker discovery has the ability to rapidly discover diagnostic tests while overcoming many limitations of genomic-based approaches. Our highly reproducible phenotype analysis technology performs as both discovery and clinical testing platform as we will demonstrate with a case study of VeriStrat, a commercially available test to help personalize therapy for lung cancer.
11:00 am-11:15 Biochip Array Technology – a Rapid Multiplex Solution for Mutation ProfilingScott McKeown, Ph.D., Research Consultant, Randox LabsRandox has developed award winning Biochip Array Technology for rapid proteomic and genomic
diagnostics across a range of human diseases. Utilizing multiplex PCR, spatially tethered specific
probes and chemiluminescence detection methods, our innovative KRAS/BRAF/PIK3CA array
simultaneously identifies 20 mutations within 3 hours.
11:15 am-11:30 Adoption of new MDx Tests – A Case Study in Clinical Utility
Carol Berry, MBA, Senior Vice President, Genomic Services Division, Asuragen, Inc.This presentation will review a real world example of establishing clinical utility of a new molecular test. Physicians need better tools for decision making in their practice but in reality many have questions on how to implement personalized medicine in the real world.
11:30 am-12:00 pm Sponsored Presentations (Opportunities Available)
Contact Ilana Quigley at firstname.lastname@example.org or 781-972-5457
12:00-1:30 Enjoy Lunch on Your Own
1:30-1:35 Chairperson’s Opening Remarks
1:35-2:00 Improving Efficacy and Reliability for Personalized Medicine: The Role of Companion Diagnostics in Drug Development
Amelia Warner, Ph.D., Director, Clinical Pharmacogenomics, Merck
Identification of subpopulations of patients who either have subclinical response or toxicity to a drug in clinical development is a challenge. Historically, identification of patients who have less than optimal response to drugs has occurred late in large global clinical trial review. However, the pharmaceutical industry is working to optimize pre-clinical models, known genetic variation across populations, and earlier inclusion of surrogate biomarkers in early development programs to allow for early identification of populations that will benefit from alternate dosing or alternate therapies. Adapting development strategies to include companion diagnostics remains a challenge, but pharmaceutical companies are developing clear working models to enable development of personalized therapies.
2:00-2:25 Drug-Diagnostic Co-Development: Navigating Open Waters
Finley Austin, Ph.D., Personalized Healthcare and Biomarker Strategy Director, AstraZeneca
This talk will examine different development scenarios outside of the “ideal” drug diagnostic co-development model. The aim will be to elucidate tensions within the oversight system and consider pros and cons of alternative approaches to co-development. Some proposals to accommodate the inherent changes in available information for decision making that occur throughout product development and review will be considered.
2:25-2:50 Towards the Future of Healthcare
Paul Kildal-Brandt, Business Alliances and Development Leader, Janssen Diagnostics
The talk will focus on advances in personalized medicine and will provide details of Janssen Diagnostics’ role in support of the pharma sector within Johnson and Johnson. We will discuss examples of past personalized medicine products commercialized by Johnson and Johnson companies as well.
2:50-3:15 Personalized Medicine in Pharmaceutical Development Is Attractive but a Very Bumpy Road
Abdel Halim, Pharm.D., Ph.D., DABCC-MDx, DABCC-TOX, DABCC-CC, FACB, Director, Clinical Biomarkers Daiichi Sankyo Pharma Development
Personalized medicine aims to predict the most efficacious and safe drug based on a laboratory test. This can be of great value to clinicians, patients, regulatory agencies, payers, pharmaceutical companies and the diagnostic industry. It requires co-development, approval, and marketing of a drug with an in vitro diagnostic test. However, a personalized medicine approach necessitates good understanding of the biomarker’s role in early phases of drug development, and a robust assay before testing in PIII trial. In this respect, the gaps are still wide between the pharmaceutical industry and biomarker labs, and between regulatory expectations and real life in drug development; also the business model for such co-development is still vague.
3:15-4:15 Refreshment Break in the Exhibit Hall with Poster Viewing
4:20-4:45 Biomarker and Companion Diagnostics Development from a Biotech Perspective
Anjan Thakurta, Ph.D., Senior Director, Translational Development Integration, Celgene
Translational research is a powerful approach that allows the discovery and development of biomarkers at all stages of drug development. While the upside is tremendous, the inclusion of translational studies poses huge challenges to the drug development process. Our ability to deal with these challenges is a critical success factor in delivering competitive advantage to our pipeline. I will discuss keys aspects of the Celgene approach in biomarker and diagnostic development addressing these challenges.
4:45-5:10 Accelerating Dx Development through the Application of Systems Thinking
Devon C. Campbell, Director, Engineering and Systems, Novartis Molecular Diagnostics
Systems Thinking is a powerful perspective and set of tools that construct a deeper, more meaningful understanding of complex systems by emphasizing relationships among a system’s parts, rather than the parts themselves. In its simplest form, it provides a more accurate picture of reality and how to utilize a system’s natural forces to achieve results. Personalized medicine diagnostics development is ripe with opportunities where the application of Systems Thinking perspectives can help accelerate the delivery of well understood, robust solutions to market. Examples will be briefly explored, demonstrating Systems Thinking’s value in the development and implementation of personalized medicine diagnostics.
5:10 End of Day 2
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