2014 Archived Content

Cambridge Healthtech Institute’s Inaugural
Leaders in Precision Medicine
Regulatory, Economic and Data Dilemmas in a Genomic Era
Part of the 21st Annual Molecular Medicine Tri-Conference

February 13-14, 2014 | Westin St. Francis | San Francisco, CA


Many believe that personalized medicine technologies, such as next-generation sequencing, have propelled us to the tipping point of a new era in healthcare - precision medicine. Stakeholders are now faced with the perplexing economic and regulatory issues of balancing the promise of new genome-driven medicines with the associated trade-offs of longer and higher development risk for Rx and CDx innovators, increased evidence standards for regulators, reimbursement concerns for patients, and unclear value for payers. This symposium will feature discussions from drug/diagnostic innovators, regulators, providers, payers, patient advocates and leaders from medical groups to discuss major challenges and potential pathways for delivering precision medicines.


Day 1 | Day 2 | Symposia Brochure | Full Event Brochure     

Friday, February 14

8:00 am Morning Coffee


TRANSLATING SCIENTIFIC POSSIBILITIES INTO PATIENT TREATMENT REALITiES 

8:25 Chairperson’s Remarks

Kip Harry, Conference Director, Cambridge Healthtech Institute

8:30 CASE STUDY 2: Kalydeco – Targeting the G552D Population in Cystic Fibrosis

Peter Mueller, Ph.D., CSO & Executive Vice President, Global Research & Development, Vertex Pharmaceuticals

To date, more than 1900 different genetic mutations are known in the gene encoding the Cystic Fibrosis Transmembrane Regulator (CFTR) protein. KALYDECO, recently approved by the FDA and EMA, is a breakthrough therapeutic approach to treat the underlying root cause of CF in patients with the G551D mutation by enhancing chloride transport using a CFTR potentiator. Relevant biological concepts and clinical development strategies addressing different aspects of the complex interplay between genotype and phenotype in patients with Cystic Fibrosis will be discussed.

9:00 Demonstrating Clinical Utility/Health Technology Assessment

Naomi Aronson, Ph.D., Executive Director, Technology Evaluation Center, Blue Cross Blue Shield

9:30 Bringing Comprehensive Molecular Information into Routine Clinical Care

Josephine N. Harada, Ph.D., MBA, Director, Strategic Alliances, Foundation Medicine, Inc.

In recent years the field of oncology has experienced a paradigm shift toward thinking about cancer as a disease of the genome. Our understanding of cancer biology has been furthered through the use of next-generation sequencing (NGS) to more comprehensively characterize the genomic alterations in an individual patient’s cancer. Once a tool used only in research, a comprehensive profiling approach utilizing NGS has been developed to enable precision medicine in clinical cancer care. Widespread use of this approach could provide more treatment options and enable more rapid accrual to ongoing and planned trials of agents targeting pathways under study, thereby continuing to accelerate the field of precision medicine.

10:00 Using a Patient’s Genetic Information in the Real World

Michael Christman, Ph.D., President and CEO, Coriell Institute for Medical Research

When a patient needs a new prescription, it will be necessary for the physician to quickly and securely access his/her genetic data to understand drug efficacy prior to dosing. Who will patients and doctors trust to store and interpret the data? Coriell and the CPMC research study have defined several of the key barriers to accelerate the adoption and routine use of genomics in medicine and have proposed solutions that are generally applicable.

10:30 Coffee Break with Exhibit and Poster Viewing

11:00 The “$1000 Genome”: Panacea or Pandora’s Box? 

Kathryn Phillips, Ph.D., Professor, Health Economics and Health Services Research, University of California, San Francisco

New technologies are enabling the arrival of the much-awaited “$1000 genome”—the ability to sequence an individual’s or a tumor’s entire genome quickly and relatively inexpensively [whole genome sequencing (WGS)]. WGS is now being offered in clinical care and is expected to become more widely used in the near future. However, this technological advance threatens to outpace our ability to use it effectively in clinical practice and to address the associated health policy issues. This talk will discuss work on evaluating the potential benefit-risk tradeoffs of WGS from the perspectives of patients, providers, the health care delivery system, and society.

11:30 PANEL DISCUSSION: The Promise of Patient Data in Healthcare

The generation and systematic utilization of vast amounts genomic data promises to be the key in enhanced decision-making for patient care, diagnostics, and the development of targeted treatments. This panel will discuss current challenges in implementing genomic data into modern-day healthcare, such as assessing the emerging role of next-generation sequencing (NGS) in clinical trial design; benefit-risk tradeoffs of NGS, clinical utility and health technology assessment; integration into EMRs, data privacy and return of patient results.

Moderator:

Kathryn Phillips, Ph.D., Professor, Health Economics and Health Services Research, University of California, San Francisco

Panelists:

Peter Mueller, Ph.D., CSO & Executive Vice President, Global Research & Development, Vertex Pharmaceuticals

Naomi Aronson, Ph.D., Executive Director, Technology Evaluation Center, Blue Cross Blue Shield

Josephine N. Harada, Ph.D., MBA, Director, Strategic Alliances, Foundation Medicine, Inc.

Michael Christman, Ph.D., President and CEO, Coriell Institute for Medical Research

12:30 Close of Symposium



Day 1 | Day 2 | Symposia Brochure | Full Event Brochure     

Japan-Flag Korea-Flag China-Simplified-Flag China-Traditional-Flag 

 

2015 MMTC Final Agenda 

Premier Sponsors:

Elsevier 


Jackson Laboratory - small logo 

Leica Biosystems 
 

 NanoString2   

 

Silicon Biosystems 

 

Singulex 

Thomson Reuters-Large 






Local Partners:

BayBio 


biocube 


Cabs 

City of SSF