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 and payers to discuss major challenges and potential pathways for delivering precision medicines.


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Thursday, February 13

7:30 am Registration and Morning Coffee


ECONOMIC AND DEVELOPMENT MODELS 

9:00 Chairperson’s Opening Remarks

Mark Trusheim, Executive in Residence & Visiting Scientist, MIT; former Special Government Employee, FDA

9:05 Precision Medicine Economics: Breakthrough or Breakdown?

Mark Trusheim, Executive in Residence & Visiting Scientist, MIT; former Special Government Employee, FDA

Precision medicines represent over half of the drugs receiving FDA Breakthrough designation. While patients are poised to receive great benefits, the economics of developing and reimbursing precision medicines may break both innovators and payers. A breakthrough in precision medicine economics that benefits all is possible, if we cleverly combine next-generation science, big data, creative business models and innovative public policies.

9:35 Collaborative Models for Rx/Dx Partnering and Impact in the Immunology Therapeutic Area

Mark Curran, Ph.D., Vice President, Systems Pharmacology & Biomarkers, Immunology Therapeutic Area, Research & Development, Janssen Pharmaceuticals, Inc.

10:05 CASE STUDY 1: A Systems Approach to Pharmaceutical R&D

Robert J. Mulroy, President & CEO, Merrimack Pharmaceuticals

Merrimack is an oncology-focused biotechnology company developing targeted therapies coupled with companion diagnostics. The company is founded on a systems biology approach to R&D that integrates big data biology, computational modeling, and systems engineering to understand the multi-dimensional interactions that regulate cellular networks. The talk will focus on how the organizational and scientific systems approaches employed by Merrimack have impacted productivity of discovery and preclinical research and is seeking to improve the productivity of clinical research to create precision medicines in cancer.

10:35 Coffee Break with Exhibit and Poster Viewing


COMPANION DIAGNOSTICS FOR STRATIFIED MEDICINES 

11:05 Diagnostics: The Silent Champion of Healthcare

Mara Aspinall, President and CEO, Ventana Medical Systems, Inc.

11:35 The Role of LDTs Before and After the Approval of a Companion Diagnostic

Terry Robins, Ph.D., former Global Director, Biomarker R&D, Quest Diagnostics

Laboratory-Developed Tests (LDTs) can play a significant role in the transition from experimental Biomarker assays to Companion Diagnostics. LDTs represent a flexible, cost-effective approach to mitigate the risks involved in developing a successful companion diagnostic strategy. Even after regulatory approval of both a drug and its companion diagnostic, LDTs serve to enhance the companion diagnostic approach and are required to improve and refine patient selection based on the most current scientific evidence.

12:05 Turbulent Change and the Reimbursement of Companion Diagnostics

Bruce Quinn, M.D., Ph.D., Senior Health Policy Specialist, Foley Hoag, LLP

Combination diagnostics target drugs to genomically defined diseases. In 2012/2013, CMS announced six policy efforts regarding diagnostic payments: The MOLDX coverage/pricing pilot program, genetic test price cuts, policies for multi-analyte test payments, and three CY2014 proposals: (a) cap the prices of advanced pathology tests like in situ hybridization, (b) bundle all diagnostic tests to physician visits or procedures for hospital outpatients, and (c) revise the entire clinical chemistry fee schedule due to technological changes. The outlook for 2014/2015 will be discussed.

12:35 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own 

1:05 Session Break

1:50 Chairperson’s Remarks

Bruce Quinn, M.D., Ph.D., Senior Health Policy Specialist, Foley Hoag, LLP

 

1:55 PANEL DISCUSSION: Precision Medicine Opportunities with Legacy Drugs

Moderator: Bruce Quinn, M.D., Ph.D., Senior Health Policy Specialist, Foley Hoag, LLP

With few exceptions, precision medicine approaches are just being applied to new therapeutics despite our knowledge that current, legacy drugs provide benefits to only about half of the patients taking them. What is preventing us from bringing precision medicine to these therapies? And if we succeed, what would be the benefits to patients, payers and the innovators?

Panelists:

Mark Curran, Ph.D., Vice President, Systems Pharmacology & Biomarkers, Immunology Therapeutic Area Janssen, Pharmaceutical Companies of Johnson & Johnson

Mara Aspinall, President and CEO, Ventana Medical Systems, Inc.

Mark Trusheim, Executive in Residence & Visiting Scientist, MIT; former Special Government Employee, FDA

 

2:55 Refreshment Break with Exhibit and Poster Viewing


TRANSFORMING THE REGULATORY PARADIGM 

3:25 PANEL DISCUSSION: Adaptive Licensing: Evolving Regulation to Enable Precision Medicine

Moderator: Mark Trusheim, Executive in Residence & Visiting Scientist, MIT; former Special Government Employee, FDA

Traditional regulatory decision-making involves the evaluation of safety and efficacy evidence from studies of phenotypically-defined patient populations. Current evidence standards for regulation reflect the predictive failure of these parameters, and other available tools, for more precisely identifying meaningful subpopulations with respect to their potential for benefit/harm. What are the implications of the emerging tools of precision medicine for regulation, and vice versa? How might we think about evidence, regulation, and, more broadly, innovation as we move from population-based drug development to treatment optimization for an N of 1? A multi-stakeholder panel from the MIT New Drug Development Paradigms (NEWDIGS) collaboration will share their perspectives from a series of live simulation exercises that have formed the foundation for a European-wide initiative now being planned for launch in 2014.

Panelists:

Hans-Georg Eichler, M.D., Senior Medical Officer, European Medicines Agency (EMA)

Kenneth Oye, Ph.D., Associate Professor Political Science & Engineering Systems, MIT

Edmund Pezalla, M.D., Ph.D., National Medical Director for Pharmacy Policy and Strategy, Aetna

Thomas Unger, Ph.D., Executive Director, Worldwide Regulatory Strategy, Pfizer, Inc.

5:25 Close of Day



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