Joseph B. Martin Conference Center at Harvard Medical School
77 Avenue Louis Pasteur, Boston, MA 02115

SPEAKER | Stephen L. Eck, M.D., Ph.D., Chief Medical Officer, Immatics U.S. Inc; Board Chair, Personalized Medicine Coalition

MODERATOR | Meg Tirrell, Reporter, CNBC

Daniel O’Day, CEO, Roche Pharmaceuticals

The pharmaceutical industry is deeply invested in commercializing personalized therapies that must recoup fixed development costs from smaller patient populations covered by health insurance companies that are increasingly concerned about rising health care costs. In that context, this diverse panel will explore the viability of the business model for developing and paying for personalized medicines, tackling issues related to costs, prices, and access.

MODERATOR | Meg Tirrell, Reporter, CNBC

Peter Juhn, M.D., M.P.H., Global Head of Value-Based Partnerships, Amgen

Nick Leschly, CEO, Bluebird Bio

Michael Sherman, M.D., Chief Medical Officer, Senior Vice President, Harvard Pilgrim Health Care

Sean Tunis, M.D., Founder, CEO, Center for Medical Technology Policy

Light refreshments provided.

Sponsored By

Recognizing that traditional randomized controlled clinical trials can only study the safety and efficacy of a single therapy in one large population of patients, researchers in personalized medicine increasingly hope that “adaptive platform trials,” which employ advanced statistical techniques to simultaneously test the effectiveness of several personalized treatments in multiple sub-populations of patients, may be the key to new drug approvals in the future. Adaptive platform trials may make drug development more efficient by revealing which of several drug candidates are most promising for which patients, but maximizing the potential of these trials requires unprecedented collaboration among the institutions conducting and sponsoring research on various personalized treatments — and no obvious business models have emerged.

During this interactive case study discussion, professors from Harvard Business School will help us examine how researchers at the Dana-Farber Cancer Institute considered and addressed myriad challenges in their effort to design and operationalize an adaptive platform trial for glioblastoma patients, a deadly disease state for which there are few existing treatment options.

PRESENTED BY

Richard Hamermesh, D.B.A., Co-Faculty Chair, Harvard Business School Kraft Precision Medicine Accelerator; and

Ariel D. Stern, Ph.D., Assistant Professor, Technology and Operations Management Unit, Harvard Business School

INTRODUCTION | Steven D. Averbuch, M.D., Vice President, Head of Precision Medicine, Bristol-Myers Squibb

• Ellen V. Sigal head of Friends of Cancer Research
• Advanced science by Diagnostics Tests
• Cancer Moonshot Program
• Revolution therapies brought to market by Sigel’s sponsorship

AWARDEE | Ellen V. Sigal, Ph.D., Chairperson, Founder, Friends of Cancer Research

Friendly conversation:

• Thanks to PMC
• sister die on breast cancer at 40 with child of 4 1/2.
• appointed to celebrate 20th year of American Cancer Association – Funding for Research, money spend in Washington is for Patients.
• After ten years, interested in measurement of achieving evaluation, FDA structure was of interest.
• Precision Medicine: biomarkers and targets for patients to define success for each patient, WHat is the right population for any drug, responders to drug therapy, if no response, change the drug.
• Patient perspective: Challenges: 90% are treated in the Community and they need a second opinion, insurance, access, clinical trials done out of the community in Academic hospitals – patients are scared to death. Patients are asking for options: Right testing, access to testing involve insurance
• combination therapy  – 6-8 months in advance,

The original architects of the personalized medicine paradigm envisioned an era in which clinicians could predict, prevent and treat disease based on an improved understanding of how human biology interacts with external environments. During this session, a panel of experts will examine our progress on each of these fronts during a wide-ranging conversation about personalized medicine’s past, present and future.

MODERATOR | Cynthia Casson Morton, Ph.D., William Lambert Richardson Professor of Obstetrics, Gynecology and Reproductive Biology, Harvard Medical School

• 1 Million volunteer participants for genome sequencing and microbiome data
• PM Past, Present and Future
• Issues that are rapidly evolving: Physician, Patients

Birgit Funke, Ph.D., F.A.C.M.G., Vice President, Clinical Affairs, Veritas Genetics; Associate Professor of Pathology (Part-Time), Harvard Medical School

• Risk prevention,
• driving DOWN operating cost curation of the Genome

Luba Greenwood, J.D., Strategic Business Development and Corporate Ventures, Verily (an Alphabet company)

• view on diagnostics from Roche, CHemist and lawyer, Venture capital, beyond Genomics, from diagnosis to prognosis,at Verily projects tapping into the entire life span aspect of health
• treatment solution therapeutics except og Oncology threatment is a strugle in the genomics field and pharmaco-genomics
• Power the patients vs Genomics in Diabetes
• Diagnostics in use to keep patients OUT of hospitals – management of chronic diseases
• Patient need to own the genome data not a Databank

Keith Stewart, M.B., CH.B., Carlson and Nelson Endowed Director, Center for Individualized Medicine, Mayo Clinic

• Hematologist, genomics apply genomics for detection of predisposition, inherited , Health genome sequencing,
• Barriers to deploy genomics: Knowledge, readiness of providers, cost of uninsured,
• Diagnostics high value low cost
• drug adherence, pharmacists to be involved in drug adherence before refill

Advocates for personalized medicine have contended that genomic sequencing can deliver clinical and economic value to patients and the health system by allowing providers to more efficiently diagnose disease and develop treatment plans. Following increased use of genomic sequencing in clinical settings, many stakeholders, including payers, have begun to examine that value proposition more closely. During this session, a pharmaceutical industry representative, a payer, and a health economist will discuss the status and future of the emerging evidence regarding the clinical and economic utility of genomic sequencing, including studies recently commissioned by the Personalized Medicine Coalition.

MODERATOR | Daryl Pritchard, Ph.D., Senior Vice President, Science Policy, Personalized Medicine Coalition

• genetic profiling, adopt policy and procedures for mass deployment of NGS
• show that it works – demonstrate value, payers and providers
• a little more that evidence exist for payer to cover
• rare diagnosed disease

Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humana

• labs, payers, providers, pharma — the GAP to be bridged
• opportunities to prevent and treat disease
• Payer, MDs, cost and impact, markers,
• Humana has a research division Use Testing to find value, pharmacogenomics  – on Medicare, Medicaid patients
• cost of doing the test vs not doing this test – assess value
• pharmacisit, economist, statisticians – CMS – provide data on what is covered and what is not Humana: any missed opportunities, MD order tests of no impact per medical record
• What test needed to be ordered? Patient stay healthy
• NGS $650 – $2000 in 2018, in 2016 it was $25,000 cost of testing, cost of drugs
• show us any value as good value – avoiding patient going to MDs Office, Hospital, ER – cost increase due to Pharmacogenomics testing $5K per test
• Guidelines on ordering genomic testing, AI can assist providers, MDs need to catch up on a weekly basis
• CMS Guideline: every test ordered must guide treatment otherwise not covered

Scott Ramsey, M.D., Ph.D., Full Member, Fred Hutchinson Cancer Research Center; Director, Hutchinson Institute for Cancer Outcomes Research

• Value and utility are interconnected
• cost effectiveness of NGS in melanoma: single gene testing – EGFR vs NGS – help clinicians to evaluate Lung Cancer
• Flariton Database, 300 centers  – 140,000 – Patients got NGS – 7% ADDITIONAL patients founded mutations beyond EGFR
• Survival in this cohort NGS vs EGFR – improved survival 6 month longer, mean survival 3 weeks long, not significant.
• Increased survivals, why? cost of sequencing  – #14 most influential – cost does not drive value
• #1 drug cost was the factor
• #2 survival
• marginal cost in platform comparison
• Pricing of Testing NGS and Targeted therapy represent a threat to adoption of Genomics in Medicine
• disparities and access – cost and patients: Partners and Mayo clinic patients are lucky

INTRODUCTION | Daryl Pritchard, Ph.D., Senior Vice President, Science Policy, Personalized Medicine Coalition

PRESENTER | Shreeram Aradhye, M.D., Head of Global Medical Affairs, Chief Medical Officer, Pharmaceuticals, Novartis; Board Member, PhRMA Foundation

#1 Prize $50,000 – Dr. Garrison, UK

#2 Prize $25,000 – Dr. Robim Hayeems, Hospital for Sick Children Institute, Toronto, Canada

#3 Prize @ $10,000  – Dr. A Le, PharmD., PhD, Western University of Health Sciences

Light refreshments provided.

Sustaining the pace of innovation in personalized medicine will require continued investment in new initiatives, but the financial outlook for the field remains unclear. In that context, this panel of investors will examine whether personalized medicine is at an impasse, an inflection point or somewhere in between.

MODERATOR | William A. Sahlman, Ph.D., Baker Foundation Professor, Harvard Business School

• market – can it sustain the opportunity – winners and losers
• innovative financial models
• Biotech IPO, VC, windows slam shut, drug failure – drivers and non
• Increasing return to scale: AI, NGS, screening, – foreign money, China
• Tsinghua University went back to China from Silicon Valley

Cary Pfeffer, M.D., Partner, Third Rock Ventures

• was a decade at Biogen, MS indication drug, no biomarkers for patients – efficacy was in 50% non respondents 25%
• Genomic sequencing to identify patient populations – no good effective medicine without target therapy
• Mayocardia – drug in CVD for patients identified by Genomics
• Genomics information needed to develop drugs

Michael Pellini, M.D., Managing Partner, Section 32; Board Member, Personalized Medicine Coalition

• Impasse or Inflection Point? it s Inflection Point NOT an Impasse
• Diagnostics component inside 4.8 Trillion in the therapeutics selection in the system as a whole
• Foundation Medicine saw Roche as Big brother with International reach
• Patients and Consumers will force in five years figuring out – every diagnosis of cancer will be sequenced and the infrastructure to interpret results and paid for

Salveen Richter, C.F.A., Vice President, Research Division, Goldman Sachs

• innovative and disruptive, orphan drugs, Health IT, US Market 3 trillion – size of the opportunity 80% genetically driven
• Cancer, CART therapy, easier to pay by performance, cost of the drug itself. profit in the 1st generation od Pharma manufacturers
• One time pricing vs further indications, annuity type system, Hemophilia – $19Million market,
• Europe successful in financing Health care — in the US — system must change – investment will flee, to fund pricing drug is key in changing the system CART Pricing is still difficult to pay for
• Sequencing cost plunged, public investors placing funding in start ups even without return in the horizon, companies with multiple modalities spurring innovation – confusing in the investment side, technologies become obsolete very fast
• Europe vs US, China is different no regulation like FDA,talent from US Pharma went back to China

SPEAKER | Edward Abrahams, Ph.D., President, Personalized Medicine Coalition