Leaders in Pharmaceutical Business Intelligence (LPBI) Group

LIVE Day One – 13th Annual Personalized Medicine Conference, From Concept to the Clinic, November 14–16, 2017, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur Boston, MA 02115

 

JOSEPH B. MARTIN CONFERENCE CENTER

HARVARD MEDICAL SCHOOL, BOSTON, MA 02115

http://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/13th_Annual_PM_Conference37.pdf

Leaders in Pharmaceutical Business Intelligence (LPBI) Group, Boston

will cover in REAL TIME the 13th Annual Personalized Medicine, From Concept to the Clinic, November 14–16, 2017, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur Boston, MA 02115

In attendance, covering LIVE using Social Media

Aviva Lev-Ari, PhD, RN

Editor-in-Chief

http://pharmaceuticalintelligence.com

@pharma_BI

@AVIVA1950

#PMConf

Agenda · Part I  NOVEMBER 15, 2017 · CONFERENCE PROGRAM | AGENDA

7:00 am Registration and Breakfast

8:00 am Opening Remarks SPEAKER | Edward Abrahams, Ph.D., President, Personalized Medicine Coalition

• 13th Annual PM Conference at HMS
• Paradigm shift from One medicine FITS ALL – 13 years ago was a promise in 2017 it is a REALITY
• Liquid biopsies, read and write gene therapy
• Value, Pricing, Access
• Evidence for reimbursement and FDA directions
• Introduction od PM to the Clinic
• Case study on Value and Healthcare System
• Future of PM Stanford, BU, Investor
• Translation to Chinese, 50 guests from 20 countries and 21 from CA

 

8:10 am The State of Personalized Medicine

INTRODUCTION |

Steven D. Averbuch, M.D., Head, Precision Medicine Research & Development, Bristol-Myers Squibb; Board Member, Personalized Medicine Coalition

• Known Tom for few decades, collecting tissue on lung cancer.
• EGFR – discovered at HMS

KEYNOTE |

Thomas J. Lynch, Jr., M.D., Executive Vice President, Chief Scientific Officer, Research & Development, Bristol-Myers Squibb

• Apply right drug to right patient
• Gefitinig (AstraZeneca) vs Carboplatin/Paclitaxel – lung cancer – advanced NSCLC
• Gene mutation – EGFR negative vs Positive – cost of sequencing inverse to Moore’s Law
• Genome Sequenced at <$1,000
• Lung Adenocarcinoma: 2016: PIK3CA, KRAS, BRAF
• 2015NEJM – Design drugs: Resistance mutation vs. negative – no mutation EGFR
• Immune -Biology of Cancer is Complex – Tumor, Effector Cells, Immune regulatory & APGs
• NEXT opportunities: Novel I-O mechanisms, patient selection is key
• Biomarkers: PDL-1, MSI – H Tumor mutation Burden (TMB) LAG-3
• Future: Gene signature
• Pembrolizumab Free survivsls – 50%
• BMS – FoundationOne – calibration used by BMS: Mutational Burden: FoundationOne assey: Exploratory analysis: High TMB (Nivoluman vs Chemo, Nivo id bettervs LowTMB
• Combination Drug Therapy: Nivo +Ipi
• Assessment of TMB: coding regions of 21K genes: WES vs FoundationOne (F1)
• TMB in Blood (bTMB) in 2L + NSCLC (POPLAR and OAK)
• bTMB <16 vs bTMB >16
• PM – molecular profiling of Hundreds of Cancers and PM of drug treatment driven by molecular profiles
• F1 is promising

8:40 am 13th Annual Leadership in Personalized Medicine Award

AWARDEE | Jay T. Flatley, M.S., Executive Chairman, Illumina

• Precision Medicine – still inpact is limited
• Regulatory and Reimbursement are BARRIERS in the HealthCare system,
• Kidney Cancer treated off-label – great
• less 10% of tumors have been sequenced, sufficient tissue needed, physician voted not to sequence deeming it un-actionable
• Cystic Fibrosis – only sequencing lead to FDA approval, POST marketing required significant resources
• Translational researchers validated 200 genes
• 400 genes at FoundationOne
• DIagnostics companies – reimbursement process is TOO LONG
• Regulatory – emerging diagnostics: Product in use in clinical trials
• Risk models to be shared with Diagnostics companies – reimbursement at end of period when results are available
• Blockchain technology is promising for handling data
• Rare diseases in Cancer  – One milion genomes sequenced
• National Genomic Service – Sequencing becoming STANDARD of Care, phynotypes, $10 million to be spent NIH said Jay T. Flatley, M.S., Executive Chairman, Illumina
• at Illimina – Consumer Advocacy added to Technology breakthroughs in genome sequencing said Jay T. Flatley, M.S., Executive Chairman, Illumina

9:10 am Networking Break

9:35 am Progress in Partnerships: A Two-Part Discussion Aligning the constructs of the health system with the principles of personalized medicine will require stakeholders to scale the most promising cross-sector partnership models. This series of conversations will examine the potential of several of the most promising models that have emerged thus far.

Discussion

Part 1 9:35 am A Model for Risk-Sharing Agreements Between Payers and the Pharmaceutical Industry Many payers are reluctant to assume that covering personalized medicines will help mitigate costs associated with major medical events that require hospitalization. During this fireside chat, however, representatives from Amgen and Harvard Pilgrim Health Care will discuss the logic and implications of their groundbreaking agreement to share the financial risks of covering a targeted medicine based on that premise. Under the terms of the agreement, Amgen agreed to cover treatment costs for patients who have a heart attack or stroke while taking its personalized therapy for familial hypercholesterolemia.

MODERATOR | Meg Tirrell, M.S.J., Reporter, CNBC

Joshua Ofman, M.D., M.S.H.S., Senior Vice President, Global Value, Access and Policy, Amgen

• Hyperlipidemia – Partnering, Amgen with Harvard Pilgrim Health Care
• Arrangements: Patients who need the medication will get access to the medication
• effective stuards: Co-Pay not too high, replacement of medication by generics
• Medicare has requirements
• Migraine medications are coming out from Amgen – novel payment
• Combination drug therapy – Payment system not ready for it yet
• 3D BioPrinting of Drugs and the innovation storm of agents — are both benefits, value based pricing, elasticities, is that price sufficient to support R&D, dynamic not linear environment said Joshua Ofman, M.D., M.S.H.S., Senior Vice President, Global Value, Access and Policy, Amgen

Michael Sherman, M.D., M.B.A., M.S., Chief Medical Officer, Senior Vice President, Harvard Pilgrim Health Care; Board Member, Personalized Medicine Coalition

• complications,  cost, outcome work vs does not work
• Gene therapies coming from Novartis and Partnership with Harvard Pilgrims
• Value proposition for one drug and one cancer – to assure access Pharma and Payers Partnership
• Drug and Outcome, high cost drug coming, very expensive, life saver for who needs them

Discussion

• Pharma’s revenue stream is international, it is in the US that payers require Value

 

Part 2 10:05 am Models for the Development of Personalized Medicine Diagnostics Pharmaceutical and diagnostics companies have responded to a host of complex scientific, regulatory and reimbursement challenges partly by developing innovative partnership models around companion diagnostics. This panel discussion will feature representatives from the pharmaceutical and diagnostics industries, who will discuss the challenges partnerships have helped industry overcome as well as the obstacles that continue to inhibit the development of the diagnostic tools upon which personalized medicine depends. Agenda ·

 

MODERATOR | Alexander Vadas, Ph.D., Managing Director, L.E.K. Consulting

Joydeep Goswami, Ph.D., M.B.A., M.S., President, Clinical Next-Generation Sequencing, Oncology, Thermo Fisher Scientific

• monitor disease , biopsy changes in 6 month and repeat is needed
• NGS – results come back in a month what the mutation mean? NOW results come in few days, data analysis  assist the MDs for action in treatment said Joydeep Goswami, Ph.D., M.B.A., M.S., President, Clinical Next-Generation Sequencing, Oncology, Thermo Fisher Scientific
• How to accelerate the need for safety and the avalangue of innovations
• Clinical sense vs research context, FDA is more comfortable with other than oncology products beyond drugs, namely diagnostics

Jacob S. Van Naarden, Chief Business Officer, Loxo Oncology

• Increase in need of drugs in NGS World, Tissue agnostics, ALL the drugs and all the tumors
• reduction in cost with technology, can’t be too expensive,
• LOXO drug development  – each testing addresses a focused medical issue  – cancer alterations  for ALL extremely aggressive cancer
• DNA and RNA based events detected by tools
• cost of test need to be low, Reference Labs need to collaborate to standarize technology and explain to the Payers
• Cost of CT Scan vs an NGS Test – Genomic testing is much cheaper said Jacob S. Van Naarden, Chief Business Officer, Loxo Oncology
• Educational

10:35 am Real-World Personalized Medicine: Examining the Role of Real-World Evidence in Personalizing Health Care FDA has offered a definition of real-world evidence, but the community continues to debate what is needed to fully integrate it into decision-making. This panel will explore what real-world evidence is, how it is being used and what regulatory requirements are needed to realize its potential.

MODERATOR | Amy Abernethy, M.D., Ph.D., Chief Medical Officer, Chief Scientific Officer, Flatiron Health; Board Member, Personalized Medicine Coalition

• Collection of evidence to accelerate from lab to the Clinic
• use data sets prospective vs retrospective studies asked Amy Abernethy, M.D., Ph.D., Chief Medical Officer, Chief Scientific Officer, Flatiron Health; Board Member, Personalized Medicine Coalition

Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA

• 3/2017 established to have an integrative approach by FDA – real world dat ais important to FDA
• Drug approved for one indication, provide new data for supplemental indications said Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA
• co-morbidities cause EXCLUSION from clinical trials, i.e., HIV patients, experience of patients excluded to learn how differently they can be treated
• drafting a document on Verify data integrity in clinical trials, detect discrepancies compromise the integrity of the data – audits by FDA said Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA
• Validation of devices- FDA Innovation Initiative,

Eric G. Klein, Pharm.D., Senior Director, Oncology, Global Patient Outcomes and Real-World Evidence, Eli Lilly and Company

• Aggregate burden of disease, existence of co-morbidities
• Why it was occurring – Genomics: WHY is explained – precise tools
• data vs intelligence – interoperability
• Past clinical trial – replicate studies with retrospective data
• finding the responding patients – pragmatic trial, not randomized, collect end point – very expensive and requires statisticians
• end pint definition changed

Eleanor M. Perfetto, Ph.D., M.S., Senior Vice President, Strategic Initiatives, National Health Council

• How patients  wish to see usage of their experience – how side effect information can be used.
• New indication
• reimbursement
• improved used of existing drug higher rating vs new indications
• clinical trial design gets input from patients, Patient can announce, dropping participation if a change is not made

Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute

• Major gene mutation and Drugs
• Drug exposure correlate with evidence, Worldwide,
• linkages vs computational techniques we do not have consistent data, data structured or not, respond to medication: symptoms, prospects vs Vital sign or WBC count – we have data standardization is evolving said Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute
• clinical decision support what is structured is data upon admission, monitoring the drugs given in this period, turning to patients willing to offer feedback and cooperate
• pre-existing autoimmune disease – not indicated for them Immunotherapy even though patients wish to try said Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute
• Standards: Toxicity criteria – library of 882 symptoms, Patient reported outcomes by Patients, Resist  criteria applied to imaging data criteria for brain tumors said Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute
• Physicians needs interfaces, dashboard information delivered to MDs, data sits unused, new tools are needed for the data display by relevance to the MDs
• Patients input and sophistication increased – IRB is not aware of the engagement of Patients and their challenging feedback say Deborah Schrag, M.D., M.P.H, Dana Farber

11:50 am Luncheon

1:00 pm The Designer Genome: Exploring the Implications of Gene Editing and Gene Therapy for the Future of Medicine and Humanity Many scientists believe the clustered regularly interspaced short palindromic repeats (CRISPR) genetic engineering tool and recent developments in gene therapy will dramatically alter the trajectory of medicine, but the specific implications of these developments for health systems around the world remain unclear. During this session, a panel of experts will discuss the status of these new technologies and how the medical community and regulatory agencies may have to adapt to keep up with forthcoming developments.

MODERATOR | Kevin Davies, Ph.D., Co-Author, DNA: The Story of the Genetic Revolution (with Jim Watson and Andrew Berry); Executive Editor, The CRISPR Journal

Katrine Bosley, CEO, Editas Medicine

• spectrum of ease to correct a mutation, some mutation are easier than others for editing,
• understand well enough  the gentic application where CRISPR will assist medicine: Retinal degeneration, two aspects one worked in Japan said Katrine Bosley, CEO, Editas Medicine

Arthur L. Caplan, Ph.D., Drs. William F. and Virginia Connolly Mitty Chair, Director, Division of Medical Ethics, New York University Langone Medical Center

• Bioethics, super babies, engineering embrios,
• Regulatory oversight on engineering embrios is coming, metric of success in recruitment of patients said Arthur L. Caplan, Ph.D., Drs. William F. and Virginia Connolly Mitty Chair, Director, Division of Medical Ethics, New York University Langone Medical Center
• cell repair is cheaper that transplantation,
• clone of super person next door
• Bioterrorism accomplished by gene engineering !!

George M. Church, Ph.D., Professor of Genetics, Health Sciences and Technology, Harvard-MIT Division of Health Sciences and Technology; Director, Harvard Medical School NHGRI-Center of Excellence in Genomic Science; Director, Harvard Medical School Personal Genome Project; Founding Member, Wyss Institute for Biologically Inspired Engineering at Harvard University

• Delivery, more precise,
• Longevity and aging – one blockbuster is needed
• Engineered mutation, machine learning
• CRISPR does not handle all mutation many require a different editing tool said George M. Church, Ph.D., Professor of Genetics, Health Sciences and Technology, Harvard-MIT Division of Health Sciences and Technology; Director, Harvard Medical School NHGRI-Center of Excellence in Genomic Science; Director, Harvard Medical School Personal Genome Project; Founding Member, Wyss Institute for Biologically Inspired Engineering at Harvard University
• over regulation – Do not touch germ line – is not desired
• Transplantation vs enhancement – resistance to senescence and pathogens to be achieved by gene editing suggests George M. Church, Ph.D., Professor of Genetics
• Bringing back genes – elephants with fur

Jeffrey D. Marrazzo, M.B.A., M.P.A., CEO, Spark Therapeutics

• Retina degeneration causes blindness, deliver drug to back of the retine, inject genetic material and achieved remarkable results, drug approval of genetic therapy in the US for a genetic disorder in Retina causing blindness
• 21st Century schema of Payment and benefits

 

2:15 pm Pricing Personalized Medicines The increasing pressure on industry stakeholders to alter their drug pricing practices has particular significance for personalized medicines, which must recoup research and development costs from smaller patient populations. This conversation will explore the pharmaceutical industry’s strategies for facilitating sustainable access to these innovative therapies.

MODERATOR | Meg Tirrell, M.S.J., Reporter, CNBC

Stephen J. Ubl, President, CEO, PhRMA

• minimum 10% invested in R&D at each Pharma
• Of 134 drugs in development – 42 have the potential to become Personalized medicine therapies, said Stephen J. Ubl, President, CEO, PhRMA
• Icer methodology – average patient aggregate data, value pricing is a better model: RA targeted to subset of patients
• Price gauging is a problem – bring solutions to the table, Patients asks for incentives
• amortizing costs like mortgage
• Outcome-based arrangements: If money-based guaranteed it negate Medicaid negotiation power. If transportation is covered – it leads to locking product in use

2:45 pm Networking Break (sponsored by GreyBird Ventures)

3:15 pm Precision Valuation: A Discussion of How Value Assessment Frameworks Can Account for Personalized Medicine Payers control access to personalized medicine, and some have begun to take an interest in findings from value assessment frameworks that are challenged to account for developments in the field. In addition to exploring their potential impact on individualized care, this session will examine how value assessment frameworks can and should consider personalized medicine as part of their processes for evaluating therapeutic options.

MODERATOR | Jennifer Snow, M.P.H., Director, Health Policy, Xcenda, AmerisourceBergen

• Quality Era moved to Value Era

 

Dane J. Dickson, M.D., CEO, Founder, CureOne (formerly MED-C); Director, Precision Medicine Policy and Registries, Knight Cancer Institute at Oregon Health and Science University

Molecular Era

• NEJM, 2017, 377, 1813-1823
• BRAF in Melanoma – 80% do not need additional therapy vs 20% benefitted in the Non-Molecular Era

data by Dane J. Dickson, M.D., CEO, Founder, CureOne (formerly MED-C); Director, Precision Medicine Policy and Registries, Knight Cancer Institute at Oregon Health and Science University

 

Robert Dubois, M.D., Ph.D., Executive Vice President, Chief Science Officer, National Pharmaceutical Council

• Value Assessment and PM: ACC, ASCO, ICER< Memorial Sloan,
• The patient: survival, QOL, Adverse events, Out of pocket cost, extra survival by disease, treatment burden,
• PAYERS: One size does not fit all – AVERAGE is meaningless
• MDs vs Patients – are different in preference

Andrea Stern Ferris, M.B.A., President, Chairman of the Board, LUNGevity Foundation

• PATIENT to be included in the conversation

Steven D. Pearson, M.D., M.Sc., Founder, President, Institute for Clinical and Economic Review (ICER)

• Precision Medicine vs Value Assessment
• Novartis CAR-T Kymriah:  relapsed B-cell precursor ALL under 25 – 5 yr survival – 10%
• Changes associated with Gene therapies: single arm trials, surrogate outcomes, less certainty safety and benefits
• Gene therapy – >> innovations Kymriah – $475,000 price
• Long term value for money vs Short term affordability

PRICE-based on Value – discount from prices after rebate to meet ICER value-based Price range

• More targeted = higher value more favorable cost-effectiveness
• Rare/ultra-rare populations: broader value range:

1. Use EARLIER
2. will it work for patients without genetic marker?

• Years of Life weighted by an INDEX of quality of life (1=perfect health;  0=dead)
• willingness to pay: WHO and ACC: 1-3x
• individual x2 salary
• Opportunity cost x1 per capita GDP in UK
• Future of value assessment and precision medicine
• CURES – CAR-T are they cures???
• A teen-ager’s Value-based Price: $475,000 x years lived suggestsSteven D. Pearson, M.D., M.Sc., Founder, President, Institute for Clinical and Economic Review (ICER)

4:30 pm The Utility Proposition: An Analysis of Case Studies in the Economic Value of Personalized Medicine Although personalized medicine’s proponents contend that the field can deliver economic value by helping doctors avoid prescribing costly but ineffective therapies, the field lacks literature testing that hypothesis. This session will highlight recent studies on the clinical and economic value of personalized medicine, shedding light on what we know about personalized medicine’s clinical and economic utility — and what we don’t.

MODERATOR | Michael Pellini, M.D., M.B.A., Chairman, Board of Directors, Foundation Medicine; Board Member, Personalized Medicine Coalition

• we know there is value in Personalized Medicine
• we need to work together to acknowledge the challenges — to prove the value in PM

Lincoln Nadauld, M.D., Ph.D., Executive Director, Precision Medicine, Precision Genomics, Intermountain Healthcare

• Interpretation by Medical Oncologists beyond: KRAS, BRAF
• Measuring the value and presenting that to the payers and inside the organizations
• 2013 –

David B. Roth, M.D., Ph.D., Simon Flexner Professor Chair, Pathology and Laboratory Medicine, Perelman School of Medicine at University of Pennsylvania; Director, Penn Center for Precision Medicine

• 5000 patients underwent genome sequencing
• Interpretation is the issue that is hard
• Health IT are still in silos: Pharmacy data, financial data, EMR are not integrated yet
• Survival of patient with mutation and targeted drug LIVE LONGER

Lotte Steuten, Ph.D., M.Sc., Associate Faculty Member, Hutchinson Institute for Cancer Outcomes Research (HICOR), Fred Hutchinson Cancer Research Center; Affiliate Associate Professor, Pharmaceutical Outcomes Research and Policy Program, School of Pharmacy at University of Washington, Seattle

• aggregate big data , models as evidence, has value to clinical, the model under development NGS Profile of Patient vs current standard of care. Model Payer advisor committee, Oncologist Advisory Committee great benefit to PM on the cost side data is for targeting treatment using the promise of PM

5:45 pm Elements Café Cocktail Reception