Planning for the Expansion of Precision Medicine · PDF file · 2018-02-23Molecular diagnostics analysis solution Precision medicine EMR ... market 2011 20162012 2013 2014 2015 Prenatal

Planning for the Expansion of Precision Medicine

Tim Remus, PhDSenior Director, Sg2

Confidential and Proprietary © 2018 Sg2 2

Precision Medicine Adoption Technology Spotlight: Gene TherapyIntegrating Precision Medicine

3Confidential and Proprietary © 2018 Sg2

Different Name, Same Definition

Aka; DNA-Based, Genomic or Molecular Medicine

Personalized Medicine:An emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person.

-National Institutes of Health

Precision Medicine: An emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person.

-National Institutes of Health


Consumers Expect An Increasing Role for Genetic Testing but Worry About Interpretation and Resources

In 5 years, what change do you expect for the use of genetic testing?

Source: UnitedHeath. Personalized Medicine: Trends and prospects for the new science of genetic testing and molecular diagnostics. March 2012.

Primary consumer concerns:─ Physician ability to interpret test results─ Confidentiality of test results and possible discrimination─ Better education and resources about the risks and benefits

How familiar are you with genetic testing?


Physicians View Genetic Testing As Essential; Despite Challenges, Genetic Services Continue to Grow

Source: UnitedHeath. Personalized Medicine: Trends and prospects for the new science of genetic testing and molecular diagnostics. March 2012 The Association of Community Cancer Program. 2015 Oncology Trends Report.

5875

47

78 7260

83 8172

Dx Testing Offered Genetic Counseling Offered Molecular Testing Offered2013 Survey 2014 Survey 2015 Survey

Do you have patients that would benefit from genetic testing?

26%

28%

35%

42%

Provider Education/Awareness

Patient Education/Awaress

Resource Intensive

Reimbursement

Implementation challenges:

75%25%

Yes No

…however, physicians report <10% of their patients having received a genetic test


Personalized Medicines comprise: 27% of the drugs approved

by the FDA in 2016 42% of all medicines in

development 73% of cancer therapies in

development

Pharma Is Betting On Precision Medicine;Integration Into Clinical Practice in Slow

Sources: Pharmaceutical Research and Manufacturers of America. Medicines in Development for Cancer: From Hope to Cure. 2015 Report.; The Personalized Medicine Report, Personalized Medicine Coalition, 2017 Report; FDA. Table of Pharmacogenomic Biomarkers in Drug Labeling. Accessed June 2016.

“… despite the steady increase in the number of clinically useful molecular diagnostics and targeted therapies, healthcare systems have been slow to

integrate personalized medicine into clinical practice.”

0%

25%

50%

75%

100%

FDA-Approved Drugs With Pharmacogenomic Labeling

Medicine (56)

Cancer (41)

Neurosciences (32)

Cardiovascular (10)


21

21

66

42

57

92

Strategy to enhance PM

Budget for PM

Offer PM services

Current Within 2 Years

HIMSS 2017 Precision Medicine Survey:

HIMSS 2017 Precision Medicine Survey

31%

31%

32%

33%

37%

41%

Pharmacogenetics

Cardiology*

Pharmacogenomics

Prenatal screening

Neurology

Oncology

Current Areas of Focus:

Source: Future Proofing Healthcare: Precision Medicine, conducted by HIMSS Analytics, on behalf of Intel, September 2017.


Essential to Support Precision Medicine

20

22

23

26

38

43

51

45

34

41

41

41

32

27

Precision medicine platform

Molecular diagnostics analysis solution

Precision medicine EMR

Outside storage/computing capabilities

Analytics/data-mining platform

Laboratory IT

Data warehouse

Current Within 2 Years

Challenges Around Precision Medicine

72%

Improvement of IT

Adequacy of existing IT

Budget/financial

Unprepared Moderately prepared Prepared

UnsureInvestUse existing IT

HIMSS 2017 Precision Medicine Survey

Source: Future Proofing Healthcare: Precision Medicine, conducted by HIMSS Analytics, on behalf of Intel, September 2017.


Precision Medicine Adoption Technology Spotlight: Gene Therapy

Integrating Precision Medicine

AVAILABLE DEVELOPING RESEARCH


Which


First FDA-approved test

Cancer Moonshot calls for “efforts to develop noninvasive tests”

Liquid Biopsy Tests Are Now ‘Mainstream’

CURRENT IMPACT

Noninvasive Inform treatment Repeatable

40 companies

$30B(US) market

2011 20162012 2013 2014 2015

Prenatal diagnostic testing

Initial liquid biopsy tests for lung cancer

Source: Chen C. Bezos, Gates chase dream of a blood test that detects cancer. Bloomberg: April 6, 2016.

AVAILABLE


How Will Liquid Biopsies Impact Lung Cancer Therapy?

SBRT = stereotactic body radiation therapy. Sources: Impact of Change®, 2017; HCUP National Inpatient Sample (NIS). Healthcare Cost and Utilization Project (HCUP) 2014. Agency for Healthcare Research and Quality, Rockville, MD; OptumInsight, 2015; The following 2015 CMS Limited Data Sets (LDS): Carrier, Denominator, Home Health Agency, Hospice, Outpatient, Skilled Nursing Facility; Claritas Pop-Facts®, 2017; Sg2 Analysis, 2017.

Growth in Lung Surgery Sg2 projects 27% growth in

lobectomy (10-year).

Growth in Lung Cancer Treatment

Targeted Therapies Sg2 projects 19% growth

in chemotherapy (10-year).

Radiation Therapy Sg2 projects 72% growth

in SBRT (10-year).


Procedures Infusion and Transfusion Volumes at Risk

Pharmaceutical Companies Biologic Drug Market

Payers High Cost Cures Compromise Margins and Sustainability

Gene Therapy is Now ‘A Thing’: How Did This Happen

Initial Failures

1990s

VC Influx

2018

Dubious BenefitToxicitiesDeaths

Quiet Progress

2000-2016

SafetyEfficiencyDelivery

FDA Approvals

2017

CAR-TBlindnessClinical Trial Successes $Many Trials

More MoneyDemand

IMMINANT DISRUPTION TO:


Gene Therapy Will Disrupt 3 Areas

Inherited Conditionshemophilia congenital blindness

Acquired Conditionscancer/car-Tchronic diseases

Augmentative Therapies‘biohacking’, DIY science

AVAILABLE DEVELOPING RESEARCH

Emerging Opportunities for Providers:Expansion of Minor Procedures and BMTPrecision Medicine can Identify/Screen Candidates‘Impatient’ patients will develop complications from unregulated therapies

Sources: DIY = “Do-It-Yourself”. This list is not meant to be exhaustive; “Trials Forbidden” = means ethically/officially forbidden to use public funds to research; number of clinical trials reported by the Alliance for Regenerative Medicine, an Industry group, Sg2 Analysis 2018.

>550 clinical trials

Trialson hold


DescriptionEnable the immune systems ability to attack B-cell malignant tumors by infusing genetically engineered T-cells back into the patient

What Is the Current Impact? Novartis – ALL; Kite (Gilead) – NHL both FDA Approved Numerous trials in CLL, MM, AML Pending long-term response rates; maybe an alternative to BMT

Novartis and Kite Break Ground with FDAApproved CAR T-cell Therapies

ALL = acute lymphoblastic leukemia; CLL = chronic lymphocytic leukemia; NHL = non-Hodgkin lymphomas; MM = multiple myeloma; AML = acute myelogenous leukemia. Source: American Cancer Society. 2017 Facts and Figures; Sg2 Impact of Change Forecast, 2017.

Tumor Type Annual Incidence

2017 IP/OP BMT Volume

% of Total BMT Volume

Leukemia’s 62,130 4371 28%Hodgkin’s Lymphoma 8,260 842 5%Multiple Myeloma 30,280 6445 42%Non-Hodgkin’s Lymphoma 72,240 3320 21%TOTAL 172,910 15,509 100%

Activated T Cell

Tumor Cell

>50% of BMT volume

AVAILABLE


Roadblocks to Solid Tumor Adoption Are Not Trivial Finding tumor specific (ie, unique) antigens and tumor heterogeneity Penetrating immunosuppressive microenvironment Delivering T-cells directly to the tumor and extending T-cell persistence

Potential Impact on Future Chemo and Radiation Therapy Volumes

Source: www.ClinicalTrials.gov.

Tumor Type Number of Clinical Trials Phase % of Total

Chemotherapy % of Total

Radiation Therapy

Gyn Onc 10 Phase I/II 5% 3%Head and Neck 2 Phase I/II 2% 9%Pancreatic 15 Phase I/II 4% 1%

Prostate 3 Phase I/II 8% 22%

Colorectal 5 Phase I/II 12% 4%Liver 18 Phase I/II 1% 2%

DEVELOPING

Breast 13 Phase I/II 17% 22%

Lung 12 Phase I/II 10% 9%

Brain 9 Phase I/II 1% 5%

What Is the Future Impact?


What Does Gene Therapy For HemophiliaLook Like?

Procedural Disruption: TransfusionsInfusion volumes

1. Precision medicine identifies patients2. Design DNA 3. Package DNA into virus4. Intravenous Injection

5. DNA delivered to liver cells6. Cells make DNA into protein7. Protein secreted into blood8. No need for bi-weekly biologic infusions

>$155,000 average annual cost per hemophilia patient DISRUPTED

Sources: Cell. Volume 171, Issue 7, p1478–1480, 14 December 2017; Am J Manag Care. 2016;22:S126-S133; Sg2 Analysis 2018.

DEVELOPING


Biblical Treatments, Goliath Costs

$850,000 Luxturna (for genetic blindness), one-time treatmentFDA Approved December 19th, 2017

$300,000 Eteplirsen (for Duchenne muscular dystrophy), per year$373,000 Yescarta (CAR-T for blood cancer), one-time drug payment$475,000 Kymriah (CAR-T for blood cancer), one-time drug payment$648,000 Strimvelis (ultra rare immune deficiency), per cure (Europe)$750,000 Spinraza (for spinal muscular atrophy), per year

$1,000,000 Glybera (ultra rare blood disease), per cure (Europe)


Description Genomic scalpel that cuts or repairs DNA at specific places

Easy to break genes; Feasible to rewrite genes

Impact Improved speed, precision, and ease raise ethical concerns Advances in basic biomedical research and drug discovery Future Impact: Advancement will initially disrupt health care in

non-obvious ways

CASE EXAMPLE: Scripps and Harvard Successful ‘humanization’ eliminated 62 pig viruses from pig

organs (kidney) using CRISPR Off-target effects were minimal; Need to make transgenic pigs!

New Genome Editing Tool Is Quickening the Pace of Research and Drug Discovery

CRISPR Technology (pronounced “CRISP·er”)

RESEARCH


Gene Therapy Strategic Points

What should you do to prepare?

What will the effect be on volumes?

How will you pay for these therapies?


Precision Medicine Adoption Technologies Spotlight: Gene TherapyIntegrating Precision Medicine


Challenge 1: Connect an Ecosystem Around Your Electronic Health Record

Sylvester Cancer Center, University of Miami Miller School of Medicine

SITUATION Providers lack the infrastructure to easily

order, store, and share test results EHRs are not designed to manage or

query genetic information; interoperability has not happened

SOLUTION – SYAPSE Connects clinical, molecular, and

outcomes data into the EHR Facilitate molecular tumor boards

SYLVESTER CC RESULTSModified treatment: ~30% Clinical trial enrollment: +1–2

patients/month

Sources: Trent J. Oncology Issues 2017; Nov/Dec:16-20; .

IMPACT OF INTEGRATION Intermountain Healthcare: doubled

progression free survival without increase total cost of care

Improved access to targeted therapies


Challenge 2: Precision Medicine Programs Must Prepare for Growing Data Management Requirements

Countless number of

data sources and formats.

Integration of patient-generated

dataNeed a data management

strategy

Dedicated IT Infrastructure

Source: Sg2 Analysis, 2018.


Challenge 3: How Do Providers Afford Precision Medicine?

Product Champion Pro forma Partnerships Philanthropy Pro bonoChallenge to AMCs: Payers are unconvinced of the benefits of PM; evidence demonstrating its value to healthcare is needed AMCs are positioned to conduct studies to demonstrate value

Design clinical studies to serve multiple purposes such as including the cost and benefits of coverage decisions in areas of unmet need

Develop an effective, universally accepted and user-friendly process to systematically collect and share treatment and outcomes data


First round• Compassionate use (payers more likely to bend to advocacy pressure)• Rare diseases (orphan drug status confers benefits) • Small markets (once approved, huge barriers to entry, trials harder)

Sells to insurer, not hospital (prevents ‘buy and bill’ practices)

Money back guarantees (outcomes-based rebate agreements”)

Financing options (payment spread over several years)

High Costs Are Changing PhRMA’s Pricing Strategy


Questions

Documents

Planning for the Expansion of Precision Medicine · PDF file · 2018-02-23Molecular diagnostics analysis solution Precision medicine EMR ... market 2011 20162012 2013 2014 2015 Prenatal