Session 95 PD, Pharmaceutical Manufacturer Perspectives
Moderator:
Gregory L. Warren, FSA, MAAA, FCA
Presenters:
Martin Marciniak, Ph.D.
Vipan Sood, RPh, MBA, MRPharmS
Nicole Yurgin, Ph.D.
LINK BETWEEN
VALUE, QUALITY
AND OUTCOMES
Society of Actuaries
2015 Health Meeting
June 17, 2015
Martin Marciniak, Ph.D.
Vice President,
GlaxoSmithKline
THIS DISCUSSION REFLECTS MY OPINIONS
AND DOES NOT REFLECT THOSE OF GSK OR
ANY ASSOCIATED ORGANIZATIONS.
SHIFT TO VALUE
Today
Tomorrow
Volume Driven Model
Value Driven Model
Fee for Service
 Visits
(FFS)
 Procedures
 Patients
 Episodes
 Fixed costs
 Variable costs
 Patient satisfaction
 Resource/case
 Outcomes
 Profit
 Profit
Per Diems
Case rates
Readmission
Bundled
Payments
Fee for Value
(FFV)
ACOs – Other Valuebased Delivery Systems
The market is shifting to value-based payment models where delivery and
payment will be based on the quality of outcomes
SHIFT TO VALUE REQUIRES PAYMENT AND DELIVERY REFORM
Transparency: Collecting and
reporting quality and cost data
to consumers enables informed
decision-making
HIT: Use of HIT tools facilitates
care integration, information
collection and improves quality
and efficiency
Delivery System
Measurement: Linking payment
to quality of care requires
improved measurement and
evaluation framework
HIT /
Supports
Incentivizes
Transparency /
System
Infrastructure
Measurement/
Evidence /
Payment Structure
Evidence: New evidence from
comparative effectiveness
research helps define delivery
and payment patterns
New payment models require a shift in the way providers deliver care: Value-based delivery models
create the relationships and structure for sharing payments and coordinating care that will be
necessary under the new payment models
Presentation title
5
Presentation title
6
HEALTHCARE REFORM PROMOTES THE ADOPTION OF HIGH
VALUE CARE, DETERMINED BY ASSESSING BOTH QUALITY
AND COST
Quality
Value
Cost
•
Provisions in the
Affordable Care Act place
a greater emphasis on the
value of care provided
•
Value incorporates both
quality and cost
components, with the goal
of promoting better care at
a lower cost
•
Several drivers within the
Act influencing provider
behavior center on this
idea of providing high
value care
7
Presentation title
HOW HEALTH OUTCOMES STUDIES ARE VALUED
IN QUALITY MEASURE DEVELOPMENT
8
COMPARISON OF NQF #1799 AND #1800
1799: Medication Management for People
with Asthma
1800: Asthma Medication Ratio (AMR)
NCQA, NQF endorsed
NCQA, NQF endorsed
HEDIS, QRS (State Exchange)
HEDIS
The percentage of patients 5-64 years of age during
the measurement year who were identified as
having persistent asthma and were dispensed
appropriate medications that they remained on
during the treatment period. Two rates are
reported: (1) The percentage of patients who
remained on an asthma controller for at least 50%
of their treatment period and (2) The percentage of
patients who remained on an asthma controller for
at least 75% of their treatment period
The percentage of patients 5–64 years of age who
were identified as having persistent asthma and had
a ratio of controller medications to total asthma
medications of 0.50 or greater during the
measurement year.
Numerator:
Medication compliance of 50% and 75% as
measured by PDC (proportion of days covered by at
least one asthma controller medication prescription,
divided by the number of days in the period)
Numerator:
The number of patients who have a medication ratio
of 0.50 or greater during the measurement year.
Denominator:
Patients 5-64 years of age during the measurement
year who were identified as having persistent
asthma.
Denominator:
Patients 5–64 years of age during the measurement
year who were identified as having persistent
asthma.
1. National
Quality Forum. http://www.qualityforum.org/Home.aspx. Accessed October 1, 2014.
9
LITERATURE REVIEWED
Broder et. al.5
Schatz et.
Description of
difference
inpatient
characteristics
and resource use
in patients with
high ratio (>0.5)
versus low ration
(<0.5), AMR,
5-56 year olds
Mixed
(2010)
al.1-3
Comparison of
asthma quality
measures
(including AMR
and HEDIS Fill)
18-56 year olds
Commercial
(2005, 2006,
2008)
Samnaliev et. al.4
Comparison of
AMR and HEDIS
adherence
measures
18-56 year olds
Medicaid
(2009)
Schatz et. al.6
Comparison of AMR
and various HEDIS
adherence measures
18-56 year olds
Mixed (Kaiser)
(2010)
Stanford et. al.7
Comparison of AMR in
various plan types and
by age,
4-17 years old & >18
years old,
Medicaid & Commercial
(2013)
NQF #1799
NQF#1800
Endorsed
July 31, 2012
AMR = Asthma Medication Ratio, HEDIS = Health Effectiveness Data and Information Set, NQF = National Quality Forum
Engelkes et. al.8
Systematic
literature review
examining
association
between asthma
controller
therapy
adherence and
serious
exacerbations
(2015)
1. Schatz, M, et al. Chest 2005;128,1968-1973. 2. Schatz M et al Chest 2006;130:43–50. 3. Schatz M and Stempel D. Ann Allerg Asthma Immunol. 2008;101(3):235-239. 4. Samnaliev M, et al.
Chest 2009;135:1193-1196. 5. Broder et al. Am J Manag Care. 2010;16(3):170-178. 6. Schatz, M et al. Am J Manag Care. 2010;16(5):327-333. 7. Stanford, R et al. Am J Manag Care. 2013;19(1):6067. 8. Engelkes M et al. Eur Respir J. 2015 Feb;45(2):396-407.
10
STUDY DESIGN
Study Period
Enrollment Period
12/01/2009
12/01/20102
1-year
pre-index period
12/31/2013
12/31/2011
15 -24 months post-index period
Index RX Date
3 -12 months marker definition
period
•Controller to total asthma
medication ratio
•Albuterol use
ED = Asthma-related Emergency Department visit
Data on File 2015N236368_00
ICU = Asthma-related Intensive Care Unit visit
12 months outcomes
assessment period
Hospitalizations, ED, ICU, OCS
OCS = Asthma-related Outpatient visit with Filled Oral
corticosteroid prescription
11
RISK OF AN ASTHMA RELATED EVENT
OR’s for cutoff >= 0.5 AMR and 50% PDC
Asthmarelated Event
Any event
ED/IP
IP
ED visit
Marker Periods
3-month
6-month
9 month
ORs (95% CIs)
ORs (95% CIs)
ORs (95% CIs)
CONTROLLER TO ASTHMA MEDICATION RATIO
0.390 (0.309 – 0.492)
0.403 (0.265 - 0.611)
0.477 (0.281 - 0.811)
0.294 (0.173 – 0.500)
0.393 (0.311 – 0.497)
0.348 (0.228 – 0.531)
0.334 (0.199 – 0.561)
0.374 (0.212 – 0.662)
0.341 (0.268 – 0.433)
0.304 (0.198 – 0.465)
0.290 (0.171 – 0.492)
0.320 (0.181 – 0.567)
12-month
ORs (95% CIs)
0.336 (0.264 – 0.427)
0.349 (0.229 – 0.533)
0.338 (0.201 – 0.569)
0.341 (0.194 – 0.601)
PROPORTION OF DAYS COVERED
Any event
ED/IP
IP
ED visit
1.057 (0.771 – 1.450)
1.361 (0.761 – 2.433)
1.550 (0.745 – 3.226)
1.138 (0.532 – 2.438)
1.201 (0.830 – 1.738)
1.204 (0.612 – 2.366)
1.817 (0.764 – 4.318)
0.940 (0.389 – 2.270)
0.966 (0.683 – 1.365)
1.050 (0.562 – 1.961)
0.950 (0.436 – 2.068)
1.123 (0.491 – 2.568)
1.047 (0.711 – 1.542)
0.874 (0.448 – 1.705)
1.046 (0.453 – 2.411)
0.681 (0.283 – 1.635)
– Any event = Combined IP/ED visit/outpatient visit with OCS fill within +/- 7 days
– Logistic regression models include baseline demographic characteristics
– Age
– Gender
– Baseline ED event
– Baseline hospitalization
AMR = Asthma Medication Ratio, PDC=Proportion of Days Covered, ED = Asthma-related Emergency Department visit, IP = Asthma-related Inpatient visit , OR = Odds Ratio, OCS = Oral
corticosteroids
Data on File 2015N236368_00
12
DISCUSSION POINTS


AMR was observed in this population of patients aged 65 years or older
to perform in a similar fashion as what has been observed in the original
AMR validation studies
PDC was not observed to be a significant predictor of asthma related
outcomes


PDC was calculated as per HEDIS and PQA recommendations
The methodology does not match past observational studies that have
observed a benefit of PDC on asthma related events.
Data on File 2015N236368_00
13
Presentation title
HOW HEALTH OUTCOMES STUDIES ARE
EVOLVING: THE EMERGENCE OF PRAGMATIC
CLINICAL TRIALS
14
“EROOM’S LAW”
Source: Scannell et al Nature Reviews Drug Discovery 2012, 11, 191
THE OPPORTUNITY FOR EFFECTIVENESS RESEARCH
IN SALFORD AND BEYOND
The Salford Lung Study is the world's first pragmatic
randomised clinical trial initiated in a pre-licence setting
16
SCALE OF THE PROJECT
88 GP
sites
128
community
pharmacies
2800 COPD and 1425
asthma subjects recruited
Bespoke eCRF
and data
monitoring system
designed, built
and working
specialist
safety team
covering 2
hospitals
Over 300
study staff
Over 3000 GP and
pharmacy staff
trained in GCP and
research-ready
17
ELECTRONIC CLINICAL MONITORING
>300
users
54,560
radiology
results
51,940
patient visits
15 data feeds per subject
4.97 million
medications
processed
9072
event
alerts in
last
12mnths
2 million
clinical
observations
>50 million rows of data
977
SAE
reports
2.8 million
biochemistry and
haematology results
18
SUMMARY


The Salford Lung Study is the first of its type in the
world
Maintains scientific rigour
•
•
•

randomised,
active control
robust primary endpoint
It has, and continues to be an enormous logistical
effort
But.....
 It will offer important information for clinicians,
healthcare decision makers and most especially
patients
 And will provide valuable information about how to
conduct real-world effectiveness studies in future
19
WHAT IS THE LINKAGE?
The U.S. is shifting to a value driven payment
model
 Increasing importance on quality of care
measures – including patient centered
outcomes
 Changing U.S. health care environment is
leading to different ways to approaches in study
development and increased data

Using Health Economics
and Outcomes Research
to Assess the Value of
New Drugs
An Example from Hepatitis C Virus
Vipan C. Sood RPh, MBA, MRPharmS
Vice President HEOR
Virology/Endocrinology/Renal
AGENDA
1. Overview of Hepatitis C Virus (HCV)
2. Role of Health Economics and
Outcomes Research (HEOR)
3. Late Phase HEOR Activities: A Case
Study from HCV
1. Overview of
Hepatitis C Virus (HCV)
Hepatitis C Virus Overview
•
Hepatitis C is a liver disease resulting from infection with the Hepatitis C virus (HCV)1
•
Severity can range from mild illness
to a serious, lifelong illness1
•
Main routes of transmission2
~3.2 M infected
individuals in the
US alone4
– Unsafe injections, inadequate sterilization
of medical equipment, blood transfusion, sexual transmission
•
Long-term complications of HCV include cirrhosis, hepatocellular carcinoma,
and death3
– HCV is the leading cause of liver transplantation in the US
– Treating 5% of the HCV+ population avoids the need for 600-1,500 liver
transplants
•
Sustained Virologic Response (SVR) following completion of treatment is reflective of
a cure3
1.
2.
3.
4.
Centers for Disease Control and Prevention. Hepatitis C for the Public. http://www.cdc.gov/hepatitis/C/ Accessed on 5/30/15.
U.S. Department of Veterans Affairs. Viral Hepatitis. http://www.hepatitis.va.gov/provider/reviews/transmission.asp Accessed on 5/30/15.
Hepatitis C: The Basics. http://www.hepmag.com/articles/2512_18755.shtml. Accessed on 5/30/15.
Centers for Disease Control and Prevention. Hepatitis C Information for Health Professionals.
http://www.cdc.gov/hepatitis/HCV/HCVfaq.htm#a4. Accessed on 5/30/15
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
4
HCV Disease Burden and Costs are Rising
Prevalence
(95% CI)
4.5
US Prevalence (millions)
3.5
$16
$12
3.0
2.5
$8
2.0
1.5
$4
1.0
0.5
0.0
1950
1960
1970
1980
1990
2000
2010
2020
Healthcare Cost ($ billions)
4.0
Health Care Cost
(95% CI)
$0
2030
US HCV prevalence is in decline, however prevalence of advanced liver
disease will continue to increase, as well as corresponding healthcare costs
Razavi H, et al. Hepatology. 2013;57:2164-2170.
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
5
History of HCV Therapies
Traditional Standard of Care
New Direct Acting Antivirals (DAAs)
1st Generation DAA
2nd Generation DAA
1998
2001
2011
2013
2014
IFN + RBV1
PegIFN + RBV2
TVR +
PegIFN + RBV3
SMV +
PegIFN + RBV5
LDV/SOF7
BOC +
PegIFN + RBV4
SOF +
PegIFN + RBV6
OBV+PRV/r+DSV8
Three times a
week injection
Weekly injection
Improved efficacy, safety, and tolerability
IFN=interferon; RBV=ribavirin; PegIFN=pegylated interferon; TVR=telaprevir; BOC=boceprevir; SMV=simeprevir; SOF=sofosbuvir; LDV=ledipasvir;
OBV=ombitasvir; PTV/r=paritaprevir/ritonavir; DSV=dasabuvir
1.
2.
3.
4.
5.
6.
7.
8.
Rebetron FDA Approval. https://www.centerwatch.com/drug-information/fda-approved-drugs/drug/442/rebetron-tm-combination-therapy
Peg-Intron FDA Approval. https://www.centerwatch.com/drug-information/fda-approved-drugs/drug/697/peg-intron-peginterferon-alfa-2b
Incivek FDA Approval. http://investors.vrtx.com/releasedetail.cfm?ReleaseID=580154
Victrelis FDA Approval. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm255390.htm
Olysio FDA Approval. http://www.jnj.com/news/all/OLYSIO-simeprevir-Receives-FDA-Approval-for-Combination-Treatment-of-Chronic-Hepatitis-C
Sovaldi FDA Approval. http://www.gilead.com/news/press-releases/2013/12/us-food-and-drug-administration-approves-gileads-sovaldi-sofosbuvir-for-the-treatment-ofchronic-hepatitis-c
Harvoni FDA Approval. http://www.gilead.com/news/press-releases/2014/10/us-food-and-drug-administration-approves-gileads-harvoni-ledipasvirsofosbuvir-the-firstoncedaily-single-tablet-regimen-for-the-treatment-of-genotype-1-chronic-hepatitis-c
Viekira Pak FDA Approval. http://abbvie.mediaroom.com/2014-12-19-AbbVie-Receives-U-S-FDA-Approval-of-VIEKIRA-PAK-Ombitasvir-Paritaprevir-Ritonavir-Tablets-DasabuvirTablets-for-the-Treatment-of-Chronic-Genotype-1-Hepatitis-C
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
6
Higher SVR and Costs with DAAs
SVR
100
90.3
96.4
15000
Cost/week, $
SVR, %
92.3
74.5
80
60
Cost/week
49.4
40
12000
9000
3000
0
0
1st generation
DAA + PR
12000
6900
6000
20
PR
12200
2nd generation
DAA + PR
2300
900
2nd generation
DAA + RBV
Two 2nd generation
DAAs (no PR)
PR=pegylated interferon; DAA=direct acting antiviral; RBV=ribavirin
Bansal S, et al. World J Hepatol. 2015;7:806-813.
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
7
Controversy Over Costs of HCV Treatment
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
8
Summary
• HCV is a disease with significant downstream morbidity and mortality
• Due to the availability of new therapies, the cure rate (SVR) has increased
significantly
• Costs of therapy may seem high, but there is value to the health system
and society in the long term
• Health Economics and Outcomes Research (HEOR) generates evidence to
assess the value of new drugs
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
9
2. The Role of Health
Economics and
Outcomes Research
(HEOR)
The Role of HEOR in Assessing a Drugs’ Value
Greater Scrutiny of Costs with the Rising Rate of
Health Care Expenditures
US Health Expenditures by Year
National Health Expenditures, Billions ($)
$3,500
$3,000
$2,500
$2,000
$1,500
$1,000
$500
$0
1960 1970 1980 1990 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013
Centers for Medicare and Medicaid Services. National Health Expenditure Data. Table 01. National Health Expenditures.
http://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-andReports/NationalHealthExpendData/NationalHealthAccountsHistorical.html Accessed 5/30/15.
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
11
If You Can’t Pay for Everything, What Do You Pay For?
Vaccination
Heart Surgery
The Ones
That Bring
the Most
Value!
Dialysis
Cancer Treatment
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
12
Drug Approval Does Not Guarantee Patient Access and
Reimbursement; Value Must Be Demonstrated
Marketing
Approval
Reimbursement Considerations
Patient
New Drug
Drug Approval Requirements
All of these components make up a
drugs value
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
13
The Role of HEOR in Assessing a Drugs’ Value
LoE
Product Launch
HEOR INPUT
L&A/Drug Discovery Clinical Development - Phase I–III
Assessing unmet need
1- Incidence/ prevalence
2- Epidemiology
3- Burden of disease
Trial Design
4- Patient-reported
outcomes
Marketed Product
Preparing For Launch Real World Effectiveness
8- Medical chart
reviews
5- Cost effectiveness
9- Prospective
observational studies
6- Budget impact
7- Long-term outcomes
10- Retrospective claims
HEOR=health economics and outcomes research; L&A=licensing and acquisition; LoE=loss of exclusivity
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
14
Summary
• With rising health care expenditures, there is increased scrutiny on costs
• HEOR uses rigorous methodology to establish evidence to demonstrate
a drug’s value through
• Budget impact models
• Cost-effectiveness studies
• Modeling long-term outcomes
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
15
3. Late-Phase HEOR
Activities: A Case Study
from Hepatitis C Virus
(HCV)
Examining budget impact, cost-effectiveness
and long-term health outcomes with new
treatments
Budget Impact Model
Introduction
Key Question
• What is the incremental impact on health care (and/or pharmacy)
budget for patients in a health plan if a new HCV regimen is
included?
Analytical Approach
• Budget impact is the difference between health care spending in
world with a new HCV regimen vs. the world without a new HCV
regimen
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
17
Example of Budget Impact of New HCV Regimen
Without New HCV Regimen
$40
Millions ($)
With New HCV Regimen
$30
$20
$10
$0
Without
With
Year 1
Without
With
Year 2
Without
With
Year 3
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
18
Cost-Effectiveness
Introduction
Key Question
• Is the value of a treatment worth its cost?
Analytical Approach
• CEA is an assessment of a therapy’s value-for-money
• Compares costs and health effects of an intervention to assess
whether it can be regarded as providing value for money
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
19
Cost-Effectiveness
Introduction
Quality-Adjusted Life Year (QALY)
• A standard measure of treatment benefit and consequence that
is represented in a common unit that can be compared across
disease stages and other interventions
• A QALY represents one year of life lived in perfect health (i.e.
without illness)
Quality of Life
1
0
QALYs without treatment
Improvement
in quality of
life with
treatment
Length of life (years)
HCV=hepatitis C virus; HCC=hepatocellular carcinoma
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
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Cost-Effectiveness
Introduction
Incremental Cost-Effectiveness Ratio (ICER)
• ICER measures the additional cost needed to achieve one QALY
with a treatment vs a comparator
• A cost-effectiveness threshold is needed to determine whether a
treatment option is cost-effective or not
• ICER < threshold suggests therapy is cost effective
• Most conservative, $50K/QALY and commonly used in US/UK
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
21
Cost-Effectiveness of All-Oral DAAs Compared to
No Treatment
ICER
threshold
of $50,000
$50,000
ICER
$40,000
$30,000
$20,000
$10,000
$0
F0
F1
F2
Fibrosis stage
F3
F4
Compared to watchful waiting, all-oral DAAs are cost effective based
on a threshold of $50,000 per quality-adjusted life-year (QALY)
among treatment-naïve genotype 1 patients with any fibrosis stage
DAA=direct acting antiviral; F=fibrosis stage (0, none; 4, cirrhosis or advanced scarring of the liver)
Johnson S, et al. Poster presented at AMCP annual meeting, April 7-10, 2015, San Diego, CA, USA.
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
22
Modeling Long-Term Outcomes
Introduction
Key Question
• What are the long-term benefits of treatment vs. no treatment?
Analytical Approach
• Use of modeling techniques to simulate patient progression with
and without treatment
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
23
Long-Term Outcomes of Patients Treated With All-Oral
DAAs Compared to No Treatment
100
All-oral DAA
80
%
No treatment
78.3
60
40
36.7
30.2
16.7
20
6.8
0
Likelihood of
cirrhosis
Likelihood of
liver cancer
1.9
9.2
7.1
Likelihood of
liver transplant
Likelihood of
death
Compared to watchful waiting, all-oral DAAs have a
lower likelihood of long-term liver morbidity
DAA=direct acting antiviral.
Johnson S, et al. Poster presented at AMCP annual meeting, April 7-10, 2015, San Diego, CA, USA.
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
24
Long-Term Reduction in Average Annual Lifetime
Medical Costs with Treatment of GT1 Patients
$10
All-oral DAA
Thousands ($)
$8
No Treatment
$6
$4
$2
$0
F0
F1
F2
F3
F4
Stage of Disease at Treatment
In GT1 patients, overall lifetime medical costs associated with all-oral DAAs are
significantly lower than those associated with no treatment
DAA=direct acting antiviral; F=fibrosis stage (0, none; 4, cirrhosis or advanced scarring of the liver); GT=genotype
Johnson S, et al. Poster presented at AMCP annual meeting, April 7-10, 2015, San Diego, CA, USA.
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
25
Summary
• Through this case study of HCV, HEOR generated evidence to demonstrate
the value of these new all-oral DAAs
• Budget-impact model: Higher cost of all-oral DAAs
• Cost-effectiveness model: All-oral DAAs are cost-effective based on ICER
threshold of $50,000
• Long-term model: All-oral DAAs lead to a reduction in downstream
morbidity, mortality, and post-treatment medical costs
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
26
Back Up
Looking Back at History
to Help Us Look Ahead
History of HIV
• Human immunodeficiency virus (HIV) was one of the most
devastating disease globally
• Highly active antiretroviral therapy (HAART) emerged in the
mid-1990s revolutionizing care
• Protests over the high price of HAART
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
30
HAART Had A Dramatic Impact on Survival
Philipson T and Jena AB. Who Benefits from New Medical Technologies? Estimates of Consumer and Producer Surpluses for
HIV/AIDS Drugs. Forum for Health Economics and Policy. 2006;9(2).
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
31
Most Of the Benefits of HAART Flowed to Patients
$63 Billion
5% of the value
creation was
returned to
innovators
$1.4 Trillion
Manufacturer revenues
Patient health benefit
Philipson T and Jena AB. Who Benefits from New Medical Technologies? Estimates of Consumer and Producer Surpluses for
HIV/AIDS Drugs. Forum for Health Economics and Policy. 2006;9(2).
Using Health Economics and Outcomes Research to Assess the Value of New Drugs | June 2015 | Company Confidential © 2015
32
Actuarial Science and Health Economics Outcomes Research
Building Bridges: Common Foundations, Divergent Approaches and Applications
June 17, 2015
Atlanta, Georgia
Gregory
Warren,
MAAA
Gregory
Warren,
FSA,FSA,
MAAA
President,
Actuarial
Consulting
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Consulting
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gregory.warren@optum.com
gregory.warren@optum.com
Actuarial Science and Health Economics & Outcomes Research
Building Bridges: Common Foundations, Divergent Approaches and Applications
Common Foundations
Calculus-Based
Statistical Theory
Measuring Results
Health Economic Impact
Dealing with Uncertainty
Divergent Approaches and Applications
Law of Large Numbers
(minimize statistical variation)
Characteristic-Matched Studies
(minimize confounding factors)
Estimate Confounding Factors
Eliminate Confounding Factors
Financial Outcomes
Clinical & Economic Outcomes
Book of Business Focus
Disease Focus
Identify Correlations
Identify Causations
Short/Intermediate-Term Horizons
Intermediate/Long-Term Horizons
Model-Building Experts
Proprietary and Confidential. Do not distribute.
2
Evaluation of Innovative
Cardiovascular Therapies
Nicole Yurgin, PhD
Executive Director, Global Health Economics
June 17th, 2015
The views expressed herein represent those of the
presenter and do not necessarily represent the views or
practices of the presenter’s employer or any other party.
2
We want meaningful innovations that
lead to better health outcomes
3
But budget constraints force tough
choices
Scarcity: Healthcare resources are limited and budgets
are constrained.
Choice: Must decide how best to fund resources to
maximize health.
Opportunity Cost: Resources deployed in one fashion
cannot be used in other ways – i.e. we give something up.
…given scarcity, choice and opportunity cost, we increasingly
need systematic ways to evaluate alternatives
A health economist considers both
value-for-money and affordability
• Economic evaluation – “the comparative analysis of
alternatives in terms of both costs and outcomes.”
• “…the basic tasks are to identify, measure, value, and compare
costs and outcomes of the alternatives”
• Perspective: typically societal and long term
• Typical activities for health economist:
•
•
•
•
•
•
Evaluate the current burden of disease
Describe areas of highest unmet need
Quantify potential value of innovative treatment
Identify the highest risk patients
Estimate value-for-money (cost-effectiveness)
Estimate affordability (budgetary impact)
Drummond et al. (1997) Methods for the Economic Evaluation of Health Care Programmes. 2nd Ed.
Cardiovascular Disease (CVD) has
received a lot of media attention lately
In The Debate About Cost And
Efficacy, PCSK9 Inhibitors May Be
The Biggest Challenge Yet
CVS Urges Cost Controls for New
Cholesterol Drugs
CVD impacts many people in the US, therefore both the costs
and the benefits for new therapies in this area could be large
CVD causes substantial healthcare
resource use and costs
CVD causes substantial healthcare resource use and costs
6M
1 out of 3
5M
In 2010, CVD lead to
almost 6M
hospitalizations and
stroke and heart
disease lead to 5M
disabilites
CVD accounted for 1 out of 3
deaths in the US each year
17
Years
Individuals with a fatal
heart attack lose an
average of 17 years
$320B
61%
65%
The total cost of CVD
is $320B annually 61%
of which were direct
costs which; the
highest of all major
diseases
65% of CVD costs
were due to heart
disease including
CHD
Mozaffarian, et al. Heart Disease and Stroke Statistics – 2015 Update: A Report from the American Heart Association. Circulation. 2015;131:e29–e322
Patients with low LDL-C have a lower
likelihood of having a major CV event
Patients with LDL-C levels of >100mg/dL are 4x more likely to have a major cardiac event
compared to patients with LDL-C levels of <50 mg/dL
Risk Estimates for Cardiovascular Events by
Category of Achieved LDL-C Level
Chance to experience Major
Cardiovascular Event (%)
35%
32.8%
30%
25%
22.0%
20%
44
15%
16.5%
16.5%
75-100
100-125
17.8%
11.4%
10%
5%
4.4%
0%
<50
50-75
125-150
150-175
>175
LDL-C Level
Boekholdt, et al. Very low levels of atherogenic lipoproteins and the risk for cardiovascular events: a meta-analysis of statin
trials. J Am Coll Cardiol. 2014: 64: 485-94.
Many patients at high risk for CVD
cannot reach target LDL-C levels
% Not Achieving LDL-C Treatment Goals
Approximately 30% - 87% of patients at high risk* for CVD cannot adequately lower their LDL-C
levels with statins and/or other available lipid-lowering agents to achieve therapeutic targets
Percent of Patients Not Achieving LDL-C Treatment Goals
100%
89%
87%
90%
78%
80%
77%
74%
73%
70%
59%
60%
56%
53%
50%
40%
39%
37%
33%
30%
36%
30%
20%
10%
0%
MI
CHD
Angina
Stroke
Patient Type
Goal of 100 mg/dL
Diabetes
Total
Secondary
Prvention
Primary
Prevention
High-Risk
Goal of 70 mg/dL
* High-Risk Patients are defined as: Patients with FH receiving background therapy; Patients unable to tolerate an effective dose of statin; Secondary prevention patients
receiving background statin therapy; Diabetic patients with high LDL-C levels
Muntner, et al. Trends in the prevalence, awareness, treatment and control of high low density lipoprotein-cholesterol among united
states adults from 1999–2000 through 2009–2010. Am J of Cardiol. 2013; 112.5: 664-670
Risk segmentation can identify highest
risk patients who benefit the most
• Each ~40 mg/dL absolute LDL-C reduction reduces the
risk of CV death, non-fatal MI or stroke by 22%1
• Suppose a new therapy reduced LDL-C by 60%
• 114 mg/dL reduction gives 63% relative risk reduction; 30% X 0.63
= 19% absolute risk reduction
1. Cholesterol Treatment Trialists’ (CTT) Collaborators. Efficacy and safety of more intensive lowering of LDL cholesterol: a
meta-analysis of data from 170 000 participants in 26 randomised trials. Lancet. 2010;376:1670–81.
Cost effectiveness analysis can also
show which patients benefit the most
• Cost effectiveness analysis is a technique for comparing the
relative value of competing clinical alternatives
Costs A
Drug A
Outcomes A
Life Years or Quality
Adjusted Life Years (QALY)
Choice
Costs B
Drug B
Outcomes B
• The ratio of the differences in costs to differences in
outcomes is also called an incremental cost effectiveness
ratio (ICER)
• Higher risk patient groups offer higher value (i.e., lower
ICERs)
Drummond et al. (1997) Methods for the Economic Evaluation of Health Care Programmes. 2nd Ed.
Economic value is increasingly being
included in clinical guidelines
• American College of Cardiology/American Heart
Association (ACC/AHA) will be issuing value guidelines for
different therapies based on cost-effectiveness.
• This is the ACC/AHA guidance for value:
Cost of a quality adjusted life year (ICER)
0
$50K
High value
$100K
Intermediate value
$150K
Low value
Anderson, et al. AHA/ACC statement on cost/value methodology in clinical practice guidelines and performance measures:
A report of the American College of Cardiology/American Heart Association task force on performance measures and task
force on practice guidelines. J Am Coll Cardiol. 2014; 63(21):2304-22.
This type of economic analysis allows us
to examine the components of value
Illustrative - All costs and values depicted in this graph are hypothetical
Net Monetary Benefit
$160,000
$140,000
Incremental
Monetary
Benefit
$120,000
Positive net
monetary
benefit
$100,000
$80,000
$60,000
$40,000
$20,000
$0
Lifetime Treatment Cost
CV Event
Costs Avoided
Incremental cost with
New Treatment
Benefit from
Additional Survival
Benefit from Additional
QoL
Incremental value with
new treatment
A health economist’s approach to
affordability
• To determine budgetary impact, we must estimate the
size of the eligible populations and potential uptake of
new therapy
• Uptake can easily be overestimated
• For example, in 2000 only 20% of adults with high cholesterol in
20001
• After considering any pharmacy cost offsets, we
estimate pharmacy budget impact from putting the drug
on formulary
• Estimate medical cost offsets from reduced events and
provide the total budget impact
1. Mann, et al. Trends in Statin Use and Low-Density Lipoprotein Cholesterol Levels Among US Adults: Impact of
the 2001 National Cholesterol Education Program Guidelines. Ann Pharmacother 2008; 42:1208-15.
Could benefit from partnering to identify
high risk CV patients
71M*
Pts with high
LDL-C
39M*
Diagnosed
and treated
16M *
High risk
LDL>70
7M *
High risk
patients with
LDL>100
*Amgen, data on file
• Risks with utilization in
large populations
• Potential for high budget
impact
• Budget impact much lower
with appropriate patient
identification
• Gains from partnering to
find the highest risk patients
Ultimately treated by
physicians
Given the large population, the CV area may be a good candidate for
a creative risk share agreement
Innovation in CV has the potential to
make a big impact on health outcomes
• The right disease area - CV is the #1 killer
• The right time – we have the methodology to choose
the right patients
• The right patient – we need work together to target high
risk patients and provide access to these patients
The goal is to improve patient outcomes and more efficiently allocate
resources
Questions
17
Back-Up
CV events are costly
CV Event
Direct Costs (SD);
First year
Subsequent years
First year: ACS (UA/MI)
$49,764 ($25,211)
$16,611 ($13,287)
First year: IS
$44,007 ($26,274)
$16,533 ($20,924)
Fatal CHD
$55,100 ($71,618)
Fatal IS
$48,895 ($64,157)
Revascularization
PCI: $39,421 ($17,930)
CABG: $77,695 ($26,581)
ACS, acute coronary syndrome; CABG, coronary artery bypass graft; CHD, coronary heart disease; CVD,
cardiovascular disease; HF, heart failure; IS, ischemic stroke ; MI, myocardial infarction; PCI, percutaneous
coronary intervention; SD, standard deviation; UA, unstable angina.
Bonafede et al. Medical costs associated with cardiovascular events among high-risk patients with hyperlipidemia. ClinicoEconomics and
Outcomes Research 2015: 7(1-9).
The need for innovative therapies in CVD
•
Hyperlipidemia is a major modifiable risk factor for CHD and ischemic
stroke1
•
Elevated LDL-C plays a critical role in atherosclerotic plaque formation,
and is a principal driver of CV risk in humans2-5
•
The main available LDL-C lowering therapies include: statins, ezetimibe,
bile acid sequestrants, fibrates, and nicotonic acid.
1. Jacobson T, Ito M, Maki K, et al. National Lipid Association recommendations for patient-centered management of dyslipidemia: part 1 - executive
summary. J Clin Lipidol. 2014;8:473-488.
2. Akram, et al. Beyond LDL cholesterol, a new role for PCSK9. Arterioscler Thromb Vasc Biol. 2010;30:1279-1281.
3. Cholesterol Treatment Trialists' (CTT) Collaborators. The effects of lowering LDL cholesterol with statin therapy in people at low risk of vascular
disease: meta-analysis of individual data from 27 randomised trials. Lancet. 2012;380:581-590.
4. Francis A, & Pierce G. An integrated approach for the mechanisms responsible for atherosclerotic plaque regression. Exp Clin Cardiol. 2011;16:77.
5. Martin S, Blumenthal R, & Miller M. LDL cholesterol: the lower the better. Med Clin N Am. 2012;96:13-26.
Better lipid management has the potential to prevent
additional MACE events in 2035 for high risk patients
Annual Projected Residual Major Adverse Cardiac Events (MACE)* in Treated Adults,
LDL>70
2.0
1.47 M
1.5
1.56 M
1.63 M
1.37 M
1.28 M
Annual MACE*1.0
(millions)
0.5
0.0
2015
2020
2025
2030
2035
*Myocardial infarction, ischemic stroke, unstable angina, coronary artery bypass graft surgery, percutaneous coronary
intervention, congestive heart failure, cardiac death
For Internal Use Only. Amgen Confidential.
21
Relationship between events and LDL
For Internal Use Only. Amgen Confidential.
22
Every ~40 mg/dL reduction in LDL-C decreases relative
risk for events by 20-25%
The relationship between LDL-C reduction and CV
event reduction is also holds for non-statin therapies
Relative Risk in CV Events vs Absolute Risk in LDL-C