ISR Reports - Comparative Effectiveness Research (CER) and Payers - Report Preview

US Payers: Comparative Effectiveness Research
and Formulary Decision-making

115 page 30 high-level 98 graphs Data collected Quantitative
report respondents and charts Dec 2011 questionnaire
with
118 questions
Use this report to…

Maximize formulary placement and reimbursement levels.
1. Develop both pre-approval and post-approval study protocols to yield data that
formulary-decision makers want to use in their evaluations.
• Learn which CER study types are gaining in importance and driving formulary
decisions.
• Learn what formulary decision-makers are skeptical of when using data from
randomized controlled trials with a placebo and plan your clinical development
studies to minimize their concerns.
2. Prioritize your CER studies based on the therapeutic areas US payers view as being most
in need of comparative effectiveness studies.
• Asthma/ COPD, Arthritis/ Osteoporosis, Oncology, Diabetes, Hypertension, Mental
health, and Heart disease

www.ISRreports.com Industry Standard Research Info@ISRreports.com

www.ISRreports.com
Industry Standard Research
info@ISRreports.com

Introduction
How important is comparative effectiveness research (CER)? If you ask AstraZeneca the answer is
likely to be: game-changing, over $2 billion USD worth of game-changing.

In early September 2011, AZ released the findings from its SATURN trial which pitted their
cholesterol-lowering product, Crestor, against Pfizer’s Lipitor in a head-to-head study with 1,300
patients. The trial was designed to investigate a sub-segment of the population that AZ thought
might benefit more from Crestor than from Lipitor. The result showed Crestor did improve the
percent atheroma volume (PAV) greater than Lipitor, but not at a level that was statistically
significant. For the secondary measure, total atheroma volume (TAV), Crestor did demonstrate a
statistically significant reduction compared to Lipitor. Sounds decent, not too bad, right? Not too
bad unless you were an investor in AstraZeneca at the time. This study sent AZ down 3.3%. Or put
another way, AZ lost over $2B USD in market capitalization the day these results were released.

It will come as a surprise to very few people that payers, managed care organizations (MCOs), are
playing more important roles in the delivery of pharmaceutical products. No longer is regulatory
approval the only hurdle to commercial success. Regulatory approval is just the beginning; especially
if you are bringing to market a me-too product or a product that is only marginally superior to the
current standard of care. Today, and likely more so in the future, the real hurdles will come from
payers in the form of where your product gets placed on their formulary. It is with this hypothesis
that ISR initiated the research that led to this report. It was developed to answer some very specific
questions around US payers and their views of comparative effectiveness research.

The AZ example we highlighted previously will not be the last you hear of a pharma company’s
fortunes moving with results of comparative effectiveness research. The data in this report highlight
that US payers desire more data from randomized controlled trials that utilize a comparator
product(s), not just a placebo. If they get their wish, it starts to put an even greater premium on
pharma companies that can create truly innovative solutions/ products and get them to market first.
If you are first, then you get to be the comparator. This puts you in the position of not having to win,
you just can’t be beat and that is definitely a position of strength you want to be in.

The following report is based on the data generated from 30 survey respondents. All respondents
have responsibility for decision-making as it pertains to formulary decision-making at their
organization. As always, this report contains all of the data collected in our survey and all data are
available in the Study Data section that follows the Study Findings section.


www.ISRreports.com
Sample Report Pages info@ISRreports.com

Formulary drivers
Study types
US payers are speaking loud and clear. They want more data from randomized controlled trials (RCTs)
with a comparator product and less from RCTs using only a placebo. An interesting aspect of the data
below is that US payers are being a bit realistic. They believe the degree of influence RCT trials with a
comparator will have over formulary decisions in two years will still likely be less than they would desire.
The graph below contains the responses when we asked respondents about their views about the
current and future degree of influence on formulary decisions. And when we asked them, across the
seven major therapeutic areas, to indicate their preferred data sources, the data becomes even more
skewed in favor of RCTs using a comparator.
Relative importance of study types – Summary
“While we understand that each evaluation is unique, over the past 24 months when you have been making decisions in
your P&T committee regarding pharmaceutical products, how much influence did data from the following sources have in
your decision-making? Please ensure your responses add to 100%. Then, over the next 24 months when you will be making
decisions in your P&T committee regarding pharmaceutical products, how much influence will data from the following
sources have in your decision-making?” (Base = 30 respondents)
Mean
Health economic outcomes research

Meta-analysis of data sets (usually data from
randomized controlled studies)

Patent-centric studies (QoL, adherence, Past 24 months
functional measures, ease of use) Next 24 months

Prospective observational studies (registry)

Randomized controlled trials with a comparator 30%
product 39%

Randomized controlled trials without a
comparator product, placebo-only

Retrospective observational studies (database
analysis, health claims)

0% 10% 20% 30% 40% 50%


www.ISRreports.com

Product types: First-in-class, Transitional, Me-too
The analysis below illustrates respondents’ views on the relative importance of the different study
types for the three classes of drugs. MCO formulary decision-makers place more importance on
on-going post-approval registry studies measuring safety for first-in-class products than for
transitional or me-too products. Along the same lines for fist-in-class products, respondents
place more importance on ongoing post-approval registry studies measuring safety than they do
for those measuring efficacy.

Study types and drug types: the importance of safety
“How important is it to your P&T committee when you are evaluating a First-in-class: Transitional or follow- on: Me-too/
marginally differentiated product that the product dossier include..?” (Base = 30 respondents)
% Extremely Important
63%
The registration filing contains data comparing the
73%
product to a leading competitor

27%
A study measuring Health Economic Outcomes/
23%
cost effectiveness 27%

13%
An ongoing post-approval registry study
23%
measuring safety 50%

10% Me-too
A retrospective data-mining analysis measuring
17%
efficacy and safety 17% Transitional

10%
An ongoing post-approval registry study
17%
measuring efficacy 27%

7%
A formal program to promote product adherence 7%
7%

0% 20% 40% 60% 80% 100%


www.ISRreports.com

This matrix plots the relative growth rate of CER study types by the degree to which respondents
indicated the importance they have in their formulary decision-making process. Studies in the
upper right of the matrix will exhibit both strong growth and have a considerable influence in
formulary decision in the next two years. Studies plotted include:
• Placebo-controlled trials demonstrating an important or unique benefit or harm of a particular drug
• Short-term head-to-head trials that use surrogate (efficacy) measures
• Cohort, case-control, or before/after studies with broad applicability and comprehensive measurement of benefits/ risks
• Long-term head-to-head controlled trials focusing on a subset of relevant benefits or risks
• Case series and case reports
• Short-term head-to-head trials focusing on tolerability and side effects
• Before/after or time-series studies demonstrating an important or unique benefit or harm of a particular drug
• Natural history (or conventionally treated history) studies that observe the outcomes of a cohort but do not compare the
outcomes among different treatments
• Patient-centric Quality of Life measures
• Adherence studies
• Health economics outcomes research
100%
Two year out use in formulary decision-making

80%
Placebo-
controlled

60%

40%

20%

0%
-20% 0% 20% 40% 60%

Two year growth in percentage terms
-20%


www.ISRreports.com
info@ISRreports.com

Sample charts and graphs

Aggregated therapeutic area view of current and desired study type impact on P&T formulary decisions
“With respect to (insert therapeutic area), when your organization is making P&T formulary decisions how much
influence do the following study types have in your decision-making?”
“With respect to (insert therapeutic area), when your organization is making P&T formulary decisions, what is your
desired mix of data?” (Base = 30 respondents)

Studies in alphabetical order
100%
• Health economic outcomes
7% research
10%
• Meta-analysis of data sets
80%
(usually data from
randomized controlled
studies)
60% • Patent-centric studies (QoL,
adherence, functional
measures, ease of use)
• Prospective observational
40%
studies (registry)
• Randomized controlled trials
with a comparator product
20%
• Randomized controlled trials
without a comparator
product, placebo-only
0% • Retrospective observational
Current influence Desired mix studies (database analysis,
health claims)


www.ISRreports.com
info@ISRreports.com

Sample charts and graphs

TAs most in need of CER
“Using the following disease conditions, please select which one you believe are most
in need of high quality comparative effectiveness research.” (Base = 30 respondents)

Extremely needed Needed Somewhat needed Not at all needed

Arthritis, osteoporosis, osteoarthritis 10% 10% 10% 10%

Asthma/ COPD 10% 10% 10% 10%

Diabetes (Type I and II) 10% 10% 10% 10% Data available
in report
(Responses appear in alphabetical order)
Heart disease, including high cholesterol 10% 10% 10% 10%

Hypertension 10% 10% 10% 10%

Mental health, including depression, bipolar 10% 10% 10% 10%

Oncology 10% 10% 10% 10%

0% 20% 40% 60% 80% 100%


www.ISRreports.com
info@ISRreports.com

Full Report Table of Contents

Copyright and Usage Guidelines 8
Methodology 9
Introduction 10
Study Findings 12
CER Market Dynamics 13
Definition 13
“In their words” 13
Influence of CER 15
Formulary placement influence 15
Data, data, and more data 16
Use of CER study types for formulary placement 16
Issues with RCT for efficacy 18
Rating the level of concern 18
Sub-segment/ population concerns 19
Summary and recommendations 20
Formulary drivers 23
Study types 23
Relative importance of study types – Summary 23
Use of CER study types for formulary placement - comparison 25
Aggregated TA view of current and desired study type impact on P&T formulary decisions 27
Importance of health economics and outcomes research 28
Product types: First-in-class, Transitional, Me-too 29
Study types and drug types: the importance of safety 29
Therapeutic areas 30
TAs most in need of CER 30
Oncology example: Current vs. desired study mix in P&T formulary decisions 31
Summary and recommendations 32
Study Data 34
Comparative Effectiveness Research trends 35
Definition “in their words” 35
CER influence in formulary placement - current 38
CER influence in formulary placement - future 39
Use of CER study types for formulary placement - current 41
Use of CER study types for formulary placement - future 43
Use of CER study types for formulary placement - comparison 45


www.ISRreports.com
info@ISRreports.com


Study type/ data source importance 49
First-in-class products 49
Importance for P&T committee product dossier 49
Transitional or follow-on products 50
Me-too/ marginally differentiated product 51
Comparison of study types and drug types 52
P&T committee decision-making 53
Study type formulary influence 53
Relative importance of study types – Past 24 months 53
Relative importance of study types – Next 24 months 55
Relative importance of study types – Summary 57
CER and therapeutic area assessment 58
Cross-therapeutic area need assessment 58
TAs in most need of CER 58
Arthritis, osteoporosis, osteoarthritis 59
Current influence of study types on P&T formulary decisions 59
Desired mix of study types on P&T formulary decisions 61
Summary: Current vs. Desired mix of study types on P&T formulary decisions 63
Asthma/ COPD 64
Oncology 69
Diabetes (Type I and II) 74


www.ISRreports.com
info@ISRreports.com


Heart disease, including high cholesterol 79
Hypertension 84
Mental health, including depression, bipolar 89
Study design/ data source analysis 94
Randomized controlled trials with a comparator product 94
Randomized controlled trials without a comparator product, placebo-only 95
Prospective observational studies (registry) 96
Retrospective observational studies (database analysis, health claims) 97
Meta-analysis of data sets (usually data from randomized controlled studies) 98
Health economic outcomes research 99
Patent-centric studies (QoL, adherence, functional measures, ease of use) 100
Aggregated TA view of current and desired study type impact on P&T formulary decisions 101
Randomized controlled trial concerns 102
Issues with RCT for efficacy 102
Rating the level of concern 102
Sub-segment/ population concerns 104
Adherence investments 105
Most valuable programs 105
Selecting the best investments 105
Respondent Demographics 108
Organization type 108
Job description 109
P&T Committee involvement: Clinical assessment 110
P&T Committee involvement: Value assessment 111
Geographic location 112
Industry tenure 113


www.ISRreports.com
info@ISRreports.com

Respondent profile

This report is based on the data generated from 30 survey respondents. All
respondents have responsibility for decision-making as it pertains to formulary
decisions at their organization.

Average industry tenure = 18 years

Respondent titles Organizations surveyed
ASSOCIATE VICE PRESIDENT CLINICAL PHARMACY ADVANTAGE HEALTH SOLUTIONS
CHIEF COMPLIANCE OFFICER AETNA
CHIEF OF PHARMACY AMERIGROUP
CHIEF PHARMACY OFFICER BCBS OF HAWAII
CLINICAL PHARMACIST BLUE CARE NETWORK OF MICHIGAN
DIRECTOR OF PHARMACY BLUE CROSS & BLUE SHIELD
DIRECTOR OF PHARMACY CENCAL HEALTH
DIRECTOR OF PHARMACY COVENTRY HEALTH CARE OF FLORIDA
DIRECTOR OF PHARMACY AND DIRECTOR OF HEALTH SERVICES COVENTRY HEALTHCARE
DIRECTOR OF PHARMACY HEALTH CENTERS AND CLINICAL DAKOTACARE SERVICES
AFFAIRS EMI HEALTH
DIRECTOR OF PHARMACY SERVICES GROUP HEALTH COOPERATIVE
DIRECTOR OF PHARMACY SERVICES HEALTH NET PHARMACEUTICAL SERVICES
DIRECTOR PHARMACY CLINICAL STRATEGIES HEALTHSPRING
DIRECTOR, PHARMACEUTICAL SERVICES HENRY FORD HEALTH SYSTEMS
DIRECTOR, PHARMACY AND MEDICARE HUMANA HEALTH CARE
DIRECTOR, PHARMACY CLINICAL STRATEGIES INLAND EMPIRE HEALTH PLAN
DIRECTOR, PHARMACY SERVICES KAISER
MEDICAL DIRECTOR MERCYCARE HEALTH PLANS
MEDICAL DIRECTOR MOLINA HEALTHCARE OF WASHINGTON
PHARMACY DIRECTOR MOUNT AUBURN CAMBRIDGE
PHARMACY DIRECTOR INDEPENDENT PRACTICE ASSOCIATION
PHARMACY MANAGER NEIGHBORHOOD HEALTH PLAN OF RI
PRESIDENT PHARMACEUTICAL OPERATIONS AND SVP PHYSICIANS HEALTH PLAN
PRESIDENT, CEO PHYSICIANS PLUS
REGIONAL CLINICAL PHARMACY DIRECTOR PREMERA BLUE CROSS
REGIONAL DIRECTOR OF CLINICAL PHARMACY PRIORITY HEALTH (N=2)
SENIOR CLINICAL PHARMACY MANAGER PROVIDENCE HEALTH PLANS
SENIOR MEDICAL DIRECTOR SELECTHEALTH
VICE PRESIDENT, PHARMACY SERVICES THE INITIAL GROUP
VP, PHARMACY SERVICES


www.ISRreports.com
info@ISRreports.com

Respondent profile

P&T Committee involvement: Clinical assessment
“Please indicate your level of involvement with your
organization’s P&T committee activities as it relates to the clinical
assessment/ review of pharmaceutical products.” (N=30)

Decision-maker, on the P&T
100%
committee for clinical review

Influencer, provide data and
0%
analysis to the P&T committee

Implementer, ensure the
decisions made in the P&T
0%
committee are administered
correctly
I have no involvement with the
clinical assessment review of 0%
pharmaceutical products

0% 20% 40% 60% 80% 100%

Job description
“Which of the following most closely matches your job description?” (N=30)

Pharmacist, Pharmacy Director 80%

Medical officer 13%

C-suite (CEO, COO, CFO) 7%

Physician 0%

Health Technology Assessment 0%

Health economics, Analytics,… 0%

Contracts/ Finance 0%

Clinical scientist/ services 0%

0% 20% 40% 60% 80% 100%


www.ISRreports.com
info@ISRreports.com

To Order US Payers: Comparative Effectiveness
Research and formulary decision-making…
VISIT the report page on our website and click “Purchase Report”

CALL us at +1.919.301.0106 x 705

E-MAIL us at info@ISRreports.com

Licensing Options
Please e-mail us at info@ISRreports.com for licensing and pricing.

About Industry Standard Research
Industry Standard Research is the premier, full service market research provider to the
pharma and pharma services industries. With over a decade of experience in the
industry, ISR delivers an unmatched level of domain expertise.

For more information about our off-the-shelf intelligence and custom research
offerings, please visit our Web site at www.ISRreports.com, email info@isrreports.com,
or follow us on twitter @ISRreports.

Send Us Your Feedback
Because we are a service organization, we enjoy receiving feedback on our work. The
good, the bad, the ugly – we encourage you to let us know what you think. Please e-
mail any comments, questions, or suggestions.


ISR Reports - Comparative Effectiveness Research (CER) and Payers - Report Preview

Recommandé

Recommandé

Contenu connexe

Plus de Industry Standard Research

Plus de Industry Standard Research (20)

Dernier

Dernier (20)

ISR Reports - Comparative Effectiveness Research (CER) and Payers - Report Preview