webinar: developing payer evidence: the role of post approval programs
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WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTHWE’RE WAITING FOR YOU AT THE GLOBAL CROSSROADS. WE KNOW THE WAY IN HEALTHCARE.™
Developing Payer-Focused Evidence:
The Role Of Post-Approval Programs
September 13, 2011
Compliments of
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTH
Jeff Trotter
Executive Vice President
Phase IV Development
Today’s Panel
2
Nathan White, CPC
Executive Director
Access & Reimbursement
Lujing Wang, MD, MPH
Practice Area Lead
Pricing & Market Access
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTHWE’RE WAITING FOR YOU AT THE GLOBAL CROSSROADS. WE KNOW THE WAY IN HEALTHCARE.™
The Global Payer Market: Programs To Support Managed
Market Strategies
Nathan White, CPC
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTH
European Landscape
• Coverage largely through government
sponsored/managed insurance
• Well-defined health technology assessment
(HTA) process
• HTA/Payer relationship is strong (i.e. UK‟s NHS
& NICE)
• Emphasis on medical innovation: “me too”
products are not favored in HTA process
• HIT is a critical part of coverage and
reimbursement systems
4
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US Landscape
• Complex evidence development and utilization
• Many national payers and PBMs have developed in-house HTA‟s
› Research could be viewed as subjective
› Rely heavily on claims data and chart review
• CMS coordinates to some degree with AHRQ on evidence needs
› AHRQ-sponsored review of evidence for colorectal screenings
› NCD for treatment of actinic keratoses
• “Me-too” products still have market potential
• National HIT standards implementation
still has room for improvement
5
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US Payers Likely To Use PRO’s In Future Decisions
6
26%
68%
5%
How likely are you to use PROs to make coverage and reimbursement policy decisions
in the future?
(on a scale of 1 to 7 where 1=Not likely, 7= Very likely)
Very Likely
Likely
Not Likely
# of lives = 4,353,435
# of lives = 51,127,435
# of lives = 19,701,655
Mean 4.5
n=22
Source: 2011 inVentiv Health Payer Study
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US Payers Likely To Follow CMS Lead
7
14%
86%
1%
If CMS publicly leverages the results of these studies, how likely are you to follow CMS’s
lead in utilizing PRO’s to guide your coverage decisions?
(on a scale of 1 to 7 where 1=Not likely, 7= Very likely)
Very Likely
Likely
Not Likely
Source: 2011 inVentiv Health Payer Study
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTH
Evidence Development: Pre- and Post-Approval
• Prospective
› Clinical study data
• May include PRO endpoints and cost-benefit analysis
› FDA approved label
• Retrospective
› Pharmacy claims analysis
› Chart review
› Budget impact modeling
› Cost effectiveness analysis (limited use in US)
› Registry
› Phase IV outcomes study with PRO
› Commercial marketing programs
8
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What Is This Evidence Used For?
• Determining relevant “access barrier” criteria
› Step therapy
› Prior authorization
› Quantity limits
• Deciding which benefit the therapy is placed in (medical v.
pharmacy v. specialty)
• Reimbursing at an appropriate rate
9
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TOMORROW?TODAY
Reimbursement Support Programs
Focused on helping patients
with reimbursement access
barriers and assisting the
underinsured
Used primarily as marketing
initiative
Captures some data which
could be valuable to managed
markets and brand teams
Typically doesn‟t capture PRO
Managed markets data could
be used to better guide NAM
tactics
Could this program type be
integrated into a Phase IV study
to reduce sponsor cost?
CHALLENGE: How do we get
all the stakeholders (vendor,
brand teams, managed markets,
etc) to share the same vision?
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TOMORROW?TODAY
Patient Assistance Programs
Focused on assisting the
uninsured (PAP)
Used primarily as corporate
awareness to the public
Captures some data which
could be valuable to managed
markets teams
Typically doesn‟t capture PRO
PAPs could begin to collect
adherence/compliance data
(especially IPAPs)
What confounding factors
would inhibit such an evolution
(ex. IRS, study population bias,
misclassification)?
Would patient advocates
object to muddying the waters of
a free drug program?
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TOMORROW?TODAY
Adherence Programs
Focused on changing patient
behavior and improving patient
health outcomes
Opt-out programs typically
administered through 3rd party
and use claims data to
intelligently message patients
High touch programs use a
clinical case management
approach
Would patients be willing to
respond to PRO questionnaires
in an opt-out program?
How can manufacturers
partner with payers and
manufacturers to utilize PRO
more effectively?
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Patient Support Programs:
The “Package” Approach
• Post-approval RCT sought to demonstrate superior effectiveness of buprenorphine medication-assisted therapy paired with interventional coaching (in opioid dependent patients)
• CAC and trained registered nurses conducted telephonic interventions designed to encourage appropriate compliance & persistency
• The study concluded that patients were more likely to take their therapy every day and less likely to abuse, compared to controls
What can we learn from this example?
• Better patient support leads to better patient outcomes, reducing overall payer spend
• Additional messaging to payers on total value of package (product + program)
13
Source: Supplement to Journal of Managed Care Pharmacy, Feb 2010
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Role of Commercial Programs In Evidence
Development
• The primary direct link to patients after approval
• Types of programs:
› Reimbursement
› Patient assistance
› Adherence
• Control arms could be added with a non-interventional survey or
interventional care coordination to demonstrate therapy or
therapy/program effectiveness to payers
14
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Observational Studies & Registries:
Strategic & Operational Considerations
Jeff Trotter
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTH
Post-approval Research Today – Safety & Value
• Requirement
› In some countries, „real world‟ post-approval experience data must be submitted to maintain market approval.
› Increasingly, some form of safety surveillance / risk management program will be mandated and enforced.
• Responsibility
› Corporate accountability for post-approval safety is increasingly expected by various constituencies.
› Documentation of clinical / economic / humanistic value is critical for commercial acceptance and accelerated product uptake.
• Opportunity
› If managed proactively, safety surveillance obligation can be controlled.
› An observational study can be a cost-effective, high ROI mechanism for fulfilling the post-approval obligation for both safety and effectiveness data.
16
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Real World Perspectives, Real World Research
17
“The conditions under which products are
examined for regulatory approval are
generally not the conditions under which
they are actually used…”
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Who Needs Real World Data?
18
• Health authorities
• Pricing commissions
• Payers
• Regulatory
authorities
• Physicians /
providers
• Policy makers
• Patients
WellPoint's CER Guide Describes How It Will Determine Usefulness Of Studies
Observational Studies Of "Real-World" Questions
The guidelines state that, "while randomized, controlled clinical trials remain the gold standard for producing reliable efficacy and safety data, WellPoint recognizes that there are circumstances in which RCTs alone may not be sufficient for decision-making. Accordingly, a well-conducted CER or observational study may complement RCT-based information by providing effectiveness data, or data on outcomes achieved in a 'real-world' setting.“
German Pharma Law Require Firms to Prove Drugs' Value
Within a Year /
Germany's Comparative Effectiveness Debate Concludes;
Dossier Refinement Begins
The holder of the marketing authorization will be required to hand in a comprehensive
dossier to the G-BA, which needs to contain information on:
• the authorized indications;
• the actual medical benefit of the product;
• the additional medical benefit of the product compared with existing therapies;
• the number of patients and patient groups for which the product is relevant;
• the cost of the therapy to the statutory health insurance funds; and
• requirements for quality-assured use of the product.
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTH
Real World Studies & Registries Are Needed
Because…
19
►…RCTs can be too artificial in intent and design, and
therefore poorly reflective of actual medical practice
► Tight inclusion criteria
► Experimental protocol
► Tight procedural control
► Randomization, blinding, placebo, etc.
► Short in duration
► Homogeneous sites
►We need to know how a product is used and how it
“performs” under real world conditions
► Safety
► Clinical outcomes (CER)
► Economic value
► Humanistic value
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But The Real World Can Be Really Messy!
►What are we trying to prove?
►What can we prove?
►Should we be trying to “prove” anything?
►Considering…
►Not typically testing a hypothesis
►Potentially shaky statistical foundation
► Inexperienced research sites
►Liberal inclusion criteria
►Strong likelihood of various biases
► Imperfect ability to identify all confounders
►Hawthorne effect
► Inconsistent understanding of observational research
…is there a “perfect” observational study? Probably not…
20
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Different Conditions Require Different Processes
21
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Key Components
22
RCT Component Observational Study
Support for approval Strategic Goal Support for “real world” data
Efficacy Measures Safety, effectiveness, value
Randomization,
inclusion/exclusion criteria,
protocol, monitored
Controls Inclusion/exclusion
Sample size based on
hypothesis
Statistical Power Possibly, based on expected
event rate, but often lacking
Investigators, subjects Participants Practitioners, patients
Consent, EC/IRB, privacy Approval Consent, EC/IRB, privacy
(notification)
As short as necessary Timeframe Longer-term (“sustain
and maintain”)
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Operational Issues & Challenges
• Site selection
• Site training and start-up
• Site “interaction”
(monitoring) and
management
› Site motivation
› Protocol “adherence”
• Inclusion
• Procedures
• Data management
› Accommodating multiple
measures
› EDC issues
› Data quality
• SDV
• Analysis
› Biases, etc.
› Findings
› Reporting (communications
23
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Observational Studies Are A Different Animal
24
So, who “owns” it…?
• HEOR
• Epidemiology
• Medical Affairs
• Marketing / Product Management
• Clinical Operations
• Development
• Safety / PV
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Highlights From Study On Observational Research
• Motivation: “Schizophrenic” RFPs
› i.e., uncertainty, inconsistency, imprecision, over-engineering, etc.
• Many functional areas have some involvement in observational research studies
• Many different purposes underlie these studies
• “Observational research” goes by many names
• Sponsors have varying levels of “comfort” with observational research
• Most sponsors do not have defined processes for observational studies
› Design, Procurement, Operational, Analytical, etc.
• Sponsors have varying expectations for the “conclusiveness” of findings from observational studies
• Sponsors are concerned that regulatory/health authorities “don‟t get it”
• Sponsors plan to become increasingly involved in observational research
25
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Operational Planning:
Building The Study From The Ground Up
26
REPORTS
LEGAL, REGULATORY, IRB REVIEW
MATERIALS PRODUCTION AND DISTRIBUTION SITE RECRUITMENT AND TRAINING
PATIENT ENROLLMENT,
OUTCOMES TRACKING,
DATA COLLECTION
SITE
SUPPORT
ANALYSES
NEWSLETTERS
PUBLICATIONS ABSTRACTS, PRESENTATIONS
MEETINGS
STRATEGY
ANALYSIS PLAN COMMUNICATIONS PLAN
DATA COLLECTION FORMS,
PROCESSES, AND LOGISTICSSCIENTIFIC ADVISORY PANEL
SITE IDENTIFICATION (FIELD
INVOLVEMENT)
• What are the strategic goals underlying the study?
› Direct impact on how the project/study should be „operationalized‟
• Direct impact on budget and ROI
• Work backwards from the deliverable
• Don‟t consider any individual component in a vacuum
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Observational Research & Registries: Best Practices
• Be realistic in study planning
• Set appropriate expectations (internally and externally)
› Observational study as part of overall “portfolio”
• Strive for organizational inclusiveness and consensus
• Develop guidelines addressing study design and SOPs addressing unique
operational requirements
• Interact with stakeholders during planning stages (and concurrently)
• Maintain a collaborative stance with research partners
› Minimize operational constraints
• Expect change: “shift” happens
27
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Payer Utilization Of Value
Evidence
Lujing Wang, MD, MPH
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTH
Pros and Cons of Late Stage Evidence Generation
29
Evidence Analysis
Very
Slight
Slight
Rele
van
ce
Credibility
Very
XEvidence
Generation
Y
Repositioning
What to generate:
study endpoints
How to generate:
study design
Pros
Real-world data with
balanced demographics
Long-term outcomes
in a large population
Ability to address
payers‟ concerns
Ability to define specific
patient (sub)population
Partnership to boost
credibility of results
Cons
Intuitive suspicion of
manufacturer-
sponsored studies
Lack of credible
adjudication of
methodology
Perceived subjectivity
of patient-reported
outcomes measures
Limited actionability of
study results
Late-stage evidence generation should aim to demonstrate how a product can provide meaningful
benefits to fulfill a justifiable need, at a reasonable and predictable cost
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Payer Communication of Value Evidence
30
Principles of Payer Communication
Simplicity
A complete story that
can be told in a
definite time window
Concise and crisp
takeaways that can
stay in memory
Transparency
Avoidance of “black
box” design and
subjective
assumptions
Key foundations for
audience to interpret
study results
Credibility
Well-accepted
methodology and
validated design
Third-party
endorsement and
KOL partnership
II IIII
Successful communication with payers requires following three principles.
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Stakeholder Engagement
31
Stakeholder Profiles Engagement PlanPotential Access
Stakeholder Groups
Pharmacy
Stakeholders
Provider Stakeholders
Financial Stakeholders
Operational
Stakeholders
Key Opinion Leaders
Societies and
Advocacies
Roles and
responsibilities
Evolving interests
and incentives
Interaction and
influence
Attitudes and
perceptions
What messages to
communicate
How to deliver the
messages
Who to own the
relationship
When to engage
the stakeholders
PULL
THROUGH
PUSH
THROUGH
The right value evidence needs to be delivered to the right audience at the right time by the right
people. Stakeholder mapping and engagement is pivotal in rolling out the study outputs efficiently
and effectively.
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Organizational Requirements
32
Hypothesis
Validation
Evidence
Generation
Value
Communication
Rigorous Scientist Credible Ambassador Strategic Visionary
The right value evidence needs to be delivered to the right audience at the right time by the right
people. Stakeholder mapping and engagement is pivotal in rolling out the study outputs efficiently
and effectively.
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTHWE’RE WAITING FOR YOU AT THE GLOBAL CROSSROADS. WE KNOW THE WAY IN HEALTHCARE.™
Panel Discussion
Nathan White, CPC
Jeff Trotter
Lujing Wang, MD, MPH
WE KNOW THE WAY IN HEALTHCARE > INVENTIV HEALTH
Panel Contact Information
Nathan White, CPC
Executive Director, Access & Reimbursement
inVentiv Patient Access Solutions
(703) 662-1851
nwhite@inventivhealth.com
Website: www.inventivhealth.com/patientaccess
Jeff Trotter
Executive Vice President, Phase IV Development
PharmaNet / i3
(847) 943-2508
jtrotter@pharmanet.com
Website: www.pharmanet.com
Lujing Wang, MD, MPH
Practice Area Leader, Pricing & Market Access
Campbell Alliance
(973) 967-2300 ext. 2343
lwang@campbellalliance.com
Website: www.campbellalliance.com
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