WEBINAR: Developing Payer Evidence: The Role of Post Approval Programs

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


The Global Payer Market: Programs To Support Managed

Market Strategies

Nathan White, CPC


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


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


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


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


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


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


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?


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?


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?


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


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


Observational Studies & Registries:

Strategic & Operational Considerations

Jeff Trotter


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


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…”


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.


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


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”)


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


Observational Studies Are A Different Animal

24

So, who “owns” it…?

• HEOR

• Epidemiology

• Medical Affairs

• Marketing / Product Management

• Clinical Operations

• Development

• Safety / PV


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


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


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


Payer Utilization Of Value

Evidence



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


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.


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.


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.


Panel Discussion

Nathan White, CPC

Jeff Trotter



Panel Contact Information

Nathan White, CPC

Executive Director, Access & Reimbursement

inVentiv Patient Access Solutions

(703) 662-1851

[email protected]

Website: www.inventivhealth.com/patientaccess

Jeff Trotter

Executive Vice President, Phase IV Development

PharmaNet / i3

(847) 943-2508

[email protected]

Website: www.pharmanet.com


Practice Area Leader, Pricing & Market Access

Campbell Alliance

(973) 967-2300 ext. 2343

[email protected]

Website: www.campbellalliance.com

Economy & Finance

WEBINAR: Developing Payer Evidence: The Role of Post Approval Programs