Transcript One Beat, One Vision

April 4, 2006
Reimbursement and Phase IV:
CRO Role In Clearing
The Fourth Hurdle
Objective
• Understand trends in payer use of
Phase IV studies and registries
• Identify considerations for adapting
Phase III and IV activities to
accommodate those trends
• U.S. reimbursement planning and
problem solving since 1998
• Former owner S&FA; Exec VP, PAREXEL
• Payer research; competitive analysis
• Strategic planning; reimbursement
forecasting
• Advocacy with major payers
Tag Client Mix
Investors/
Advisors
15%
Biotech/
Biologicals
40%
Ad/PR/PA
Agencies
10%
Devices/
Diagnostics
15%
Pharmaceuticals
20%
Current Assignment Include
• Sepsis
• Immune globulins
• PDT
• Osteoporosis
• HIV/AIDS
• Personalized cancer
immunotherapy
• Genetic testing
• Bleeding disorders
The Fourth Hurdle
1.
Proof of efficacy
2.
Acceptable safety
3.
GMP
4. Reimbursement
4x4: 4th Hurdle’s Link to Phase IV
• U.S. payers routinely require outcomes
research to support coverage of high
cost technologies
• High cost = “On my radar per case or
in total”
Link – cont’d
• Tech developers are often reluctant to
include in Phase III more than what is
needed for FDA. That’s OK because …
• Payers want to know how new tech
affects real populations, not protocoldriven clinical trial subjects
• But Phase III design should anticipate
Phase IV data collection
Payers Want Practical Clinical
Trials (PCTs)
• Evidence-based coverage policy will
require PCTs
– E.g. ICD
• Study design is formulated to enable
treatment decision making
– Distinguish from explanatory clinical trials
designed to maximize the chance that a
biological effect of a new tx will be revealed
PCT Characteristics
• Compare clinically relevant
interventions
– E.g. Enroll based on presenting
symptoms rather than confirmed
diagnosis
• Enroll a diverse population of study
participants
– E.g. Elderly not excluded
Characteristics – cont’d
• Recruit from a variety of practice
settings
• Collect data on a broad range of health
outcomes beyond mortality and
morbidity
– E.g. QoL, symptom severity, cost, patient
satisfaction
MCO Views on Outcomes Data
• Economic data
Most persuasive
• Clinical data
Sometimes useful
• Quality of life
Interesting but
seldom compelling
Risks in Post Approval Trials
• Failure
– Pfizer funded trial comparing its calcium
channel blocker Norvasc to other
antihypertensives
– Generic diuretic (chlorthalidone) was
shown to be superior in preventing
certain cardiovascular outcomes
Risks – cont’d
• Credibility
– Payers assume study lacks scientific
rigor
Active Controlled Trials
• Payers want to know how the new tech
compares to standard of care, not to
absence of care
• Some manufacturers willing to risk
active trials in Phase III because of
payer, not FDA, pressure
Amgen Oncology and Osteoporosis
• Head-to-head trials of AMG-706 and
Avastin
• Comparative trials of denosumab
against Fosamax and Zometa
• “If not superior, we’d rather know
now than later.”
AHRQ Payer Registry Guide
• Agency for Healthcare Research and
Quality is developing “how to” guide for
payers who create patient registries as
part of coverage with evidence
development
• On contract to Outcome Sciences, Inc.
Registry Guide – cont’d
• National workshop to be scheduled –
date TBD
• Monitor at ahrq.gov
Medicare Evolving to Be National
Treatment Policy Manager
• CMS process for evaluating new
technology is rigorous and evidence based
• Adverse Medicare coverage policy decision
is routinely followed by private payers
• Part D benefit and Coverage With
Evidence Development (CED) are
accelerating the evolution
Part D
• More difficult for manufacturers to
differentiate products via detailing
• Part D benefit design drives utilization
toward generics, forcing undifferentiated
products to lower net selling price
Part D – cont’d
• Part D Plan P&T committees rely
heavily on comparative effectiveness
data
– Coverage decisions will likely migrate to
Plans’ non-Medicare businesses
Medicare CED
• Coverage with evidence development
for FDA approved drugs, biologicals,
devices
• CMS can require evidence collection,
including Phase IV trials and patient
registries, as a condition for Medicare
coverage
Features
• Will be used only where Medicare
coverage would otherwise be denied as
not reasonable or necessary
• Systematic, protocol-driven data
collection
• No reimbursement for data collection
Most Likely To Be Used For …
• Drugs in new classes with novel
mechanisms
• Treatments that may be ineffective or
unsafe in some patient subgroups
• Off label uses
Awaiting New Guidance Document
• CMS intends to issue revised guidance
by summer 2006
– Clarify Common Rule and IRB
application to CED
One Current Use of CED
• Expand coverage of Eloxitin,
Camptosar, Erbitux and Avastin off label
for colorectal cancer
• Patient must be enrolled in NCI
sponsored trial
• “Sufficient inference of benefit” +
safeguards inherent in NCI sponsored
trials
ICDs: Another Example of
Evidence-Based Coverage Policy
Situation:
• Trials of implantable cardioverter
defibrillator (ICD) showed it to be
effective in some patients but not in
others
• High cost, large population, unsettled
medical evidence resulted in adverse
coverage policies
ICD Coverage
Response:
• Manufacturer sponsored Phase IV trial:
Sudden Cardiac Death in Heart Failure
Trial (SCD-HeFT)
• Medicare expanded coverage to include
study population
Outcome
• Trial provided evidence on survival
benefit with simple, single lead ICD
• Medicare issued new national coverage
determination
How CRO Can Prepare Client
for The Fourth Hurdle
• Design Phase III for beyond Phase III
• Conduct primary research to understand
what important payer(s) want to see in
that therapeutic class
– E.g. What metrics are relevant?
• PMPM
• Cost/savings
• Drug budget impact; Overall budget impact
How To Prepare – cont’d
• Press to have Phase IV studies be at
least as rigorous and credible as Phase
III
• Identify new tools to address payer
needs
– E.g. – validated biomarkers
Hypothetical Case
• New ADD/ADHD tx in development
• Will universally be 3rd tier
– Inherent safety concerns – pediatric –
complicated by conflicting, government
sponsored class findings
– Not life saving
– Many alternatives whose PMPM costs are
well understood
– Competitor performance contracts in
place
Manufacturer Objective: Begin
Moving To Tier 2 In Year 2
• How will drug move from 3rd to 2nd tier?
– Significant price concessions
– Outcomes research (OR)
• Which is the better choice for client? If
OR, then need to identify
– Endpoints that are relevant to payers
– Tools to accelerate conclusions
Example of Tool:
Validated Cognitive Biomarkers
• How can validated cognitive
biomarkers:
– Identify patients who are likely
responders to this drug?
– Use response data to suggest cost
effectiveness?
– Produce data that is credible to the
payer?
Summary
• U.S. payers, led by Medicare, now
require post-marketing outcomes data
to grant or continue coverage of new,
high cost technologies
• Many pharma companies do not yet
– Recognize the extent of the trend
– Prepare adequately pre-launch
– Find out from payers what they really
need to see
Summary – cont’d
• Significant opportunity exists for CROs
to fill the knowledge gap by
– Determining payer data needs
– Perfecting clinical trial tools to address
those needs (e.g. validated biomarkers)
– Helping clients understand that
• Market access does not end with FDA
approval
• Phase III planning is essential to streamline
the Phase IV work
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