Slide 1

Evaluating & Applying
What Works Best
Leaders’ Project Policy Forum
Kathy Buto, VP Health Policy
April 24, 2008
Comparative Effectiveness
- Help establish value of treatments
- Help physicians and patients make better decisions
- Promotes higher quality, value-based care
- Way for developers of products to differentiate truly
innovative products
Research investment will focus on finding higher
value treatments if include incentives (like
streamlined approvals, higher reimbursement)
Comparative Effectiveness
Concerns and implications
- Expectations the CE is “silver bullet”
- Won’t address major drivers of costs
- Population-based decisions trumping individual
patient needs (cost/QALY metric)
- Consider alternatives to treat condition not just drug
to drug, device to device
- Could be used as a “fourth hurdle”
- For devices, role and training of operator/surgeon,
procedure volume, ease of use, shorter lifecycle must
be considered
- Can be misleading (CATIE study)
- If costs, should be costs/savings over time not input
Comparative Effectiveness
Best way to go about it:
- Undertake trust-building among stakeholders
- Public-private entity or “enterprise”
- Transparent processes, findings, limitations,
ways to disseminate
- Begin with conditions where wide variation in
treatment patterns, high burden of illness
- Acknowledge the role of population-based studies but
build in consideration of individual patient variation
- Consider incentives to create “clamoring” for CE
- Focus on comparative clinical value not costs
Risk Sharing – the Velcade Case
 Final NICE Recommendation, October 2006
“Bortezomib monotherapy, in its licensed indication,
is not recommended for the treatment of patients
with multiple myeloma…….
…….the Committee concluded that bortezomib
monotherapy for the treatment of relapsed multiple
myeloma is clinically effective compared with HDD,
but that it has not been shown to be cost effective.”
Risk Sharing – Velcade Case
Only licensed drug for multiple myeloma, available in
Scotland, Wales, N. Ireland
Clinically effective but did not meet cost effectiveness
threshold – uncertainty about patients who would benefit
Patient and media response, characterized as a “death
Company appealed but prior to Velcade, only 1 appeal
had succeeded in overturning guidance
Company proposed “risk sharing” to reduce uncertainty
Risk Sharing – Velcade Case
 NHS funds treatment at first relapse in patients who
achieve a complete or partial response during first 4
cycles of treatment (serum M protein test)
 Patients who fail to respond discontinue Velcade
 Complete rebate or free stock direct to hospital units for
patients with less than a partial response
 Interim bridging to provide access ahead of new
evidence – not a permanent reimbursement
Other Risk Sharing
 Performance agreements – restenosis rates,
anti-psychotic compliance
 Guaranteed Savings – utilization rates
 “Coverage with evidence development” – U.S.
Drug Safety Monitoring
Evaluate benefit-risk profiles continuously, with
important information gained in real world use
Pre-approval, knowledge based on thousands of patients
in controlled clinical setting
In study with 3000 patients, one can identify adverse
reactions that occur at a rate of 1 in 100 patients
Not possible to identify an adverse reaction that occurs
in fewer than 1 in 1000 patients
In post-approval, rely on post-marketing reports,
additional research, including epidemiological studies
and targeted trials
Good risk management requires cooperation between
regulatory agencies and the company and timely
communication of benefit-risk information to health
professionals and patients.
Drug Safety Monitoring
Under FDA Amendments, FDA will have ability to develop
partnerships with public and private payers and
academia to increase access to data on drug safety
Companies will play an important role in assessing the
data in partnership with FDA
Question of whether these data can provide information
to help improve targeting of appropriate patients,
improve understanding of effectiveness as well as safety

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