Transcript Slide 1

NOPR 2006-2009
and the New Era of
Comparative Effectiveness
Bruce E. Hillner, M.D.
Eminent University Scholar and Professor
Virginia Commonwealth University
Richmond, VA
1
Medicare (CMS) Coverage of New Technologies
• Standard for reimbursement is “reasonable and
necessary”
• In 1990s, CMS adopted a new evidence-based
approach for making coverage determinations
– Requires peer-reviewed scientific evidence to document
that new technology leads to changes in patient
management and improved health outcome
• CMS elected not to broadly consider oncologic indications
for PET, but rather to evaluate the evidence on a cancerspecific and indication-specific basis
2
Medicare’s Coverage of PET
• CMS initially covered PET on a cancer-specific and
indication-specific basis
– This approach rapidly became unwieldy
– Decisions became, de facto, by cancer type
• From 2006-2009, CMS used NOPR to provide access
for PET to patients with not-previously-covered cancers
– About 20% of CMS oncology PET scans (50,000/year)
• In April 2009, CMS modestly expanded the “covered
cancers,” while simplifying the plan for NOPR 2
– NOPR 2 will be about 10% of CMS oncology scans
3
NOPR’s Goals and Objectives
• Assess the effect of PET on referring physicians’ plans of
intended patient management
– across a wide spectrum of cancer indications for PET
that are currently not covered by the Medicare
program, and
– in relation to cancer-type, indication, performance
status, physician’s role in management, and scan type
• Provide access to the service
• Minimize the burden to the patient, the PET center, and
referring physicians
• Generate evidence of reasonable quality to assist CMS in
deciding whether to expand coverage of PET
NOPR Workflow
Referring MD
requests PET
Pre-PET
Form
Ask patient
for consent
PET
done
PET
interpreted
& reported
Post-PET
Form sent,
including question for referring
MD consent
Ongoing
patient
management
Post-PET Form
completed.
Claim submitted
Timing of PET in Cancer Natural History
Suspected
Cancer
(Diagnosis)
Initial
Staging
Restaging
Treatment
Monitoring
Suspected
Recurrence
Later
Suspected
Recurrences
Treatment
Monitoring
Pre-PET Form – 5 Questions
• Reason for the PET Scan
• Cancer Site/Type
• Summary of Disease Stage
– NED, Localized, Regional, Metastatic, Unknown
• Performance Status
– Asymptomatic, Symptomatic, Bedridden
• Intended Patient Management Plan
Example of Question Detail:
Intended Patient Management Plan
5. If PET were not available, your current management strategy
would be (select one)?




Observation (with close follow-up)
Additional imaging (CT, MRI) or other non-invasive diagnostic tests
Tissue biopsy (surgical, percutaneous, or endoscopic).
Treatment (if treatment is selected, then also complete the following)
Treatment Goal: (check one)  Curative  Palliative
Type(s): (check all that apply)
–  Surgical  Chemotherapy (including biologic modifiers)
–  Radiation  Other  Supportive care
Strengths of the NOPR Data
•
•
•
•
•
“Real world” data
Timely data
Very large patient cohorts
Current technology (≥ 85% PET/CT)
Good observational studies usually match controlled
studies in magnitude and direction of effect
(Concato NEJM 2000; Benson NEJM 2000; Ionnanidis JAMA 2001)
• Results similar to more tightly managed single-institution
studies (e.g., Hillner 2004) and to new Australian studies
with outcome validation
Limitations of the NOPR Data
• Collected change in “intended” management, not
actual management
• Unknown if management changes are in the correct
direction or improve long-term outcomes
• Defining the relevant long-term “outcomes” for a
diagnostic (instead of therapeutic) procedure is
controversial
• Potential that physicians may have been influenced
by the knowledge that future Medicare reimbursement
might be influenced by their responses
Limitations (2)
• NOPR does not address:
– Whether PET should be used in lieu of or as a
complement to other imaging techniques
– The optimal sequencing of CT, MRI and PET.
– How much ‘better’ PET is than next best method
NOPR Results
Overall Impact on Patient Management
– Diagnosis, Staging, Restaging, Recurrence
– Data on 22,975 scans from May 8, 2006 – May 7, 2007
– J Clin Oncol 2008; 26:2155-61
Impact on Patient Management by Cancer Type
– Confirmed Cancers
– Staging, Restaging, Recurrence
– Data on 40,863 scans from May 8, 2006 – May 7, 2008
– J Nucl Med 2008; 49:1928-35
Treatment Monitoring
– Data on 10,447 scans from May 8, 2006 – Dec 31, 2007
– Cancer 2009:115:410-18
Top Ten NOPR Cancer Sites/Indications
•
•
•
•
•
•
•
•
•
•
Ovary / Uterine Adnexa – Recurrence (Covered)
Ovary / Uterine Adnexa – Treatment Monitoring (Covered)
Ovary / Uterine Adnexa – Restaging (Covered)
Prostate – Initial Staging (Non-covered)
Prostate – Recurrence (CED)
Pancreas – Initial Staging (Covered)
Stomach – Initial Staging (CED)
Bladder – Initial Staging (CED)
Prostate – Restaging (CED)
Small Cell Lung – Restaging (CED)
Cohort Profile
• First year of NOPR
(5/8/06 to 5/7/07)
• 22,975 “consented” cases
from 1,519 facilities
• Technology profile
– 84% PET/CT
– 71% non-hospital
– 76% fixed sites
Hillner et al., J Clin Oncol 2008
PET Changed Intended Management in 36.5% of Cases
Clinical Indication for PET Study (Percent)
Pre-Pet
Plan
Post-PET
Plan
Treat
Dx
Staging
Restaging
Recurrence
All
n=5,616
n=6,464
n=5,607
n=5,388
n=22,975
Same
16.0
46.5
15.8
20.4
25.5
Non-Treat
Same
52.9
14.0
48.0
40.7
37.9
Non-Treat
Treat
23.2
31.6
28.6
29.2
28.3
Treat
Non-Treat
7.9
7.9
7.5
9.7
8.2
31.1
39.5
36.1
39.0
36.5
Patients with change
post-PET (%)
Hillner et al., J Clin Oncol 2008
Changes in Intended Management (%)
Stratified by Pre-PET Plan
Pre-PET Plan
Image
n=9,518
Biopsy
n=3,552
Watch
n=2,199
Treatment
n=7,706
Image
5.8
6.0
4.6
3.0
Biopsy
9.5
24.0
9.0
6.8
Watch
37.2
33.6
62.3
15.6
Same Rx
NA
NA
NA
42.4
New or Major
Change in Rx
47.6
36.3
24.1
8.7
Minor change Rx
NA
NA
NA
23.5
Post-PET Plan
Hillner et al., J Clin Oncol 2008
Change in Management by Cancer Type
Bladder (CED)
Brain (CED)
Cervix (Covered)
Kidney (CED)
Staging
Restaging
39.9
(1,461)
--
36.4
(1,239)
--
36.1
(341)
41.1
(895)
26.9
(353)
34.4
(979)
Suspected
Recurrence
36.7
(878)
40.5
(222)
35.9
(290)
32.4
(1,003)
% (patients)
Hillner et al., J Nucl Med 2008
Change in Management by Cancer Type
Ovary (Covered)
Pancreas (CED)
Prostate (CED)
Small Cell Lung
(CED)
Myeloma
(CED)
Staging
Restaging
Suspected
Recurrence
43.2
(378)
39.2
(1,491)
32.0
(2042)
43.3
(1,082)
52.2
(402)
37.7
(1,971)
38.3
(1,021)
34.0
(1,477)
40.8
(1,357)
46.4
(1009)
44.5
(2,160)
39.3
(802)
39.4
(1,790)
38.1
(544)
50.9
(373)
Hillner et al., J Nucl Med 2008
PET for Treatment Monitoring
• PET during a planned course of cancer treatment
• NOPR did not dictate or collect data on when during
treatment PET was done
• 82% Chemotherapy, 12% chemoXRT, 6% XRT
• Ovarian, pancreas, NSCLC, SCLC most frequent
• Metastatic disease in 54%
Hillner et al., Cancer 2008
PET Used for Treatment Monitoring
Switching to Another Therapy
Effect of Year and Assessment of Prognosis
Cancer
Type
Overall
2006
Overall
2007
Overall
2008
2006
If worse
2007
If worse
2008
If worse
Pancreas
30.9
28.7
25.0
66.3
58.1
55.6
Prostate
26.4
22.0
22.4
62.2
48.8
48.3
Kidney
21.9
24.0
23.1
53.7
56.6
54.2
Bladder
34.2
27.1
28.4
67.2
58.8
64.0
Stomach
20.6
25.2
24.4
46.9
59.4
59.3
No evidence supporting learning curve
Hillner et al., J Clin Oncol 2008
Summary of NOPR Results
• Change in intended management associated with PET in
previously non-covered cancers similar to that reported in
single-institution studies of covered cancers
• ~1/3 of older patients undergoing PET for cancer types
covered under Medicare’s CED policy had a major change
in intended management, including type of treatment
• Examination of individual cancers did not find a significant
difference in treatment changes between cancer
• NOPR has not yet examined if PET actually changed
patient management or if PET improved outcome
CMS Coverage with Evidence Development
Goals for NOPR 2
• Determine whether oncology care that is supported by PET
improves health outcomes, as demonstrated by:
– Improved survival,
– Improved quality of life, or
– Improved palliative care
• NOPR data show both strengths and limitations when evaluated
against CMS goals
Institute of Medicine Top 100 Priorities for
Comparative Effectiveness Research
• #17 “Compare the effectiveness of imaging
technologies in diagnosing, staging, and monitoring
positron emission tomography (PET), magnetic
resonance imaging (MRI) and computed tomography
(CT).”
The 2009 Challenge
• Such ‘comparative effectiveness’ evaluations must
move beyond the "if" to the “how" by addressing the
relative value of
– Sequencing
– Frequency
– Timing (during treatment monitoring)
– Combinations of PET, MRI and CT
• Measure actual (vs. intended management)
• Complementary prospective and retrospective studies
The Challenge to Registry-based Studies:
Defining appropriate comparison control groups
Options a) Historical controls to Non-PET care when PET not available
b) Contemporary controls to Non-PET when PET was available
Both face:
• Indication Bias
– Differ in presentation
– Differ in probability of metastasis
– Differ in potential extent of metastasis
• Provider Bias (MDs and hospital)
– Patterns of care by referring MDs and hospitals using PET likely to
differ from non-PET users
• Spectrum Bias: For non-PET imaging, clinical indication not available
Could there be a patient selection bias in NOPR?
Penetration of NOPR PET Scans for Advanced Disease
NOPR PET for
Restaging or
suspected
recurrence (% of
Annual Deaths)
CDC 65+
Incidence (2005)
CDC 65+
Deaths (2005)
NOPR 65+
Restaging or
suspected
recurrence 2007
Prostate
116,659
26,327
1,856
7.0%
Pancreas
22,252
23,397
1,035
4.4%
Kidney
21,571
8,147
1,126
13.8%
Bladder
32,800
7,627
1,202
15.7%
Stomach
12536
8,045
834
10.4%
Cancer
Grand Opportunity (GO) Grant
• Collaboration of Dartmouth, Brown, ACRIN and NOPR
• Starts 10/1/2009 (2 years)
• Proposed Projects
– Validation of Intended vs. Actual Management
– End-of-Life Care associated with PET vs. Non-PET
– Regional associations between PET use and
intensity of non-PET advanced imaging
GO Study 2: End-of-Life care in 12 to 18 months before
death, ± PET (CMS claims)
Stratify by
Comparator (Usual)
Care Group
-- NOPR years
(no PET)
-- 2004-2005
(Historical)
Control for
Cancer Type
Initial Stage
Known metastatic
disease at DX
NOPR
PET
(Metastatic dis. Pre-PET)
Comparator
Imaging
Specific cancer +
ICD-9 for
metastatic disease
Frequency & accuracy?
- 2 years
Usual Care
Deaths from
Pancreas
Bladder
Kidney
Prostate
GO Study 3:
Evaluate Geographic and Temporal Variation
1) Aggregate use for
approved cancers (e.g.
lung, colon, lymphomas)
Use Dartmouth Atlas
Hospital Referral Regions
(DA-HRR)
2) Compare pre-NOPR
(04-05) and NOPR (07-08)
utilization
Compare utilization by age
strata, gender, cancer type
HRR specific means and
rates
Study whether PET is
3) Examine rates of CT
predominantly an additive
and MRI for these cancers
or a replacement relative to
for same periods
other advanced imaging
Treatment Monitoring Revisions for NOPR 2
•
•
•
–
–
–
Updated NOPR data collection forms:
Continue collecting data on palliative v. curative goal
New questions to assess:
timing during the planned course of treatment
planned duration of therapy
Further clarify the referring physician’s impression of
response
– More clearly ask what the alternative management plan
during treatment would be if PET were not available
Final Comments
• It has taken 20-30 years for one “knowledge turn” to show
that PET has unique value in cancer management
• NOPR has shown the feasibility of performing largescale, policy-relevant imaging research that is minimally
intrusive to patients and imagers
• For current advanced imaging, the policy and economic
questions going forward are when, how often, and in what
sequence should advanced imaging be used in patients
with suspected and confirmed cancer
• Prospective multi-center investigator-initiated evaluations
are needed to confirm ‘relative’ comparative value