nopr 2006-2009 and the new era of comparative effectiveness
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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. Medicare (CMS) Coverage of New Technologies. Standard for reimbursement is “reasonable and necessary” - PowerPoint PPT PresentationTRANSCRIPT
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NOPR 2006-2009 NOPR 2006-2009 and the New Era of and the New Era of
Comparative EffectivenessComparative Effectiveness
Bruce E. Hillner, M.D.Eminent University Scholar and Professor
Virginia Commonwealth UniversityRichmond, VA
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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 cancer-specific and indication-specific basis
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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
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
Referring MD requests PET
Pre-PET Form
PETdone
PET interpreted& reported
Post-PETForm sent,
including question for referring MD consent
Post-PET Form completed.
Claim submitted
Ongoingpatient
management
NOPR Workflow
Ask patient for consent
SuspectedCancer
(Diagnosis)Restaging Suspected
Recurrence
TreatmentMonitoring
LaterSuspected
Recurrences
Timing of PET in Cancer Natural History
TreatmentMonitoring
InitialStaging
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
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
5. If PET were not available, your current management strategy would be (select one)?
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 ResultsOverall 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
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
Patients with change post-PET (%) 31.1 39.5 36.1 39.0 36.5
Hillner et al., J Clin Oncol 2008
Clinical Indication for PET Study (Percent)
Pre-Pet Plan
Post-PET Plan
Dxn=5,616
Staging n=6,464
Restaging n=5,607
Recurrence n=5,388
Alln=22,975
Treat Same 16.0 46.5 15.8 20.4 25.5
Non-Treat Same 52.9 14.0 48.0 40.7 37.9
Changes in Intended Management (%) Stratified by Pre-PET Plan
Image n=9,518
Biopsy n=3,552
Watch n=2,199
Treatment n=7,706
Post-PET Plan
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
Pre-PET Plan
Hillner et al., J Clin Oncol 2008
Change in Management by Cancer Type
Staging Restaging Suspected Recurrence
Bladder (CED) 39.9(1,461)
36.4(1,239)
36.7(878)
Brain (CED) -- -- 40.5(222)
Cervix (Covered) 36.1 (341)
26.9 (353)
35.9(290)
Kidney (CED) 41.1 (895)
34.4 (979)
32.4(1,003)
% (patients)
Hillner et al., J Nucl Med 2008
Change in Management by Cancer Type
Staging Restaging Suspected Recurrence
Ovary (Covered) 43.2(378)
37.7 (1,971)
44.5(2,160)
Pancreas (CED) 39.2(1,491)
38.3 (1,021)
39.3(802)
Prostate (CED) 32.0(2042)
34.0 (1,477)
39.4(1,790)
Small Cell Lung(CED)
43.3(1,082)
40.8 (1,357)
38.1 (544)
Myeloma(CED)
52.2(402)
46.4(1009)
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 MonitoringSwitching to Another Therapy
Effect of Year and Assessment of Prognosis
CancerType
Overall2006
Overall2007
Overall2008
2006If worse
2007If worse
2008If 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
Hillner et al., J Clin Oncol 2008No evidence supporting learning curve
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 groupsOptions a) Historical controls to Non-PET care when PET not available
b) Contemporary controls to Non-PET when PET was availableBoth 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
CancerCDC 65+
Incidence (2005)CDC 65+
Deaths (2005)
NOPR 65+ Restaging or
suspected recurrence 2007
NOPR PET for Restaging or
suspected recurrence (% of Annual Deaths)
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%
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
Deaths from PancreasBladderKidney
Prostate
Specific cancer +ICD-9 for
metastatic disease
Comparator (Usual) Care Group-- NOPR years (no PET)-- 2004-2005 (Historical)Control forCancer TypeInitial StageKnown metastatic disease at DX
GO Study 2: End-of-Life care in 12 to 18 months before death, ± PET (CMS claims)
Stratify by
- 2 years
NOPR
Usual Care
PET(Metastatic dis. Pre-PET)
ComparatorImaging
Frequency & accuracy?
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 typeHRR specific means and rates
3) Examine rates of CT and MRI for these cancers for same periods
Study whether PET is predominantly an additive or a replacement relative to 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 large-scale, 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