NOPR 2006: Results and Lessons Learned (Text Version)

On September 29, 2010, Bruce Hillner made this presentation at the 2010 Annual Conference. Select to access the PowerPoint® presentation (415 KB). Free PowerPoint® Viewer (Plugin Software Help).

Slide 1

NOPR [National Oncologic PET Registry] 2006: Results and Lessons Learned

2010 Annual AHRQ Conference

Bruce E. Hillner, M.D.
Eminent University Scholar and Professor
Virginia Commonwealth University
Richmond, VA

Slide 2

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

Slide 3

PET Changed Intended Management in 36.5% of Cases

Hillner et al., J Clin Oncol 2008.

Slide 4

Changes in Intended Management (%) Stratified by Pre-PET Plan

Pre-PET Plan

Hillner et al., J Clin Oncol 2008.

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Change in Management by Cancer Type

Hillner et al., J Nucl Med 2008.

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Global Summary

• Change in intended management associated with PET in previously non-covered cancers was 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.
• The relative impact of PET on intended management was observed across the full spectrum of indications for PET.

Hillner et al., J Clin Oncol 2008.

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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.
• Results similar to more tightly managed single-institution studies (e.g., Hillner, J Clin Oncol 22:4147, 2004) and to Australian studies with outcome validation (Scott, J Nucl Med 49:1451, 2008),

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NOPR Limitations (1)

• Data "quality."
• Potential that physicians may have been influenced by the knowledge that future Medicare reimbursement might be influenced by their responses.
• Collected change in "intended" management, not actual management.
• Unknown if management changes were in the correct direction or improve long-term outcomes.
• Defining the relevant long-term outcomes for a diagnostic (instead of therapeutic) procedure is controversial.

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NOPR 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.

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Lessons Learned 1: Must Do Even if Painful

• Preparing a formal "operations manual" similar in structure to a clinical trial protocol document.
• Project annual registry enrollment for relevant time frame.
• Prepare a statistical plan even if multiple definitions of 'meaningful' change in endpoints are considered.
• If registry includes multiple diseases (e.g. different types of cancer) or sub-groups (e.g. past myocardial infarctions vs. angina), define the priority areas for first analysis.

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Lessons Learned 2

• Design your case report forms such that all data fields must be complete before accepting record.
• Web-based entry of data to 'center' minimizes costs and assists data integrity.
• No evidence that being the treating physician was associated with higher rates of change in the post-PET (treatment) strategies.

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Lessons Learned 3

• The primary endpoint of 'intended' vs. "actual" management was a compromise.
• Define and concurrently implement 'validation' strategy from the onset of the registry:
  • Prospective claims collection.
  • Prospective selected chart audits ($).
• Consider how proximate imaging is to preferred 'hard endpoint'.

Slide 13

CMS Decision Memo
CAG-00181R
April 3, 2009

New Framework differentiates PET imaging into uses informing initial treatment strategies from uses guiding subsequent treatment after completion of initial treatment.

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IOM Priority 17/100

"Compare the effectiveness of imaging technologies in diagnosing, staging, and monitoring positron emission tomography (PET), magnetic resonance imaging (MRI) and computed tomography (CT)."

Image: Cover of Institute of Medicine (IOM) report titled Initial National Priorities for Comparative Effectiveness Research.

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Prospective Concurrent Claims Database of Registry Participants is Necessary but Not Necessarily Sufficient for Proving Better Outcomes

• Relevant outcome rarely proximate to diagnostic test.
• Discordance between intended vs. actual management is more likely to vary depending on the type of intended management than on the value of the test information.
• Defining control group is problematic.

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The Challenge to Registry-based Studies: Defining appropriate comparison control groups

Options:

1. Historical controls to Non-PET care when PET not available.
2. 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.

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2010 CER 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
• Comparisons between imaging types less likely to benefit from registry design.
• Complementary prospective and retrospective studies.

Slide 18

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.
• Going forward the policy and economic questions for advanced imaging 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.