Databases or Registries? Points to Consider Mary Lou Skovron, DrPH



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Databases or Registries? Points to Consider

  • Mary Lou Skovron, DrPH

  • Group Director, Global Epidemiology

  • Bristol-Myers Squibb

  • FDA/Industry Statistics Workshop

  • September 29, 2006


Overview

  • Claims databases

    • Types
    • Advantages
    • Limitations
  • Registries

    • Types
    • Advantages
    • Limitations
  • Examples

  • Conclusions



Claims Databases



Claims Databases Types

  • Open-Plan eg UHC, PharMetrics

    • Pharmacy-based
    • Practice-based
    • Hospital-based
  • HMO, eg Kaiser-Permanente

  • Government eg Medicare, Medicaid



Claims Databases: Advantages

  • Already exist, accruing patient information…useful when a relatively quick answer is needed

  • Potentially large populations from which to draw treated patient and comparison samples…statistical power not usually an issue

  • May include subgroups not included in clinical trials…expand knowledge



Claims Databases: Limitations

  • ICD-9 coding… potential for misclassification

    • Limited sensitivity/specificity
    • Coding affected by reimbursement
  • Short ‘residence’ in the database

  • Clinical, lab, imaging data not usually present

  • Rare events in rare diseases require huge “electronic” populations to identify adequate numbers treated

  • May not represent important sub-populations

  • Surveillance bias and channeling bias

  • Inpatient drug exposures not usually recorded



Claims Databases: Application

  • Acute events (short-term events)

  • Events that come to medical attention, eg CVA

  • Validated algorithm to identify indication and event of interest OR medical record review to verify events

  • Statistical approaches for confounders (eg propensity scores, instrumental variables, risk factor scores, multivariate analyses)



Registries



Registries

  • “A systematic collection of defined events or product exposures in a defined patient population for a defined period of time”1

  • “A registry per se is not a study. It is an organized collection of data in humans within a particular disease group or other special group…”2



Registries: Types

  • Drug/Device Registry: Includes subjects receiving the drug or device regardless of indication

  • Disease Registry: Includes patients with the disease regardless of drug or device exposure



Registries: Strengths

  • Richer data than in electronic databases: Patient SES, history, treatment, clinical data can be collected

  • Define encounter frequency and follow-up duration

  • Event ascertainment does not depend on ICD-9 coding

  • Can address additional questions in the data



Registries: Limitations

  • Accrual can be slow if insufficient sites engaged

  • Generalizability must be established

  • Practical limits on number of patients followed



Registries: Application

  • Long-term outcomes

  • Rare diseases

  • Potential confounders important

  • Multiple objectives



Usefulness of Registries

  • Characteristics of patients in the target population for the new drug

  • Clinical course of the disease

  • Treatment patterns, health care utilization

  • Frequency of adverse events



Registry Lifecycle

  • Early: Describe patient demographics, clinical characteristics, practice patterns (usually cross-sectional analyses); incidence of AEs with short lag times

  • Intermediate: Analyze relationships pf patient characteristics, treatment with outcomes; incidence of less common AEs, AEs with longer lag times; assess risk factors for AE incidence

  • Advanced: Evaluate changes in practice patterns; impact on outcomes and AE incidence; assess rare AE incidence



Registry Quality

  • DeCIDE Network currently developing a reference document on developing, conducting and evaluating registries

  • Sponsored by AHRQ

  • Document in draft, Outline available on the web

  • Report currently in draft



Examples



Example: Cox-2 Inhibitors and Cardiac Events

  • Cox-2 use frequent in population

  • Primary care drug

  • Cardiac events not rare in target population

  • Short-term (< 2 years) events

  • Clinical history important



One Solution Cox-2 Inhibitors and Cardiac Events

  • Claims database analysis1

    • Advantages:
      • Data already accrued when study need identified
      • Large population
    • Limitation offset
      • Multivariate regression to control confounding
      • Verified cardiac events by chart review
    • Remaining limitations
      • Under-represented > 65 yo


Key Feature of the Solution

  • Numbers readily available:

    • Exposure: ~ 425,000 eligible subjects available for study
      • at least one dispensing of the 5 study medications during preceding 18-month period
      • first dispensing after minimum of six months without any of the medications
    • Endpoint: ~ 725 confirmed MI/ACS
  • Verification of endpoints

    • Medial record review applying accepted criteria


Example: Thrombolytics And Bleeding

  • Important focus: subpopulations eg ethnicity, gender, age

  • Short-term event

  • Benefit and risk

  • Hospital-based treatment



Solution Thrombolytics and Bleeding

  • Registry approach: National Registry of Myocardial Infarctions

  • Salient strengths:

    • Clinical and lab data ascertained
    • Data from medical records
  • Limitation Offset

  • Remaining limitations

    • Short-term data cannot answer long-term questions


Key Feature of the Solution

  • Large number of hospitals participate order to gather data on~200,000 MIs per year

  • Rich patient, clinical, treatment and outcome information

  • Answered the question about safety in subgroups under-represented in the clinical trials

  • In its > 10 year lifecycle has contributed to broad understanding and improvement of medical practice

  • External investigators can propose research; external advisory group reviews and approves all research



Example: Safety of Orencia®, a New Biological for RA

  • Short-term (infections) and longer-term (NHL and other malignancies) events

  • Low general population prevalence of RA candidates for biologics treatment

  • RA a specialty-treated disorder

  • Proactive approach



Solution: Complementary Approaches

  • Claims database study in UHC data

    • Event validation
    • Large pool of potential comparators
    • Population treatment patterns
    • Infections, other possible short-term events
  • Registry building on the independently conducted National databank for Rheumatic Diseases

    • Large pool of participating rheumatologists
    • 5,000 Orencia® initiators comparison to >=15,000 initiators/switchers of other treatments
    • Patient self-report and event verification
    • Enrollment and retention enhancements
    • Short and long-term events


Key Features of the Solution

  • External scientific advisory group

    • Individual protocols
    • Statistical analysis plans
    • Interpretation of results including approaches to integration


Conclusions



Conclusions

  • One size does not fit all: evaluate options

  • Registries: a useful alternative to electronic databases

  • Consider complementary approaches



Useful References

  • Strom BL, ed; Pharmacoepidemiology 4th Edition; UK; John Wiley and Sons Ltd;2005

  • AHRQ DeCIDE Network upcoming publication

    • http://effectivehealthcare.ahrq.gov/decide/


Thank You




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