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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.

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Presentation on theme: "Databases or Registries? Points to Consider Mary Lou Skovron, DrPH Group Director, Global Epidemiology Bristol-Myers Squibb FDA/Industry Statistics Workshop."— Presentation transcript:

1 Databases or Registries? Points to Consider Mary Lou Skovron, DrPH Group Director, Global Epidemiology Bristol-Myers Squibb FDA/Industry Statistics Workshop September 29, 2006

2 Overview Claims databases Claims databases TypesTypes AdvantagesAdvantages LimitationsLimitations Registries Registries TypesTypes AdvantagesAdvantages LimitationsLimitations Examples Examples Conclusions Conclusions

3 Claims Databases

4 Claims Databases Types Open-Plan eg UHC, PharMetrics Open-Plan eg UHC, PharMetrics Pharmacy-basedPharmacy-based Practice-basedPractice-based Hospital-basedHospital-based HMO, eg Kaiser-Permanente HMO, eg Kaiser-Permanente Government eg Medicare, Medicaid Government eg Medicare, Medicaid

5 Claims Databases: Advantages Already exist, accruing patient information…useful when a relatively quick answer is needed 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 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 May include subgroups not included in clinical trials…expand knowledge

6 Claims Databases: Limitations ICD-9 coding… potential for misclassification ICD-9 coding… potential for misclassification Limited sensitivity/specificityLimited sensitivity/specificity Coding affected by reimbursementCoding affected by reimbursement Short residence in the database Short residence in the database Clinical, lab, imaging data not usually present Clinical, lab, imaging data not usually present Rare events in rare diseases require huge electronic populations to identify adequate numbers treated Rare events in rare diseases require huge electronic populations to identify adequate numbers treated May not represent important sub-populations May not represent important sub-populations Surveillance bias and channeling bias Surveillance bias and channeling bias Inpatient drug exposures not usually recorded Inpatient drug exposures not usually recorded

7 Claims Databases: Application Acute events (short-term events) Acute events (short-term events) Events that come to medical attention, eg CVA Events that come to medical attention, eg CVA Validated algorithm to identify indication and event of interest OR medical record review to verify events 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) Statistical approaches for confounders (eg propensity scores, instrumental variables, risk factor scores, multivariate analyses)

8 Registries

9 Registries A systematic collection of defined events or product exposures in a defined patient population for a defined period of time 1 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 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 1 Arlett P, Moseley J, Seligman PJ p 119 in Pharmacoepidemiology Fourth Edition, Strom BL, ed CIOMS V Section II.h.

10 Registries: Types Drug/Device Registry: Includes subjects receiving the drug or device regardless of indication 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 Disease Registry: Includes patients with the disease regardless of drug or device exposure

11 Registries: Strengths Richer data than in electronic databases: Patient SES, history, treatment, clinical data can be collected Richer data than in electronic databases: Patient SES, history, treatment, clinical data can be collected Define encounter frequency and follow-up duration Define encounter frequency and follow-up duration Event ascertainment does not depend on ICD-9 coding Event ascertainment does not depend on ICD-9 coding Can address additional questions in the data Can address additional questions in the data

12 Registries: Limitations Accrual can be slow if insufficient sites engaged Accrual can be slow if insufficient sites engaged Generalizability must be established Generalizability must be established Practical limits on number of patients followed Practical limits on number of patients followed

13 Registries: Application Long-term outcomes Long-term outcomes Rare diseases Rare diseases Potential confounders important Potential confounders important Multiple objectives Multiple objectives

14 Usefulness of Registries Characteristics of patients in the target population for the new drug Characteristics of patients in the target population for the new drug Clinical course of the disease Clinical course of the disease Treatment patterns, health care utilization Treatment patterns, health care utilization Frequency of adverse events Frequency of adverse events

15 Registry Lifecycle Early: Describe patient demographics, clinical characteristics, practice patterns (usually cross-sectional analyses); incidence of AEs with short lag times 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 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 Advanced: Evaluate changes in practice patterns; impact on outcomes and AE incidence; assess rare AE incidence

16 Registry Quality DeCIDE Network currently developing a reference document on developing, conducting and evaluating registries DeCIDE Network currently developing a reference document on developing, conducting and evaluating registries Sponsored by AHRQ Sponsored by AHRQ Document in draft, Outline available on the web Document in draft, Outline available on the web Report currently in draft Report currently in draft

17 Examples

18 Example: Cox-2 Inhibitors and Cardiac Events Cox-2 use frequent in population Cox-2 use frequent in population Primary care drug Primary care drug Cardiac events not rare in target population Cardiac events not rare in target population Short-term (< 2 years) events Short-term (< 2 years) events Clinical history important Clinical history important

19 One Solution Cox-2 Inhibitors and Cardiac Events Claims database analysis 1 Claims database analysis 1 Advantages:Advantages: Data already accrued when study need identified Data already accrued when study need identified Large population Large population Limitation offsetLimitation offset Multivariate regression to control confounding Multivariate regression to control confounding Verified cardiac events by chart review Verified cardiac events by chart review Remaining limitationsRemaining limitations Under-represented > 65 yo Under-represented > 65 yo 1 Velentgas P et al 2006

20 Key Feature of the Solution Numbers readily available: Numbers readily available: Exposure: ~ 425,000 eligible subjects available for studyExposure: ~ 425,000 eligible subjects available for study at least one dispensing of the 5 study medications during preceding 18-month period 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 first dispensing after minimum of six months without any of the medications Endpoint: ~ 725 confirmed MI/ACSEndpoint: ~ 725 confirmed MI/ACS Verification of endpoints Verification of endpoints Medial record review applying accepted criteriaMedial record review applying accepted criteria

21 Example: Thrombolytics And Bleeding Important focus: subpopulations eg ethnicity, gender, age Important focus: subpopulations eg ethnicity, gender, age Short-term event Short-term event Benefit and risk Benefit and risk Hospital-based treatment Hospital-based treatment

22 Solution Thrombolytics and Bleeding Registry approach: National Registry of Myocardial Infarctions Registry approach: National Registry of Myocardial Infarctions Salient strengths: Salient strengths: Clinical and lab data ascertainedClinical and lab data ascertained Data from medical recordsData from medical records Limitation Offset Limitation Offset Validated completeness against another data sourceValidated completeness against another data source Large number of hospitals to assure adequate subpopulationsLarge number of hospitals to assure adequate subpopulations Remaining limitations Remaining limitations Short-term data cannot answer long-term questionsShort-term data cannot answer long-term questions

23 Key Feature of the Solution Large number of hospitals participate order to gather data on~200,000 MIs per year Large number of hospitals participate order to gather data on~200,000 MIs per year Rich patient, clinical, treatment and outcome information Rich patient, clinical, treatment and outcome information Answered the question about safety in subgroups under-represented in the clinical trials 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 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 External investigators can propose research; external advisory group reviews and approves all research

24 Example: Safety of Orencia®, a New Biological for RA Short-term (infections) and longer- term (NHL and other malignancies) events Short-term (infections) and longer- term (NHL and other malignancies) events Low general population prevalence of RA candidates for biologics treatment Low general population prevalence of RA candidates for biologics treatment RA a specialty-treated disorder RA a specialty-treated disorder Proactive approach Proactive approach

25 Solution: Complementary Approaches Claims database study in UHC data Claims database study in UHC data Event validationEvent validation Large pool of potential comparatorsLarge pool of potential comparators Population treatment patternsPopulation treatment patterns Infections, other possible short-term eventsInfections, other possible short-term events Registry building on the independently conducted National databank for Rheumatic Diseases Registry building on the independently conducted National databank for Rheumatic Diseases Large pool of participating rheumatologistsLarge pool of participating rheumatologists 5,000 Orencia® initiators comparison to >=15,000 initiators/switchers of other treatments5,000 Orencia® initiators comparison to >=15,000 initiators/switchers of other treatments Patient self-report and event verificationPatient self-report and event verification Enrollment and retention enhancementsEnrollment and retention enhancements Short and long-term eventsShort and long-term events

26 Key Features of the Solution External scientific advisory group External scientific advisory group Individual protocolsIndividual protocols Statistical analysis plansStatistical analysis plans Interpretation of results including approaches to integrationInterpretation of results including approaches to integration

27 Conclusions

28 Conclusions One size does not fit all: evaluate options One size does not fit all: evaluate options Registries: a useful alternative to electronic databases Registries: a useful alternative to electronic databases Consider complementary approaches Consider complementary approaches

29 Useful References Strom BL, ed; Pharmacoepidemiology 4 th Edition; UK; John Wiley and Sons Ltd;2005 Strom BL, ed; Pharmacoepidemiology 4 th Edition; UK; John Wiley and Sons Ltd;2005 AHRQ DeCIDE Network upcoming publication AHRQ DeCIDE Network upcoming publication

30 Thank You


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