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How to Design
Effective
and
Efficient
Real World Trials
Todd Berner MD
Executive Medical Director, Urology
Astellas Scient...
Muller RW, ECRI Institute Conference 11.28.2012
Providers
Assuming
Risk:
• Nationwide, about 120 provider-
sponsored health plans are owned by
hospitals or health systems...
Transforming Care Delivery
Transformation
of Care
Primary
Care
Snyder R, ECRI Institute Annual Conference Nov 28,2012
Listening to the Patient Voice in
Research
PCORI Determinants of Success in
Involving Patient’s in Research
 Are we producing high-quality, timely, useful,
trustwor...
Efficacy
7
Real World Evidence
Effectiveness
Value Information= Valued Information
• Generate valued information regarding cost-
effectiveness useful to payers and/or ...
Value Evidence Generation
Effectiveness
Efficacy
Adherence
Individual
Patient
Characteristics
Tolerability
Access
Hypothesis
Question
Methodology
Data
Generation
Using Evidence to Inform
Development of Clinical Trials
• Retrospective Claims and Medical Records Database Analysis
• Ana...
RWE within the product lifecycle
Pre-launch Product Launch 1 yr post-launch 2+ yrs post-launch
Market
Shaping
• Burden of
...
Real World Evidence:
Efficacy vs. Effectiveness
Example-
 Conditions with significant patient subjective symptom
outcome ...
Inception Point:
The ‘Representativeness’ Issue
Robert M. Califf, MD
Considerations in Designing Acute
Decompensated Heart...
Pragmatic Clinical Trials
Brass EP Clin Pharmacol Ther 2010
How much could we save if
RWE replaced just a single clinical trial?
Cutting Edge Survey
Phase IIIB/IV Clinical Trials 2011
Capturing Stakeholder Voices
Uncovering the true needs
• In this changing landscape, they may not have a
clear idea
• Use...
Complexities of Defining Quality
 Measurement Challenges
• Disparate data sources needed
• What data should be reported t...
Quality Metrics
Benefit from a focused clinical set of proven,
evidenced-based process and outcome
measures that are most...
Evidenced Based Solutions
Measurement is not the endpoint
 Need to find ways to improve on performance,
using tested pro...
Performance Improvement Research Collaborative:
Blueprint
Phase I Phase II Phase III Phase IV
Research:
Burden of Illness
...
Mapping the Patient Journey
Baker GR, Schwartz F
Healthcare Quarterly, 8(3) May
2005.doi:10.12927/hcq.2005.17194
 Patients newly initiating therapy
Index event captured by HCP Investigator
 Patient then engages with Registry and pro...
Phase 1
• Descriptive analysis (baseline characteristics, treatment
patterns (adherence (primary and secondary),
discontin...
Performance Improvement within a Health System with
Significant ‘excursions’ of Patient Care Outside the System
• Assess t...
EMR Based Condition Specific Prompts and
HCP Clinical Decision Support
National Quality Strategy--The Future of Quality Me...
EMR Based Condition Specific Prompts and
HCP Clinical Decision Support
• Clinical Decision Support (CDS)
 Detect potentia...
What Does Good Look Like?
Toward Continued Excellence
• More patient-centered care
• More predictable outcomes
• Low toler...
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How to design effective and efficient real world trials TB Evidence 2014 10.23.14

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How to design effective and efficient real world trials TB Evidence 2014 10.23.14

  1. 1. How to Design Effective and Efficient Real World Trials Todd Berner MD Executive Medical Director, Urology Astellas Scientific & Medical Affairs, Inc. October 23, 2014
  2. 2. Muller RW, ECRI Institute Conference 11.28.2012
  3. 3. Providers Assuming Risk: • Nationwide, about 120 provider- sponsored health plans are owned by hospitals or health systems or are in the process of applying for a license to own health plans • Few provider-owned health plans participate in exchanges • About 15% of hospitals had PPOs, 13% HMOs and 5% fee-for-service products in 2011, with percentages relatively flat over a decade, according to AHA’s latest data Health Plan Week August 19, 2013 Volume 23 Issue 28 There are limitations for smaller provider organizations in taking full risk and becoming an insurance plan, “because you need large numbers in terms of how capitated rates are set.”
  4. 4. Transforming Care Delivery Transformation of Care Primary Care Snyder R, ECRI Institute Annual Conference Nov 28,2012
  5. 5. Listening to the Patient Voice in Research
  6. 6. PCORI Determinants of Success in Involving Patient’s in Research  Are we producing high-quality, timely, useful, trustworthy information?  Is the information we produce being used?  Are other (funders of) researchers following our lead?
  7. 7. Efficacy 7 Real World Evidence Effectiveness
  8. 8. Value Information= Valued Information • Generate valued information regarding cost- effectiveness useful to payers and/or policy makers • Real World Evidence Development • Follows from clinical findings, real-world observations – Retrospective data studies – Models of care – Prospective observations – Measures of Patient Experience(PROs) “Value is in the eyes of the Beholder”
  9. 9. Value Evidence Generation Effectiveness Efficacy Adherence Individual Patient Characteristics Tolerability Access
  10. 10. Hypothesis Question Methodology Data Generation
  11. 11. Using Evidence to Inform Development of Clinical Trials • Retrospective Claims and Medical Records Database Analysis • Analysis of treatment patterns and utilization • Analysis of prevalence and population trends • Burden of Illness • Adherence and persistence assessments • Economic and resource utilization • Effectiveness studies and outcomes analysis • Pharmacoepidemiology • Prospective Research • Patient Reported Outcomes • Treatment patterns and resource utilization • Quality of Life • Productivity
  12. 12. RWE within the product lifecycle Pre-launch Product Launch 1 yr post-launch 2+ yrs post-launch Market Shaping • Burden of Illness • Gaps in Care • RCT on-label communication • Establish place in treatment • Analysis of clinical trial subpopulations • Economic evaluations • Budget impact • CE using RCT data • PRO Measures of Patient Experience RWD generation • Adherence/ persistence • Healthcare Resource Utilization • Clinical Outcomes • Patient Satisfaction
  13. 13. Real World Evidence: Efficacy vs. Effectiveness Example-  Conditions with significant patient subjective symptom outcome measures (OAB, IBD, migraine) • RCT data Extremely high placebo response rates Difficult to show efficacy for drug compared to placebo It is essentially all non-pharmacologic therapy compared to non-pharmacologic therapy + drug • Real World data All of the behavioral, non-pharmacologic intervention associated with the RCT moves over to the drug side of the ledger This becomes a comparison of activated, engaged Rx recipients vs. those with just an Rx
  14. 14. Inception Point: The ‘Representativeness’ Issue Robert M. Califf, MD Considerations in Designing Acute Decompensated Heart Failure Clinical Trials
  15. 15. Pragmatic Clinical Trials Brass EP Clin Pharmacol Ther 2010
  16. 16. How much could we save if RWE replaced just a single clinical trial? Cutting Edge Survey Phase IIIB/IV Clinical Trials 2011
  17. 17. Capturing Stakeholder Voices Uncovering the true needs • In this changing landscape, they may not have a clear idea • Use therapeutic expertise to generate ideas • Build on those ideas they do have • But this might not be enough • May need to actually work together to figure it out • Collaborative Research The “We don’t have the bandwidth ” Challenge
  18. 18. Complexities of Defining Quality  Measurement Challenges • Disparate data sources needed • What data should be reported that will be useful to stakeholders?  to achieve behavior change  for patient choice  for payer market decisions • Criteria for Outcome Measures  Risk adjustment  Capture few unintended consequences The Joint Commission National Patient Safety Goals 2012
  19. 19. Quality Metrics Benefit from a focused clinical set of proven, evidenced-based process and outcome measures that are most likely to drive quality outcomes and have utility for patients Tie to the power of payment policies Appreciate that clinical measures not credible to clinicians don’t get measured well, nor do they result in quality improvement Many measures may not even contain the information needed to make QI changes The Joint Commission National Patient Safety Goals 2012
  20. 20. Evidenced Based Solutions Measurement is not the endpoint  Need to find ways to improve on performance, using tested proven solutions that are sustainable because they are imbedded into practice & are widely accepted Bring process improvement into solutions Solutions must be customizable to setting Improvements must be measurable to know if they work and at what cost The Joint Commission National Patient Safety Goals 2012
  21. 21. Performance Improvement Research Collaborative: Blueprint Phase I Phase II Phase III Phase IV Research: Burden of Illness Predictive Model Project Phases Recruit Health Pilot Sites Design Change Package Implement Change Package in Pilots Collect Data Disseminate Best Practices Learned Analyze Align Implement Spread Partnering to deliver improved care at a reduced cost.
  22. 22. Mapping the Patient Journey Baker GR, Schwartz F Healthcare Quarterly, 8(3) May 2005.doi:10.12927/hcq.2005.17194
  23. 23.  Patients newly initiating therapy Index event captured by HCP Investigator  Patient then engages with Registry and provides ePRO data throughout the course of 12 month observation period, independent of whether they:  Remain on treatment Switch or add on additional treatments Change or consult with multiple providers Naïve sites; Weighted PCP representation;  Linkage to multipayer/allpayer claims data of meaningful scale is still lacking The Patient Journey- An Observational Registry
  24. 24. Phase 1 • Descriptive analysis (baseline characteristics, treatment patterns (adherence (primary and secondary), discontinuation, and switchers) and healthcare resource use. • Identify risk factors associated with treatment patterns Phase 2 • Survey patients newly initiated on therapy who subsequently Persist, Switch, or Discontinue Phase 3 • Implement Quality Improvement intervention using results from Phase II in Health System Patient population with the condition Primary Nonadherence within a ‘Closed’ Healthcare System
  25. 25. Performance Improvement within a Health System with Significant ‘excursions’ of Patient Care Outside the System • Assess treatment approaches, Explore several measures:  Compare the number of visits during which condition was:  Appears in the problem list  Listed as a diagnosis  A drug was prescribed for the condition  Examine appropriateness of referral patterns:  PCP should try at least one drug for this condition– but only one – before referring to a specialist  Referrals made without trying any drug or after prescribing more than one will be classified as potentially inappropriate or suboptimal  Subgroup analysis of referrals from the Health System’s primary care clinic network vs others  Examine the use of diagnostic testing and imaging  Examine the use and documentation of validated symptom assessment tools.  Compare presentation of treatment options to patients among those that we can identify via the EHR
  26. 26. EMR Based Condition Specific Prompts and HCP Clinical Decision Support National Quality Strategy--The Future of Quality Measurement ONC, AHRQ, CMS Presentation. September 14, 2012
  27. 27. EMR Based Condition Specific Prompts and HCP Clinical Decision Support • Clinical Decision Support (CDS)  Detect potential safety and quality problems and help prevent them  Detect inappropriate utilization of services, medications, and supplies  Foster the greater use of evidence-based medicine principles and guidelines  Organize, optimize and help operationalize the details of a plan of care  Help gather and present data needed to execute this plan  Ensure that the best clinical knowledge and recommendations are utilized to improve health management decisions by clinicians and patients Osheroff JA, Pifer EA, Teich JM, et al. Improving Outcomes with Clinical Decision Support: An Implementers' Guide Chicago: HIMSS; 2005.
  28. 28. What Does Good Look Like? Toward Continued Excellence • More patient-centered care • More predictable outcomes • Low tolerance for unwarranted deviations from the norm • Collective Mindfulness • Greater efficiencies, less waste • More appropriate/evidence based care that meets shared expectations • Evolving “Learning” Systems The Joint Commission National Patient Safety Goals 2012

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