Development of Harmonized Outcome Measures for Use in Research and - PowerPoint PPT Presentation

Development of Harmonized Outcome Measures for Use in Research and Clinical Practice Richard Gliklich, MD Elise Berliner, PhD Michelle Leavy, MPH Agency for Healthcare Research and Quality OM1, Inc. April 12, 2019

PROJECT PURPOSE & OBJECTIVES 2

Question • Can we collect detailed and standardized information across patients, settings and treatments to understand which factors lead to improved outcomes? 3

What is a Patient Registry? “an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure and that serves one or more pre-determined scientific, clinical, or policy purposes” Gliklich R, Dreyer N, Leavy M, eds. Registries for Evaluating Patient Outcomes: A User’s Guide. Third edition. Two volumes. (Prepared by the Outcome DEcIDE Center [Outcome Sciences, Inc., a Quintiles company] under Contract No. 290 2005 00351 TO7.) AHRQ Publication No. 13(14)-EHC111. Rockville, MD: Agency for Healthcare Research and Quality. April 2014. https://effectivehealthcare.ahrq.gov/topics/registries-guide-3rd- edition/research/. 4

Traditional Uses for Registries • Observe the natural history of a disease/condition • Understand variations in treatment and outcomes • Examine factors influencing prognosis, quality of life • Describe care patterns, including appropriateness of care and disparities in the delivery of care • Assess effectiveness • Monitor safety and harm • Measure quality of care 5

New Applications for Registries • Providing infrastructure for embedded / nested studies (e.g., randomized trials, pragmatic trials) • Supporting value-based care efforts (e.g., ACOs, alternative payment models) • Providing evidence for coverage and reimbursement • Combining data with other sources as part of networks to support new research or safety surveillance (e.g., PCORnet, Sentinel) • Providing decision support at the point of care – particularly when integrated with EHRs 6

Registries & Learning Health Systems Registries: Registries: • Observe natural • Track long-term patient history of disease outcomes Patient • Assess effectiveness • Collect PROs Research Outcomes • Meet post-marketing commitments Learning Health System Registries: Registries: • Collect & transmit data • Support Quality Clinical for quality reporting reimbursement and Improvement Practice • Provide tools to value-based care support quality • Support accreditation improvement • Provide decision support 7

Current Investment in Registries • Thousands of registries exist – ► Over 4,500 have registered voluntarily on ClinicalTrials.gov ► They cover hundreds of condition areas ► They range from a few patients to >20 million • Existing registries represent a: ► Powerful resource for new research ► Enormous investment in data infrastructure ► Tool to support value-based care ► Potential foundation for learning health systems and embedded trials 8

Improving Registry Utility & Value • Registries, even within the same clinical area, define and capture different outcome measures • This makes it difficult to connect data across registries and across health IT systems • Question : how do we improve the ability of registries to connect to other registries and other health IT systems? 9

Outcome Measure Harmonization • Harmonization of outcome measures is key: ► To compare and aggregate results between and among registries, clinical research, quality reporting, etc. ► To facilitate performance and value-based measurement 10

OUTCOME MEASURES FRAMEWORK A standard, common model for patient and provider relevant outcome measures within and across condition areas 11

Variation in Outcome Definitions Exacerbation Definitions Used in Asthma Registries 2017 GINA Report 2 NIH Workshop 1 ATS/ERS Statement 3 An exacerbation is a worsening Exacerbations of asthma are Severe asthma exacerbations of asthma requiring the use of episodes characterized by a are events that require urgent systemic corticosteroids (or for progressive increase in action on the part of the patient patients on a stable symptoms of shortness of and physician to prevent a maintenance dose, an increase breath, cough, wheezing or serious outcome, such as in the use of systemic chest tightness and progressive hospitalization or death from corticosteroids) to prevent a decrease in lung function, i.e., asthma. Severe asthma serious outcome. they represent a change from exacerbations include at least the patient’s usual status that is OR one of the following: sufficient to require a change in (a) Use of systemic treatment. corticosteroids or an increase from a stable maintenance dose, for at least 3 days . (b) A hospitalization or ER visit 1 Fuhlbrigge et al. J Allergy Clin Immunol. 2012 Mar;129(3 Suppl):S34-48. because of asthma, requiring 2 GINA. Global Strategy for Asthma Management and Prevention, 2017. systemic corticosteroids. 3 Reddel et al.. Am J Respir Crit Care Med. 2009 Jul 1;180(1):59-99 12

Why Is This So Hard? • Different views on what constitutes an outcome measure • Different goals in different studies • Continuous reinventing of the wheel • An industry that has grown up on quality and process measures • The centrality of the patient not always considered • No roadmap • No organized way to harmonize differences 13

Outcome Measures Framework (OMF) • Goal: Common, conceptual model for classifying the range of outcomes that are relevant to patients and providers across most conditions • Process: Stakeholder-driven (~400) process incorporating iterative rounds of review and revision across multiple condition areas 14

Characteristics Treatment Outcomes Participant Survival Demographics Overall Mortality Genetics Cause-Specific Mortality Family/Participant/Social History Disease Free Survival Functional/Performance Status Other Health Behaviors Clinical Response Environmental Exposures Recurrence/Exacerbation/ Preferences for Care Improvement/Progression/ Type Change in Status/Other Disease Surgical Diagnosis Medical Events of Interest Risk Factors Device Adverse Events/Exacerbations/ Staging Systems Alternative Complications/ Genetics of Disease Education Other Tissue or Infectious Agent Patient Reported Biomarkers Intent Functioning Comorbidities/Symptoms Palliative/Management vs. Quality of Life Assessment Scales Curative Other Physical Findings Severity Resource Utilization Disease Understanding Inpatient Hospitalization/ Office Visits/ED Visits/ Provider Productivity/ Training/Experience Additional Treatments/ Geography Procedures/Direct Cost/Other Practice Setting ------------------------------ Academic vs. Community Impact on Non-Participant 15 Gliklich RE, Leavy MB, Karl J, Campion DM, Levy D, Berliner E. A framework for creating Experience of Care standardized outcome measures for patient registries. J Comp Eff Res. 2014;3(5):473‐480.

Building on Existing Efforts And other efforts… 16 16

OMF Harmonization Project Goals • Assess whether harmonized outcome measures can be developed for a sample set of 5 clinical areas: Atrial fibrillation 1. Asthma 2. Depression 3. Lung cancer 4. Lumbar Spondylolisthesis 5. • Translate narrative harmonized definitions into standardized terminologies to facilitate consistent capture and extraction of measures from EHRs, registries, and other research studies • Develop final report on policies and best practices for harmonization and development of standardized libraries of outcome measures 17

HARMONIZATION USING THE OMF An example from the Depression Workgroup 18

Methodology Overview 2. Collected & 1. Recruited 3. Built proposed categorized registries & minimum measure outcome measures stakeholders set using OMF 4. Harmonized 5. Identified key definitions for characteristics to 6. Produced final measures in support risk standardized library minimum set adjustment Completed with 5 workgroup meetings over 8 months 19

Participating Registries: Variations in Purposes, Patient Populations & Data Narrow patient • UTSW Depression Cohort population, • Dallas 2K consistently collected • Mood Network (PCORnet) detailed data • NNDC Mood Outcomes Program Research • Treatment-Resistant Depression Quality Improvement • MN Community Measurement* • PRIME Registry* *Qualified Clinical • PsychPRO* Data Registry (QCDR) Broad patient population, variation • Dept. of Veterans Health System / Population in data consistency & detail Management Affairs 20

Participating Stakeholders Professional Societies Patient Advocacy Organizations Patient Advocacy Organizations Professional Associations • Depression and Bipolar Support • American Psychological Association Alliance • American Psychiatric Association • International Foundation for Research • American Board of Family Medicine and Education on Depression • National Alliance on Mental Illness Federal Agencies Payers Payers Federal Agencies • FDA • CMS • National Institute of Mental Health • Blue Cross Blue Shield of • National Library of Medicine Massachusetts • SAMHSA • National Cancer Institute (PROMIS)