NDARC Annual Symposium Embedding Evaluation and Learning into the Delivery of Routine Services Louis Fiore MD, MPH lfiore@bu.edu NDARC Annual Symposium 8 October 2018

Roadmap Learning versus discovery Requirements for learning Examples of learning healthcare system activities Lessons learned

Chapter 1: Learning versus Discovery

Traditional Observational Health Research Cohort Identification Data Cleaning Data Collection Data Analysis Healthcare System Research Enterprise Knowledge Generation Publication Translation Gap

Local Learning for Care Optimization

Local Learning with Implementation Cohort Identification Data Collection Data Cleaning Data Analysis Knowledge Generation Patient Specific Decision Support Prediction Algorithm Results Individual Patient Data

Chapter 2: Requirements for Learning Engagement of care providers “Bottom Up” Data availability and provenance Of critical data elements only Data science The easy part Ability to experiment Randomization in usual care Implementation Implement as you learn

Chapter 2: Requirements for Learning Engagement of care providers “Bottom Up” Data availability and provenance Of critical data elements Data science The easy part Ability to experiment Randomization in usual care Implementation Implement as you learn

The VA VistA/CPRS National EHR RDW RDW RDW CDW RDW

Chapter 2: Requirements for Learning Engagement of care providers “Bottom Up” Data availability and provenance Of critical data elements Data science The easy part Ability to experiment Randomization in usual care Implementation Implement as you learn

Chapter 2: Requirements for Learning Engagement of care providers “Bottom Up” Data availability and provenance Of critical data elements Data science The easy part Ability to experiment Randomization in usual care Implementation Implement as you learn

Point of Care (Embedded) Clinical Trials Cohort Identification Enroll & Consent Randomize Intervention Decision Support Data Capture Study DB Analysis

Point of Care Clinical Trial A clinical trial with a substantial portion of its operations conducted by clinical staff in the course of providing patient/subject’s routine clinical care and where the choice of treatment is between two “equivalent” options

POCR Advantages Pragmatic qualities address issues of Clinical Effectiveness Faster (immediate) Integration of results into practice thereby lowering the T2 translation barrier Enhanced acceptance by providers (locally selfish) Conversion to a decision support node Improved logistics – scalable

Use of EHR Application Layer

Chapter 2: Requirements for Learning Engagement of care providers “Bottom Up” Data availability and provenance Of critical data elements Data science The easy part Ability to experiment Randomization in usual care Implementation Clinicians generate knowledge and implement change in care delivery Decision support modules

Chapter 3: Examples of Learning Healthcare System Activities Weight-based versus sliding scale insulin administration Diuretic comparison study Phenobarbital versus benzodiazepines for alcohol withdrawal syndrome Precision Oncology Program Spinal Stenosis Registry and SOLID study

LBP with Spinal Stenosis Conservative Therapy Surgery 55% 45% Spondylolisthesis 18% 82% No Spinal instability Spinal Instability No Spondylolisthesis Randomize POC-CT Decompression with fusion Objective 1: Observational Study Decompression with fusion Decompression alone Re-operation No re-operation Re-operation No re-operation (14 - 22%) ( 21 - 34%)

Chapter 3: Examples of Learning Healthcare System Activities Weight-based versus sliding scale insulin administration Diuretic comparison study Phenobarbital versus benzodiazepines for alcohol withdrawal syndrome Precision Oncology Program Spinal Stenosis Registry and SOLID study Each of these projects addresses compelling clinical issues and was proposed by clinical staff (not researchers).

Chapter 4: Lessons Learned Learning activities should be supported by clinical care services (not research services) Engaged clinicians must have a strong voice in determining the questions that are addressed (not researchers or regulators) Answers to the questions must be meaningful to providers and patients (not regulators) ‘Patient centric’ Researchers are critical partners but not owners of the process

Chapter 4: Lessons Learned continued… Experimentation (randomization) critical to validate important findings Should be designed to minimally perturb the clinical ecosystem Interventions studied must be proof-tested for implementation before study starts It is hard work to move the needle for issues relating to human subjects protection: Informed consent Engagement in research Patient privacy

Let’s start destroying those silos Clinical Hospital Administration Clinical Research Pharma

Avoid bedside-to-bookshelf activities

The Free Rider Dilemma