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Patient Registries: Ditch the Silos and Create an Ecosystem of Discovery


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Siloed thinking, practices and technology greatly undermines potential to advance research, treatments and cures for most diseases. This is a shot at a vision to address this challenge, starting with a disease called primary ciliary dyskinesia (PCD).

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Patient Registries: Ditch the Silos and Create an Ecosystem of Discovery

  1. 1. Building A Discovery Platform to Advance Research, Diagnosis, Care and Cures
  2. 2. Initial Goals • Track the natural history and clinical outcomes • Improve and inform clinical practice and patient care quality • Expand evidence-based research on genetic, molecular and physiological basis • Provide a framework for recruitment, participation/surveillance in/of clinical trials and treatment development • Inform stakeholder decision-making through tracking patient, family and caregiver experiences
  3. 3. State-of-the-Art Platform To collect, analyze and disseminate information on demographics, genotype/phenotype, natural history, health outcomes and clinical care. • Phased (“building block”) approach as resources, insights, network, technology and healthcare/IT standards evolve • Interoperable, open-standards-based foundation to ensure collaboration, transferability, integration and secure communication across systems and applications as needed • Organized system to collect uniform data to evaluate specified outcomes based on observational study methods • Store, retrieve and disseminate data collected on identifiable individuals • Leverage best-practices to maximize validity and representativeness of data • Capitalize on functionality/insights to serve as clinician training tool
  4. 4. PCDF Registry: Discovery Platform To Advance Research, Diagnoses, Care & Cures • Annual Reports • Program Dev • CRCN Progress • New Insights Single Point of Truth/ Central Repository for ClinROs, PROs, Research Studies, Integration with External/Third Party Applications Quality Control Patients (incl. families, caregivers): PROs, Care Insights Clinicians (PCDF Clinical and Research Centers): ClinROs Researchers, Industry (Studies, RxTx Dev) : Approved Access Clinical, Aggregate Insights Consent, Surveys, Education, Engagement Recruitment Patient Insights Reporting Analytics Genotype/ Phenotype Genetics Biobank EMR/EHR Clinical Tr Mgmt Registry (Others) For more information on the PCDF Foundation or our Clinical and Research Centers Network (CRCN), please visit
  5. 5. • Basic Demographics and Vital Statistics 1. Where are patients located? 2. What does the patient group look like? 3. What are the vital statistics including life expectance, age of transplant/disability/death, etc.? • Longitudinal Natural History and Clinical Outcomes 1. How does the disease progress over time? 2. What microbes are most commonly seen? 3. When does lung damage occur? Under what circumstances? 4. What’s the status of hearing loss and sinus disease? 5. Are these issues genotype-dependent? Questions To Answer (Samples)
  6. 6. • Clinical Practice Improvement 1. What are the care patterns? 2. What are the clinical practices and associated outcomes? 3. What therapies are most effective? 4. Where are the gaps/disparities in care/access? 5. What are the attributes of patients for whom therapy appears most effective? 6. How do centers’ outcomes compare by patient population, treatments, etc.? • Research Insights 1. What are the areas where research is needed to fill gaps in evidence? 2. What are the genotype-phenotype correlations? 3. What can we translate from the lab into therapeutics? For which mutations? 4. Are there ways to classify mutations functionally/clinically for targeted therapies Questions To Answer (cont’d)
  7. 7. • Clinical Trials and Treatment Pipeline 1. Where do therapeutic targets come from? 2. What is the clinical effectiveness of X? For which mutation(s)? 3. What is the comparative effectiveness of X and Y? for which mutation(s)? 4. What side effects are patients experiencing with X? • Informed Patient, Care team, Public Health and Quality of Life 1. What’s the rate of misdiagnosis? 2. What factors delay a correct diagnosis? 3. How long does it take to diagnose a patient? 4. How often are patients treated for the wrong condition? 5. How does my health compare with the health of others with PCD? 6. What can I do to improve my health? 7. What factors influence prognosis and quality of life (PROs)? Questions To Answer (cont’d)
  8. 8. Questions, Comments, Collaborative Opportunities Carey Kauffman, PCDF VP, Board Member e: t: +1 404.840.7151