1. PFF Teal = 0+160+175 MAIN COLORS PFF Green = 120+162+47 Light Green = 193+216+47 Red = 242+102+73 HIGHLIGHT COLORS Light Grey = 220+220+210 Dark Grey = 100+98+76 Black = 0+0+0 PATIENT-CENTERED RESEARCH: PCORI AND THE PATIENT VOICE SONYE K. DANOFF, MD, PHD CLINICAL SCIENCE: PATIENT-CENTERED ENDPOINTS  SYMPTOMS AND FUNCTIONAL STATUS NOVEMBER 13, 2015

2. Research support from PCORI as part of PaTH CDRN (clinical data research network) Grant under review at PCORI 2

3. Building Patient Driven Research Review PCORI Mission PaTH CDRN (Clinical Data Research Network) PaTH-IPF Clinician-Patient Partnership Cohort Other PCORI funded research 3

4. The Patient-Centered Outcomes Research Institute (PCORI) An independent nonprofit, nongovernmental organization located in Washington, DC, was authorized by Congress in 2010 Improve the quality and relevance of evidence available to help patients, caregivers, clinicians, employers, insurers, and policy makers make informed health decisions Fund comparative clinical effectiveness research, or CER, as well as support work that will improve the methods used to conduct such studies 4 http://www.pcori.org/about-us

5. Engaging Patients in Research Meaningful involvement of patients, caregivers, clinicians, and other healthcare stakeholders throughout the research process—from topic selection through design and conduct of research to dissemination of results 5 http://www.pcori.org/about-us

6. PaTH: Towards a Learning Health System in the Mid-Atlantic Region University of Pittsburgh/UPMC and UPMC Health Plan (western PA, OH, WV) Penn State College of Medicine/Hershey Medical Center (central PA) Temple University School of Medicine/Temple Health (eastern PA, DE, NJ) Johns Hopkins University/Johns Hopkins Health System and Johns Hopkins Health Care (MD, VA, DC) 6 To develop and test a multi-center EHR-based clinical research platform capable of investigating common and rare disease J Am Med Inform Assoc. 2014 Jul; 21(4): 633–636.

7. PaTH: Data Infrastructure 7 J Am Med Inform Assoc. 2014 Jul; 21(4): 633–636.

8. Estimated Prevalence 8 J Am Med Inform Assoc. 2014 Jul; 21(4): 633–636.

9. 9

10. PaTH-IPF Network Develop computable phenotype to identify IPF from EHR Validate computable phenotype (Poster-S. Charles) Create PaTH Network IPF Clinician-Patient Partnership Cohort Begin study of Pre-determined Patient Related Outcomes (PROs) Expand the study to evolving areas of relevance (Poster-I. Hollin) 10

11. Building a Computable Phenotype Based on diagnostic and billing codes Modeled on strategy used in Raghu et al. –Incidence and prevalence of idiopathic pulmonary fibrosis. –Am J Respir Crit Care Med. 2006 Oct 1;174(7):810-6. Specific goals –Diagnostic accuracy –Patient not deceased or transplanted –Patient potentially available for subsequent recruitment to Clinician-Patient Partnership Cohort (at least 2 visits) 11

12. Validation of Computable Phenotype (Hopkins Cohort) Validate diagnosis of individuals identified by computable phenotype (v.1) 12 55 Patients Meeting all Inclusion Criteria35 (64%) Deceased7 (13%) Transplanted7 (13%) Incorrect diagnosis6 (11%)

13. Other PCORI Projects 13

14. Bringing Patient Voice to Research Integration of patient in developing questions, study design, review of instruments, recruitment, analysis, dissemination 14

15. To the Lisa Sandler Spaeth and Cecilia Fisher-Rudman Funds for Pulmonary Fibrosis at Johns Hopkins & To our patients who keep us focused on what really matters With Gratitude