1. Bringing the Patient Voice into Cancer Care and Research Ethan Basch, MD, MSc November 19, 2015

2. Symptoms Common and often debilitating in cancer Recognizing symptoms is a key role of doctors and nurses – During cancer treatment – In clinical trials

3. Symptoms Often Go Undetected Up to half of patients’ symptoms are missed during cancer treatment Nausea Fatigue Months Anorexia Vomiting Months Patient- reported Clinician- reported 3

4. Serious Implications Under-management of symptoms during cancer treatment Under-documentation of symptoms and side effects in clinical trials

5. Research on “Patient-Reported Outcomes” Develop questionnaires and software to enable patient self-reporting of symptoms, physical functioning and “quality of life” Test these approaches during routine chemotherapy care and in clinical trials nationally Work closely with NCI, FDA, pharmaceutical industry, etc.

6. UNC is a National Leader in this Area Angie Smith, MD Arlene Chung, MD Bryce Reeve, PhD Antonia Bennett, PhD Bill Wood, MD Hy Muss, MD Grant Williams, MD

7. Examples

8. Pain Common in cancer, not well controlled – Reported by 2/3 patients with advanced disease Inadequate analgesics among 1/3

9. Particularly True in Prostate Cancer More than half with advanced disease report clinically meaningful pain (>4 on 0-10 NRS)

10. 1996 Approved based on an old patient pain questionnaire

11. 1997-2014 Only 1 other oncology drug approved by FDA based on patient-reported information (Ruxolitinib, 2011) Surprising, given how common symptoms and functional impairment are in oncology

12. FDA introduced a “PRO Guidance” Outlined standards - Patient interviews - Testing - Relevance to trials - Implementation, analysis Groundbreaking… but overly stringent, and industry was largely unwilling and unable to adhere in most cases Why So Rare? U.S. Food & Drug Administration

13. Researcher Role Worked with FDA on approach to PROs in oncology – Outcome: pain intensity – Measure: 0-10 NRS (BPI) – Recall: 24 hours – Meaningful change: 30% or 2 points – Analgesic use: integrated into design – Analysis approaches

14. Work with Industry

15. Work with Consensus Groups

16. Work with National Clinical Practice Guidelines

18. A Second Example Measuring symptom side effects in clinical trials – Clinicians miss many of these – UNC researchers led development of approach for patients to report their own side effects Developed for the National Cancer Institute Called the “PRO-CTCAE”

19. Developed/Tested Questionnaire and Software

20. Clinician Reporting Patient Reporting Shows Better Symptom Detection in Trials

21. Improves Outcomes during Treatment Improved QOL, longer chemotherapy, fewer ER visits

22. Recent Publications on PRO-CTCAE

23. Support PRO Software for Many Trials UNC Cancer Center “PRO-Core"

24. Patient-Centered Approaches to Quality of Care Standards for Patient- Reported Outcome-based Performance Measures

25. PROs in Geriatrics Key to measure the patient experience – risk of comorbidities; functional impairments – E.g., the GA Older patients are enthusiastic to self-report Enables communication Methods research considerations – What outcomes, how ask questions, how often, how use the information, how report results? – Requires interdisciplinary collaboration

26. Thank You