Bringing the Patient Voice into Cancer Care and Research

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 Anorexia Fatigue • Up to half of patients’ symptoms are missed during cancer treatment Nausea Vomiting Patient- reported Clinician- reported 3 Months Months

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. Why So Rare? 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 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. Shows Better Symptom Detection in Trials Clinician Reporting Patient Reporting

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