Transcript Presentation - UNC Lineberger Comprehensive Cancer Center

Bringing the Patient Voice into
Cancer Care and Research
Ethan Basch, MD, MSc
November 19, 2015
Symptoms
• Common and often debilitating in cancer
• Recognizing symptoms is a key role of doctors
and nurses
– During cancer treatment
– In clinical trials
Symptoms Often Go Undetected
• Up to half of
patients’ symptoms
are missed during
cancer treatment
Patientreported
Fatigue
Anorexia
Nausea
Vomiting
Clinicianreported
Months
3Months
Serious Implications
• Under-management of symptoms during
cancer treatment
• Under-documentation of symptoms and side
effects in clinical trials
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.
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
Examples
Pain
• Common in cancer, not well controlled
– Reported by 2/3 patients with advanced disease
• Inadequate analgesics among 1/3
Particularly True in Prostate Cancer
More than half with
advanced disease report
clinically meaningful
pain (>4 on 0-10 NRS)
1996
• Approved based on an old patient pain questionnaire
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
Why So Rare?
FDA introduced a “PRO
Guidance”
U.S. Food &
Drug
Administration
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
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
Work with Industry
Work with Consensus Groups
Work with National Clinical Practice Guidelines
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”
Developed/Tested Questionnaire and Software
Shows Better Symptom Detection in Trials
Clinician Reporting
Patient Reporting
Improves Outcomes during Treatment
• Improved QOL, longer chemotherapy, fewer ER visits
Recent Publications on PRO-CTCAE
Support PRO Software for Many Trials
• UNC Cancer Center “PRO-Core"
Patient-Centered Approaches to Quality of Care
Standards for PatientReported Outcome-based
Performance Measures
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
Thank You