Lecture #1
Introduction to Outcomes and
Effectiveness Research
January 5, 2015 (9:00-11:50 am)
HPM 214 http://hpm214.med.ucla.edu/
911 Broxton Avenue
Los Angeles, CA 90024
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HPM 214 Schedule
01/05/15
Introduction to Outcomes and
Effectiveness Research, and
Fung & Hays (2008); Reeve et al.
(2013)
Patient-Reported Outcomes
01/12/15
HRQOL Profile Measures
Hays & Revicki (2005 chapter); Hays
& Reeve (2010)
01/19/15
MLK Holiday
Ganz et al. (2014)
01/26/15
HRQOL Preference-Based Measures
(Quiz)
Feeny (2005, chapter); Kaplan et
al. (2011)
02/02/15
02/09/15
Designing HRQOL Measures
McColl (2005 chapter)
Hays & Fayers (2005 chapter);
Hays & Revicki (2005 chapter)
02/16/15
Presidents’ Day Holiday
Evaluating HRQOL Measures
Improving HRQOL in
Dialysis patients
http://www.kidney.org/professio
nals/CFCs.cfm
02/23/15
PROMIS/IRT/Internet Panels
Cappelleri et al. (2014); Cella et al.
(2010)
03/02/15
Responding to reviews of submitted
manuscripts;
Cognitive Interview questions
Example of reviews received,
resubmitted article and cover letter
03/09/15
Course Review (Cognitive Interview writeups due)
---------------------------------
03/16/15
Final Exam* 9-11:50am
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HPM 214 Assignments
• Class participation (25%)
• Two class assignments (25%)
– Complete the SF-36 v2 survey at http://www.sf36.org/demos/SF-36v2.html
– Conduct and summarize 5 cognitive interviews with
a self-administered HRQOL survey.
• Extra credit (2-page critique of published
HRQOL article).
Final Exam (50% of grade)
•
Which of the following could be a patient-reported measure (PRM)?
– Social support
– Satisfaction with health care
– Income
– Adherence to medical recommendations
– All of the above
U.S. Health Care Issues
• Access to care
– ~ 50 million people without health insurance
• Costs of care
– Expenditures ~ $ 2.7 Trillion
• Effectiveness (quality) of care
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How Do We Know If Care Is Effective?
• Effective care maximizes probability of
desired health outcomes
– Health outcome measures indicate whether
care is effective
Cost ↓
Effectiveness ↑
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What Are Health Outcomes?
• Traditional clinical endpoints
– Death, disease occurrence, other
adverse events
– Clinical measures/biological indicators
• Blood pressure
• Blood hemoglobin level
• Symptoms (e.g. fever)
• Health-Related Quality of Life
HRQOL is Multi-Dimensional
HRQOL
Physical
Mental
Social
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Health-Related Quality
of Life (HRQOL)
How the person FEELs (well-being)
• Emotional well-being
• Pain
• Energy
What the person can DO (functioning)
• Self-care
• Role
• Social
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HRQOL is Not
Quality of environment
Type of housing
Level of income
Social Support
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Social Support
People sometimes look to others for companionship,
assistance, or other types of support. How often were each of
the following kinds of support available to you when you
needed it during the past four weeks?
– Someone to turn to for help with a personal problem?
– Someone to help with daily chores if you were sick?
– Someone to love and make you feel wanted?
All of the Time
Most of the Time
Some of the Time
A Little of the Time
None of the Time
Patient-Reported Outcomes (PROs)
“Any report coming from patients about a health
condition and its treatment”
(U.S. FDA, 2006)
Including
• Health-related quality of life (HRQOL)
• Satisfaction with treatment
• Patient reports about care
• Needs assessment
• Adherence to treatment
Patient-Reported Outcomes (PROs)
“Any report coming from patients about a health
condition and its treatment”
(U.S. FDA, 2006)
Including
• Health-related quality of life (HRQOL)
• Satisfaction with treatment
• Patient reports about care
• Needs assessment
• Adherence to treatment
Patient-Reported Measures (PRMs)
• Background characteristics
– Age, education, income
• Health care experiences
– Reports about care (e.g., communication)
• Behavior
– Adherence to physician recommendations
• Outcomes
– Satisfaction with care
– HRQOL
Satisfaction
With Care
Patient
Characteristics
Health
Behaviors
(Adherence)
Quality
of Care
Technical
Quality
HRQOL
Patient
Reports
About Care
Uses of HRQOL Measures
•
•
•
•
Monitoring population (and subgroups)
Observational studies
Clinical trials
Clinical practice
HS 214, Winter 01·11·10
Behavioral Risk Factor
Surveillance System (BRFSS)
• Telephone interview (random digit dialing) of
nationwide survey of U.S. adults
• % reporting poor or fair health about 16%
Greater % of fair or poor health reported
by older adults (33% for 75+ vs. 9% for 18-24)
Greater % of fair or poor health reported
by females (17%) than males (15%)
Uses of HRQOL Measures
•
•
•
•
Monitoring population (and subgroups)
Observational studies
Clinical trials
Clinical practice
Observational Study
•Observation of groups
•(non-random assignment)
Outcomes
Casemix adjustment needed
• + Conditions/comorbidity
• + Severity
• + Demographics
•
•Clinical
•Complications
•Survival
•HRQOL
Observational Studies
Process
Of Care
Expert
Consensus
PatientReported
Outcomes
Clinical
PatientReported
Process of Care
• Expert Consensus
– Quality of Care “If, Then” Indicators
• % of patients with diabetes with one or more HbA1c
tests annually
• Patient reports about communication
– In the last 12 months, how often did your doctor
explain things in a way that was easy to
understand?
Outcomes of Care
• Clinical
– % of patients with diabetes with most recent HbA1c
level >9.0% ( poor control)
• Patient global rating of health
– Would you say that in general your health is:
Excellent | Very good | Good | Fair | Poor
Uses of HRQOL Measures
•
•
•
•
Monitoring population (and subgroups)
Observational studies
Clinical trials
Clinical practice
Randomized Trial Design
• Outcomes
Intervention
– Clinical
Group
– Complications
– Survival
Study
Randomize – HRQOL
Population
Control
Group
• Little control for
case-mix required
Uses of HRQOL Measures
•
•
•
•
Monitoring population (and subgroups)
Observational studies
Clinical trials
Clinical practice
HRQOL Assessment by
Providers May
• Facilitate patient-physician communication
• Improve clinician understanding of patients’ problems
(particularly those of a psychosocial nature)
Detmar SB, Aaronson NK. Quality of life assessment in daily clinical oncology practice: a feasibility study. Eur
J Cancer. 1998;34(8):1181-6.
Detmar SB, Muller MJ, Schornagel JH, Wever LD, Aaronson NK. Health-related quality-of-life assessments
and patient-physician communication: a randomized controlled trial. J Am Med Assoc. 2002;288(23):3027-34.
Hess R, Tindle H, Conroy MB, et al. A randomized controlled pilot trial of the Functional Assessment
Screening Tablet to engage patients at the point of care. JGIM. 2014; 29(12):1641-1649.
Velikova G, Brown JM, Smith AB, Selby PJ. Computer-based quality of life questionnaires may contribute to
doctor-patient interactions in oncology. Br J Cancer. 2002;86(1):51-9.
Velikova G, Booth L, Smith AB, et al. Measuring quality of life in routine oncologypractice improves
communication and patient well-being: a randomized controlled trial. JClin Oncol. 2004;22(4):714-24.
Questions?
Contact Information:
drhays@ucla.edu (310-794-2294)
Powerpoint file posted at:
http://hpm214.med.ucla.edu/
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