Patient-Reported Outcomes (PROs)
in Ovarian Cancer Clinical Trials
Sheryl M. Pease and Alexandra I. Barsdorf
1
Pfizer, Inc. New York, NY USA
1
1
OBJECTIVE: To assess use of Patient-Reported Outcomes (PROs) in studies of Ovarian Cancer treatment.
METHODS
RESULTS
• A search of Citeline’s Trialtrove was conducted using the search
criteria of “Ovarian Cancer” as the disease, drugs tested was
“any,” and trial phase was II/III, III, and IV.
• Of the 189 studies reviewed, 102 (54%) included PROs.
• This resulted in 252 trials. Studies were excluded if the disease
type was other than ovarian cancer.
• A total of 15 different PROs were identified.
• Study designs were clinical trial and observational studies, and
both double-blind and open-label studies were included.
• Target subject accrual for all studies was ≥53.
• Study start dates ranged from 1990 to 2014.
• The remaining 189 trials were then assessed for prevalence and
type of PROs.
• For these 102 trials, a total of 148 PROs were included, and an average
of 1.5 PROs were used per study with a range of 1 to 4 instruments.
• One hundred twenty-six (85%) of these PROs measured HealthRelated Quality of Life (HRQL), 10 (7%) measured QoL/Utilities, 9
(6%) measured Symptoms, 2 (1%) measured Activities of Daily Living
and 1 (1%) was categorized as other.
• Within the HRQL measures, 35 (28%) were ovarian cancer specific
measures, 27 (21%) were cancer general measures, 6 (5%) were
cancer treatment specific, 2 (2%) were generic measures and 1 (1%)
was chemotherapy treatment specific. The remaining 55 (43%) HRQL
endpoints were unspecified.
Citeline’s Trialtrove Search
Ovarian
Cancer+Phase
II/III, III &
IV+drugs
tested-any
Removed studies
with any disease
type listed other
than Ovarian Cancer
Results
252 trials
Total number
of trials
Trials that include
PRO (%)
189
54%
Out of the
189 trials,
102 studies
included PROs
Results
189 trials
Reviewed
102 trials for
PRO specific
information
HRQL (n=126)
Ovarian Cancer
Specific
35
Cancer General
Cancer Treatment
Specific
27
6
Generic
Chemotherapy
Specific
Unknown
2
1
55
HRQL Broken Down by Type
PRO Classification
1%
55
1%
6%
7%
35
HRQL (e.g., SF-36)
QOL/Utilities (e.g., EQ-5D)
27
Symptoms (e.g., FOSI)
Activities of Daily Living
Other (e.g., FACT-BRM)
6
85%
2
1
Cancer General
Cancer Treatment
Specific
Chemo Treatment
Specific
Ovarian Cancer
Specific
Generic
Unknown
Ovarian Cancer PROs Frequently Used
and Common PRO Combinations
Ovarian Cancer Specific HRQL and Symptom PROs
FOSI
14%
FACT-O
2%
EORTC QLQ-C30
FACT-O + EQ-5D
43%
EORTC QLQ-OV28 (HRQL)
FACT-O (HRQL)
EORTC QLQ-C30 + EORTC QLQ-OV28
MDASI-OC (Symptom)
0
10
15
20
25
30
Total number of times PRO was used out of 102 studies
FOSI (Symptom)
41%
5
•
•
•
•
•
•
EORTC QLQ-C30 was used 9 times, 9% of the time (9/102 studies)
FACT-O was used 11 times, 11% of the time (11/102 studies)
When EORTC QLQ-C30 was used, EORTC QLQ-OV28 was used with it 18 times
When FACT-O was used, EQ-5D was used with it 4 times
When FOSI was used, EQ-5D was also used 2 times
EORTC QLQ-OV28 was never used alone, always in combination with EORTC QLQ-C30
CONCLUSION: PROs are included in ovarian cancer clinical studies approximately half the time, which is low compared to other disease areas.1
The majority of measures assess HRQL and are specific to ovarian cancer. Progression free survival (PFS) tends to be the common primary
endpoint because it can be easily measured and it is clinician reported. Including HRQL in ovarian cancer studies can support the value of
progression free survival to patients. The goal of many patients with advanced cancer is improved quality of life.
When designing clinical trials, one question researchers should ask is how PROs can help clinicians and patients better evaluate the efficacy
and safety results? Early planning, design, implementation and analysis are critical to obtaining meaningful clinical trial data. Where practical,
engaging experts, clinicians, and developers in the planning and design can also help ensure PRO data supports the objectives of the study. Finally,
researchers should ask themselves if they have considered what is important to the patients. The benefit of PFS is not always apparent to the
patient, but if it is accompanied by improved HRQL, the benefit of the treatment becomes much more meaningful. PROs are an important tool to
show the value of new cancer treatments.
References: Gnanasakthy A, Mordin M, Clark M, DeMuro C, Fehnel S, Copley-Merriman C. A review of patient-reported outcome labels in the United States: 2006 to 2010. Value Health. 2012;15(3): 437-442.