FEATURE
II,4 with primary endpoints of reduction in
spleen volume. COMFORT-I also included a
secondary endpoint based on a PRO instrument that researchers developed in conjunction
with the FDA.
“The drug would not have been approved if
it did not make people feel better; the reduction in spleen volume on its own was not a
meaningful outcome,” Dr. Basch said.
According to Dr. Abernethy, it was key that
the FDA understood how the PRO measurement was developed and could trust in the implications of a measurable change on the scale.
“In addition, the measurement tool made sense
in terms of symptom profile in that it went
along with what patients expected to be feeling
and experiencing in terms of developing spleen
problems and other issues associated with that
disease,” Dr. Abernethy added.
Payers are also taking a greater interest in
PROs.
For example, in the cardiology realm, when
discussing treatments for heart failure, the
metric of hospital readmission is often used
to measure a successful treatment. However,
patient symptoms such as shortness of breath,
chest pain, sleep quality, and physical functioning are also important elements of heart failure
treatment.
“People who have symptoms and functional
status problems are more likely to use a lot
more services,” Dr. Basch said.
In addition, medical associations are starting to use PROs in clinical practice guidelines
as a way to prioritize one product compared
with another in terms of clinical benefit and
potential harms or side effects.
“Some of the very large payer organizations
are starting to prioritize treatment based on
these guidelines and the quality-of-life information,” Dr. Basch added.
Incorporating How Patients Feel
into Research
As PROs gain attention, many in the research
world have begun to develop PRO measurement tools or incorporate them into therapeutic trials.
“There has been development and use of
PRO tools for many decades, but more recently
there has been consolidation in this space as
urged by the National Institutes of Health and
its PROMIS program,” Dr. Abernethy said.
PROMIS is the Patient Reported Outcome
Measurement Information System and it funded investigators to develop a “psychometrically
validated, dynamic system to measure PROs
efficiently in study participants with a wide
range of chronic diseases and demographic
characteristics.”
Currently, PROMIS tools measure physical health, mental health, and social health
for adults and children. However, there is a
large and wide portfolio of other instruments
available to researchers interested in measuring
PROs, Dr. Abernethy said.
ASHClinicalNews.org
In its guidance document, the FDA outlined some of the attributes that define a quality PRO measurement tool including content
reliability, ability to detect change, and validity.
Reliability is a key factor. When administered to different people it is important that the
tool can reliably get the same type of estimate
for people with similar problems, Dr. Abernethy said. It is also important that the questions are understandable, meaning that people
from different backgrounds can understand the
questions and provide similar answers.
“For doctors,
patient-reported
data are the
beginning of
every story; it
is the history
of the patient’s
present illness.”
—AMY P. ABERNETHY, MD, PHD
Responsiveness to change must also be
evident in a quality PRO measurement tool. For
example, if the use of a certain drug makes a
difference to a patient’s life, it is important that
the employed tool shows an evident change.
Validity – that an instrument measures what it is
supposed to be measuring, when compared to a
gold standard – is at the crux of any useful tool.
“My first advice to anybody looking to
develop a PRO measurement is to try to use
something that is already out there and has already been validated, provided it fits your goal,”
Dr. Abernethy said.
How to Use PROs in Clinic
According to Dr. Abernethy, physicians can use
routinely collected PROs as a way of monitoring treatments and signaling what interventions a patient may need. Symptom-focused
PROs are particularly helpful.
“For example, when you see a patient in the
clinic, you can systematically measure pain,
which is a PRO,” she said. “If a patient comes
in with hip pain and you ask them to rank the
pain on a scale from zero to 10 and then apply
a steroid injection, on their next visit you can
revisit that pain and any shift in score could
indicate that the intervention is working, not
working well enough, or not working at all.”
Second, PROs can be used as a way to triage
patients, when applicable. A patient presenting
with chest pain and shortness of breath may
need to go to the emergency department, or
someone in psychological distress may need
the aid of a social worker or a psychiatrist.
Depending on how, and if, each practice
collects and stores PRO data, it can be used
for a series of secondary purposes as well, Dr.
Abernethy said.
The question of how physicians can use
PROs in their everyday clinical practice is one
that Dr. Abel thinks is deserving of greater
research.
“I worry that in our busy hematology
practices we do not have a lot of time to tease
out what a patient’s quality of life truly is,” Dr.
Abel said.
For example, for patients with myelodysplastic syndromes (MDS), fatigue is a big issue.
“Some patients with MDS think that being
tired is just part of getting old, and miss out
on treatments that could help because their
providers don’t always probe the extent of
their fatigue,” he said. “Measuring all patients’
quality of life in a rigorous way would take out
some of the guesswork.”
Dr. Abel said there is a great need to
continue to develop methods to measure and
administer PROs, especially in certain specific
disease states. One method that may be helpful
is the use of electronic medical records with
patient portals, which can allow physicians
to email patients prior to an appointment to
assess PROs and compare that information to
PROs at prior visits or to other patients with
the same disease.
Although PRO research is still a relatively
young field, a recent systematic review published
by Kotronoulas et al. in The Journal of Clinical
Oncology showed that the use of PRO measures
in cancer care w