34
THE ROLE OF PATIENT VALUE AND PATIENT-CENTRED
CARE IN HEALTH SYSTEMS
at the arguments they present as part of the
drug approval process. “There is no regular,
systematic and transparent documentation on
patient preferences,” he adds.Accordingly, Mr Mühlbacher, says “a steadily
growing stream of patient preference studies”
has appeared. These attempt to provide more
robust patient input on questions such as
which unmet needs deserve priority in drug
development, the most appropriate things to
measure in clinical trials, and how to balance
potential benefits and risks of new products.The field, though, remains at a very early stage.
Mr Mühlbacher notes that currently the US
Food and Drug Administration is furthest
ahead in the use of patient preference studies
as part of its efforts on patient-focused drug
development, issuing draft guidelines oncollecting patient input,^51 while the European
Medicines Agency has engaged in pilot projects
in collecting such data.One issue is potential cost. Mr Mühlbacher
explains that various methodologies for
patient preference studies are available, with
different benefits and drawbacks, but that a
reasonable study would normally cost around
US$200,000-US$300,000, while one with the
best available techniques in several countries
would reach about half a million. Whether this
is expensive, though, is a matter of perspective.
Mr Mühlbacher believes it is a small price
relative to the high cost of clinical trials in order
to get an accurate idea of whether patients
think a product is worth approving. “If we
pay 1% of the costs of clinical trials in order
to translate the clinical effects into value to
patients, it doesn ́t seem a lot to me.”Data bubble: whose data gets to matter?
People can now get a range of monitors for condition-specific symptoms, such as blood glucose levels
to more general tools, such as blood pressure meters. A personal electrocardiogram machine is easy to
acquire and home DNA testing (for both health and ancestry) is increasingly common. Fitness trackers
are also ubiquitous. Their integration into smart watches and other mobile devices, though, means
that most of the 115m wearables sold globally in 2017 alone^52 can generate data on exercise levels, sleep
patterns and other aspects of health.Mr Graham points out that “people have access to a fantastic richness of data, way beyond the details
of what professionals would have wanted.” The potential benefits of using patient-generated health
data (PGHD) in healthcare are substantial. Some doctors who have done so report deeper insight into
patients’ medical conditions, more accurate information and improved understanding of how patients
lived between clinical visits, allowing better care plans.^53On the other hand, PGHD presents challenges. With so many equipment and software producers, for
example, data quality can be uncertain. The way in which information is presented may not adhere to
standard clinical formats. The data are also sometimes incomplete, and patients may abandon devices
or regimens. Moreover, given the wide range of data falling under PGHD and its relatively recent
appearance as a major source of information, widespread evidence of its utility remains scant. Finally,51 FDA, “Patient-Focused Drug Development:
Collecting Comprehensive and
Representative Input Guidance for Industry,
Food and Drug Administration Staff, and
Other Stakeholders DRAFT GUIDANCE,”
June 2018.
52 IDC, “Global Wearables Market Grows 7.7%
in 4Q17 and 10.3% in 2017 as Apple Seizes
the Leader Position,” press release, March
1st 2018.
53 Deborah J Cohen et al, “Integrating Patient-
Generated Health Data Into Clinical Care
Settings or Clinical Decision-Making,”
JMIR Human Factors, 2016; US Office
of the National Coordinator for Health
Information Technology, Conceptualizing
a Data Infrastructure for the Capture, Use,
and Sharing of Patient-Generated Health
Data in Care Delivery and Research through
2024 , 2018; AM Lai et al, “Present and Future
Trends in Consumer Health Informatics
and Patient-Generated Health Data,” IMIA
Yearbook of Medical Informatics, 2017.