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Utilizing Patient Registries to Support Health Economics Research: Integrating Observational Data with Economic Analyses, Models, and Other Applications
Les Noe RPh, Leanne Larson MPh, and Jeffrey Trotter MBA, Ovation Research Group, Highland Park, IL, USA
Patient registries have become a hot topic in today’s
pharmaceutical, biotech, and medical device industries. Many
companies have initiated them. Many others are contemplating them.
But even with all the interest, there still exists much uncertainty
around how to make the most of these important and complex programs.
Much of a registry’s intrinsic value lies in its ability to
address, in one comprehensive program, a variety of organizational
objectives: collecting post-marketing safety information, gathering
clinical effectiveness data, and understanding long-term outcomes,
to name a few. Compared to phase III trials, registries typically
involve a larger and more diverse group of patients; as such, they
may better reflect real-world management practices and outcomes. In
addition to gathering the more typical clinical and safety evidence,
registries represent an opportunity to prospectively collect and
report on important pharmacoeconomic outcomes, such as
health-related quality of life (HRQoL), health care resource
utilization, and patient satisfaction. Considered as part of an
overall strategy to demonstrate value, data from registries may also
be used to support other health economics/outcomes research (HE/OR)
initiatives, such as cost-ofillness studies, economic modeling, and
costeffectiveness analyses. Indeed, HE/OR staff, in companies with
dedicated personnel, can lead the way organizationally in bringing
together commercial and clinical perspectives by coordinating the
design and execution of these important research initiatives.
Registries - What are
they?
Perhaps the most straightforward definition of a patient
registry describes it as a prospective, observational cohort study
of patients with a particular disease and/or receiving a particular
treatment/intervention. Registries are, in fact, typically
categorized as either disease or product registries. That
definition, however, doesn’t adequately communicate the true value
of a registry - the ability to integrate organizational objectives,
crossing disciplines and departments, providing a long-term
opportunity to generate important disease and product data, and
supporting a product in the marketplace throughout it’s life cycle.
Indeed, objectives for initiating a registry range along a continuum
from learning more about a product’s real-world performance to
gathering additional safety data; in all cases, though, the registry
seeks to advance science by spotlighting what really happens in
actual medical practice, in contrast to the artificial environment
of a controlled clinical trial. Designing a registry to meet BOTH
commercial and safety objectives can be challenging, but can
dramatically improve the return on the project’s investment. The
HE/OR group can be an important bridge between research and
marketing, often opening a dialogue and facilitating discussions
that can lead to a registry design with broader objectives than
either group might have conceived independently. And along the way,
important HE/OR data can be compiled, further improving a company’s
return on investment (ROI).
Disease registries provide an opportunity to assess the natural
history of a disease, its management, and its outcomes. In a disease
registry, most, if not all, treatment approaches and alternatives
are evaluated. Because the focus is not on any specific product,
these programs can be initiated well before, potentially years
before, a company plans to bring a product to market. This proactive
approach allows a sponsor to collect valuable information prior to
product launch, including data on disease epidemiology, prescribing
patterns, competitive therapies, and standard of care. Once the
product is marketed, then, companies can continue to collect
information on its utilization and outcomes relative to other
treatments. More commonly, though, registries (including disease
registries) are initiated after product launch, to allow sponsors to
collect a combination of clinical, safety, and HE/OR data. Disease
registries, in particular, represent rich sources of information for
outcomes researchers.
 Product
registries are often undertaken to gain a more in-depth
understanding of utilization and outcomes associated with a specific
product. In a product registry, only data on a specific product are
typically collected; competitive therapies are not assessed. Because
product registries are focused on a particular treatment, they
cannot be initiated until after the product is approved and
launched. Product registries are more often used when companies need
to collect post-marketing safety data or want to collect performance
data in actual practice settings. Product registries extend the
clinical and safety evidence base gained during registration trials,
and can generate real-world outcomes data used to support economic
analyses. Both approaches have merit, and can be highly credible and
well-accepted, provided the program’s objectives and methodologies
are consistent with current understanding and practice. In the
post-approval setting, undertaking a registry in a less-controlled,
naturalistic setting can provide important insight into safety,
effectiveness, potential drug interactions, cost-effectiveness,
patient risk profiles, and other factors only observable in larger
and more diverse patient populations. Registries, in fact, may be
the only practical way to gather evidence in certain patient
populations, such as the elderly, or patients with significant
comorbid conditions.
Importance of Registries
to HE/OR
In one sense, a registry could be considered the ultimate outcomes
research project, with the opportunity to prospectively collect
real-world data on effectiveness, safety, HRQoL, satisfaction,
resource utilization, costs, work loss and productivity, and other
relevant outcomes, often over a longer time frame than might
otherwise be studied. Every registry, in fact, should be evaluated
for the opportunity to collect additional data that could be
important for demonstrating product value. It may not always be
possible, as there are practical limits with any study, but the
strategic discussion is, nonetheless, vital. Table 1 describes some
of the endpoints that might be of interest from a HE/OR perspective.
Ideally, HE/OR staff will be part of the registry team, involved
in developing the program’s strategy and objectives. Indeed, given
their broad perspectives, HE/OR may be the most logical registry
“champion.” This isn’t always the case, however, as registries are
initiated from a variety of functional areas within a company, and
there may be resistance to including other groups with potentially
different agendas. Nevertheless, the registry team should be
encouraged to think broadly about the program’s analytical
opportunities, and HE/OR must clearly communicate their objectives
and needs, why the results are important, how they will be used, and
the expected benefits.
Consideration must also be given to the potential burden on
participating sites and patients. While it would be ideal to collect
more data, overloading the study with elements of marginal interest
may negatively affect physician participation and patient
enrollment, cause internal conflict, and can lead to higher overall
study costs.
If HE/OR data are collected as part of a registry, those
endpoints can be analyzed and reported along with other data. As
registries are not randomized trials, however, care must be taken in
analyzing and interpreting the results, given the limitations of
observational studies; a priori analysis plans are critical in
establishing expectations. Nevertheless, there are a variety of
insightful analyses that can be informative, as shown in Table 2.
Using Registries for
HE/OR Research
Ideally, a registry is but one aspect of an overall HE/OR strategy
designed to build evidence of value. As such, registries can be used
to gather additional information that builds on the evidence gained
through other initiatives (Figure 1). For instance, if a preliminary
cost-effectiveness model has already been developed based on phase
III trial data, then registry data can be used to update the model
to provide a more realistic picture of a product’s value. This will
be an increasingly critical activity as payors, pricing authorities,
guideline developers, and agencies involved in technology appraisal
begin reassessing a product’s cost-effectiveness within a few years
after it is introduced.
HE/OR can benefit greatly from a well-designed registry. Registries
can provide important information to help HE/OR groups address a
variety of needs, as described in Table 3.
Not only can registries benefit HE/OR projects, HE/OR projects
can, in turn, be instrumental in shaping the registry design and
objectives. Early modeling efforts often identify information gaps
that need to be filled. While some of these gaps can be filled with
trial data, a registry may be able to capture endpoints that are
not, for a variety of reasons, included in clinical trials, such as
long-term outcomes and patient utilities. Understanding what
information gaps need to be filled is the key step in articulating
HE/OR needs to the registry team, and thereby maximizing the value
of the registry. |
