Manufacturers are becoming more dependent on using observational studies to meet new requirements. Because observational studies
can be used to meet many different needs, they are being implemented by many different departments and functions within life
sciences organizations, including epidemiology; safety and risk management; health economics and outcomes research; evidence-based
medicine; regulatory affairs; medical affairs; clinical operations; market access; and health policy. The growing importance
and uses of these studies necessitates a cross-functional approach to design and implementation. Manufacturers are also now
approaching real-world observational studies as a key aspect of product development.
For industry, observational studies are not only crucial for meeting post-marketing commitments and receiving initial reimbursement,
but for also expanding market access, gaining a competitive advantage, and understanding the history of disease. Researchers
can use observational studies to understand the real-world benefits of different dosing, adherence to a medication, and sometimes
(with a compliant design) even outcomes from off-label use, since more than 50 percent of drugs and devices are used off-label
in many disease areas.
Like the government initiatives mentioned above, industry and issues-based groups are developing best practice principles
for conducting observational studies, including STROBE (STrengthening the Reporting of OBservational studies in Epidemiology),
one of the earliest attempts to promote guidelines for observational research, and the GRACE Principles (Good ReseArch for
Comparative Effectiveness). STROBE, although widely known, addresses reporting, but not the quality of these studies. The
GRACE Principles, sponsored with seed funding from the National Pharmaceutical Council and involving contributors from academia,
government, and industry, provide high-level guidance to decision-makers to help them evaluate the quality of observational
studies around comparative effectiveness.
Providers and Patients
Healthcare providers and patients are naturally concerned with the effectiveness and safety of medical products, and without
their participation, observational studies wouldn't work. Providers are beginning to view these studies as a way to make evidence-based
decisions on what to prescribe and to whom. Patients can use the data to make informed decisions about their own medical treatment.
Healthcare reform efforts in the US have put additional pressure not only on manufacturers but also on providers. They too
are important pieces in the puzzle of how to improve care and reduce healthcare costs. Providers are being encouraged to participate
in registries and other studies to help create an evidence-based, outcomes-focused system of care. These programs occur at
the local, regional, and national level across a wide range of disease states, and are funded at different levels ranging
from the individual hospital or practice to national medical associations.
The next wave of change may come from organized patients. Increasingly, disease-focused patient associations have organized
their own programs to better understand the natural history of disease and the impact of new therapies. Organizations such
as the Cystic Fibrosis Foundation have pioneered the role of the patient organization in the research enterprise, and many
other organizations are taking notice and developing their own infrastructures for registries and other studies.
Over time, multiple stakeholders will converge and collaborate around these programs to both reach critical mass and reduce
duplication of effort. Collaboratively building or leveraging existing networks of information will be an important direction
for all stakeholders as the need for, and benefits of, real-world studies increases. For example, manufacturers are already
leveraging certain registries managed for rare-disease-focused patient organizations or learning to partner with medical associations
to meet common goals. And, different agencies of the US government are working with organizations such as the American Academy
of Ophthalmology and Outcome Sciences to leverage new and existing registries from purposes such as safety assessment of intraocular
lens implants to comparative effectiveness of glaucoma treatments.
Richard Gliklich, MD, is President and CEO of Outcome. He can be reached at firstname.lastname@example.org