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International Clinical Trials
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Healthcare is increasingly subject to standards of value, but
appropriate data are needed in order to evaluate treatment outcomes and
clinician performance, and to support reimbursement decisions
The global healthcare system is under rising scrutiny as both the need
for healthcare, and the cost for its delivery, continue to increase. In
the US, agencies dedicated to providing healthcare (Centers for Medicare
and Medicaid Services (CMS)) and improving medical quality, safety,
efficiency and effectiveness (Agency for Healthcare Research and
Quality’s (AHRQ)) are mandating an evidence-based approach to medical
treatment (evidence-based medicine), including comparative effectiveness
research (CER), in order to improve the quality of care and to support
better-informed reimbursement decisions about available treatments.
By definition, evidence-based medicine requires data. But how does one
measure the effectiveness of treatment? For certain medical conditions, a
quantitative assessment can reliably be used to determine patient
status. Examples include: measuring blood pressure for hypertension,
cholesterol levels for hypercholesterolaemia, or glycosylated
haemoglobin (haemoglobin A1c) for diabetes. For other conditions, such
as chronic pain, assessing the effectiveness of treatment is more
complex and relies upon information provided by patients to describe how
pain affects various key domains of their lives, including: pain
interference with essential activities, status of physical functioning,
emotional well-being, satisfaction, and potential risk for prescription
opioid abuse and misuse. When collected consistently and accurately,
these patient-reported outcome (PRO) data can provide improved and more
comprehensive characterisation of the disease state and enable informed,
individualised treatment decisions.
More than a decade ago in the US, the Institute of Medicine (IOM) of the
National Research Council mandated a patientcentred approach to
healthcare that would use clinically meaningful outcome measures to
monitor treatment effectiveness and caregiver performance (1). Progress
toward this goal is occurring at pace. The Patient-Centered Outcomes
Research Institute (PCORI), established by the US Congress through the
2010 Patient Protection and Affordable Care Act, recently issued its
first draft National Priorities for Research and initial Research Agenda
for public comment (2). The report prioritises five areas that are
intended as a broad base for research initiatives; these items emphasise
the importance of a patient-centred, comparative effectiveness
methodology.
A developing view in clinical research is that a truly patientcentred
approach entails the assessment of individual patient outcomes
throughout the course of treatment; this is the basis of
measurement-based care (MBC). With MBC, information on safety and
effectiveness is collected periodically for each patient and is used to
assess – and, where appropriate, modify – that patient’s treatment. MBC
represents a step up from standard clinical practice, where clinicians
typically rely upon global judgments rather than specific, more accurate
and comprehensive symptom assessments. MBC can help improve
decision-making by clinicians, resulting in a more consistent and
effective delivery of care.
A recent research initiative successfully employed MBC to evaluate and
compare treatment effectiveness in real-world patients with major
depressive disorder: the Sequenced Treatment Alternatives to Relieve
Depression (STAR*D) study (3). This large study, involving over 4,000
patients, validated the usefulness of the MBC approach overall, in
addition to assessing the effectiveness of specific pharmacological
agents.
These results have helped to stimulate wider adoption of MBC methods in
other clinical studies, with the ultimate goal of incorporating them
into clinical practice. MBC is especially valuable in complex
conditions, such as chronic pain, where PROs are needed to fully
evaluate the effects of treatment, both positive and negative.
Management of Chronic Pain
The management of chronic pain has proven especially problematic within
the setting of the US healthcare system: although numerous pharmacologic
therapies have been introduced over the past few decades, patterns of
care are inconsistent, patient functioning often remains impaired, abuse
is commonplace and treatment costs continue to rise (4). Current
recommendations for improvement include the identification and use of
appropriate PROs to evaluate treatment response, and the use of
evidence-based interventions within an individualised plan of care
(5,6).
Clearly, better management of chronic pain requires a multifaceted
approach, including initiatives such as establishing treatment
guidelines, education of primary care providers and collaboration with
pain specialists, actively engaging patients in the management of their
disease through individual education and routine collection of PROs, and
ultimately, evaluation of treatment success and costs as part of
healthcare delivery. MBC is an essential tool in this improvement
effort.
Case Study: CPAIN™
The International Network for Outcome Research is working with
Registrat-MAPI on the Chronic Pain Impact Network (CPAIN™), a programme
that facilitates MBC by collecting data on pain treatments and
associated PROs, and by providing detailed clinical assessments to
physicians. Patients with any diagnosis of chronic pain may participate:
low back pain, arthritis, neuropathic pain, fibromyalgia, complex
regional pain syndrome, headache, cervical neck pain, cancer pain,
muscle pain, vascular disease or injury, temporomandibular joint
disorder, and so on.
Patients in the CPAIN programme undergo routine assessments of symptoms
and side effects, and data are collected and presented in
easily-understood patient profiles that document change in status over
time. In this patientcentred system, patients have the opportunity to
document their pain experiences, set personal functional goals, and
observe their own progress over time. Treating clinicians also enter
information for each patient and use the patient profile to manage each
individual course of treatment. In addition to the individual patient
profiles, clinicians can have access to de-identified, collective data
for the programme, and thus can compare their own observations to the
aggregate results.
The CPAIN programme has two parts: a ‘patient profiler’ and an
observational research study. In particular, patients and clinicians can
use the patient profiler component of CPAIN without participating in
the research study.
Patient Profiler
The CPAIN patient profiler contributes to routine clinical care by
providing pain clinicians with longitudinal patient profile reports
containing comprehensive assessments that guide pain management and
monitor treatment effectiveness over time. The patient profiler allows
clinicians to measure and assess pain in a standardised fashion; it is
important to note that measuring pain includes not only the assessment
of pain intensity, but also measuring physical and emotional function,
and overall quality of life.
Patients enter their information into the patient profiler at each
clinic visit. After a patient completes an assessment, a patient profile
report is generated, summarising the findings; this report becomes part
of the patient’s medical record. A patient profile report (measuring
pain in multiple domains) is intended to be analogous to an MRI report
(measuring anatomical structures), serving as a regularly performed
assessment of the disease state.
At baseline and at each follow-up visit, after patients complete their
assessments in the CPAIN patient profiler, they are asked whether they
would like to participate in the research study. Only data from patients
who provide their informed consent are included in the CPAIN research
study.
Research Study
The CPAIN research study is designed as a very large, prospective,
observational, non-interventional study capturing information on routine
clinical care. The study provides a data collection network that can be
utilised for large-scale assessments of pain management, such as
comparative effectiveness research (CER) analyses, enhanced
postmarketing safety surveillance, assessments of risk evaluation and
mitigation strategies (REMS), sub-studies, and hypothesis generation
that may lead to additional clinical studies.
The recruitment goal is to include data on hundreds of thousands of
patients at hundreds of sites. Patients are the primary subjects in this
study and healthcare providers at the clinical sites are the secondary
subjects, providing background information on their clinical training
and experience.
The CPAIN programme allows clinicians to monitor appropriate PROs (for
example, pain intensity, physical function, emotional function and
quality of life), as well as assess outcomes of pharmacological and
non-pharmacological treatments, for individual patients. It is also a
useful tool to help address the increasing societal problems of misuse,
abuse, and diversion of prescription analgesics, specifi cally with
opioids. CPAIN also tracks disease costs through records of medical
events and resource utilisation, as well as prescription data. By
applying MBC to pain management, the CPAIN programme will help to
improve understanding of a complex disorder, reduce unnecessary practice
variability, lower risks of abuse and overdose, improve patient
outcomes, and promote improved satisfaction of both providers and
patients.
CPAIN has been launched nationally at 12 academic and community-based
sites, including the University of Washington’s Division of Pain
Medicine, which has implemented an MBC model for management of chronic
pain using the CPAIN programme (4). As of Q2 2012, 36 participating
physicians are actively enrolling patients, with over 10,000 individual
patients enrolled.
Measurement-Based Care: A New Standard
Delivery of healthcare in the US is increasingly subject to standards of
value, and clinical research incorporating MBC is needed to accurately
evaluate treatment outcomes and clinician performance for a wide range
of diseases. Properly designed and conducted studies that incorporate
MBC can be useful to address governmental and industry mandates through a
variety of investigational objectives, for example:
- Assessing comparative effectiveness of available treatments
- Matching patient subgroups to the most effective treatment alternatives
- Developing new, evidence-based treatment guidelines/ treatment algorithms
- Identifying better predictors for disease/ treatment outcomes
- Developing optimal systems for evaluating and rewarding clinician performance
The integration of MBC into healthcare research and delivery may provide
additional benefits. Because of the real-world, and often very large,
patient base, the data collected through late-phase MBC research may
yield new and comprehensive summaries of disease characteristics,
subgroup issues (including, but not limited to: age, sex, racial
differences, undertreated patient populations, and so on), and other
information for dissemination to disease-centered societies and patient
advocacy groups, as well as the medical community, resulting in
improved, patient-centered treatment standards and better-informed
patients
Furthermore, when MBC is included in routine clinical practice,
individualised treatment for each patient becomes the norm – an asset
when the practice of ‘mainstream’ medicine is viewed by many as
increasingly specialised, automated and depersonalised. The perception
that healthcare providers are aware of, and responsive to, the
individual patient’s condition, and the sense that the patient maintains
an active role in the treatment process, may promote patient compliance
and retention, especially in the management of problematic, chronic
diseases.
Treating each patient as an individual can optimise the quality of care
while reducing costs. As a key component of evidencebased medicine, MBC
will play an increasing role in clinical research, and ultimately in
established medical practice.
References
- National Research Council, Crossing the Quality Chasm: A New
Health System for the 21st Century, 2001 available at
www.pcori.org/assets/ PCORI-D
raft-National-Priorities-and-Research-Agenda.pdf
- Selby JV, Beal AC and Frank L, The Patient-Centred Outcomes
Research Institue (PCORI) national priorities for research and initial
research agenda, JAMA; 307 (15): pp1,583-1,584, 18 April 2012
- Huynh NN and McIntyre RS, What are the implications of the STAR*D
Trial for primary care? A review and synthesis, Prim Care Companion J
Clin Psychiatry 10(2): pp91-96, 2008
- Cahana A, The future of pain management: Creating value through
measurement, Integrative Pain Practitioner 21(4): pp34-36, Winter 2011
- Portenoy RK, Treatment of cancer pain, Lancet 377(9784): pp2,236-2,247, 2011
- Turk DC, Wilson HD and Cahana A, Treatment of chronic non-cancer pain, Lancet 377(9784): pp2,226-2,235
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Stephen Webb has 25 years of
experience in pharmaceuticals, academic and clinical research, and
strategic design and development of numerous peri- and post- approval
studies. Stephen is responsible for North America business and
operations, global affiliates, strategic consulting, product
commercialisation, and client and business development activities for
the company. Stephen has held previous positions at Glaxo
Pharmaceuticals, Research Triangle Park, NC working in gastro-infectious
disease and CNS products and ClinTrials Research. He is a graduate of
the University of Kentucky where he majored in Business Administration
and Biology, completed his premedical curriculum, and conducted basic
research at the Center for Biomedical Engineering and Department of
Anatomy and Neurobiology. He is a Fellow of the Howard Hughes Medical
Institute.
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