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International Clinical Trials

The Patient as an Individual



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

  1. 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
  2. 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
  3. 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
  4. Cahana A, The future of pain management: Creating value through measurement, Integrative Pain Practitioner 21(4): pp34-36, Winter 2011
  5. Portenoy RK, Treatment of cancer pain, Lancet 377(9784): pp2,236-2,247, 2011
  6. 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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