Improving Patient-Centered Care in Diabetes With Comparative Effectiveness Research
Published Online: September 30, 2013
Douglas K. Owens, MD, Wade Aubry, MD, Roy Beck, MD, Joshua Benner, MD, Jan E. Berger, PharmD, Michael E. Chernew, PhD, Felicia Forma, BS, Dana P. Goldman, PhD, William H. Herman, MD, Rebecca Killion, MA, Darius Lakdawalla, PhD, and Anne L. Peters, MD
The complexity of glycemic management in type 2 diabetes mellitus (T2DM) has increased dramatically in the past 15 years. In 1995, the drugs available for treatment of T2DM were insulin and sulfonylureas. Since then, 9 new drug classes have become available, significantly increasing the number of clinical options for physicians and patients. The expanded treatment options currently available, in turn, have produced more opportunities for individualized, patient-centered treatment approaches, while creating additional challenges. For example, among T2DM patients, there is substantial heterogeneity in clinical outcomes and patient preferences regarding which health outcomes and treatment effectsmatter to them most. For the physician who seeks an approach that maximizes an individual patient’s likelihood of responding favorably to treatment while optimizing other considerations (eg, quality of life, functional ability, healthcare spending), the challenge is made greater by an insufficient evidence base to inform clinical decision making.
At a minimum, such an evidence base would include data on the comparative effectiveness of various treatment options—both overall and for specific subgroups of patients—as well as data on patient preferences that drive treatment decisions and, often, health outcomes. Comparative effectiveness research (CER) plays an important role in generating evidence for patients, physicians, and payers; it is increasingly conspicuous in discussions about optimizing patient-centered care for T2DM. CER compares the benefits and harms of alternative treatment options to determine “what works best for which patients under what circumstances.”1 By also assessing utilization and costs, CER can provide a foundation for cost-effectiveness analysis,2 an important approach for identifying high-value health care.3
The hallmark of CER is the comparison of clinically relevant alternative diagnostic or management strategies in representative clinical practice populations. With its multiple treatment alternatives and heterogeneity of patient outcomes, T2DM management is well-suited to this type of research. Accordingly, CER is increasingly used within the diabetes arena. For example, a form of CER was used to evaluate available drug therapies for T2DM.4 However, without an economic evaluation or measurement of comparative clinical effectiveness in real-world settings, the findings are limited. In another example, a major pharmaceutical company developed its phase III clinical trials program using CER. Drawing on insights of an expert panel, the company developed a clinical research approach to provide clinical and economic data once the trials were completed, with a particular focus on enhancing liraglutide’s entry into the market and integration into formularies.5
Finally, a recent comprehensive review of randomized control trials (RCTs) and observational studies by the Agency for Healthcare Research and Quality (AHRQ) identified several gaps in the
evidence on the effectiveness of oral agents for T2DM. These gaps will limit clinicians in providing patient-centered care.6,7
Comparative Effectiveness Research Working Group
To better understand how CER may be used to help improve patient-centered T2DM care, we convened a multidisciplinary working group that included patient representatives as well as a range
of experts in: diabetes care, technology assessment, pharmacology, health economics, evidence synthesis, systematic reviews, clinical decision making, guideline development, epidemiology,
clinical trials, and public policy. The group considered the following questions:
1. What are the limitations in the available evidence for patient-centered T2DM care in diabetes?
2. What outcomes are important to patients and, therefore, should be included in studies of diabetes management?
3. How should RCTs be modified to improve the evidence base for patientcentered care?
4. How should observational studies be designed to improve the evidence base for patient-centered care?
The working group made recommendations, by consensus, for how CER could be used to improve the evidence base for patient-centered diabetes care in order to make results of future diabetes management studies more useful. The final recommendations are summarized here.
Limitations of the Evidence Base for Patient-Centered Diabetes Care
The working group highlighted 5 gaps in the evidence base for T2DM patient-centered care: (1) limited evidence on long-term and patient-reported outcomes; (2) the nonrepresentativeness of patient populations and clinical settings— particularly in clinical trials; (3) the dearth of systematic data on patient subgroups; (4) the insufficient attention paid to social, cultural, and economic factors that influence care; and (5) the comparatively few direct comparisons among alternative treatment strategies. We discuss each in turn.
Limited Evidence Regarding Long- Term and Patient-Reported Outcomes
The comparatively little evidence on long-term outcomes is striking: many outcomes important to clinicians and patients are not tracked or reported. The AHRQ review, for example, found insufficient evidence to conclude that alternative T2DM treatments result in improvements in total mortality, cardiovascular mortality or morbidity.6,7 No study in the AHRQ analysis addressed retinopathy. Only 3 studies evaluated neuropathy, and these had significant methodological flaws.6,7 The working group also noted a lack of postmarketing surveillance, which limits the likelihood of identifying adverse events. Another limitation of the available evidence identified by the working group is the comparatively little attention paid to results that patients find most important, which we call “patientcentered outcomes.” The working group highlighted the importance of outcomes such as satisfaction with care, functional ability, and quality of life. Other outcomes that may be significant to patients include therapeutic side effects (such as weight gain and hypoglycemia), convenience, and cost. Patient- centered outcomes are important because they can influence adherence to care, among other things. Adherence is particularly challenging when patients may not fully believe in the value of the prescribed medications or if they find the regimens difficult to follow. To prevent long-term complications, diabetes care also often includes treating patients who are asymptomatic.
Nonrepresentativeness of Patient Populations and Clinical Settings
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