Comparative Effectiveness Research in Lung Diseases and Sleep Disorders

Abstract

The Division of Lung Diseases of the National Heart, Lung, and Blood Institute (NHLBI) held a workshop to develop recommendations on topics, methodologies, and resources for comparative effectiveness research (CER) that will guide clinical decision making about available treatment options for lung diseases and sleep disorders. A multidisciplinary group of experts with experience in efficacy, effectiveness, implementation, and economic research identified (a) what types of studies the domain of CER in lung diseases and sleep disorders should include, (b) the criteria and process for setting priorities, and (c) current resources for and barriers to CER in lung diseases. Key recommendations were to (1) increase efforts to engage stakeholders in developing CER questions and study designs; (2) invest in further development of databases and other infrastructure, including efficient methods for data sharing; (3) make full use of a broad range of study designs; (4) increase the appropriate use of observational designs and the support of methodologic research; (5) ensure that committees that review CER grant applications include persons with appropriate perspective and expertise; and (6) further develop the workforce for CER by supporting training opportunities that focus on the methodologic and practical skills needed.

Core Domains for Clinical Research in Acute Respiratory Failure Survivors: An International Modified Delphi Consensus Study

“…Thus, we re-named the domain, which may have resulted in lower ratings among clinical researchers (vs. patients/caregivers). Consistent with prior expert groups (6, 9, 48), we believe it is important to include a measure addressing survivors’ subjective quality of life in a COS. We also encourage clinical researchers to consider assessing outcome domains that failed to meet the criteria for inclusion in a COS, but were rated as critical by the vast majority of patient and caregiver representatives, such as financial impact on patients (88%) and impact on family and/or caregivers (76%).…”

“…Each domain was rated using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) scale (30), which is a 9-point scale that is commonly divided into 3 categories for COS projects: Not Important (1 – 3), Important but Not Critical (4 – 6), and Critical (7 – 9). Additionally, panel members were provided an “Unable to Score” response and instructed to use it if they did not feel comfortable rating any specific domain.…”

Do-Not-Resuscitate Status and Observational Comparative Effectiveness Research in Patients With Septic Shock*

“…Observational CER may be particularly useful in the area of critical care medicine because of the unique challenges involved in studying the critically ill, such as the need for surrogate consent in the face of time-sensitive treatment decisions. However, an important limitation in observational CER is the potential for unmeasured confounding variables to produce spurious effect estimates (4). For example, an unmeasured variable associated with disease severity that influences the selection of a treatment of interest may bias findings of observational studies through confounding by indication.…”

Comparative effectiveness research in chronic obstructive pulmonary disease

“…The Federal Coordinating Council for Comparative Effectiveness defines CER as research that “provides information on the relative strengths and weakness of various medical interventions. Such research will give clinicians and patients valid information to make decisions that will improve the performance of the USA healthcare system” [17,18]. And specifically this cross-institute council indicated that CER is the “conduct and synthesis of research comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in ‘real world’ settings.…”

“…Moreover, CER suggests that interventions for comparison are equally broad, spanning such therapies as medications, procedures, medical and assistive devices and technologies, diagnostic testing, behavioral change and delivery system strategies. In addition, the field of CER and its champions, such as the IOM and PCORI, are engaged in expanding data sources and methods of assessment to achieve efficient, impactful studies with active dissemination of results that can and will be useful in healthcare [18,20–22]. Implicit across the definitions of CER is the need for novel methodology to include broad stakeholder input to prioritize research topics and engage in subsequent practical translation.…”