One size does not fit all: Insights for engaging front-line clinicians in pragmatic clinical trials.

Introduction: Despite the proliferation of pragmatic clinical trials (PCTs) conducted in health care delivery settings, we know relatively little about how practicing clinicians perceive their potential roles in such research. Empirical evidence and practical guidance concerning clinician engagement in research is needed to inform the design and successful implementation of PCTs.

Methods: We conducted a two-phase qualitative study to better understand how and to what extent practicing clinicians should be involved in PCTs and to develop guidance for researchers on engaging front-line clinicians in PCTs.

A multistakeholder Delphi consensus core outcome set for clinical trials in moderate-to-severe asthma (coreASTHMA).

Background: Treatments for long-term control of asthma have improved and include a promising but expensive class of biologic therapies. However, the clinical trials evaluating these and other novel treatments have used a variety of different outcomes to evaluate efficacy. The evolution of asthma care calls for a re-examination of outcomes that are most important to patients and other stakeholders.

Proposed framework for patient-centered outcomes-based measures in alternative payment models.

Objectives: Alternative payment models (APMs) are part of a growing shift from volume-based, traditional fee-for-service payment models toward payment for value. To date, however, patients have been largely omitted from efforts to design new payment models. We sought to identify key characteristics of outcomes-based quality measures to inform future APMs that are more patient-centered.

The asthma evidence base: a call for core outcomes in interventional trials.

Objectives: Biologic therapies are emerging as an option to treat a subset of patients with severe asthma, however no direct comparison between these agents has been conducted. Furthermore, heterogeneity of outcomes in clinical trials makes it difficult to compare these agents and traditional therapies. The extent to which this heterogeneity exists has major implications for evidence-based decisions and is yet to be fully reported.

The increasing complexity of the core outcomes landscape.

In this project, we set out to identify ways to increase the uptake of core outcome sets in clinical research. In doing so, we uncovered a growing recognition, across many different health care sectors, of the need for common, relevant outcomes to improve the quality of decision-making. This has led to a plethora of projects, initiatives, and new organizations all intended to develop standardized outcomes and outcome measures for their particular fields.

Stakeholder perspectives regarding alternate approaches to informed consent for comparative effectiveness research.

Introduction: Traditional informed consent approaches, involving separate discussions and lengthy consent forms, may be an imperfect fit for comparative effectiveness research (CER) that is integrated into usual care and compares non-investigational treatments. However, systematic efforts to collect broad stakeholder perspectives about alternative streamlined approaches to disclosure and consent in this context have been limited.

Methods: We used a deliberative engagement method to solicit the views of a multi-stakeholder group regarding 3 alternative models of disclosure, consent, and authorization in CER studies: Opt-In, Opt-Out, and "General Approval".

Framing the conversation: use of PRECIS-2 ratings to advance understanding of pragmatic trial design domains.

Background: There continues to be debate about what constitutes a pragmatic trial and how it is distinguished from more traditional explanatory trials. The NIH Pragmatic Trials Collaborative Project, which includes five trials and a coordinating unit, has adopted the Pragmatic-Explanatory Continuum Indicator Summary (PRECIS-2) instrument. The purpose of the study was to collect PRECIS-2 ratings at two points in time to assess whether the tool was sensitive to change in trial design, and to explore with investigators the rationale for rating shifts.

Tobacco Smoking and Tuberculosis among Men Living with HIV in Johannesburg, South Africa: A Case-Control Study.

Setting: Although there is ample evidence that smoking increases the risk of tuberculosis (TB), the magnitude of impact on TB risk among HIV-infected persons is poorly described. Given that a high proportion of patients with TB are co-infected with HIV in South Africa, the risks arising from the intersection of smoking, TB, and HIV/AIDS have key relevance for tobacco control policies.

Objective: To evaluate the association of pulmonary tuberculosis (PTB) with current tobacco smoking among men with HIV in South Africa.

Understanding practice-based research participation: The differing motivations of engaged vs. non-engaged clinicians in pragmatic clinical trials.

Background/aims: Pragmatic clinical trials (PCTs) represent an increasingly used strategy for "real-world" trials. Successful PCTs typically require participation of community-based practices. However, community clinicians often have limited interest or experience in clinical research.

PAYER PERSPECTIVES ON FUTURE ACCEPTABILITY OF COMPARATIVE EFFECTIVENESS AND RELATIVE EFFECTIVENESS RESEARCH.

Objectives: Our objective was to gather perspectives from payers on how comparative effectiveness research (CER) in the United States and relative effectiveness (RE) research in Europe will impact evidentiary standards for access decisions of new drugs by 2020.

Methods: We conducted semi-structured interviews with fourteen senior officials representing public and private payers, health technology assessment groups, and pricing and reimbursement bodies in the United States and Europe. An online survey assessed current use of CER/RE evidence and potential trends that might influence its use for decision making by 2020.

Incubation periods of mosquito-borne viral infections: a systematic review.

Mosquito-borne viruses are a major public health threat, but their incubation periods are typically uncited, non-specific, and not based on data. We systematically review the published literature on six mosquito-borne viruses selected for their public health importance: chikungunya, dengue, Japanese encephalitis, Rift Valley fever, West Nile, and yellow fever viruses. For each, we identify the literature's consensus on the incubation period, evaluate the evidence for this consensus, and provide detailed estimates of the incubation period and distribution based on published experimental and observational data.

Improving the efficiency and effectiveness of pragmatic clinical trials in older adults in the United States.

Pragmatic clinical trials (PCTs) seek to improve the generalizability and increase the statistical power of traditional explanatory trials. They are a major tenet of comparative effectiveness research. While a powerful study design, PCTs have been limited by high cost, modest efficiency, and limited ability to fill relevant evidence gaps.

Incorporating stakeholder perspectives in developing a translation table framework for comparative effectiveness research.

This project used a stakeholder-driven process to understand the factors that drive the selection of study designs for comparative effectiveness research (CER). The project assembled a diverse stakeholder committee to explore the basis of a translation framework and gathered input through surveys, interviews and an in-person meeting. Stakeholders recommended different study designs for the CER topic areas and identified different outcomes as the most important outcomes to study in each area.

Academic medical centers and underserved communities: modern complexities of an enduring relationship.

Background: Most private academic medical centers are located in underserved areas, yet fiscal pressures have led many to struggle with balancing their commitment to surrounding communities with other missions.

Objective: To explore stakeholders' views regarding the ethical, legal, and financial obligations of private academic medical centers to their surrounding neighborhoods.

Design, Participants, And Measures: QualitatiVe analysis of key informant interviews during 2008 with medical students, faculty and community physicians, administrators, and community health leaders at a large urban academic medical center.

Physicians' views on the importance of patient preferences in surrogate decision-making.

Objectives: To explore the degree to which physicians report reliance on patient preferences when making medical decisions for hospitalized patients lacking decisional capacity.

Design: Cross-sectional survey.

Setting: One academic and two community hospitals in a single metropolitan area.

U.S. physician knowledge of the FDA-approved indications and evidence base for commonly prescribed drugs: results of a national survey.

Purpose: The Food and Drug Administration (FDA) regulates prescription drug marketing, not prescribing. However, off-label use is common, often lacks supporting evidence, and may expose patients to unwarranted risk. We sought to determine physicians' knowledge of the FDA-approved indications of commonly prescribed drugs, and to assess whether physicians' belief that an indication is FDA-approved increases with level of evidence supporting such use.

Physicians' experience with surrogate decision making for hospitalized adults.

Background: Hospitalized patients frequently lack decision-making ability, yet little is known about physicians' approaches to surrogate decision making.

Objective: To describe physicians' experiences with surrogate communication and decision making for hospitalized adults.

Design: Cross-sectional written survey.

National trends in treatment of type 2 diabetes mellitus, 1994-2007.

Background: Diabetes mellitus is common, costly, and increasingly prevalent. Despite innovations in therapy, little is known about patterns and costs of drug treatment.

Methods: We used the National Disease and Therapeutic Index to analyze medications prescribed between 1994 and 2007 for all US office visits among patients 35 years and older with type 2 diabetes.