Development of a patient-reported outcome instrument to assess complex activities of daily living and interpersonal functioning in persons with mild cognitive impairment: The qualitative research phase.

Introduction: As drug development research efforts move toward studying patients earlier in the course of Alzheimer's disease (AD), it is important to incorporate the patient's perspective into measurement of outcomes.

Methods: This article summarizes the qualitative work of the Patient-Reported Outcome Consortium's Cognition Working Group in the development of a new self-reported outcome measure in persons with mild cognitive impairment (MCI) due to suspected AD, herein referred to as MCI.

Results: The draft measure captures the patient's voice for two functional domains, complex activities of daily living and interpersonal functioning.

Network analysis of RE-AIM framework: chronology of the field and the connectivity of its contributors.

The reach, effectiveness, adoption, implementation, and maintenance (RE-AIM) framework has been widely used for translational research. We used social network analysis (SNA) to explore how innovative research frameworks, such as RE-AIM, have diffused over time in academic literature. A structured literature review was conducted on RE-AIM between 1999 and 2012.

Futurescapes: expectations in Europe for relative effectiveness evidence for drugs in 2020.

Aim: Explore key factors influencing future expectations for the production of evidence of relative effectiveness (RE) for drugs in Europe in 2020; construct three plausible future scenarios for RE evidence generation.

Materials & Methods: Semi-structured key informant interviews and three rounds of modified Delphi to gather expert perspectives and develop future scenarios.

Results & Conclusion: Most influential factors were degree of regulator use of postmarketing authorization (postlaunch) efficacy studies and adaptive licensing; degree of pan-European health technology assessment body coordination in reviewing prelaunch evidence and demanding postlaunch studies; the nature of regulator - health technology assessment body interaction.

Futurescapes: evidence expectations in the USA for comparative effectiveness research for drugs in 2020.

Aim: Explore key factors influencing future expectations for the production of evidence from comparative effectiveness research for drugs in the USA in 2020 and construct three plausible future scenarios.

Materials & Methods: Semistructured key informant interviews and three rounds of modified Delphi with systematic scenario-building methods.

Results & Conclusion: Most influential key factors were: health delivery system integration; electronic health record development; exploitation of very large databases and mixed data sources; and proactive patient engagement in research.

The future of comparative effectiveness and relative efficacy of drugs: an international perspective.

Drug development takes place in a global marketplace, albeit with the USA and EU markets currently dominating. In the USA, demands for comparative effectiveness research have gained traction against a backdrop of health delivery reform, while European stakeholders deliberate the role of relative effectiveness in health technology assessment, trying to reduce the duplication of effort by regulators and health technology assessment bodies. In both arenas, drug-makers are faced with mounting drug development costs, and uncertainty over the types of evidence acceptable for a growing list of stakeholders.

Patient and clinician views on comparative effectiveness research and engagement in research.

Aims: Describe and compare patient and primary care clinician attitudes about comparative effectiveness research (CER) and inclusion of patients and clinicians as partners in clinical research.

Materials & Methods: Online survey of patients (n = 900) and primary care clinicians (n = 750) from opt-in survey panels.

Results: Nearly half (45%) of clinicians were not familiar with CER, but after reviewing a definition of CER, most (76%) agreed that it can improve patient care.

Conceptual and practical foundations of patient engagement in research at the patient-centered outcomes research institute.

Purpose: To provide an overview of PCORI's approach to engagement in research.

Methods: The Patient-Centered Outcomes Research Institute (PCORI) was established in 2010 to fund patient-centered comparative effectiveness research. Requirements for research funding from PCORI include meaningful engagement of patients and other stakeholders in the research.

A systematic review of approaches for engaging patients for research on rare diseases.

Background: Patients with rare diseases have limited access to useful information to guide treatment decisions. Engagement of patients and other stakeholders in clinical research may help to ensure that research efforts in rare diseases address relevant clinical questions and patient-centered health outcomes. Rare disease organizations may provide an effective means to facilitate patient engagement in research.

Apolipoprotein L1 gene variants associate with prevalent kidney but not prevalent cardiovascular disease in the Systolic Blood Pressure Intervention Trial.

Apolipoprotein L1 gene (APOL1) G1 and G2 coding variants are strongly associated with chronic kidney disease (CKD) in African Americans (AAs). Here APOL1 association was tested with baseline estimated glomerular filtration rate (eGFR), urine albumin:creatinine ratio (UACR), and prevalent cardiovascular disease (CVD) in 2571 AAs from the Systolic Blood Pressure Intervention Trial (SPRINT), a trial assessing effects of systolic blood pressure reduction on renal and CVD outcomes. Logistic regression models that adjusted for potentially important confounders tested for association between APOL1 risk variants and baseline clinical CVD (myocardial infarction, coronary, or carotid artery revascularization) and CKD (eGFR under 60 ml/min per 1.

Patient-powered research networks aim to improve patient care and health research.

The era of big data, loosely defined as the development and analysis of large or complex data sets, brings new opportunities to empower patients and their families to generate, collect, and use their health information for both clinical and research purposes. In 2013 the Patient-Centered Outcomes Research Institute launched a large national research network, PCORnet, that includes both clinical and patient-powered research networks. This article describes these networks, their potential uses, and the challenges they face.

PCORI at 3 years--progress, lessons, and plans.

The Patient-Centered Outcomes Research Institute (PCORI) has established distinctive pathways for funding and conducting practical research and has awarded over $318 million for studies covering a wide range of conditions, locations, and socioeconomic characteristics.

Social support, self-efficacy for decision-making, and follow-up care use in long-term cancer survivors.

Objective: Cancer survivors play an important role in coordinating their follow-up care and making treatment-related decisions. Little is known about how modifiable factors such as social support are associated with active participation in follow-up care. This study tests associations between social support, cancer-related follow-up care use, and self-efficacy for participation in decision-making related to follow-up care (SEDM).

Role of oncologists and primary care physicians in providing follow-up care to non-Hodgkin lymphoma survivors within 5 years of diagnosis: a population-based study.

Purpose: The growing population of non-Hodgkin lymphoma (NHL) survivors living longer with high physical and psychological treatment burden, in combination with the projected shortage of medical professionals, necessitates redesigning cancer follow-up care. This study examined follow-up care patterns, factors associated with follow-up care, and attitudes towards follow-up care among NHL survivors.

Methods: We surveyed survivors of aggressive NHL 2 to 5 years post-diagnosis (N = 363) using a population-based sample from the Los Angeles County Surveillance Epidemiology and End Results registry.

How the Patient-Centered Outcomes Research Institute is engaging patients and others in shaping its research agenda.

Clinical research has been driven traditionally by investigators, from generating research questions and outcomes through analysis and release of study results. Building on the work of others, the Patient-Centered Outcomes Research Institute (PCORI) is tapping into its broad-based stakeholder community--especially patients, caregivers, and their clinicians--to generate topics for research, help the institute prioritize those topics, select topics for funding, and ensure patients' involvement in the design of research projects. This article describes PCORI's approach, which is emblematic of the organization's mandate under the Affordable Care Act to seek meaningful ways to integrate the patient's voice into the research process, and describes how it is being used in selection of research that PCORI will fund.