Framework of the strengths and challenges of clinically integrated trials: An expert panel report.

The limitations of the explanatory clinical trial framework include the high expense of implementing explanatory trials, restrictive entry criteria for participants, and redundant logistical processes. These limitations can result in slow evidence generation that is not responsive to population health needs, yielding evidence that is not generalizable. Clinically integrated trials, which integrate clinical research into routine care, represent a potential solution to this challenge and an opportunity to support learning health systems.

Real-world experiences with generating real-world evidence: Case Studies from PCORI's pragmatic clinical Studies program.

Background: Over the last decade, randomized studies evaluating outcomes of health care interventions conducted in real-world settings-often termed "pragmatic trials"-have come to be seen as an important means of obtaining relevant, actionable evidence to guide health care decisions. Despite extensive writing on methodological considerations in pragmatic trial design, limited information exists regarding the practical and logistical challenges encountered in carrying out rigorous randomized evaluations in highly representative, real-world contexts.

Methods: The Patient Centered Outcomes Research Institute (PCORI) convened an expert panel in 2017 to examine common tradeoffs in study design and implementation through 3 "case studies" of in-progress, PCORI-funded pragmatic trials.

Patient-centered priorities for improving medication management and adherence.

Objective: The Centers for Education and Research on Therapeutics convened a workshop to examine the scientific evidence on medication adherence interventions from the patient-centered perspective and to explore the potential of patient-centered medication management to improve chronic disease treatment.

Methods: Patients, providers, researchers, and other stakeholders (N = 28) identified and prioritized ideas for future research and practice. We analyzed stakeholder voting on priorities and reviewed themes in workshop discussions.

Risk minimization practices for pregnancy prevention: understanding risk, selecting tools.

According to the March of Dimes, approximately 4% (1/28) of babies are born in the US each year with a birth defect. For the majority of birth defects the etiology is unknown, although chemicals, including drug exposures, probably account for less than 1% of all birth defects. The identification of potential human teratogenicity during drug development is important because drug-induced adverse fetal effects are potentially preventable with the application of risk assessment strategies and risk minimization tools and programs to minimize risk of pregnancy exposure while preserving access to drug benefits; risk assessment and risk minimization together comprise risk management.

Changes in isotretinoin prescribing before and after implementation of the System to Manage Accutane Related Teratogenicity (SMART) risk management program.

Purpose: To assess changes in isotretinoin prescribing following the implementation of the System to Manage Accutane Related Teratogenicity (SMART) risk management program.

Methods: Using nationally representative commercial data resources on prescription drug dispensing patterns, surveys of office-based physician practices, and a large, claims database from a pharmacy benefits manager (PBM), we examined the total number of isotretinoin prescriptions (new and refill), prescriber speciality, and patient characteristics (age, gender, severity of acne indication) in the year before (April 2001-March 2002) and the year following (April 2002-March 2003) implementation of the SMART program.

Results: In the 12-months prior to SMART, 1 508 000 prescriptions were dispensed for isotretinoin, declining approximately 23% to 1 160 000 prescriptions in the year following SMART.

The relationship of asthma therapy and Churg-Strauss syndrome: NIH workshop summary report.

The Churg-Strauss syndrome (CSS) is a distinct form of vasculitis that is notable for its eosinophilia and frequent associations with asthma and sinusitis. Because there has been an increasing recognition that CSS can develop in patients with asthma and that CSS might be associated with specific asthma treatments, the National Heart, Lung, and Blood Institute, the National Institute of Allergy and Infectious Diseases, the Office of Rare Diseases, National Institutes of Health, and the US Food and Drug Administration jointly sponsored a workshop to consider interrelationships among CSS, asthma, and asthma therapeutics and to assess what is known about underlying mechanisms of CSS. Issues related to the criteria for defining and diagnosing CSS were reviewed, including the contemporary understanding that diagnostic biopsies need only reveal eosinophilic perivascular infiltrates and that asthma need not be present when CSS develops.

Monitoring the health status and impact of treatment on Americans: the Medicare Beneficiary Health Status Registry.

Objective: A major new survey program, the Medicare Beneficiary Health Status Registry (MBHSR), has been proposed to improve the monitoring of the health status of Medicare beneficiaries. The MBHSR would collect data by mail with telephone follow up of nonrespondents to permit economical assessment of a total Registry of approximately 200,000 Medicare beneficiaries, approximately 54,000 of whom would be surveyed in any given year. (Surveys would be conducted of samples of new enrollees who would be reinterviewed every five years.

Mammography use by elderly women: a methodological comparison of two national data sources.

Purpose: Estimates of mammography utilization vary considerably, depending on the data source. Among women aged 65 years and older, recent estimates of annual mammography derived from the 1992 National Health Interview Survey (NHIS) were 50% higher than estimates from Medicare claims. We investigated possible reasons for the different estimates.

Evaluation of the 1990 Centers for Disease Control and Prevention smoke-free policy.

Purpose: To determine the prevalence of tobacco use among Centers for Disease Control and Prevention (CDC)/Agency for Toxic Substances and Disease Registry (ATSDR) employees and the effect of the smoke-free policy on smoking behavior and air quality at work.

Design: A stratified telephone survey of 1181 CDC/ATSDR employees randomly selected from employee rosters.

Setting: CDC/ATSDR work sites in Atlanta, Georgia, and other major CDC locations throughout the United States and Puerto Rico.