Value and Implementation of the Aggregate Safety Assessment Plan.

Aggregate safety assessment involves evaluation of the totality of safety data to characterize the emerging safety profile of a product. The Drug Information Association-American Statistical Association Interdisciplinary Safety Evaluation scientific working group recently published an approach to developing an Aggregate Safety Assessment Plan (ASAP). Creation of an ASAP facilitates a consistent approach to safety data collection and analysis across studies and minimizes important missing data at the time of regulatory submission. A critical aspect of the ASAP is identification of the Safety Topics of Interest (STOI). The STOI, as defined in the ASAP, comprises adverse events (AEs), which have the potential to impact the benefit: risk profile of a product and typically require specialized data collection or analyses. While there are clear benefits to developing an ASAP for a drug development program, multiple concerns may be encountered with implementation. This article uses the examples of two STOIs to demonstrate the benefits and efficiencies gained with implementation of the ASAP in safety planning as well as in optimally characterizing the emerging safety profile of a product.

Kidney-Related Research in the United States: A Position Statement From the National Kidney Foundation and the American Society of Nephrology.

Kidney disease is an important US public health problem because it affects over 37 million Americans, and Medicare expenditures for patients with chronic kidney disease now alone exceed $130 billion annually. Kidney disease is characterized by strong racial, ethnic, and socioeconomic disparities, and reducing kidney disease incidence will positively impact US health disparities. Due to the aging of the US population and an unabated obesity epidemic, the number of patients receiving treatment for kidney failure is anticipated to increase, which will escalate kidney disease health expenditures. The historical and current investment in kidney-related research via the National Institute of Diabetes and Digestive and Kidney Diseases has severely lagged behind ongoing expenditures for kidney disease care. Increasing research investment will identify, develop, and increase implementation of interventions to slow kidney disease progression, reduce incidence of kidney failure, enhance survival, and improve quality of life. This perspective states the urgent reasons why increasing investment in kidney-related research is important for US public health. The National Kidney Foundation and the American Society of Nephrology are working together to advocate for increased funding for the National Institute of Diabetes and Digestive and Kidney Diseases. The long-term goal is to reduce the burden of kidney disease in the US population and improve the quality of life of patients living with kidney disease.

Returning to Growth: One Academic Medical Center's Successful Five-Step Approach to Change Management.

The University of Alabama at Birmingham academic medical center (UAB AMC) had achieved great success and growth during the 50 years since its founding. However, the challenging and more competitive environment of the 2000s left the UAB AMC on a downward trajectory. The UAB AMC had to overcome difficult internal cultural and structural barriers that stood in the way of the transformational change needed to remain competitive. Competition rather than collaborative and strategic financial investment were the primary cultural barriers for the UAB AMC, while people were the primary structural barrier. Leadership identified 5 steps that were critical for the transformation that occurred between 2013 and 2018: alignment of leadership; creating a compelling and credible shared vision; identifying cultural and structural barriers; creating a thoughtful, data-driven intervention; and improved communication and accountability. Following these steps enabled the UAB AMC to transform its institutional structure and culture. As a result, the UAB AMC thrived, returning to substantial growth in research and clinical care. UAB AMC School of Medicine grew by $100 million in National Institutes of Health funding and moved up 10 spots in ranking. In 2018, UAB Hospital had 10 specialties ranked by U.S. News & World Report, 7 more than in 2013. This article outlines the approach taken and provides a conceptual framework for other AMCs eager to transform their structure and culture and position themselves for growth.

Current State and Future of Research in Nephrology.

The focus of this article is to review the available funding opportunities for the nephrology workforce at all career levels and review the current challenges involved in the career of a physician-scientist. While the scarcity of nephrology fellows for training programs is a continuing challenge, increased funding for the National Institutes of Health is encouraging particularly for early career investigators. In addition to National Institutes of Health funding, other funding sources are also discussed as they provide much needed bridge funding during key transition periods for young careers. Recent initiatives such as the Advancing American Kidney Health, KidneyX, and National Institute of Diabetes and Digestive and Kidney Diseases' Kidney Precision Medicine Project offer new research opportunities for bringing much needed innovation to improve lives of people with kidney diseases. The time is now for us to seize the opportunity and ensure that a strong workforce will be able to take advantage of these potential game changers for nephrology.

Ethical considerations in industry-sponsored multiregional clinical trials.

During the last several decades, the scientific and ethics communities have addressed important ethical issues in medical research, resulting in the elaboration and adoption of concepts, guidelines, and codes. Ethical issues in the conduct of Multiregional Clinical Trials have attracted significant attention mainly in the last two decades. With the globalization of clinical research and the rapid expansion to countries with a limited tradition of biomedical research, sponsors must proactively address local ethical issues, the adequacy of oversight as well as the applicability and validity of data, and scientific conclusions drawn from diverse patient populations. This paper highlights some core ethical principles and milestones in medical research, and, from an industry perspective, it discusses ethical issues that the clinical trial team may face when conducting Multiregional Clinical Trials (MRCT, clinical trials conducted at sites located across multiple geographic regions of the world). This paper further highlights the areas of consensus and controversies and proposes points to consider.