Diversity, Disability, and Addressing the Varied Needs of Learners

This chapter presents a thorough examination of international and U.S. legal and educational foundations which justify instructional variation and diversification for all students, particularly for those with disabilities. With the exploration of various instructional frameworks, objective setting, methods and strategies, materials, and outcomes assessment, the reader develops an understanding of instructional diversification and why it is important for students with and without disabilities. The diverse educational approaches of differentiated instruction, multi-sensory instruction, Bloom's taxonomy of learning, understanding by design, and universal design for learning are presented and explained.

Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework

Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.