Generating health technology assessment evidence for rare diseases

Objectives: Rare diseases are often heterogeneous in their progression and response to treatment, with only a small population for study. This provides challenges for evidence generation to support HTA, so novel research methods are required. Methods: Discussion with an expert panel was augmented with references and case studies to explore robust approaches for HTA evidence generation for rare disease treatments. Results: Traditional RCTs can be modified using sequential, three-stage or adaptive designs to gain more power from a small patient population or to focus trial design. However, such designs need to maintain important design aspects such as randomization and blinding and be analyzed to take account of the multiple analyses performed. N-of-1 trials use within-patient randomization to test repeat periods of treatment and control until a response is clear. Such trials could be particularly valuable for rare diseases and when prospectively planned across several patients and analyzed using Bayesian techniques, a population effect can be estimated that might be of value to HTA. When the optimal outcome is unclear in a rare disease, disease specific patient reported outcomes can elucidate impacts on patients’ functioning and wellbeing. Likewise, qualitative research can be used to elicit patients’ perspectives, with just a small number of patients. Conclusions: International consensus is needed on ways to improve evidence collection and assessment of technologies for rare diseases, which recognize the value of novel study designs and analyses in a setting where the outcomes and effects of importance are yet to be agreed.</p

Beyond the Bubble: Technology and the Future of Student Assessment

Provides an overview of information technology's potential to enable better assessments of student achievement. Outlines promising models for testing complex skills, cognition, and learning and for utilizing such assessments to improve instruction

Coordinating Local Adaptive Strategies through a Network-Based Approach

As the impacts of climate change become increasingly destructive and pervasive, climate adaptation has received greater political and academic attention. The traditional top-down model for mitigating climate change, however, is ill-suited to implementing effective adaptation strategies. Yet, local communities most impacted by climate change seldom have the tools and resources to develop effective adaptive strategies on their own. This note argues that a bottom-up, network-based approach could be a promising paradigm towards implementing effective adaptive strategies and empowering affected communities

The State of Adaptation in the United States: An Overview

Over the past two decades the adaptation landscape has changed dramatically. From its early days as a vague theoretical concept, which was often viewed as a threat to advocating for the reduction of greenhouse gas emissions, it has developed into a widely, albeit not universally, recognized governmental mandate to reduce societal vulnerability to climate change. While it is important to appreciate the progress that we are making on this issue, it is impossible to ignore the urgent need to do more. Smart investment can be made by reflecting on what is already underway in order to determine where to build on existing efforts and where to innovate new approaches to fill the gaps in the path forward. In this report we provide illustrative examples of the variety of work on climate change adaptation that is underway in the United States. This is by no means an exhaustive survey of the field; however it does provide insight into the dominant focus of work to date, the resultant gaps, and the opportunities available for advancing this essential aspect of sustainability. We focus on four areas of activity -- agriculture, natural resources, human communities, and policy. The general trends relevant to these sectors can be applied more broadly to other sectors and countries. Adaptation can be thought of as a cycle of activities that ultimately -- if successful -- reduces vulnerability to climate change. This process starts with identifying the impacts of climate change to determine the types of problems climate change might pose. This includes all of the research on the causes and the global, regional, and local manifestations of climate change, often referred to as impacts assessments

Scaling Success: Lessons from Adaptation Pilots in the Rainfed Regions of India

"Scaling Success" examines how agricultural communities are adapting to the challenges posed by climate change through the lens of India's rainfed agriculture regions. Rainfed agriculture currently occupies 58 percent of India's cultivated land and accounts for up to 40 percent of its total food production. However, these regions face potential production losses of more than $200 billion USD in rice, wheat, and maize by 2050 due to the effects of climate change. Unless action is taken soon at a large scale, farmers will see sharp decreases in revenue and yields.Rainfed regions across the globe have been an important focus for the first generation of adaptation projects, but to date, few have achieved a scale that can be truly transformational. Drawing on lessons learnt from 21 case studies of rainfed agriculture interventions, the report provides guidance on how to design, fund and support adaptation projects that can achieve scale

Navigating the Turbulent Waters of School Reform Guided by Complexity Theory

The goal of this research study has been to develop, implement, and evaluate a school reform design experiment at a continuation high school with low-income, low-performing underrepresented minority students. The complexity sciences served as a theoretical framework for this design experiment. Treating an innovative college preparatory program as a nested complex adaptive system within a larger complex adaptive system, the school, we used features of complex adaptive systems (equilibrium, emergence, self-organization, and feedback loops) as a framework to design a strategy for school reform. The goal was to create an environment for change by pulling the school far from equilibrium using a strategy we call “purposeful perturbations” to disrupt the stable state of the school in a purposeful way. Over the four years of the study, several tipping points were reached, and we developed agent-based simulation models that capture important dynamic properties of the reform at these points. The study draws upon complexity theory in multiple ways that have supported improved education for low-achieving students

Emotional and Adrenocortical Responses of Infants to the Strange Situation: The Differential Function of Emotional Expression

The aim of the study was to investigate biobehavioural organisation in infants with different qualities of attachment. Quality of attachment (security and disorganisation), emotional expression, and adrenocortical stress reactivity were investigated in a sample of 106 infants observed during Ainsworth’s Strange Situation at the age of 12 months. In addition, behavioural inhibition was assessed from maternal reports. As expected, securely attached infants did not show an adrenocortical response. Regarding the traditionally defined insecurely attached groups, adrenocortical activation during the strange situation was found for the ambivalent group, but not for the avoidant one. Previous ndings of increased adrenocortical activity in disorganised infants could not be replicated. In line with previous ndings, adrenocortical activation was most prominent in insecure infants with high behavioural inhibition indicating the function of a secure attachment relationship as a social buffer against less adaptive temperamental dispositions. Additional analyses indicated that adrenocortical reactivity and behavioural distress were not based on common activation processes. Biobehavioural associations within the different attachment groups suggest that biobehavioural processes in securely attached infants may be different from those in insecurely attached and disorganised groups. Whereas a coping model may be applied to describe the biobehavioural organisation of secure infants, an arousal model explanation may be more appropriate for the other groups

Evaluating Conservation Effectiveness and Adaptation in Dynamic Landscapes

Rissman talks about evaluating conservation easement effectiveness requires interdisciplinary research that reaches beyond legal analysis to examine how easements influence human behaviors, which subsequently influence environmental conditions. Conservation easement effectiveness is not a fixed target, but is influenced over time by social and ecological landscape change. The promise of perpetuity is central to the appeal of conservation easements within the conservation movement