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

Advocacy & Public Policy Grantmaking: Matching Process to Purpose

Over the past five years, advocacy and public policy grantmaking has moved away from supporting individual grantees to achieve their particular policy goals toward a more targeted, proactive approach designed to achieve the funder's policy goals. More recently, some funders have begun to explore new ways of designing advocacy and public policy grantmaking to achieve longer-term and more substantial changes in the policy landscape at large. As foundations consider how to approach their advocacy and public policy grantmaking in the future, a better understanding of this variety of approaches, and pros and cons of each among foundations with a long history of policy work, may provide options for framing and focusing such efforts.Building on research conducted in 2007 by Coffman and Campbell, this brief summarizes advocacy and public policy grantmaking approaches and their implications for grant portfolio composition and management, auxiliary supports and evaluation. "Advocacy and public policy grantmaking" refers to grantmaking in support of a wide range of advocacy activities that are intend to trigger, block, maintain, support and/or monitor changes in public policy at any level of government. The findings below emerged from an extensive literature review, as well as interviews with staff at 14 foundations and three independent consultants with in-depth policy experience. Interviewees were selected to represent an array of foundation sizes, content areas and structures, all with mature policy portfolios. Foundation staff were asked to describe their foundation's approach to advocacy and public policy grantmaking, including:The foundation's goals for its advocacy and policy grantsWhat the foundation has learned about how best to structure grantmaking and construct a portfolio to achieve its goalsWhat auxiliary supports (e.g., convenings, technical assistance, etc.) the foundation provides that have proven indispensable to its workHow the foundation defines the role of its policy staff, and how these staff interact with other staffHow the foundation designs reporting and conducts (or would like to conduct) evaluationHow the foundation thinks about its role in the larger field of "actors" in the advocacy field and in relation to other funders.The assessment revealed two approaches to advocacy and public policy grantmaking for which foundation practices and processes are fairly well established: a policy target approach to achieve the passage, successful implementation and maintenance of a funder's specific policy goal; and an advocacy niche approach to strengthen the presence or influence of a particular strategic function (i.e., policy analysis and research or grassroots mobilization) within the policymaking process. In addition, the assessment identified an emerging approach to advocacy and public policy grantmaking that has yet to be well defined: a field-building approach to develop the stability and long-term adaptive capacity of a group -- or field -- of advocacy organizations. Many funders choose a combination of these approaches, and some prefer to be more strongly positioned within a single approach. Approaches are not mutually exclusive, and may even be mutually supportive; but each approach has unique implications for grantee selection, organizational processes, program officer roles and evaluation.This report frames a series of options for advocacy and public policy grantmaking and explores implications for foundation practices by drawing on the best experiences from the field. It presents a particular focus on what it means for foundations to design their grantmaking to build the capacity and influence of a field of advocates to tackle a wide range of policy challenges over time. As such, this brief can help foundation staff and leadership establish a common language and understand trade-offs among the different advocacy strategy paths

Breakthroughs in Shared Measurement and Social Impact

A surprising new breakthrough is emerging in the social sector: A handful of innovative organizations have developed web-based systems for reporting the performance, measuring the outcomes, and coordinating the efforts of hundreds or even thousands of social enterprises within a field. These nascent efforts carry implications well beyond performance measurement, foreshadowing the possibility of profound changes in the vision and effectiveness of the entire nonprofit sector. This paper, based on six months of interviews and research by FSG Social Impact Advisors, examines twenty efforts to develop shared approaches to performance, outcome, or impact measurement across multiple organizations. The accompanying appendices include a short description of each system and four more in-depth case studies

Key Lessons from the Field of Cultural Innovation

The primary purpose of this knowledge synthesis is to provide the Rockefeller Foundation staff with a broader context for considering the findings of an independent evaluation of the Foundation's Cultural Innovation Fund (CIF). The synthesis has been designed to help key audiences understand the state of play, common concepts, challenges, questions, and key lessons from arts organizations who are already engaging innovative strategies and practices

A Case Study on Formal Verification of Self-Adaptive Behaviors in a Decentralized System

Self-adaptation is a promising approach to manage the complexity of modern software systems. A self-adaptive system is able to adapt autonomously to internal dynamics and changing conditions in the environment to achieve particular quality goals. Our particular interest is in decentralized self-adaptive systems, in which central control of adaptation is not an option. One important challenge in self-adaptive systems, in particular those with decentralized control of adaptation, is to provide guarantees about the intended runtime qualities. In this paper, we present a case study in which we use model checking to verify behavioral properties of a decentralized self-adaptive system. Concretely, we contribute with a formalized architecture model of a decentralized traffic monitoring system and prove a number of self-adaptation properties for flexibility and robustness. To model the main processes in the system we use timed automata, and for the specification of the required properties we use timed computation tree logic. We use the Uppaal tool to specify the system and verify the flexibility and robustness properties.Comment: In Proceedings FOCLASA 2012, arXiv:1208.432

A list of websites and reading materials on strategy & complexity

The list has been developed based on a broad interpretation of the subject of ‘strategy & complexity’. Resources will therefore more, or less directly relate to ‘being strategic in the face of complexity’. Many of the articles and reports referred to in the attached bibliography can be accessed and downloaded from the internet. Most books can be found at amazon.com where you will often find a number of book reviews and summaries as well. Sometimes, reading the reviews will suffice and will give you the essence of the contents of the book after which you do not need to buy it. If the book looks interesting enough, buying options are easy

Behavioral Approaches to Corporate Law

This chapter reviews the challenges associated with developing a plausible theory of why psychological heuristics and biases might persist in high-stakes business settings. Specific attention is given to issues of loyalty on corporate boards, behavioral finance, and corporate cultures

Resilience Capacity and Strategic Agility: Prerequisites for Thriving in a Dynamic Environment

organizational resilience, strategic agility, competitive dynamics

Twenty first century standards for thermal comfort : fostering low carbon building design and operation

Nearly 50% of energy consumed in the developed world is consumed in buildings. Despite regulation intent, many new buildings are energy profligate. Thermal comfort standards are partly responsible for this increase in consumption. In this volume, Roaf et al. have described the evolution of current comfort standards and problems inherent in buildings they shape, and have discussed two new methods of regulating thermal comfort in buildings which recognize human adaptation and have potential for reduced energy demand. These new methods incorporate adaptation through a fixed heating and cooling threshold approach (similar to Japanese Cool-Biz) or through heating and cooling setpoints calculated based on outdoor conditions(using CEN standard equations). The impact on comfort and energy demand of these new approaches is investigated for a London office building. Variables such as future climate, future building upgrades, setback temperatures, internal gains and ventilation are also explored. Adoption of the new approaches gave a 50% reduction in heating and cooling energy for the simulated office. The new approach together with optimized setback temperatures, ventilation strategies and higher efficiency equipment gives predicted heating and cooling energy demand close to zero. Recommendations for future regulation, design and operation of buildings are proposed