Too Ultra-Rare for Care? Orphan Drug Availability for People Living With Ultra-Rare Respiratory Diseases in the Pre, Present and Post Pandemic Worlds

Objectives: To compare the current and potential availability of treatments for seven index ultra-rare respiratory diseases before and after the peak of the COVID-19 pandemic. Method(s): At the 2019 ISPOR EU Conference, a landscape review of available treatments for ultra-rare respiratory diseases was presented. Therefore, 3 years on, we sought to explore if treatment access has improved for ultra-rare respiratory diseases. A landscape review was undertaken to seek evidence of treatment developments for ataxia telangiectasia (AT), lymphangiomatosis (LYMF), pulmonary alveolar proteinosis (PAP), pleuroparenchymal fibroelastosis (PPFE), pulmonary alveolar microlithiasis (PAM), pulmonary dendriform ossification (PDO), and light chain deposition disorders (LCDD). Information from clinicaltrials.gov, orpha.net, the EMA and FDA archives and websites of five health technology assessment (HTA) bodies was narratively synthesised. Result(s): The 2019 clinicaltrials.gov search identified 24 studies and the 2022 search 25 studies at various stages of the clinical trial process. They concerned PAP (2019/2022: 11/10), AT (6/10), LCDD (5/4), LYMF (1/0) and PPFE (1/1). No studies were identified for PAM or PDO. The 2019 review found treatments for AT and PAP were granted orphan status by the EMA and the FDA, and in 2021 the FDA granted orphan status to another AT treatment. Neither search found reimbursement submissions for any of the ultra-rare respiratory disease. Conclusion(s): There still remains an obvious lack of proven and available treatments for ultra-rare respiratory diseases. Over the past 3 years, only 1 new treatment has been granted regulatory approval and no treatments have yet to result in therapies that are licensed or approved by HTA bodies. It is our hope that as we enter the post-pandemic world, we as industry professionals and researchers start to adequately address the treatment needs of people living with these debilitating conditions. Copyright © 2022

Moderate support for the use of digital tracking to support climate-mitigation strategies.

The use of digital tracking of individuals throughout the coronavirus 2019 (COVID-19) pandemic renewed societal debates on the efficacy and ethics of digital surveillance to mitigate collective crises. While digital emissions tracking is being used to support climate-mitigation strategies, to date there has been limited exploration of the opportunities and challenges of deploying it at the individual level. Here, we assess temporal and regional differences in levels of support for the use of digital surveillance in times of crisis, such as climate change. Results from a global survey indicate moderate support for the use of digital tracking, including for personal carbon footprints. Response varied regionally, with the lowest support in North America and Europe. This study raises key questions-if digital surveillance tools could be part of a socially acceptable response to the climate crisis, is it worth exploring? Or is this an unacceptable risk for society?

Ten new insights in climate science 2021: a horizon scan

Non-technical summaryWe summarize some of the past year's most important findings within climate change-related research. New research has improved our understanding about the remaining options to achieve the Paris Agreement goals, through overcoming political barriers to carbon pricing, taking into account non-CO2 factors, a well-designed implementation of demand-side and nature-based solutions, resilience building of ecosystems and the recognition that climate change mitigation costs can be justified by benefits to the health of humans and nature alone. We consider new insights about what to expect if we fail to include a new dimension of fire extremes and the prospect of cascading climate tipping elements.Technical summaryA synthesis is made of 10 topics within climate research, where there have been significant advances since January 2020. The insights are based on input from an international open call with broad disciplinary scope. Findings include: (1) the options to still keep global warming below 1.5 °C;(2) the impact of non-CO2 factors in global warming;(3) a new dimension of fire extremes forced by climate change;(4) the increasing pressure on interconnected climate tipping elements;(5) the dimensions of climate justice;(6) political challenges impeding the effectiveness of carbon pricing;(7) demand-side solutions as vehicles of climate mitigation;(8) the potentials and caveats of nature-based solutions;(9) how building resilience of marine ecosystems is possible;and (10) that the costs of climate change mitigation policies can be more than justified by the benefits to the health of humans and nature.Social media summaryHow do we limit global warming to 1.5 °C and why is it crucial? See highlights of latest climate science.

Mass SARS-CoV-2 Testing in a Dormitory-Style Correctional Facility in Arkansas.

Objectives. To assess SARS-CoV-2 transmission within a correctional facility and recommend mitigation strategies.Methods. From April 29 to May 15, 2020, we established the point prevalence of COVID-19 among incarcerated persons and staff within a correctional facility in Arkansas. Participants provided respiratory specimens for SARS-CoV-2 testing and completed questionnaires on symptoms and factors associated with transmission.Results. Of 1647 incarcerated persons and 128 staff tested, 30.5% of incarcerated persons (range by housing unit = 0.0%-58.2%) and 2.3% of staff tested positive for SARS-CoV-2. Among those who tested positive and responded to symptom questions (431 incarcerated persons, 3 staff), 81.2% and 33.3% were asymptomatic, respectively. Most incarcerated persons (58.0%) reported wearing cloth face coverings 8 hours or less per day, and 63.3% reported close contact with someone other than their bunkmate.Conclusions. If testing remained limited to symptomatic individuals, fewer cases would have been detected or detection would have been delayed, allowing transmission to continue. Rapid implementation of mass testing and strict enforcement of infection prevention and control measures may be needed to mitigate spread of SARS-CoV-2 in this setting.

[Intensive care back up for infectious disease disasters]. / Intensivmedizinisches Back-up bei infektiologischen Katastrophen.

BACKGROUND: Outbreaks of infectious diseases pose particular challenges for hospitals and intensive care units. OBJECTIVES: Typical infectiological scenarios and their significance for modern intensive care medicine are presented. MATERIALS AND METHODS: Selected pathogens/infectious diseases that have significantly strained the resources of intensive care units are described. RESULTS: Intensive medical care is necessary in severe cases of many infectious diseases. In the context of epidemics/pandemics, many critically ill patients have to be admitted within a short time. Examples are the 2009 H1N1 influenza pandemic, the 2011 enterohemorrhagic Escherichia coli (EHEC) outbreak in northern Germany, the 2014/2015 Ebola fever outbreak and the 2020 coronavirus disease 19 (COVID-19) pandemic. Multidisciplinary teams, protocol development, adequate staffing, and training are required to achieve optimal treatment outcomes, including prevention of healthcare worker infections. CONCLUSIONS: Pandemics and epidemics are unique challenges for intensive care unit preparedness planning.