RWE in Europe Paper II: The use of Real World Evidence in the disease context

Real World Evidence (RWE), the use of data not collected via traditional randomised controlled trials (RCT) for decision-making, is becoming more interesting to market-access and reimbursement decision-makers, despite potential methodological issues around its use. This paper, the second in a series looking at the use of Real World Evidence (RWE) in Europe, analyses the opinions of a number of key experts in pricing and reimbursement from a selection of countries across Europe. Discussion centred on the use of RWE in licensing, commissioning, clinical decision-making and patient and outcome related decision-making in the context of three different treatment areas – chronic disease, oncology and rare diseases. Results of discussion sessions with ‘RWE experts’ indicated that the associated benefits of RWE are becoming more relevant but there is a need for a well-organised, high quality system for data generation, interpretation and use. It is likely that different treatment areas will have differing RWE requirements and differing levels of utility. In the rare disease arena, RWE may have a role in licensing based decisions, but this is unlikely for chronic disease or oncology. In order to enhance the role of RWE, and to ensure it meets its full potential in all treatment areas, a multi-stakeholder approach at the EU level is required, with collaboration between national and supranational organisations and all stakeholders including patient organisations, manufacturers and reimbursement agencies

RWE in Europe Paper IV: Engaging pharma in the RWE Roadmap

This paper summarises discussions held at the fourth round-table style meeting with a group of expert stakeholders with experience in specialist disease areas and commissioning of care plus prior experience in the field of real world evidence (RWE). The aim of these meetings was to gain an understanding of the use of RWE across Europe and to develop a road map of initiatives for the pharmaceutical industry in order to enhance their use of RWE. This, the fourth and final paper in this series, outlines the future potential of the stakeholder group involved in the project to date

100 Years of Immunotherapy: The Monaco Charter

Aims of the Monaco Charter: (1) to present the current evidence on the efficacy and safety of allergen-specific immunotherapy (SIT) and to address the reasons for its underuse in clinical practice; (2) to develop strategies to increase the awareness about the benefits and the hazards of SIT in allergic patients, lay public and healthcare professionals not trained in allergy, and (3) to make SIT accessible and affordable to eligible patient

RWE in Europe Paper III: A Roadmap for RWE

Real world evidence (RWE) has been touted as a remedy for current market access issues, facilitating quicker approvals and increased odds of reimbursement at a good price. It is therefore an attractive avenue for pursuit for manufacturers today. This paper, the third in a series looking at the use of RWE in Europe, outlines the discussions held between key opinion leaders in pricing and reimbursement across a number of European countries at a roundtable-style meeting. The aim of the meeting was to develop a 3-year roadmap, and resulting action plan, of initiatives for the enhanced use of RWE in decision-making in the pharmaceutical industry. Following a series of brainstorming sessions across the areas of commissioning and access, clinical evidence and patients and outcomes, contributors were asked to prioritise the importance of a refined set of initiatives identified in these brainstorming sessions to develop the three-year road map. Finally, four key points from the roadmap were identified for initial action: actively engage in early dialogue with payers on RWE needs; consensus exercise on RWD/E in clinical decisions, develop a definition of patient reported/relevant outcomes and develop a model approach for the collection of patient reported/relevant outcomes data. These action points are seen as the most imperative steps for enhancing the role of RWE. If its use is to become more common addressing these steps, as quickly and efficiently as possible, will be vital for all stakeholders in the pharmaceutical arena

A charter to improve patient care in severe asthma

Severe asthma is a subtype of asthma that is difficult to treat and control. By conservative estimates, severe asthma affects approximately 5-10% of patients with asthma worldwide. Severe asthma impairs patients' health-related quality of life, and patients are at risk of life-threatening asthma attacks. Severe asthma also accounts for the majority of health care expenditures associated with asthma. Guidelines recommend that patients with severe asthma be referred to a specialist respiratory team for correct diagnosis and expert management. This is particularly important to ensure that they have access to newly available biologic treatments. However, many patients with severe asthma can suffer multiple asthma attacks and wait several years before they are referred for specialist care. As global patient advocates, we believe it is essential to raise awareness and understanding for patients, caregivers, health care professionals, and the public about the substantial impact of severe asthma and to create opportunities for improving patient care. Patients should be empowered to live a life free of symptoms and the adverse effects of traditional medications (e.g., oral corticosteroids), reducing hospital visits and emergency care, the loss of school and work days, and the constraints placed on their daily lives. Here we provide a Patient Charter for severe asthma, consisting of six core principles, to mobilize national governments, health care providers, payer policymakers, lung health industry partners, and patients/caregivers to address the unmet need and burden in severe asthma and ultimately work together to deliver meaningful improvements in care.Funding for this study, the article processing charges, and the open access charge was provided by AstraZeneca

ARIA digital anamorphosis : Digital transformation of health and care in airway diseases from research to practice

Digital anamorphosis is used to define a distorted image of health and care that may be viewed correctly using digital tools and strategies. MASK digital anamorphosis represents the process used by MASK to develop the digital transformation of health and care in rhinitis. It strengthens the ARIA change management strategy in the prevention and management of airway disease. The MASK strategy is based on validated digital tools. Using the MASK digital tool and the CARAT online enhanced clinical framework, solutions for practical steps of digital enhancement of care are proposed.Peer reviewe

Cabbage and fermented vegetables : From death rate heterogeneity in countries to candidates for mitigation strategies of severe COVID-19

Large differences in COVID-19 death rates exist between countries and between regions of the same country. Some very low death rate countries such as Eastern Asia, Central Europe, or the Balkans have a common feature of eating large quantities of fermented foods. Although biases exist when examining ecological studies, fermented vegetables or cabbage have been associated with low death rates in European countries. SARS-CoV-2 binds to its receptor, the angiotensin-converting enzyme 2 (ACE2). As a result of SARS-CoV-2 binding, ACE2 downregulation enhances the angiotensin II receptor type 1 (AT(1)R) axis associated with oxidative stress. This leads to insulin resistance as well as lung and endothelial damage, two severe outcomes of COVID-19. The nuclear factor (erythroid-derived 2)-like 2 (Nrf2) is the most potent antioxidant in humans and can block in particular the AT(1)R axis. Cabbage contains precursors of sulforaphane, the most active natural activator of Nrf2. Fermented vegetables contain many lactobacilli, which are also potent Nrf2 activators. Three examples are: kimchi in Korea, westernized foods, and the slum paradox. It is proposed that fermented cabbage is a proof-of-concept of dietary manipulations that may enhance Nrf2-associated antioxidant effects, helpful in mitigating COVID-19 severity.Peer reviewe

Nrf2-interacting nutrients and COVID-19 : time for research to develop adaptation strategies

There are large between- and within-country variations in COVID-19 death rates. Some very low death rate settings such as Eastern Asia, Central Europe, the Balkans and Africa have a common feature of eating large quantities of fermented foods whose intake is associated with the activation of the Nrf2 (Nuclear factor (erythroid-derived 2)-like 2) anti-oxidant transcription factor. There are many Nrf2-interacting nutrients (berberine, curcumin, epigallocatechin gallate, genistein, quercetin, resveratrol, sulforaphane) that all act similarly to reduce insulin resistance, endothelial damage, lung injury and cytokine storm. They also act on the same mechanisms (mTOR: Mammalian target of rapamycin, PPAR gamma:Peroxisome proliferator-activated receptor, NF kappa B: Nuclear factor kappa B, ERK: Extracellular signal-regulated kinases and eIF2 alpha:Elongation initiation factor 2 alpha). They may as a result be important in mitigating the severity of COVID-19, acting through the endoplasmic reticulum stress or ACE-Angiotensin-II-AT(1)R axis (AT(1)R) pathway. Many Nrf2-interacting nutrients are also interacting with TRPA1 and/or TRPV1. Interestingly, geographical areas with very low COVID-19 mortality are those with the lowest prevalence of obesity (Sub-Saharan Africa and Asia). It is tempting to propose that Nrf2-interacting foods and nutrients can re-balance insulin resistance and have a significant effect on COVID-19 severity. It is therefore possible that the intake of these foods may restore an optimal natural balance for the Nrf2 pathway and may be of interest in the mitigation of COVID-19 severity

ARIA digital anamorphosis: Digital transformation of health and care in airway diseases from research to practice

Digital anamorphosis is used to define a distorted image of health and care that may be viewed correctly using digital tools and strategies. MASK digital anamorphosis represents the process used by MASK to develop the digital transformation of health and care in rhinitis. It strengthens the ARIA change management strategy in the prevention and management of airway disease. The MASK strategy is based on validated digital tools. Using the MASK digital tool and the CARAT online enhanced clinical framework, solutions for practical steps of digital enhancement of care are proposed