112th Edition – May 2021

New institute to act as catalyst for wider use of real-world evidence in healthcare decision-making in Europe

A new non-profit organisation called the GetReal Institute, an outgrowth of the IMI-funded GetReal Initiative project, has been set up to drive the adoption of real-world evidence (RWE) in healthcare decision-making in Europe. The Netherlands-based institute will offer an independent, neutral meeting point where regulatory agencies, health technology assessment (HTA) bodies, patient organisations, partners from academia and industry and data partners can work on reducing barriers to the secondary use of data sources for healthcare decision-making.

The institute will tackle challenges related to data discoverability, quality, and accessibility, and issues relating to bridging the gap between RWE and conventional randomised controlled trial approaches. It will act as an ‘incubator and design lab’ where scientific and operational uncertainties relating to generating, evaluating and using RWE can be worked out, while also offering education and training resources through the GetReal Academy.

Any organisation involved in the generation, evaluation, or use of RWE may join the GetReal Institute. It will operate as a not-for-profit member association, and will be supported through a combination of membership fees, revenue from the GetReal Academy, and other programmes and grants.

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The real cost of RSV, a common but often fatal disease

The RESCEU project has put together the most comprehensive cache of evidence to date on the true burden of respiratory syncytial virus (RSV), a disease that still claims millions of mostly infant victims every year. In an interview with the IMI Programme Office, project coordinator Harish Nair of the University of Edinburgh talks about the project’s recent supplement published in the Journal of Infectious Diseases, which he says represents an important source of evidence for policy makers.

‘The publications from RESCEU are certainly the most comprehensive evidence that has been put together in recent times that present the burden of disease of RSV,’ he says. The data gathered includes the first local estimates of RSV-associated acute respiratory infections in older adults; the first global estimates of the economic burden of RSV in children under five; estimates of the RSV-related hospitalisation burden in children under five in seven European countries, and the first estimates of the risk period for death in children under five after RSV illness.

‘This is the beginning of the process to generate evidence to influence policy specifically for the EU,’ explains Professor Nair. The team has been working with the European Centre for Disease Prevention and Control and (ECDC) national public health agencies to develop guidance on RSV surveillance at country level, which would be required prior to the introduction of RSV immunisation.

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EHDEN health data academy welcomes 1 000th student

Over 1 000 people have signed up to the EHDEN Academy, which provides the global scientific community with free educational resources on real world data and real world evidence.

The aim of EHDEN is to make the large-scale analysis of health data in Europe a reality. At the heart of the project is a growing group of trained, certified small and medium-sized enterprises (SMEs) responsible for transforming data owned by hospitals, and others, to a common data model.

The training materials designed for these SMEs formed the basis of the EHDEN Academy, expanding to methods, skills and tools for working with real world data to generate real world evidence. The Academy was launched in April 2020 and has now attracted over 1 000 enrollees from 46 countries worldwide.

Today, the academy features 9 courses and a faculty of 10 expert instructors. The courses are freely available on demand and represent an important source of training for anyone who generates and uses data, works with it, or is involved in the development and use of analytical tools.

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Digital biomarkers: an initiative to help researchers navigate new reality and roles

Mobilise-D and IDEA-FAST have created the Digital Health Catalyst (DHC) in response to the growing need for research and application in the area of real-world digital measurements. According to a Lynn Rochester, coordinator of Mobilise-D and Wan Fai Ng, coordinator of IDEA-FAST, the DHC intends to foster the next generation of early career researchers and clinicians in the field of digital healthcare. It will bring together ‘the collective expertise in digital health across the two IMI consortia for maximum learning, collaboration and impact’.

Accepted methods like questionnaires are subjective and don’t quite capture changes in the severity of a person’s condition. If remote assessment and the use of digital biomarkers as indicators of health are to be adopted widely, priorities will have to be established with regards to education and training, career pathways, cross-disciplinary opportunities and requirements for support and development. During the DHC launch event, early career researchers listed their priorities as skills development, exchange programmes and internships, engaging with the various relevant stakeholders and attending seminars.

The DHC will be a standalone sustainable entity that can attract funding and expand its remit to additional areas to promote and catalyse the field of digital health.

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‘The scale of the funding enables real impact’ – IMI Associated Partner the Children’s Tumor Foundation

The Children's Tumor Foundation is one of IMI's Associated Partners. In an interview with the IMI Programme Office, foundation president Annette Bakker says that the unique IMI model ensures alignment between industry and public partners.

The Children’s Tumor Foundation is a renowned international non-profit organisation that seeks to advance research into a disease called neurofibromatosis, a genetic disorder of the nervous system. They are involved in EU-PEARL and also contributed to the work of PARADIGM. In addition, the organisation has been a member of the Strategic Governing Group (SGG) on oncology, which feeds topics to IMI.

Asked about the benefits of IMI, Dr Bakker says: ‘The scale of the funding enables real impact. Also the unique business model ensures alignment between industry and the public partners on what matters most. The commitment of all the stakeholders is also a great benefit.’

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COVID exacerbates healthcare inequalities for autistic people

Researchers from AIMS-2-TRIALS have published a report that shows that the COVID-19 pandemic has further exacerbated existing healthcare inequalities for autistic people and has likely contributed to disproportionate increases in morbidity and mortality, mental health/behavioural difficulties and reduced quality of life. The researchers arrived at this conclusion after studying COVID-19 policies from 15 countries and analysing data from a survey of over 1 000 autistic people and caregivers. Writing in BMJ OPEN, the team explains that autistic people experienced four significant barriers accessing COVID-19 services:

• despite being at elevated risk for severe illness due to co-occurring health conditions, there was a lack of accessibility of COVID-19 testing (e.g. lack of reasonable adjustments/provision of preparatory materials);
• many COVID-19 outpatient and inpatient treatment services were reported to be inaccessible – predominantly resulting from individual differences in communication needs;
• ICU triage protocols in many European countries may have (directly or indirectly) resulted in discriminatory exclusion from lifesaving treatments;
• interruptions to standard health and social care left over 70% of autistic people without everyday support, in the absence of appropriate mitigation measures.

Dr Bethany Oakley of Kings College London said: ‘Increasing service capacity and investing in resilience planning should be the primary response for decision makers to make sure that there is equal access to hospital and intensive care resources, across the population, and that steps are taken to make sure that autistic people and all those with developmental differences are not put at a disadvantage.’

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Got a promising potential antibiotic? Our GNA NOW project wants to hear from you!

IMI’s GNA NOW project is looking for a novel antibiotic programme to progress to Investigational New Drug (IND). The deadline for expressions of interest is 18 June.

The aim of GNA NOW is to advance the development of antibiotics designed to treat infections caused by Gram-negative bacteria. These bugs are particularly tough to treat, as they are encased in a tough outer membrane which effectively stops many antibiotics from getting into the bacteria and killing it.

Now the project has launched a call for expressions of interest for potential antibiotics with a novel mode of action designed to address severe hospital infections caused by certain drug-resistant, Gram-negative bacteria.

Those interested in getting involved should submit a short, non-confidential submission of interest to the project by 18 June. Expressions of interest will be assessed by the GNA NOW Review Committee, which is made up of industry, independent and academic experts and antibiotic drug discovery and development.

The teams behind the top three expressions of interest will then be invited to submit a full dossier to the committee.

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