- New report highlights socio-economic impacts of IMI projects
- IMI Scientific Committee issues opinions on drug repurposing and equitable access
- It's time to close the paediatric gap – an opinion piece by Pierre Meulien
- c4c’s paediatric trial network gets put to the test
- Lab-grown tumours will help end drought of childhood cancer drugs
- INNODIA kicks off clinical trials for type 1 diabetes treatments
- COMBACTE-MAGNET launches AMR travel tool
- IMI pain project NGN-PET creates new business for Italian SME Axxam
- EUPATI launches new e-learning platform for patients
New report highlights socio-economic impacts of IMI projects
Are IMI projects delivering socio-economic benefits? Yes, but it often takes time for these to become apparent, concludes a new report on the outcomes of 44 IMI1 projects. The report, by Clarivate’s Centre for Innovation in Regulatory Science (CIRS), delves into the public summaries of the final reports of 44 IMI1 projects in the hunt for project results and impacts. The authors identified project outcomes in four categories: innovation; infrastructure and resources for further research; structuring the European research area; and dissemination of information. This revealed a wealth of results covering diverse disease areas (e.g. neurodegenerative disease, rheumatoid arthritis, pain, cancer, and infectious diseases) as well as cross-cutting issues such as safety, manufacturing issues, knowledge management, and education and training.
They then analysed how these outcomes could contribute to six potential socio-economic impact types:
- increase robustness and reproducibility of research
- reduce the time and cost of research
- improve manufacturing processes
- support environmental sustainability
- improve standards of care
- consolidate and expand knowledge base.
The study reveals that while some projects have delivered concrete socio-economic benefits, in many more the benefits are not yet tangible, and this is in line with the nature of IMI projects and the medicines development process more broadly. ‘It requires time to produce innovative solutions, often happening in the later phases of the project lifecycle and very often even beyond the end date (after projects have been completed),’ the report reads. ‘As a result, the impact of IMI projects is expected to evolve with time.’
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IMI Scientific Committee issues opinions on drug repurposing and equitable access
IMI’s Scientific Committee has issued opinions on two topics: drug repurposing and equitable access.
Drug repurposing refers to the use of existing medicines or active substances for different diseases. Because these medicines have reached an advanced stage of development or even been approved, extensive data on them already exists, and this can speed up their further development for new disease areas. A lot of research into COVID-19 treatments has focused on this approach. There are however challenges in the repurposing field, and the Scientific Committee suggests that a public-private partnership approach could help to make progress on certain questions. Here they highlight in particular the use of repurposed drugs in precision medicine for diseases such as cancer.
On equitable access, the committee writes: ‘Reducing inequity in health is an important global health objective. This is also true for the European Union as based on many studies, there is a significant health gap between higher and lower income European Member States.’ They suggest that in future programmes, including public-private partnerships, the EU should take into account how research projects can facilitate the reduction of health gaps between countries with different patient subgroups, economic status or advancement in health system design.
They conclude by recommending that future EU research initiatives, including PPPs, address inequity in health by:
- taking into account the heterogeneity in patient populations and assessing differences in unmet medical needs in pharmaceutical R&D;
- developing innovative concepts for equitable patient access to medical innovation across EU Member States;
- requesting data on transferability of health policy recommendations to lower income countries with less advanced health systems.
Find out more
- Visit the Scientific Committee web page
- Read the Scientific Committee opinions on drug repurposing and equitable access
It's time to close the paediatric gap – an opinion piece by Pierre Meulien
Innovation has been slow to reach the youngest patients. Thankfully, that is changing, writes IMI Executive Director Pierre Meulien in a new opinion piece on the IMI website.
Today, half of new medicines are not tested in children, and in the absence of data from clinical trials, clinicians have traditionally administered medicines off-label; dosing is guesswork, with per-kilo-values used as a marker. But children are not simply small adults; they don’t metabolise drugs in the same way adults do. Yet despite growing support for greater testing of medicines in children, the how remains a challenge.
At IMI, a growing number of projects are supporting the development of medicines for children. For example, ITCC-P4 is developing preclinical models of high-risk paediatric cancers to advance research, while c4c is setting up standards and a network for clinical trials involving children. They’ve recruited advisory groups made up of young people with different diseases who, for example, have helped draw up child-friendly consent forms.
Looking to the future, IMI is preparing a project on the genetic screening of newborn babies for rare diseases. Again, though this topic has been under discussion for decades, it is fraught with ethical concerns.
‘Given the kind of infrastructure and that would be needed to build such a screening platform, and given the challenges in terms of acceptance and safety, ethics, policy, rights and the law that it would surface, a public-private partnership seems to be the best option,’ writes Dr Meulien. ‘Reaching any kind of consensus on such a topic will require a lot of cooperation with partners inside and outside of the IMI world. Nobody can do it alone, and it’s crucial that we get it right.’
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c4c’s paediatric trial network gets put to the test
c4c aims to enable hospitals and clinical sites across Europe to become ‘trial ready’ for paediatric clinical trials. The project has just embarked on a number of proof of viability (PoV) studies, where they use clinical trials to build and test the network.
‘The studies are testing the ability of the c4c network to provide information and advice that is not always available to sponsors (the organisation responsible for overseeing and/or funding the study),’ explained the project leadership team in an interview with the IMI Programme Office.
‘Investigators (the person/people responsible for carrying out the study at a site) sometimes find interaction with sponsors and contract research organisations (the organisation contracted by the sponsor to perform some of their duties) difficult. By allowing clinicians to talk with clinicians, the c4c network facilitates site identification, feasibility, and setup, including troubleshooting delayed ethics applications, site understanding of budgets and contracts, and trial procedures at site level.'
The project has also set up advice groups in as well as a public involvement database comprising individual parents / patients and patient organisations.
‘The c4c network promotes the central role of participants in paediatric trials by supporting children, young people, and their families in activities such as the review of study protocols, to ensure their opinions and needs are incorporated in the design and implementation of studies,’ the project said.
Find out more
- Read the article in full
- If you're interested in getting involved in the project, you can sign up to the c4c patient database.
Lab-grown tumours will help end drought of childhood cancer drugs
Because childhood cancer is rare, it receives relatively little scientific attention. Pharma companies have in their possession molecules that hold promise as drugs for use in child cancer patients, but there has been a lack of tools and animal models that can be used to test the molecules’ effects on tumours. ITCC-P4 was conceived to fix this problem; the project is creating hundreds of different childhood tumours in mouse models in order to study them in detail and, ultimately, use them to test out promising molecules that will lead to new drugs.
‘Our most fervent hope is that the tumour models within the platform represent the heterogeneity across paediatric tumour types. So, let’s say for instance, there are 30 ‘ways’ for a normal cell to essentially go haywire and become a childhood sarcoma. We expect to have in our models those 30 ‘ways’ represented,’ explains project leader Louis Stancato of Eli Lilly.
‘In other words, a patient comes into a clinic, a chunk of his or her tumour is taken and then sequenced, and it’s found that, for example, the cell has gone awry in this particular way. The idea is that we have models like that in our repertoire and we can test molecules against that particular mode of tumorigenesis. We can see which drugs are efficacious, which ones are active. If the child has a certain mutation, well we hope to have that mutation represented in our collection, and hopefully we can identify drugs in the pipeline - or maybe even on the market already - that might target that particular mutation.’
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- Read the article in full
INNODIA kicks off clinical trials for type 1 diabetes treatments
IMI’s INNODIA and INNODIA HARVEST projects have launched four clinical trials designed to test treatments to prevent and cure type 1 diabetes. The trials focus on children, adolescents and adults aged from 5 to 45 years who have been diagnosed within the past 6 weeks.
The first trial, MELD-ATG, was launched by INNODIA in December 2020, and investigates whether ATG (anti-thymocyte globulin) antibodies targeting the immune system can stop the body´s immune system from attacking the cells in the pancreas that produce the hormone insulin. Insulin is responsible for regulating blood sugar levels. Three further clinical trials have since been launched by INNODIA’s sister project, INNODIA HARVEST. The trials all follow the INNODIA master protocol for clinical trials, which received the green light from the European Medicines Agency (EMA) earlier in 2020.
‘This is a very important moment for us,’ said INNODIA coordinator Professor Chantal Mathieu of University Hospital Leuven. ‘We bring our INNODIA network to the next level, by going from biomarker discovery to clinical interventions.’
All the trials focus on people who have just been diagnosed with type 1 diabetes because research has shown that in the newly-diagnosed, half of the cells in the pancreas that produce insulin are still working (this is known as the honeymoon). The hope is that by treating these people with drugs designed to protect these cells, they will retain the ability to produce their own insulin, and be spared the need to inject themselves with insulin.
‘With a strong community of people with type 1 diabetes and big pharma companies involved, we have high hopes to discover products that will increase the quality of life for people living with type one diabetes,’ said Dr Olivier Arnaud, a member of INNODIA’s Patient Advisory Committee (PAC).
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COMBACTE-MAGNET launches AMR travel tool
Antimicrobial resistance (AMR) project COMBACTE-MAGNET has launched a new, freely accessible online tool that allows healthcare professionals and the public to assess the risk of international travellers acquiring (and spreading) antimicrobial-resistant bacteria.
Travel plays a major role in the spread of AMR, and the new AMR Travel Tool will make it easier for healthcare professionals to evaluate patients’ travel history and associated AMR risk factors. The healthcare professional’s section of the website highlights differences in resistance levels between the travel destination and the host country, for example.
Meanwhile, travellers can enter their travel destination and receive general advice on infection control as well as pathogen-specific advice on the infection risks related to their destination (and tips on how to avoid them).
Find out more
- Visit the AMR Travel Tool
IMI pain project NGN-PET creates new business for Italian SME Axxam
Neuropathic pain is a chronic condition that affects around 8 % of the population and can be triggered by things like surgery, injury, and chemotherapy. Current treatments only work in a number of patients. IMI project NGN-PET explored how the interactions between neurons and other cells in the nervous system as well as immune cells contribute to neuropathic pain. Among other things, the project delivered a range of tools that allow researchers to screen, identify and evaluate potential drugs to treat neuropathic pain.
Now, Italian SME Axxam, which coordinated NGN-PET, has incorporated many of these tools into its research services. The tools can be accessed via research collaboration agreements or commercial agreements.
For example, one key project output was a procedure to produce pain-relevant in vitro models (including sensory neurons and immune cells), derived from primary cells and human induced pluripotent stem cells (hiPSCs), in miniaturised in vitro formats – something that is technically highly challenging.
Disseminating the NGN-PET results at congresses and meetings has helped to attract a number of new customers and contracts for Axxam on both sides of the Atlantic.
Explaining how NGN-PET gave Axxam a competitive edge, Scientific Innovation Manager Dr Paola Tarroni said: ‘Other companies offer similar services in the same sector; however, NGN-PET gave us resources to have hands-on new technologies and know-how, and to produce case studies demonstrating Axxam’s capabilities and skills in the field.’
Find out more
- Read the full news story
- Read the NGN-PET factsheet
- Visit the NGN-PET website
- Visit the Axxam website
EUPATI launches new e-learning platform for patients
Patient education initiative EUPATI has launched the EUPATI Open Classroom, an e-learning platform that will expand access to the EUPATI Patient Expert Training Programme. The course started life under the IMI project EUPATI as a 14-month course on medicines research and development combining online and face-to-face learning. Graduates receive a certificate and are known as EUPATI Fellows, and the skills and knowledge gained allow them to contribute to medicines R&D in a wide range of ways.
EUPATI received additional funding from EIT (European Institute for Innovation and Technology) Health to transform the training content into smaller units following a MOOC (‘massive open online course’) format. This new version of the programme is now available via the EUPATI Open Classroom.
Access to the EUPATI Open Classroom is open to anyone in the world. Those who want to become a EUPATI Fellow can still do so by registering as a learner and following the full programme and attending two training events. However, it is now also possible to register as a learner and choose and complete individual courses. There is no fee for accessing the courses, however, those who want to complete the assessment (and receive a certificate if they pass) will have to pay a small fee. Registered learners can follow courses at their own pace, and track their progress via a personalised dashboard.
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