Aligned with the idea of the IC Permed launched this week, IMI has been paving the road towards personalised medicines since 2008

The realisation that “one size does not fit all” – that is, not every medicine is suitable for every patient – dawned on pharma quite a while back, but translating this sentiment into reality is taking time.

That does not mean that we have not come a long way since this concept initially came to light. Less than a decade ago, sequencing the complete genome of a single individual took an eternity and threatened to break the bank. Nowadays this can be accomplished in under 24 hours and costs less than €1000.

Moreover, the advent of the big data/smart data/real world data revolution has set us on a clear path towards more precisely-targeted or even personalised medicine. The ability to collate, store, share and link data from a variety of sources, including health records, disease-specific registers and other health data repositories, will change dramatically the way in which we deliver quality healthcare and develop life-saving medicines.

Nevertheless, this type of evolution require drivers and one of the key motivators in this area is the world’s largest public-private partnership in the life sciences sector, the Innovative Medicines Initiative. Completely aligned with the ideas behind the International Personalised Medicines consortium that the Commission is launching this week, and financed jointly by the European Union and EFPIA, several of IMI’s projects, which are outlined here, aim to take personalised medicines development to the next level.

Currently, many drugs fail late in drug development because the tests introduced early on simply do not reflect what happens in real life when the drug is administered in patients, partly because these early tests rely heavily on animal cells. The STEMBANCC project looks to generate and characterise 1,500 high-quality, human, induced pluripotent stem (iPS) cell lines that can be used to study disease biology and detect disease markers, as well as to help to identify potential drug targets. The will help accelerate and refine drug development, ensuring that patients benefit from more effective and safer drugs.

STEMBANCC is complemented by EBiSC, whose goal it is to establish a centralised facility where stakeholders can store and access high-quality, well-characterised iPS cells covering a range of disease areas as well as cells from healthy donors. EBiSC aims to be a centre of excellence for applied science, championing the most advanced methods for characterising, processing, storing and distributing iPS cells.

‘Medicines adaptive pathways to patients’ (MAPPs) is an approach that is set to provide patients with a more timely access to beneficial medicines, beginning with a small group of well-identified patients. ADAPT-SMART will create a platform where the conditions and feasibility of MAPPs implementation within the EU regulatory/legal context can be discussed openly and also to ensure that MAPPs-related work in IMI is well coordinated. Engaging in a dialogue with relevant stakeholders, the ADAPT-SMART consortium will help to align understanding of the impact of MAPPs. This will increase the probability of successful innovation and accelerate access to crucial therapies.

Recent years have seen an explosion of databases containing medical and research data, from Electronic Health Records (EHRs), cohort studies, disease-specific studies, and biobanks, to name a few. Nevertheless, this data is scattered across diverse platforms, so can’t be exploited fully. Linking up the data would allow scientists to significantly advance medical R&D.

EMIF’s focus is on the development of a common information framework of patient-level data that will link up and facilitate access to diverse medical and research data sources, opening up new avenues of research for scientists. By bringing together this data from across different populations, we can increase sample sizes and facilitate the study of rare or highly specific subgroups.

The development and integration of Electronic Health Record systems (EHRs) for medical research can: enable substantial efficiency gains; make Europe more attractive for R&D investment; and provide patients better access to innovative medicines and improved health outcomes. EHR4CR aims to provide adaptable, reusable and scalable solutions for reusing data from Electronic Health Record systems for Clinical Research. Electronic Health Record (EHR) data offer large opportunities for the advancement of medical research, the improvement of healthcare, and the enhancement of patient safety.

Some of the IMI projects are scrutinizing more specific disease areas.

Existing literature, public databases, and private companies, provides us potentially with vast amounts of data that could be used to pave the way for a better classification of patients, based on underlying causes instead of symptoms. AETIONOMY is set to pave the way towards a new approach to this classification of neurodegenerative diseases, particularly Alzheimer’s and Parkinson’s diseases.

The AETIONOMY team will examine how to organise and structure different types of data and apply this knowledge to construct a new classification of patient groups, based on the underlying causes of disease. This will help drug companies identify the most promising targets for new drugs and, crucially, the patients mostly likely to benefit.

The goal of EU-AIMS is to generate tools to enhance our understanding of autism spectrum disorders, for which no drugs currently treat its main symptoms. The project identifies the biological causes of autism and has developed innovative approaches to speed up the development of new, more effective treatments. Once the various biological causes of autism are identified, a physician could alter the treatment to suit an individual’s specific condition.

NEWMEDS is a unique project that partners top scientists with nearly all the world’s major medicines manufacturers. The NEWMEDS project has shed light on the underlying causes of schizophrenia and depression, developed new tools to aid in the development of new drugs, and proposed novel, more efficient ways of carrying out clinical trials. NEWMEDS also demonstrated that early clinical trials of potential medicines for schizophrenia could be made shorter and more efficient by including more women, more people with certain types of symptoms, and younger patients.

A range of different forms of severe asthma, caused by different mechanisms of disease, make this condition difficult to address. Indeed, patients with different types of asthma may react differently to new or existing treatments. U-BIOPRED wants to create and validate innovative testing methods to classify patients into distinct severe asthma types. Using a sophisticated systems biology approach, researchers will bring together genetic data from patients, results from tissue samples, blood tests and breathing tests, as well as clinical findings and patient-reported symptoms. This will be linked to results of preclinical models, in order to facilitate future drug development.

Not all patients are at equal risk for vascular diabetic complications, so to better predict, monitor and treat those patients at risk, scientists of the SUMMIT project will identify biomarkers that indicate in advance if a patient is likely to develop vascular complications. The SUMMIT team will identify genetic factors that make some patients more susceptible to vascular diabetic complications than others and will develop novel computer simulations and models for laboratory tests, so they can better predict the outcome and development of complications in human populations.

IMI is contributing insightfully and comprehensively across a range of areas, towards making personalised medicine a reality. The route is littered with hurdles – among them, disparate clinical trial regimes, scientific complexities, ethical concerns and market incentives – and the challenges cannot be underestimated. Nevertheless IMI’s concerted drive to deliver better, more targeted healthcare is a step in the right direction along a path that ultimately may lead to tailor-made solutions for different patient populations and therefore greater benefits for individual patients.

Magda Chlebus

Magda Chlebus, Executive Director of Science Policy & Regulatory Affairs at the European Federation of Pharmaceutical...
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