Rare Disease Day and the need for personalised medicine

By European Alliance for Personalised Medicine (EAPM) Executive Director Denis Horgan

28 February is Rare Disease Day, which throws into sharp relief the fast-developing concept of personalised medicine, with its goal of bringing the right treatment to the right patient at the right time, as well as attendant issues such as access for patients to the best possible care and the question of how Europe should run its clinical trials for the benefit of relatively small groups.

The European Commission has stated: “Rare diseases are a significant health problem in the EU. Up to 36 million EU citizens live with a rare disease. However, the number of patients affected by each particular rare disease is, by definition, limited. There are diseases which may affect only very few patients, in particular in smaller member states. This, together with the fragmentation of knowledge across the EU, makes rare diseases a primary example of where working at European level is both necessary and highly beneficial.”

The European Alliance for Personalised Medicine – EAPM – has always advocated much stronger cross-border collaboration in all sectors of the health-care arena and, in the run-up to Rare Disease Day, it will be focusing on how to bring the dream of truly individualised treatment closer to reality.

On the morning of 25 February, close to the Brussels seat of the European Parliament, EAPM will be running a workshop to discuss perceptions of the value of diagnostics.

Companion diagnostics are complex and unique even within the field of diagnostic tools, yet they are critical for the appropriate prescription of personalised therapies.

EAPM has worked with its membership to engage patients, payers, policymakers, academia, and industry to examine various approaches to value assessment. This workshop will showcase the different views – including those from an EAPM cross-stakeholder survey - with the objective of hosting a critical discussion. The emerging results and wider perceptions will have an undeniable impact on the future of companion diagnostic access and innovation in Europe.

Later that day, a second meeting will address some of the aspects of where personalised medicine is going in the EU, where it needs to be, and how it can get there.

There is a reason why the phrase “prevention is better than cure” is so well known - and personalised medicine goes a long way towards addressing this. EAPM believes that the case for prevention as treatment – as well as treatment as prevention – is overwhelming in a Europe struggling to deal with the demands that a population of 500 million is putting on health-care systems.

Earlier diagnostics and earlier treatment has many benefits, among them fiscal, because while cost is a major issue – and there are key questions about the cost-effectiveness of new and even existing treatments – better diagnostics will ease the burden on health care systems and lead to a healthier and, thus, wealthier, Europe.

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