Is precision medicine the route to a healthy world?

By EAPM Executive Director Denis Horgan; Gordon McVie, European Institute of Oncology; Angelo Paradsio, National Cancer Centre ‘Giovanni Paolo II’, Bari, Italy; Angela Brand, Institute for Public Health Genomics (IPHG), Maastricht University; Mark Lawler, Queens University Belfast

In response to a recent letter in The Lancet, Is precision medicine the route to a healthy world?, stakeholders of the Brussels-based European Alliance for Personalised Medicine (EAPM) feel duty- bound to state their case.

The authors of the original letter argue: “Although precision medicine will almost certainly be used in niche applications, if widely implemented, it could be a distraction from low-cost and effective population-wide interventions and policies,” adding “we believe precision medicine is not the route to a healthy world and instead urge a renewed and increased focus on public health and prevention.”

Personalised medicine is all about “public health and prevention”, with an added emphasis on patient empowerment. The latter involves an assessment of lifestyle – as personalised medicine does not only rely upon genetics, but also on information about, as the authors put it, “physical activity and dietary habits”.

It does not necessarily mean the genetic profiling of millions of people – with all the attendant costs - and it most certainly does not aim to shift emphasis from the application of drugs and treatments that work for the majority. It seeks, instead, to develop better outcomes for the minorities (becoming larger all the time) for whom a blanket treatment does not work, and on whom substantial sums of money are therefore being wasted by adherence to a one-size-fits-all methodology.

Spending vast amounts of money on treatments that are inappropriate and ineffective for many people in subgroups, due to a lack of understanding of their conditions – which could be facilitated by new laboratory tests that is becoming cheaper all the time - is counter-productive and will actually mean that money will be lost for other health priorities.

In the future, the emphasis in personalised medicine may in fact shift away from patients with clinical symptoms, and towards healthy citizens. This is about as preventative as medicine can get and will lead to long-term savings, better quality of life (less time spent in expensive hospitals and clinics) as well as more ongoing home-based treatment, using wearables, ‘smart’ pill boxes and more.

From an empowerment point of view, today’s patients are well-informed and also have high expectations regarding being treated for their ills. When it comes to choosing the correct course of treatment, in tandem with the patients, the knowledge that the latter are unlikely to respond to a one-size-fits-all treatment, and/or may suffer significant side effects, is clearly important.

Also, when targeted options are known to be available, the patient will be able to make a considered choice taking into account many lifestyle issues – including the aforementioned physical activity and dietary habits.

On top of this, the use of genetic information and so-called Big Data be able to flag up early signs of a disease as well as, in certain cases, a predisposition to, say, a particular cancer.

On the topic of inherited cancers which, the authors state, are in the minority, they concede that genetic information can give some answers to how patients will respond to a particular therapy. This is true but, crucially, the information can also offer insights into any secondary effects (which may enable doctors to selectively give preventative treatment – and more cash savings).

Of course, there are “complex ethical, legal, financial, and social issues,” in respect of genetic (and other medical) information and these are currently being debated at European Union level, but the fact is that this type of biomarker-based approach will have a huge impact on research into many diseases if a robust legal and ethical framework is brought into play.

But this framework must clearly balance the protection of the patients’ information against the need for permission to use the data for the benefit of society as a whole.

Meanwhile, the development of precision medicine will also permit the interpretation of several pathogenetic phenomena not otherwise interpretable, thus permitting us to activate approaches to old problems.

And while the costs of such genomic approaches are indeed a concern at the moment, an attempt should be made to develop modern approaches in a way strictly related to the true and urgent needs of the world – such as Europe’s ageing population of 500 million potential patients across 28, very different, Member Sates.

To summarise: Personalised medicine’s rapidly developing science-driven approach to healthcare has potentially very high benefits for patients, clinicians and healthcare systems alike. This involves better treatments on the one hand, and long-term cost savings on the other.

Advantages include the ability to make more informed medical decisions; a higher probability of desired outcomes thanks to better-targeted therapies; a reduced probability of adverse reactions to medicines; a focus on prevention and prediction of disease rather than reaction to it; earlier disease intervention than has been possible in the past; and improved healthcare cost-containment.

Collaboration and the breaking-down of silo walls must, of course, occur among universities, industry, healthcare providers and other actors but, while there is a long way to go the fact remains that, yes, precision medicine is “the route to a healthy world”.

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