Making the most of the personalised medicine revolution

By European Alliance for Personalised Medicine Executive Director Denis Horgan

Personalised (or precision) medicine is a fast-moving field that sees treatments and medicines tailored to a patient’s genes, as well as his or her environment and lifestyle.  In a nutshell it aims to give the right treatment to the right patient at the right time, and can also work in a preventative sense.  These cutting-edge sciences and ‘omics’ hit the news recently when the US’s President Obama launched the Precision Medicine Initiative earlier this year, sending dollar upon dollar in the direction of research, clinical trials and DNA sequencing.  Unfortunately, because we are only at the beginning of the personalised medicine revolution, it is less wide-spread than it could and should be. It is also currently less precise than it will be in the future. 

But the technology is marching on and seems unstoppable. For example, the human genome was first sequenced little more than a decade ago and took some time to process at great expense. Today, the process takes a few hours and is relatively inexpensive, at around 1,000 dollars a time.  When it comes to that serial killer cancer, a field in which personalised medicine has already had major effects, treatments that target cancerous mutations have become a reality. These involve examining proteins and receptors, as well as looking at how the tumour tissue has mutated. This allows healthcare professionals (HCPs) to find the best way of attacking the cancer.  Let’s say that a patient is diagnosed with lung cancer. The doctor will take a small sample of the cancer tumour and run the sequence of the genes found in the cells. In most cases only a small sample of genes will be used to look for mutations that are known to drive cancer, although sometimes more background knowledge is required, which requires examining more genes.

After the drivers/mutations are found and identified, drugs can be targeted to effectively zap only the mutated cells, rather than killing all swiftly reproducing cells – cancerous and non-cancerous - as happens in chemotherapy. In most cases this results in less side effects, faster recovery and a better sense of well-being in the patient.  Due to great leaps in science and the steady expansion of personalised medicine, researchers now know that cancers with the same driving mutation have plenty in common, so it’s no longer a case of putting lung, breast, liver etc into their own separate boxes. So-called basket clinical trials in the United States have already tested new treatments on patients carrying the same mutation but in different parts of the body. These trials have also included treatments for rare cancers.  Even more exciting are the recent breakthroughs in immuno-oncology which its proponents say will revolutionize cancer care. A new class of drug has arrived that is designed to encourage the body’s own immune system to attack malignant cells.  The World Health Organization has predicted that the number of people dying from cancer will rise from 8.2m in 2012 to 14.6m in 2035, yet these treatments are already coming up with the goods even in advanced melanoma and lung cancer.

There is currently a lot of optimism around and these drugs have been hailed in some quarters as the beginning of the end of cancer. And with nearly 300 clinical trials of immunotherapies happening right now in the US, there are clear indications that pharmaceutical companies have a huge amount of faith in immuno-oncology.  So with all of these new developments, what needs to be done to turn the potential of personalised medicine into reality in a European Union of 500 million people who will all be patients at some stage in their lives?  The importance of access to medicines and innovative treatments across the 28 member states is undergoing particular scrutiny at the moment and the Brussels-based European Alliance for Personalised Medicine (EAPM) believes that access for patients to medicines that efficiently treat illness is one of several important issues that must be addressed both at national and EU level.   Access involves availability – meaning that new medicines are developed or existing products are adapted - and accessibility, which means bringing the products to patients who need them.

It is also about affordability – ensuring that patients, healthcare providers and governments can afford the products; and lets not forget ensuring quality, so that medicinal products work as intended and are efficient and safe. The European Commission has created the Expert Group on Safe and Timely Access to Medicines for Patients (STAMP), which began its work in January 2015, while the Commissioner for Humanitarian Aid & Crisis Management outlined a strategy on EU cooperation on health technology assessment near the end of 2014. EAPM welcomes these initiatives, but maintains that they are not enough. Among the basic tenets of the EU are equality and access to the best healthcare for all, regardless of who or where they are. This is clearly not the case at the moment. Unfortunately, despite the existence of innovative new drugs, new technologies and developments in medical science, many citizens are not able to access them, often due to high costs.

Other issues include overly bureaucratic reimbursement procedures and a lack of implementation of the Cross-Border Health-Care Directive. EAPM believes that EU policymakers should ensure that regulatory decisions on the value of innovative therapies are based on what matters most to patients, and ensure that they get access to innovative treatments following a centralised cost-benefit analysis by the European Medicines Agency. The Alliance maintains that the Commission, in tandem with the European Parliament, needs to create a regulatory environment which allows early patient access to novel medicines and treatments.  Meanwhile, there is a need for greater collaboration between Member States in areas such as data sharing, a removal of the silo mentality between different medical disciplines, the solving of inter-operability issues and up-to-the-minute training for HCPs.  Finally, medicine should be all about the patient and this will entail better communication on the part of HCPs to truly allow each patient to share equally in decision-making about his or her own treatment.  It’s a long road, but at least the journey has begun.

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