Taking STEPs towards patient-centric treatment

By Tony Mallett

Personalised medicine (PM) starts with you and me. It’s all about empowering the patient and giving the right treatment to the right one at the right time. Sound simple? Well, it isn’t, for a variety of reasons, but the concept is already starting to revolutionise medicine and the way treatment is delivered.


First a bit of background: In practice, rather than having a unique treatment for each individual, patients are sub-divided into groups based on their molecular make up, by using biomarkers. These are characteristics that can be used as indicators to measure, for example, pharmacological responses to a particular treatment.

So, while you and I may have the same disease per se, our molecular make-ups may mean that one of us responds to a certain treatment, while the same treatment won’t work for the other.

By such stratification it becomes possible to create a medical model using molecular profiling to tailor the right therapeutic strategy for the right person at the right time. It can also flag up a predisposition to a disease as well as allowing for timely prevention. All good stuff.

Championing this revolution is the European Alliance for Personalised Medicine (EAPM), which brings together healthcare experts, legislators and patient advocates involved with major chronic diseases. The aim is to improve patient care by accelerating the development, delivery and uptake of PM and diagnostics.

Although only around for two years, EAPM has already gathered support from cross-party MEPs and many key figures in the health arena, including the former European Commissioner for Health, David Byrne.

The mix of its members provides extensive scientific, clinical, caring and training expertise in PM and diagnostics, across patient groups, academia, health professionals and industry. Relevant departments of the Commission have observer status, as does the European Medicines Agency, so it really is in the vanguard of this rapidly developing science-driven approach to health care.

PM clearly has potentially very high benefits for patients, clinicians and healthcare systems alike. And this has already been recognized by the Commission, which stated: "With the emergence of new technologies…personalised medicine is now on the horizon. In the long term, doctors may be able to use genetic information to determine the right medicines, at the right dose and time. This field is already affecting companies’ business strategies, the design of clinical trials and the way medicines are prescribed."

And those are not just words, as PM is being taken into account in EU legislative initiatives, including the rules on medical devices and clinical trials, and the new pharmacovigilance regime.

To help push the agenda further, EAPM is this week (19 February) launching its STEPs campaign in the Brussels seat of the European Parliament. STEPs stands for Specialised Treatment for Europe’s Patients and aims to highlight, to current and potential MEPs, the possibilities surrounding PM and the advantages for their constituents (that’s you and me), in the run-up to this year’s European elections. You can read about it here.

Essentially, it outlines five STEPs towards a healthier Europe by aiming to secure patients’ quality of life through PM. The goals are to ensure a regulatory environment which allows early patient access to novel and efficacious PM; increase research and development for PM, while recognising its value; improve the education and training of health care professionals; support new approaches to reimbursement and HTA assessment, required for patient access to PM, and; increase awareness and understanding of PM.

Those involved in the field are convinced that achieving these goals will improve the quality of life for patients across Europe.

But let’s be clear – there are real challenges to be faced and hurdles to overcome. Cost, as ever, is a massive issue. As is patient access to clinical trials (or the current lack thereof). Education of patients and clinicians is another challenge, as is the debate over the use of data. Collaboration between stakeholders is yet one more…the list goes on.

At least when it comes to education, the experts are clear. "It is impossible to empower patients unless they can understand the information being given to them, It needs to be simple and effective," Ian Banks, Chair of the ECCO Patient Working Group, told EU Reporter. He was backed by the chairman of the EAPM Working Group for Research, Professor Ulrik Ringborg, who agreed that transmitting information was key to patients’ involvement in their own treatment.

But it seems that Europe’s health policies must change too, with sometimes-unwilling stakeholders required to get their act together, and quickly.

"While there might not be a Tea Party in Europe, we certainly feel the need for change,’ says Professor Louis Denis, director of the Oncology Centre Antwerp, adding that: "Our system of health policies must change, but a number of stakeholders don’t feel like changing."

Professor Denis also called for greater and better European collaboration in basic research and was supported by the director of the European Organisation for Research and Treatment of Cancer (EORTC), Denis Lacombe, who told this website: “All stakeholders should leave their comfort zone. We are heading for new forms of clinical research for personalised medicine and all of us – that’s pharma, academia, payers, regulators – need to move forward to a new form of collaboration."

Lacombe added: "Patients are waiting for therapeutic improvement and asking us - while we have good technologies are we really bringing them the best new drugs? And if we look hard in the mirror the fact is that we are not using technology optimally. There needs to be more collaboration, new models…and that means we have to think outside the box."

Taking up the theme, Professor Per-Anders Abrahamsson of the European Association of Urology (EAU) said: ‘The medical profession needs to transform what is going on in the laboratories, all the way to the doctors and then, in the end, to our patients.

"We have to work together with other specialists in order to make a difference. We need to think, study, research, discover, evaluate, teach, learn and approve. That is our vision for the future – a future that is already here."

One of his EAU colleagues, Didier Jacqmin, is a professor of urology based in the European Parliament-city of Strasbourg, France. He acknowledges that two major challenges with PM involve getting patients to clinical trials and the cost of producing drugs that work for sub-groups. Once genetic profiles have been selected the trials that are then held are by definition smaller. Getting patients involved is already an issue with bigger trials.

"There is a fear of trials among some patients," said Didier, "and also a lack of awareness that they are taking place. We need to get patients more involved, better informed, and advertise these trials to the public. Even a lot of GPs don’t know that trials are ongoing."

On that topic, Mary Baker, MBE, who is president of the European Brain Council, highlights a further issue: ‘Patients often don’t want to hand over sensitive personal information that is vital for research. We need to be able to communicate with patients to explain the benefits. There needs to be a debate in society and that is currently lacking.

"Nevertheless," she added, "there is tremendous hope hanging on personalised medicine."

The next issue is getting a drug that works for a sub-group onto the market. Said Jacqmin: "It’s difficult for companies because it’s costly in terms of R and D. One way to soften the blow is to give the pharma companies longer exclusivity with a new, sub-group-targeted product to allow them to get their money back."

And Baker added: "Development costs are rising. Effort is high for what is by definition a smaller market. Basically, when it comes to PM, we need to find new ways to make it work."

But it’s not just a new structure to get the drugs to market that’s required. How ready are individual health care systems for what could be a PM explosion?

"Overall there is a lack of forethought and long-term vision, certainly in some member states. The NHS, for example, is great at fire drills but no good at DNA drills," Baker insisted. "Science must be underpinned by infrastructure, communication, knowledge and socio-economics."

Dagmar Roth-Berhendt agreed that member states have work to do. She has been an MEP since 1989 and, among other duties and interests, has been a leading light on the institution’s Committee on the Environment, Public Health and Food Safety, known as ENVI.

She has very recently been involved in the debate over in vitro diagnostic medical devices – an important piece of legislation in the field of PM.

"Personalized medicine is certainly a great hope for many people and in the efforts to find cures for diseases in the future," she said. "But I’d like to see a general approach across member states to make things comparable.

"And the longer I look, the less confident I am that the patient is actually at the centre where he or she should be. That certainly needs to change."

These are major issues but there’s another large one that many in the frontline are tackling head on: PM also involves working hard on interacting with patients, educating them and, crucially, listening to them.

Said Jacqmin: "It‘s not only about understanding and using the science. PM is also about adapting to the patient in front of you. He or she may have several options - such as surveillance, surgery, radiotherapy etcetera - and we have to take into account the patient’s choices.

"We are not in the patient’s place and do not know his or her lifestyle, family circumstances and so on. It’s important to listen and we always send them home with plenty of advice and written information that will help them make the right choice for their unique circumstances.’

"The fact is," Jacqmin insisted "that a well-informed patient is happier and has a better quality of life." Which is pretty much where we came in.

So, it seems as though PM still has a long way to go. But if solutions can be found – and they will be – this revolutionary approach will have a massive impact on how patients are treated (in all senses of the word) into the near future and beyond. And that can only lead to a healthier Europe.

Tony Mallett is a Brussels-based freelance journalist. [email protected]

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