EAPM German Presidency Conference

Building a Decentralised, Data-Rich Biomarker Space to Speed Better Cancer Care
17-18 November 2020


Looking ahead to the next two decades, there will be a massive increase in cases of cancer in Europe.

Regulators, industry and healthcare professionals really need to step up to this new reality, and this also applies to citizens, who have a certain degree of responsibility for their own healthcare.

Lifestyle changes will be paramount, and could amount in many cases to the best form of prevention.

Stopping smoking, cutting down on alcohol, getting off the sofa and exercising, eating the right food – it all helps. Adhering to medicine regimes isn’t a bad plan either…

However, biomarkers have a job to do, too, in modern personalised medicine. We need more of these that are relevant to treatment.

Certainly, we’re churning out lots of data, but as more clinical trials and large scale epidemiological studies take place, new technologies such as blockchain will be urgently needed to handle the data.

And this has to be done without infringing regulations surrounding data protection (namely the General Data Protection Regulation (GDPR).

Unfortunately, the barriers in respect of data sharing mean that risks related to data security and privacy can have a paralysing effect on progress.

One of the goals of EAPM events is to engage politicians and lawmakers in the fast-growing field of personalised medicine, and deliver political asks through our consensus-based process, while also aiming to showcase developments and new ideas.

The sessions at the German Presidency conference include the following:

The framework for Health is an EU Framework of Cooperation

It’s all about the teamwork… We hear that again and again when bosses praise their staff, when football managers are happy after a good result, when someone picks up an Oscar for Best Director.

But is there really enough teamwork and collaboration happening?

The fact is, certainly in a healthcare sense, there is certainly too little cooperation to tackle the problems that beset our ageing society, which include under-resourced health services, an age spent getting new drugs to market, an increase in chronic diseases, et al.

It’s difficult to solve these issues as a cohesive unit, of course, as health is a national rather than EU competence under the Treaties.

But, still, there are solid arguments that what we need is more, not less, Europe – and for practical purposes that means less silo thinking and more cooperation, across borders and across disciplines.

Healthcare needs modernising and, while top-down legislation on clinical trials, IVDs and data protection and sharing has helped in recent times, arguably the EU should be doing more from a centralised point, at the very least in encouraging Member States to share more information on health from data banks, cooperate way-more effectively, work to avoid research duplication and so on, for the benefit of the citizenry.

Multi-stakeholder approach – privacy

The key question here is how can we create a system that allows for biomarker-data exchange that addresses patient privacy concerns?

Meanwhile, there is a need to ensure that sharing complies with regulatory requirements, and also the question of how we ensure that all of regulatory requirements of industry partners are met.

All of this must, of course, be done against the modern-day backdrop of increasing cyber threats.

This has to be a cross-industry, cross-country and cross-disciplinary process – basically, every stakeholder has to be involved, emerge from their respective silos, and make progress interoperable in human terms.

So how do we reach a target of agreement between all partners?

Trust and Policy Making – the need for Cooperation

We’re talking a lot about collaboration and cooperation at this conference. But there needs to be a framework in which it can happen to its highest potential. Not least in policy making.

Obviously, a step in the right direction is the European Commission’s proposal on Health Technology Assessment (HTA) cross Member States. This outlines plans for future cooperation among EU countries on determining the added value of new therapies but, unsurprisingly, ran into trouble over suggested mandatory aspects.

Do Member States trust the EU not to overstep its mandate? Will it even allow it to stretch it a little (or a lot)?

Several issues were highlighted on HTA, including differences in methodology and procedures across Member States, while it is noted that limitations to current systems – despite a decade of a certain level of cooperation – include low uptake of joint work and no sustainability of the current co-operative model.

However, on the plus-side, achievements thus far include the development of trust between HTA bodies, capacity building, the development of joint tools and earlier dialogues.

We are in desperate need of innovation and it is clear that progress has brought about a greater need for adaptation through appropriate frameworks that must be designed by experts, in consensus – albeit with plenty of necessary input from regulatory bodies.

Within an environment of trust.

The need for the EU to act for its Citizens and for the Economy

During a recent EU Presidency, a large emphasis was put on ‘the Economy of Wellbeing’.

The idea is not new, of course, and it certainly won’t go away. Nor should it.

This ongoing process aims to put people and their well being at the centre of policy- and decision-making, including, obviously in health.

Healthy citizens contribute to economic growth, rather than their taking more-and-more from ‘the pot’. Health means wealth, and statistics show it.

The EU is duty bound under the Treaties to employ effective and efficient policy measures and structures ensuring access to all to public services including health services and promotion of health and preventive measures.

The European Pillar of Social Rights provides a compass for renewed upward convergence towards better living conditions in Europe. Delivering on the Pillar is a shared political commitment and responsibility of both the European Union and the Member States, and requires continuous progress and in-depth action by both sides.

Where the EU cannot act as a body, it must nevertheless encourage and recommend.

Fortunately, a large number of Europeans live prosperous lives. The majority of people in the EU are wealthier, healthier and more educated than ever before. The life expectancy of new-borns is much longer than ever.

Yet there is still work to do. And, as argued elsewhere at this conference, in healthcare what is needed now is ‘more Europe, not less’.

Diagnosis and therapeutics

Early diagnostics, obviously, have a major part to play in healthcare and there has never been a better time to grasp the opportunities in, for example, cancer prevention using the latest discoveries in “omics” – including genomic science.

Due to these advances, our knowledge of common variants related to cancer risks has leaped from five to more than 450 and, genetically, we know a great deal more about what makes individuals susceptible.

As we know, personalised medicine utilises research, data and up-to-the-minute technology to provide better diagnostics and follow-up for citizens than is currently the case. It uses genetic information to discern whether a particular drug or regime will work for a particular patient and assists clinicians in deciding which treatment will be the most effective. It can also have a huge impact in a preventative sense.

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 healthcare systems.

But better and equitable access to these diagnostics is key.

There is also a major issue surrounding the on-going training of healthcare professionals (HCPs). Personalised medicine is now at the centre of most, if not all, aspects of patient care. It is not limited to rare diseases or cancer, but spans all medical specialties. This exciting new way of treating patients is, however, based on specific concepts or biological pathways in a field which is continuously moving.

This means that all HCPs in close contact with patients or their families need to possess a solid knowledge of the current aspects of personalised medicine and its latest breakthroughs, in order to better understand patients’ concerns.

These HCPs are being asked to move beyond traditional reactive medicine towards proactive healthcare management, employing screening, early treatment, and prevention, and to classify and treat diseases in a new way, interpreting information from across sources that blur the traditional boundaries of individual specialties.

Professionals will need to be confident of the science behind targeted therapies, including greater understanding of the immune system and molecular medicine, and knowledge of the mechanisms of action and interaction of targeted therapies, as well as common adverse events. This means ongoing education and training.

Member State sovereignty and shared competence in the delivery of healthcare

We are all aware that there is only so much the EU can do in respect of healthcare across the bloc in the context of the closely guarded Member State competence.

Yet it still has a huge part to play as it has shown with legislation on data protection, clinical trials, HTA, invitro diagnostics, cross-border healthcare and more.

Will umbrella action is needed at the highest EU levels, individual Member States (and the regions within them) are vital to the process of driving healthcare forward.  Many of these specialise in certain areas (breast cancer, lung cancer etc), including in vital research for the discovery, diagnosis and treatment of rare diseases.

In the field of modern medicine, innovation and its translation into healthcare systems is vital. They are essential components to sustainability, as is speedy delivery to market.

If these elements are not achieved, it will act as a disincentive to innovators, industry and researchers, for example, eventually leading to a lack of new developments across Europe.

Add to this inevitable loss of life, quality of life, demotivation of healthcare professionals and others, plus an impact on the economy, and it is clear that we cannot afford to fail.

What is required is better and bigger partnerships between Member States, organisations and regional set-ups, identifying needs and common values, as well as employing much more horizontal collaborations.

All EU Member States vary in size so even with enhanced cooperation and collaboration it is undeniably difficult, but a model should be developed that works at every population level and across the whole EU.

The role of cooperation between regions (eg Nordics, Central Eastern Europe, South Europe) to deliver healthcare

Healthcare systems need to encourage and harness innovation, and this means they need to cooperate as they all have national competence and will get nowhere fast if they fail to move forward together in the era of personalised medicine.

Innovation and the incentives for it are vital to health and wealth in the EU. It also encourages investment from outside of the EU, clearly good for business and jobs.

Countries acting in concert as a region can play a huge part in this process, as indeed can regions within a single Member State.

Personalised medicine has advanced hugely, and the reason for this is not least through developments in sciences in areas such as genetics, which have had a profound effect on the treatment of cancers, for instance.

One problem, however, is that Europe’s current healthcare systems are out of date and personalised medicine is struggling to be embedded.

Collaboration, innovation, deliverability, a solid evidence base and sustainability are all required.

While existing systems were certainly designed and developed to support innovation and access for patients to innovative medicines and treatment, these systems are falling short and need to be reassessed.

And they need to better collaborate at Member States and regional level. Agreed standards, interoperability, EU-wide programmes – none of this can happen without, internal, regional and EU-wide cooperation and joint action.

The Need for Evidence to Develop Healthcare Solutions –  Data, Data, Data

Data is everywhere among us and around us and, as part of this, Europe has the chance to create and harness a data-rich, decentralised, secure, and GDPR-compliant block chain system.

But, as ever, nothing is that simple, as all healthcare data will need to be evidence-based data as the questions of what constitutes ‘value’ refuse to go away.

Real-world evidence is necessary to generate comparative effectiveness studies using data from patients with, for example, common cancers, who are treated in different environments. This in order to enable the evaluation of care outcomes provided when specific biomarkers are used to direct patient care with targeted management.

One key challenge for Europe is the need for a number of translational scientists to ensure that clinical data, cancer biomarkers, blockchain technology, ISO standards, and data science can come together in one, cohesive project.

Another is to bring together huge amounts of biomarker data using the decentralised nature of blockchain technologies, its security and privacy characteristics.

But how?

Bring it now….the future is here

During this conference there will be much talk of ‘trust’, ‘collaboration’ and ‘cooperation’.

Big Data and how we use it is a case in point.

The potential availability of data from a large number of individuals increases the ability to investigate questions across a large number of diseases in different populations and also provides more information for understanding the results for clinical care in an individual.

Researchers would potentially be able to access millions of genetic markers and accelerate science towards better understanding of diseases and specific patients. Crucially, this would guide choice of therapy, prevention and screening programmes, increasing overall healthcare efficiency and patient outcomes.

It would be a perfect example of coordination, cooperation and teamwork. The fostering of meaningful, trusted and effective partnerships between Member States, the health stakeholder community and, of course, those at the absolute centre of healthcare – Europe’s patients – is necessary.

We cannot u-turn on personalised medicine processes, we cannot u-turn on genome sequencing, and we cannot u-turn on the mass of data now available.

Our only real recourse is to accept that what was once the future is here, right now, today.