By Prof Denis Horgan, EAPM Executive Director
The European Society of Medical Oncology Congress conference in September was, as is now customary, a major event in the European scientific and medical calendar, as the attendance of 30,000 delegates testified.
The questions posed and answers provided across the range of cancer diagnosis and treatment highlighted the rapid evolution that continues in tackling this monumental challenge to health.
But what the proceedings also revealed was the continuing gap between potential and realisation.
The scope for improving health is limited not only by the intrinsic complexities of disease, but by the persistent lack of adequate frameworks for turning possibilities into practicalities.
The outcome is often that invaluable innovations are insufficiently developed or deployed, depriving patients of benefits that might in a better-organised world be available to them.
As has become a familiar mantra in healthcare, a therapy that never reaches the patient is of little value.
In many respects, healthcare suffers because the allocation of resources remains sub-optimal.
More effective integrated approaches to care are often neglected.
And the tendency within healthcare of operating in isolation rather than in collaboration still blunts the ability of science to influence the all-important policymaking sphere that dictates so much of the entire healthcare context.
This is why EAPM's contribution during ESMO was devoted largely to exploring how policy can play a fuller role alongside science in turning potential into personal care.
Over the course of three events, EAPM tackled the evident implementation gap and explored how resources might be better allocated and links more helpfully established.
These well- attended events – with more than 200 attendees – covered leading-edge issues, ranging from novel complementary biomarkers to accelerating the development and validation of liquid biopsy, and from tackling mantle cell lymphoma cancer and thyroid cancer to anticipating the future to accelerate change in healthcare management.
In each event, the discussion centred on the role of innovation and the impact of technology in bringing benefit to patients and citizens.
The underlying theme was the sharper definition that technology progressively brings, to the accuracy and precision of diagnosis, the development of treatment pathways and of patient monitoring.
In concrete terms, for example, the development of science has avoided unnecessary surgery for breast cancer patients.
But while the improved quality of technology has permitted breakthrough possibilities, it has not always been accompanied by parallel improvements in communication or understanding among all the multiple actors in the healthcare complex.
Closer and more organised collaboration at the level of politics, policy, patients, clinicians and technology developers will maximise the chances of innovation being adopted into health systems, and delivering on its potential.
There are clear and recent examples in Europe of imperfect coordination resulting in imperfect policy – as with the complications for healthcare that have arisen with legislation on data protection or in vitro diagnostics, where insufficient attention to the broader picture has led to adverse consequences that were not always unforeseen, but were insufficiently considered.
The same fate appears to hover over the upcoming review of the EU's pharmaceutical legislation, where the European Commission’s own quality audit team has emitted a negative evaluation of the proposal.
The risk from inadequate linking across the healthcare sector is that much of the courageous work on science and innovation that was presented at ESMO could, for all its merits, face a wait of 10-15 years to be widely adopted into healthcare systems.
Getting this right means achieving the right links between essential factors – by deploying science, technology and policy in combination.
The predictive capacity of digital technologies and advanced molecular diagnostics can not only prefigure the health status of target populations, but also assess their likely engagement with influential practices such as prevention or adherence to treatment.
Offering genuine personalised medicine through health technologies allows patients to express their own experience of illness and health as guides to the choice and development of care pathways.
Preventive measures over a life-long process, to tackle health issues in advance, are possibilities opening up to patients and policymakers. Participation by all actors – patients and doctors, of course, but also, with due regard for patients' privacy choices, with the wider healthcare world, feeding into and from the wealth of predictive and preventive data through the advanced communication features of health technologies.
And account must be taken of psycho-cognitive factors: health technologies, as well as medical treatment as a whole, should be designed and developed with a focus on the patients’ perspective, so as to allow them to act as protagonists of their own healthcare journey.
This interlocking approach of five key elements for effective personalised medicine, summarised in the acronym Per5 (five factors for personalised medicine), was a recurrent theme throughout the EAPM events.
A session on “Developing a Personalised Precision Medicine Strategy" focused on anticipating the future as a way of accelerating changes, recognising that despite the potential of personalised medicine for better outcomes, its integration into clinical practice is still limited, and in many countries faces obstacles – not just around the world, but even across EU member states.
A reflection process was launched at ESMO, placing the patients and stakeholders at its centre, between decision makers in public health on how countries can contribute to integrating PM into clinical practice and enhancing patients' access.
A session on "Tackling Rare Cancers" looked at the specific challenges in tackling mantle cell lymphoma cancer and thyroid cancer, and how to overcome the challenges of late or incorrect diagnosis, the paucity of tissue banks, the lack of access to appropriate therapies and expertise, the lack of commercial feasibility in developing new therapies and the difficulties in conducting well-powered clinical studies.
Policy makers can provide support both through governance, clinical standardisation, and awareness and education, and through provision for equitable reimbursement, infrastructure for conducting and validating tests, and testing access.
There were hopes – if not yet expectations – expressed in a session on liquid biopsies that the EU Beating Cancer Plan can make a significant contribution to advancing their development and use by bringing together the range of healthcare actors in closer and more effective coordination.
The potential is there, but needs more collaborative exploration, for screening for the early detection of multiple cancers, for tailoring treatment, such as guiding post-operative adjuvant systemic treatment in colorectal or breast cancer, and for selection of targeted treatment and monitoring progressive disease and treatment response.
Similarly, an EAPM event examined the prospects of creating an international index to assess the status of NGS implementation across different regions - another area of huge potential but dependent on collaboration at numerous levels to take real advantage of what it can deliver.
Many healthcare systems may need wide-ranging adaptations to facilitate the necessary innovation, investment and incentives.
The recommendation proposed is to combine the work of expert panels on issues as diverse as clinical organisation, genomics guidelines, data management or workforce skills and organisation to formulate a strategy involving decision makers, patient organisations, medical professionals, payers and regulators to integrate NGS and personalised medicine into clinical practice while enabling much greater access for patients.
The need for adaptation of healthcare systems was also conspicuous in the EAPM session on lung cancer screening.
Crucially, identifying the population at risk for lung cancer is not simply based on age and sex, as it is in the majority of breast or colon cancer screening, and Europe needs to involve all key groups in developing recommendations and guidelines for implementation, adapted according to the healthcare landscape of individual countries.
The need was identified for a coordinated plan, and a declaration was launched with the aim of gathering support for principles that could influence the decisions that will in due course emerge from discussions in the European Commission and the European Parliament on what action to take.
This declaration stresses that it is possible to reduce avoidable deaths by implementing effective lung cancer screening, and that the targeting of individuals at high risk of lung cancer with low-dose CT scans has a demonstrated positive benefit-risk ratio.
It highlights the need for a policy with clear metrics built on targets for early detection and treatment, with sharing of good practices to ensure continual improvement and maximised use of all available data sources to enhance programmes, and with support for continuing research into and development of screening technology and pathways, including new laboratory tests and biomarkers to refine risk assessment, risk stratification techniques, and the use of artificial intelligence in interpretation of results.
The declaration is open for signature, and follows up work that EAPM put on the political map as far back as 2016.
These events demonstrated clearly the role of EAPM as a bridge to policy makers, based on its expert input from the broadest range of healthcare actors.
Its engagement in ESMO was consistent with its permanent engagement with the medical, patient and regulatory community and its role in conveying well-informed perspectives to policy makers at the Member State and EU level.
As the scientific and technological advances accelerate, and the evolving political and regulatory context for healthcare in Europe continues to take shape, the work of EAPM continues and is itself gathering pace.