The European Union is recognizing the need to make better provision for health, and has started to put in place ambitious frameworks to remedy the deficiencies exposed by the COVID 19 pandemic and to create longer-term strategies for harnessing innovation in the quest for better care of its citizens. But the structures, for all their merits, are not enough, writes European Alliance for Personalised Medicine (EAPM) Executive Director Denis Horgan.
They remain in many cases mere scaffolding, and the hard work must still be undertaken of constructing the entire edifice, of fashioning an integrated ecosystem that takes full advantage of the opportunities that science and technology are every day presenting for better and more efficient care. What is needed is specific actionable agendas, concrete detailed targets and timelines, and clear links among all stakeholders. Otherwise, the fine and laudable ambitions will not be realised.
A video-conference in Brussels on March 8 brought together prominent actors from across the European health sector to identify where progress needed a determined push. As Denis Horgan, the executive director of EAPM, which organised the conference, put it: “There is an implementation gap in translating Europe’s grand plans into concrete actions. It is necessary to set up mechanisms and investment and instruments that can deliver on the obvious potential.”
In particular, given EAPM’s core interest of furthering precision medicine, he suggested that the realisation of that promise depended heavily on the necessary elements being in place across the health-care systems of Europe. Health-care professionals, decision makers, patient organisations, and European umbrella organizations representing interest groups and associations actively engaged in the field of personalised medicine took up the theme.
Europe’s admirable scaffolding was on full display. Recent demonstrations of wide support for EU initiatives such as its Beating Cancer Plan or its Cancer Mission, as well as numerous declarations made by the EU institutions both before and during the coronavirus crisis, suggest a growing recognition of the need to innovate – at the level of both policymakers and of the health community.
The renewed attention to disparities in health care and access across Europe is also driving new assessments of obstacles and new pursuits of solutions and promoting greater networking and collaboration among cancer institutions. A new spirit of readiness among European officials to embrace change and technology in a new decade is perceptible, and it is now up to policymakers to incentivise innovation, and investment as levers to improve European citizens’ prospects in a renaissance in thinking about healthcare in Europe
Ceri Thompson, Deputy Head of Unit DG CNECT H3 in the European Commission, responsible for eHealth, Well-being and Ageing, listed the ambitions in digital health, with the upcoming Data Governance Act, Digital Market Act, Implementing Act for the Open Data Directive, and the Data Act – each scheduled to bring new advantages to the health sector.
The European Health Data Space was scheduled to appear before the end of this year, and will be followed by a cascade of other initiatives, in cancer imaging, diagnostics and treatment, genomics, cybersecurity, and work on digital twins over the next five years, she added. She outlined the EU’s four-pillar strategy for data access and use, with cross-sectoral governance, an enabling infrastructure, adequate skills and competences, and the roll-out of common European data spaces.
Solange Peters, Chair of Medical Oncology at Lausanne University, spelled out how ESMO (of which she is currently President) is contributing to the EU’s Beating Cancer Plan, with its goal of marshalling technologies, research and innovation into cancer prevention, treatment and care along the entire disease pathway. It is a whole-of-government approach that focuses on the patient and maximises the potential of new technologies and insights; strengthens co-operation and opportunities for EU added value, eradicates inequalities in access to cancer knowledge, prevention, diagnosis and care, and delivers improved health outcomes to patients.
Christine Chomienne, Vice-Chair of the Mission Board Cancer at the European Commission and Professor of Cellular Biology at the Université de Paris, described the joint work of the Cancer Mission as “an unprecedented level of cooperation.”
Ciaran Nicholl, Head of the Health in Society Unit, Joint Research Council outlined the evolution of the Knowledge Centre on Cancer, scheduled for launch in mid-2021, as an example of working across the Commission services and with stakeholders to build an aligned approach to tackling cancer.
Overall, the EU is on the brink of a new era in cancer prevention and care, where patients have access to high-quality screening, treatments and the latest state of the art technologies, with support at EU level that allows scale and specialisation.
Daria Julkowska, Co-ordinator of the European Joint Programme on Rare Diseases, reported on progress with new approaches to data coordination, working with stakeholders towards the creation of a virtual platform that can offer federated, standardised GDPR- compliant, sustainable and quality assessed data.
Stephen Hall, European Regional Director for Precision Oncology at Novartis, expressed industry’s full support for the move towards developing personalised medicine and its greater selectivity of treatments to match individual patients’ responsiveness.
But – and it was a big but – there was also plenty of evidence of where the scaffolding remains disconcertingly bare, and opportunities are not being fully taken advantage of.
Former European health commissioner Vytenis Andriukaitis pointed to a continuing lack of appreciation among national governments of the merits of working together on health – typified, he said, by the spectacle of member states cutting spending on the Europe4Health programme in the teeth of a pandemic. He spoke of a long history of member states failing to coordinate, paying only lip-service to the concept – as the still-unresolved three-year stand-off in agreeing on joint health technology assessment demonstrated.
- Member states should co-ordinate and collaborate more
Ortwin Schulte, Health Attaché at the Permanent Representation of Germany to the EU, offered a ray of hope in respect of HTA, relating the recent progress on the EU plan for a more coordinated approach, and which might, he revealed, reach the stage of joint talks between Commission, Parliament and Council during March, after three years of discussion among member states. But he also observed how despite the impulse that COVID 19 had given to EU political coordination that could lead to a new level of integrated health policy, national reservations remained about the division of competences.
- Member states should seek resolution of their issues with greater EU engagement in health policy
Different Approaches to Personal Data Protection
Ceri Thompson’s vision of EU data strategy was still subject to diverse views on how to protect the personal data that should play such a part in its implementation. She acknowledged that disparate national implementation of the General Data Protection Regulation was a fact of life, and “different member states with their different health systems have their own approaches, and this is a task for them.”
- Member states should take into account the cross-border implications of their choices in implementing GDPR
Solange Peters of ESMO, for all her endorsement of the Beating Cancer Plan and the scope this could offer for advancing personalised medicine in cancer care, nonetheless listed the formidable bottlenecks that still stand in the way, in terms of patient education, sample acquisition, next generation sequencing assay, bioinformatics, reporting on clinical relevance, the need for tumour boards, and attaining the right match to available drugs. She also highlighted the persistent wide variability in access to biomarker testing across the EU member states, and called for progress on GDPR, rare cancers, ERNs, funding, and the HTA regulation.
Francesco Florindi, Strategy & Partnership Manager of BBMRI-ERIC, said data banks and repositories “have some elements that can help avoid reinventing the wheel”, and posed the question as to “How can we make it happen? How can we implement it?”
Rapid and continuing advances in biomarker testing are not being matched by uptake in health systems, hampering both patient care and innovation, and costing health systems the opportunity to make their services more efficient and, over time, more economical. The potential that genomics has brought to biomarker testing in diagnosis, prediction and research is being realised, pre-eminently in many cancers, but also in an ever-wider range of conditions.
But the implementation of genetic testing in routine clinical settings is still challenging. Development is impeded by country-related heterogeneity, data deficiencies, and lack of policy alignment on standards, approval and reimbursement. Europe should already have in place a guarantee of universal access to a minimum suite of biomarker tests and be planning for integrating a wider range of biomarker tests into a more sophisticated health system articulated around personalised medicine.
- Concerted action is needed to improve patient education, sample acquisition, next generation sequencing assay, the use of bioinformatics, the creation of tumour boards, and access to the right drug in personalised care.
- Member states should all provide a minimum suite of accessible biomarker tests.
- The potential of biobanks should be exploited in harmonisation, standardisation and information exchange
Ciaran Nicholl at the JRC admitted it was a persistent challenge for many stakeholders to work together: “We know what the needs are, but the question is whether we can work together to meet them.”
Christine Chomienne said success of the Cancer Mission would depend largely on ensuring effective links with all stakeholders – “and that takes time”, she said. “It is important to bring all member states on board – not just the ‘usual suspects'”.
Anna Middleton, Head of Society and Ethics Research in Cambridge, issued a call for action to engage patients and public. She reported on studies showing low awareness of data sharing and the potential of genomics testing, and urged a large and professional campaign of public communications to defuse distrust and even to enlist support for progress.
Ettore Capoluongo, Professor of Clinical Biochemistry and Clinical Molecular Biology at Federico II University in Naples, called for better processes for evidence generation, arguing that there were significant improvements in outcomes when targeted treatments are deployed in oncology. He saw a major role for the EU in catalysing a more harmonised and standardised approach across member states. “There is significant work to be done to create a favourable policy environment, particularly in heterogeneous countries like Italy,” he said. His recommendations included implementing the level of information relevant to personalised oncology in training of healthcare professionals, and strengthening the education and knowledge of scientific advances among practising clinicians.
At the same time, he said, all eligible patients should have access to fully reimbursed biomarker testing built into standardised patient pathways at diagnosis and during disease progression. The EUnetHTA model should incorporate a personalised oncology pathway specifically focused on new models for evidence generation. Patient associations., groups and clinicians should collaborate, giving health literacy a higher priority, so patients feel empowered as advocates for the integration of personalized oncology into their care. And there is a need to support countries with difficulties in implementing population genomics with adequate financing.
- Links between all stakeholders – including member states – should be boosted to develop a common sense of engagement in bettering Europe’s healthcare offerings for all its citizens.
- Education and information for general publics and healthcare professionals should be developed with appropriate professionalism.
- The EU should exert more influence on national provision of healthcare and relevant services.
- Reimbursement systems should provide for coverage of biomarker testing.
- Health literacy should be developed for patients and clinicians.
- Genomic testing should be supported.
Daria Julkowska, pleading for a common approach to removing barriers to innovation, recognised that coordination on rare disease “is difficult because of the intrinsic complexity of the issues and the wide range of stakeholders.”
Birute Tumiene, Lithuanian representative on the ERN Board of Member States Board of the European Reference Networks, told the meeting that rare diseases are still “often ignored”, and the ERNs conceived of as a remedy continue to suffer from insufficient funding and “vary varied participation” across Europe. “Is the European Commission capable of responding to such diverse challenges?”, she asked, suggesting that there is currently huge waste of resources in much cancer spending, and the right innovation could help. “Our ability to reap the benefits depends on member states and on the Commission,” she said.
Alastair Kent, former Director of Genetic Alliance UK, insisted that high quality information was needed to develop innovative therapy for rare disease, and at present there was still “huge unmet medical need and an urgent demand for innovation.” He said it was necessary to create an environment where research and development is not only possible, but actively encouraged. Data of itself, he added, is of no value until and unless it is transformed into knowledge – and that requires efficient and effective systems.
The discussion of rare diseases inevitably raised questions about the EU’s current reflections on updating its orphan drug rules, designed in 2000, as “necessary to stimulate the research, development and bringing to the market of appropriate medications by the pharmaceutical industry”. Any review of the operation of the regulation should take full account of imperfections that have been identified as inimical to its objectives, and in light also of the often dramatic changes in science and technology and that have opened up new dimensions to the understanding of disease and of therapy.
Maciej Gajewski, Head of International Government Affairs & Policy at Alexion, urged a greater sense of realism in policy discussions of rare diseases. Close analysis of the gap in coverage of rare diseases reveals that 95% are currently without treatment, and that 90% of known diseases have a prevalence of less than one in 100,000. At the same time, 98% of patients suffer from one of most prevalent diseases. These figures should influence the planning of how to fill the gaps. “If 90% of rare diseases remain extremely rare, we may need new models for those that fall outside current attempts,” he said. It may need new incentives, new collaborations and new partnerships involving industry. He urged a check on “the level of commitment to tackling rare diseases”. Are all member states engaged, he queried, suggesting that inequality of access to treatments across Europe is in part a measure of commitment. “These factors won’t be solved by any shift in regulation – we need a working ecosystem for rare diseases,” he said.
- Funding for ERNs should be increased to allow wider participation across member states.
- Expensive cancer treatments that are deployed in the absence of adequate testing should be avoided, to reduce waste of resources.
- Research and development need active encouragement.
- New models of support are needed for research and development into the rarest diseases.
Rigid Regulation and Regulation Refinement
Stephen Hall pointed to the contrasting priorities of the industry and of society on the development and deployment of biomarkers, and cautioned against excessively rigid interpretation of the upcoming new EU legislation on diagnostics. “We will have to work with policy makers to modify it to obtain more flexibility,” he said.
Benjamin Horbach of Roche, responsible for Personalised Healthcare and Health Systems Strategy, highlighted the need of how to support governments and public policy in more effective assessment of the value and benefits of personalised health care, with investments in data and health infrastructure, with adequate legal frameworks and standards, and relevant funding options.
- Different perspectives of the private and public sector should be recognized .
- Regulation should take account of the on-the-ground realities and resources of innovative product development.
- More sophisticated methods are needed to assess the value of innovative treatment approaches.
Speaker after speaker identified bottlenecks and unresolved issues that still demanded a stronger sense of coordination, and more senior-level policy support. But Daria Julkowska struck an upbeat tone that resonated with many of the panellists. She said that with so much change going on, it is: “A great moment to be working on these challenges, and working together.”
The constructive change that the health care context requires will not emerge merely by accident. Change will result only from vigorous debate among all stakeholders, and agreement on recommendations of a technical and political nature that will result in a better deal for patients and a more sustainable approach to healthcare. Horgan summed it up in his conclusions to the meeting: “There is an implementation gap that must be filled,” he said. “To translate the grand plans into concrete actions, support will be needed in terms of systems and investment and instruments, and a greater sense of engagement by all member states. Successful development and deployment of healthcare innovation depends on a policy framework in which countries would find it easier to reach consistent decisions and to provide clearer funding arrangements, thus boosting access and continued development.”