Bringing Onco-Innovation to Europe’s Healthcare Systems: The Potential of Biomarker Testing, Real World Evidence, Tumour Agnostic Therapies to Empower Personalised Medicine

Rapid and continuing advances in biomarker testing are not being matched by uptake in health systems, and this is hampering both patient care and innovation. It also risks 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—notably BRCA1/2 testing in ovarian, breast, pancreatic and prostate cancers. Nevertheless, the implementation of genetic testing in clinical routine setting is still challenging. Development is impeded by country-related heterogeneity, data deficiencies, and lack of policy alignment on standards, approval—and the role of real-world evidence in the process—and reimbursement. The acute nature of the problem is compellingly illustrated by the particular challenges facing the development and use of tumour agnostic therapies, where the gaps in preparedness for taking advantage of this innovative approach to cancer therapy are sharply exposed. Europe should already have in place a guarantee of universal access to a minimum suite of biomarker tests and should be planning for an optimum testing scenario with a wider range of biomarker tests integrated into a more sophisticated health system articulated around personalised medicine. Improving healthcare and winning advantages for Europe’s industrial competitiveness and innovation require an appropriate policy framework—starting with an update to outdated recommendations. We show herein the main issues and proposals that emerged during the previous advisory boards organised by the European Alliance for Personalized Medicine which mainly focus on possible scenarios of harmonisation of both oncogenetic testing and management of cancer patients.

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Bringing onco-innovation to Europe’s healthcare systems: the unexploited potential of biomarker testing, real world evidence and the potential of Tumour Agnostics. The lesson from BRCA1/2 genetic testing

10.20944/preprints202008.0307.v1 ◽

2020 ◽

Author(s):

Denis Horgan ◽

Gennaro CILIBERTO ◽

Pierfranco Conte ◽

Giuseppe CURIGLIANO ◽

Luis Seijo ◽

...

Keyword(s):

Health Systems ◽

Real World ◽

Universal Access ◽

Personalised Medicine ◽

Policy Framework ◽

Real World Evidence ◽

Biomarker Testing ◽

Policy Alignment ◽

Prostate Cancers

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Bringing Greater Accuracy to Europe’s Healthcare Systems: The Unexploited Potential of Biomarker Testing in Oncology

Biomedicine Hub ◽

10.1159/000511209 ◽

2020 ◽

Vol 5 (3) ◽

pp. 1-42

Author(s):

Denis Horgan ◽

Gennaro Ciliberto ◽

Pierfranco Conte ◽

David Baldwin ◽

Luis Seijo ◽

...

Keyword(s):

Health System ◽

Health Systems ◽

Universal Access ◽

Personalised Medicine ◽

National Policy ◽

Healthcare Systems ◽

Policy Framework ◽

Colon Cancers ◽

Biomarker Testing ◽

The Many

Rapid and continuing advances in biomarker testing are not being matched by take-up in health systems, and this is hampering both patient care and innovation. It also risks costing health systems the opportunity to make their services more efficient and, over time, more economical. This paper sets out the potential of biomarker testing, the unfolding precision and range of possible diagnosis and prediction, and the many obstacles to adoption. It offers case studies of biomarker testing in breast, ovarian, prostate, lung, thyroid and colon cancers, and derives specific lessons as to the potential and actual use of each of them. It also draws lessons about how to improve access and alignment, and to remedy the data deficiencies that impede development. And it suggests solutions to outstanding issues – notably including funding and the tangled web of obtaining reimbursement or equivalent coverage that Europe’s fragmented health system implies. It urges a European evolution towards an initial minimum testing scenario, which would guarantee universal access to a suite of biomarker tests for the currently most common conditions, and, further into the future, to an optimum testing scenario in which a much wider range of biomarker tests would be introduced and become part of a more sophisticated health system articulated around personalised medicine. For exploiting genomics to the full, it argues the need for a new policy framework for Europe. Biomarker testing is not an issue that can be treated in isolation, since the purpose of testing is to improve health. Its use is therefore always closely linked to specific health challenges and needs to be viewed in the broader policy context in the EU and more widely. The paper is the result of extensive engagement with experts and decision makers to develop the framework, and consequently represents a wide consensus of views on how healthcare systems should respond from push and pull factors at local, national and cross-border and EU level. It contains strong views and clear recommendations springing from the convictions of patients, clinicians, academics, medicines authorities, HTA bodies, payers, the diagnostic, pharmaceutical and ICT industries, and national policy makers.

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Integration of Registries with EHRs to Accelerate Generation of Real-World Evidence for Clinical Practice and Learning Health Systems Research

The Journal of Bone and Joint Surgery (American) ◽

10.2106/jbjs.19.01464 ◽

2020 ◽

Vol 102 (19) ◽

pp. e110

Author(s):

Patricia D. Franklin ◽

Jon Lurie ◽

Tor D. Tosteson ◽

Anna N.A. Tosteson

Keyword(s):

Clinical Practice ◽

Health Systems ◽

Real World ◽

Health Systems Research ◽

Systems Research ◽

Real World Evidence

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Use of Real-World Data Sources for Canadian Drug Pricing and Reimbursement Decisions: Stakeholder Views and Lessons for Other Countries

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462319000291 ◽

2019 ◽

Vol 35 (03) ◽

pp. 181-188

Author(s):

Don Husereau ◽

Edward Nason ◽

Tarun Ahuja ◽

Enkeleida Nikaï ◽

Eva Tsakonas ◽

...

Keyword(s):

Health Systems ◽

Administrative Data ◽

Real World ◽

Round Table ◽

Real World Data ◽

Common Data Elements ◽

World Data ◽

Real World Evidence ◽

Pricing And Reimbursement ◽

Data Elements

AbstractBackgroundCanada has a long history of the use of clinical evidence to support healthcare decision making. Given improvements in data holdings and analytic capacity in Canada and stakeholder interest, the purpose of this study is to reflect on perceptions of the value of real-world evidence in pricing and reimbursement decisions, barriers to its optimal use in pricing and reimbursement, current initiatives that may lead to its increased use, and what role the pharmaceutical industry may play in this.Methods/ResultsTo capture stakeholder perceptions, ninety-one participants identified as key stakeholders were identified according to background roles and geography and invited to participate in four round table discussions conducted under Chatham House rule. Important themes emerging from these discussions included: (i) the need to understand what “real world” evidence means; (ii) barriers to using real world evidence from differences in access, governance, inter-operability, system structures, expertise, and quality across Canadian health systems; (iii) differing views on industry's role.ConclusionsThe use of real-world data in Canada to inform pricing and reimbursement decisions is far from routine but nascent and slowly increasing. Barriers, including interoperability concerns, may also apply to other federated health systems that need to focus on the networking of healthcare administrative data across provincial jurisdictional boundaries. There also appears to be a desire to see better use of pragmatic trials linked to these administrative data sets. Emerging initiatives are under way to use real world evidence more broadly, and include identification of common data elements and approaches to networking data.

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