The convergence of cutting-edge technologies and breakthroughs in the scientific understanding of cancer have enabled unprecedented opportunities for Precision Oncology (PO). At the core of PO is the ability to identify optimal treatments tailored to each patient’s unique biological characteristics, breaking away from conventional one-size-fits-all approaches centered around tumor location.

This revolutionary approach brings significant advantages for both individual patients and healthcare systems. Patients experience improved response rates, elevated survival rates, and enhanced long-term outcomes, minimizing unnecessary side effects and complications. For healthcare systems, PO offers an avenue to reduce costs and alleviate the strain on an already burdened healthcare workforce.

Recognizing the progress in cancer care through initiatives like Europe’s Beating Cancer Plan (EBCP) and the EU Cancer Mission, urgent alignment among stakeholders is needed for a holistic vision to ensure equitable, sustainable, and innovative PO application across Europe. Failure to do so may result in patients left behind in the rapidly advancing treatment landscape, and Europe struggling to keep pace with life science innovation. This becomes particularly critical as we approach the European Parliament elections in 2024, the conclusion of the EBCP Implementation Roadmap in 2025, and engage in discussions about the next Multiannual Financial Framework.

In response, the EFPIA Oncology Platform presents a vision for PO in Europe beyond 2025 built on four pillars: (1) access; (2) health systems sustainability; (3) research and innovation, and (4) data.

Access

Access to PO, including biomarker testing and medicines, varies across Europe. Several challenges persist which contribute to unequal access to PO between and within countries, and addressing these challenges requires a comprehensive approach.

Firstly, one of the primary challenges is the absence of simultaneous regulatory and reimbursement approval for PO and its associated biomarker test. PO medicines and biomarker testing work in tandem. Yet, in numerous countries, significant delays exist between the approval of PO medicines and corresponding biomarker tests, including their listing on reimbursement rosters. Furthermore, different reimbursement levels for biomarker testing across the EU countries lead to further complexities in access to PO.

Secondly, there is a need to adopt flexible regulatory frameworks such as conditional approvals or accelerated access programs, which allow for facilitated approval of life-saving medicines, while guaranteeing that comprehensive data for safety, efficacy, and quality is generated post-approval.

Likewise, reimbursement frameworks encounter challenges with PO due to traditional models not designed for it. The rapid advancements and continuous discoveries in PO demand an adaptive approach which allows for uncertainty to be managed while enabling conditional reimbursement linked to additional evidence collection, both within the EU General Pharmaceutical Legislation and in national legislation.

Finally, there is an imperative need for a comprehensive and holistic value framework for PO therapeutics in health technology assessments (HTA). This will help to unlock the full value of PO, extending beyond initial cost considerations and recognizing value and the significance of Oncology Relevant Endpoints (OREs), contributing to a patient-centric evaluation and a thorough understanding of PO’s benefits.

Healthcare system sustainability 

The revolutionary shift from a conventional one-size-fits-all approach to personalized strategies in cancer treatment has amplified the critical role of PO in bolstering sustainable healthcare systems. PO has proven to reduce unnecessary hospitalizations and the risks associated with ineffective / over-treatment, thus optimizing resource utilization, and cutting costs within the healthcare ecosystem. Nonetheless, that is not possible if it is not underpinned by strong infrastructure.

Currently, realizing the full potential of PO is impeded by significant disparities in the necessary infrastructure in Europe including variations in diagnostic capacities and capabilities, accreditation, and healthcare professional trainings.   

These disparities lead to differences in patient access to PO tests and treatments, with patients in Eastern and Southern Europe waiting 500-800 days more than their counterparts in Northern and Western Europe for the same tests and treatments [1]. Additionally, disparities in patient literacy further contribute to the variation in access to PO.

To address these challenges and facilitate improved infrastructure for PO across the EU, appropriate financial support is needed. To that end, the next Multiannual Financial Framework must allocate appropriate financial resources, such as the European Regional Development Fund, to ensure the availability of strong PO infrastructure in all Member States.

Research and innovation

In the ever-evolving landscape of PO, the engine driving progress is continuous research, development, and innovation.

Within Europe’s R&D landscape, challenges arise with the EU’s In Vitro Diagnostic Medical Devices Regulation (IVDR), significantly impacting patients’ access to oncology clinical trials. This not only hinders treatment development but also restricts access for patients who have exhausted all available treatment options. Beyond these challenges, a broader concern looms—the potential decline in Europe’s attractiveness for PO clinical trials. It is therefore essential that going forward a harmonized PO trial regulations are put in place to ensure transparency and predictability.

Moreover, fostering interdisciplinary collaboration and public-private partnerships in PO to unite various research disciplines, including oncologists, geneticists, pathologists, and data scientists, is crucial to integrate diverse expertise, enhancing efficiency, accelerating innovation, and ensuring the direct translate of PO advancements into improved patient outcomes. Thus, as we look beyond the post-2025 priorities of EBCP and into Horizon Europe in 2027, policymakers must commit both resources and a clear vision to ensuring timely patient access to cutting-edge treatments while fortifying Europe’s global competitiveness in shaping the future of PO.

Data

The success of PO is linked with access to individual data as it enables the identification of the most appropriate treatment for patients, and thus allows for a truly personalized approach.

The use of personal data in PO involves assessing great amounts of genomic profiles and biomarkers, often coming from different sources. The diverse origins of such data pose challenges related to compatibility, interoperability, and privacy. Recognizing that several initiatives are already in place, it is crucial that policies enable collaborative data-sharing through data interoperability, privacy-centric frameworks, and common standards that enable seamless cross-border data exchange.

Moreover, the establishment of real-world evidence (RWE) registries is crucial for gathering extensive data and deriving meaningful RWE endpoints that enhance the understanding of treatment effectiveness in real-world settings. This, in turn, informs clinical decision-making and contributes to a comprehensive HTA of the impacts of PO. Leveraging EU initiatives like EP PerMed could play a pivotal role in this process and could contribute to the development of RWE registries.

Call to Action

Envisioning a future where the potential of PO is not just theoretical but tangibly transformative necessitates aligning policy priorities and investments with the evolving needs of patients and the healthcare ecosystem. The EFPIA OP provides its vision to build a common European path towards a future where every individual grappling with cancer can benefit from the transformative power of PO. Through concerted efforts, strategic collaborations, and an unwavering commitment to innovation, we can build a Europe where PO is not merely a treatment option but a symbol of hope for all those affected by cancer.

Our proposal aims to maintain an attractive and innovation-friendly environment for research, development, and production of medicines in Europe. PO represents the cutting edge of scientific progress, and it is imperative that EU legislation keeps pace with the rapid advancements in this field. Let us embark on this journey of transformation and solidarity to ensure that no one is left behind in the fight against cancer.

[1] EFPIA, The root cause of unavailability and delay to innovative medicines, available here: https://www.efpia.eu/media/636822/root-cause-unavailability-delays-cra-report-april-2022-final.pdf, pp.6