Despite significant advances in cancer care, the disease remains a major burden for European citizens and patients and for our healthcare systems. Personalised oncology – giving the right treatment at the right dose to the right person at the right time – is contributing to these gains and providing renewed hope for patients, but more progress could be achieved if its full value were unleashed.

Access to Personalised Oncology in Europe, a new report by LSE consulting commissioned by EFPIA, identifies the barriers to better deployment of personalised cancer treatment for European cancer patients and makes concrete recommendations on how to advance and implement personalised oncology in Europe. Policymakers in Europe need to be more aspirational in improving equity in access to cancer care Europe-wide (as articulated in the recently launched European Code of Cancer Practice), as well as advancing and implementing personalised medicine approaches for all cancer patients in Europe. This will not only benefit patients, their care givers and physicians, but also strengthen the European research and innovation landscape and contribute societal benefits.

The report offers policy recommendations in three key areas: Stronger collaboration delivering transparent and well-informed, decision-making processes to advance and implement personalised oncology in Europe; investing in appropriate infrastructure that expedites the delivery of personalised oncology; and improvements in institutional structures to secure access to new personalized oncology medicines and biomarkers. 

Accelerating progress

The need for action is clear. The incidence of cancer across Europe has increased from around two million to three million cases since the end of the last decade and is projected to increase to four million over the next twenty years. Mortality is expected to hit two million by 2040, almost double that seen at the end of the twentieth century.

While these projections may be daunting, there is a strong case for optimism if we embrace innovative diagnostic, therapeutic and supportive care tools. Evidence of success is already building, but the benefits have not been felt across all cancer types nor are they equally available to all people in Europe. Equity must be a driving principle of our cancer health systems across Europe.

Advances in diagnosis, treatment and supportive care have led to improved outcomes for cancer patients. However, the latest innovations in personalised oncology can help drive further improvements. These tools are predicted to lead to better outcomes and reduced risk of adverse effects for the patient, while improving the sustainability and efficiency of healthcare systems. 

Policy recommendations

So, what’s holding us back?

The report calls for improved regulatory and reimbursement pathways for personalised oncology. The existing system for securing marketing approval is ill-suited to innovative treatments that warrant accelerated development approaches and require novel trial designs.

Improving post-marketing monitoring systems will reduce uncertainty. The use of real-world evidence – as a key component of adaptive pathways – and risk-sharing between payers and manufacturers can help provide faster and more equitable access to innovative medicines. In addition, greater coordination between medicines regulators and diagnostics-competent authorities would ensure that companion diagnostics become available in tandem with new therapies. Currently, we have the paradoxical situation that an innovative medicine may be approved but its companion diagnostic is not, nullifying the benefit of a personalised approach. This is not in the best interest of patients and must be addressed.

Alongside the regulatory pathway, the method by which value is determined, and the way pricing and reimbursement decisions are made, needs a step change. Again, collaboration between decision-makers is needed, while it is essential that HTA bodies expand their capacity to use and accept data (including real world evidence) generated via novel clinical trials designs.

The report highlights the need to move away from short-term assessments of value. Instead, overall economic value should be considered, including the indirect financial benefits to health budgets and society as a whole. Costs offset by ‘ruling out’ ineffective treatment options should also be taken into account.

Rethinking pricing

Pricing and reimbursement systems also need to adapt to personalised medicines as we move towards an era of medicines with multiple indications. The development of indication-based pricing (IBP) may be a step forward in this context. In this situation, price is permitted to vary according to indication and value – moving towards a situation where price is associated with each use of a medicine.

Support for bioinformatics and big data will also play a central role in how efficiently Europe can tap into the potential of personalised cancer care. ICT developments will allow the further use of real-world evidence, for example, via life-long real-time monitoring and data collection. This will be key to collecting and applying insights from patient-reported outcomes. EU funding could play a role in supporting progress in this field. Additionally, data sharing must be incentivised, moving away from the siloed approach to data, so that the benefits that big data and data analytics bring are made available for the benefit of patients.

Enhancing education in personalised medicine among clinicians and data scientists will be central to their future collaboration. Equally, improving health literacy among patients and the wider population is essential if patients are to be active participants in personalised oncology. The European Cancer Patient Coalition’s “Personalised Medicine: A Guide for Patients” is a good exemplar of a patient-focussed educational tool. The report also sets out a number of areas where personalised oncology can link with the European Beating Cancer Plan and the EU Cancer Mission. It calls on the European Parliament to push for a ‘new social health contract’ with patients.

Finally, as with many personalised medicines, diagnostics have a key role to play. Innovation in targeted therapies is ineffective without innovation in diagnostics. Availability of companion diagnostics must be the norm for personalised medicine delivery. Timely access to both diagnostics and innovative therapies is fundamental to the success of this personalised approach to cancer care across Europe.

Building momentum

The report is a substantive contribution to stakeholders’ conversations on personalised oncology. It comes as momentum gathers behind Europe’s Beating Cancer Action Plan and EU institutions step up efforts to better coordinate health policy. With the help of the policy recommendations outlined here, the Commission can ensure that the European Union and its Member States do not lag behind in medical progress and that advances in innovative science are integrated rapidly into health systems for the benefit of citizens, patients and society.

Given the burden of cancer in Europe and the potential of medical innovation to reduce this burden on patients and health systems, personalised oncology must be central to any vision of future health policies in Europe.