Treatment Optimisation - Improving access to better cancer care for all
Across Europe, many in the cancer research community have voiced concerns about how cancer treatments are currently used. Too often, we see expensive new therapies introduced on the back of limited data, with approval based on surrogate markers like tumour shrinkage rather than survival or quality of life. This leaves healthcare providers and healthcare systems without clear guidance on how best to use these treatments, including the right dose, duration, combination, or which patients benefit most. This can lead to unnecessary side effects for patients, huge financial strains for healthcare systems, without clear added value in return.
Why academic research matters in treatment optimisation
At EORTC, we've spent decades running clinical trials focused on helping cancer patients get not just new treatments, but the right treatments, for use in the right way. These treatment optimisation studies – often run by non-profit researchers – explore practical questions like whether a lower dose is just as effective but causes fewer side effects. Many of these are ‘de-escalation studies’ designed to reduce the burden of treatment while maintaining outcomes. This kind of work doesn’t just help doctors make better decisions; it is a public health necessity. But running these trials in everyday clinical settings isn’t easy. Regulatory red tape and logistical hurdles often get in the way.
Working together to improve access
To move things forward, EORTC helped launch the Cancer Medicines Forum (CMF) within the European Medicines Agency. The goal: bring all the key players together – regulators, researchers, payers, and health technology assessors – to have an honest discussion about how to make treatment optimisation a routine part of drug development. As key questions are identified, EORTC and its partners design pilot studies to explore how treatments could be better used across real-world healthcare systems.
What’s happening at the EU level
The European Union is slowly catching on. The new pharmaceutical legislation (specifically Article 12) acknowledges treatment optimisation. But decisions about access still rest with each country, making implementation patchy and uncertain. At the same time, the new EU regulation on health technology assessment (HTA) is pushing for a stronger focus on whether new treatments really deliver better outcomes. That’s a step in the right direction. As joint clinical assessments become more common, it will become easier to pinpoint where we need more data – especially post-approval data – to guide real-world use.
What we need from policymakers
EORTC believes this work cannot succeed without long-term commitment from EU and national policymakers. We need frameworks that make it easier to run these trials in everyday settings, outside of pharmaceutical company pipelines. That means adapting the Clinical Trials Regulation to support independent, non-commercial research that puts patients first.
Professional societies and independent research groups like EORTC have a crucial role to play. By designing rigorous but practical studies that show how best to use cancer therapies, we ensure innovation doesn’t just reach a few but delivers real benefits for all.
Denis Lacombe,
Chief Executive Officer
EORTC
