The first day of the CDDF Multi-Stakeholder Workshop on Enabling smarter clinical trials to optimise patient care Deploying data to design and deliver better trials 17 – 18 November 2025, Hybrid Workshop – Brussels, BE set the stage for insightful discussions on the evolving landscape of oncology treatments. The event brought together experts from regulatory bodies, pharmaceutical companies, academia, and patient advocacy groups to explore the opportunities and challenges associated with innovative treatment modalities. Here are the key takeaways from the first three sessions of the day.
SESSION 1: Clinical trials and real world evidence: what really matters
Key challenges identified
- Need for proportionality and harmonisation, and more patient centricity Over-complex and uneven clinical trial implementation: trials remain administratively heavy and inconsistently proportionate across member states, limiting participation; successful national models shared by Juan Estevez show advances are possible but not yet harmonised.
- Data quality challenges: Variable quality and fitness-for-purpose of real-world data: Oncology RWD often lacks essential variables and consistent mapping, reducing confidence in their use for regulatory and HTA decisions.
- Cancer medicine shortages: fragmented evidence for shortages and critical medicines
The EU remains vulnerable due to external manufacturing dependence and a lack of integrated data, while China benefits from vertically integrated, strategically supported production. - Misalignment of expectations concerning cancer medicines across stakeholders: regulators, HTA bodies, clinicians, industry and patients lack a shared standard on when RWE or pragmatic trials are sufficient, slowing adoption of more efficient, patient-centred evidence approaches.
Potential next steps
- Co-develop guidance on proportionate, patient-centred trial design: CDDF can convene a multi-stakeholder group to produce practical, oncology-focused guidance on risk-based, decentralised and pragmatic trial elements.
- Invest in oncology-ready RWE infrastructure and methods for data collection that is fit per purpose: efforts such as DARWIN EU and ONCONET should be expanded to improve and incentivise data completeness and run pilots where RWE is assessed alongside clinical trial evidence.
- Build an EU collaborative, competitiveness and resilience framework for critical medicines: Governments should invest in onshoring and diversifying manufacturing while creating an integrated early-warning system combining supply, regulatory and real-world utilisation data.
- Create a shared learning agenda on RWE acceptability and impact, including among patients: CDDF can lead iterative pilots and workshops to define clear criteria for RWE use and produce consensus principles on what constitutes decision-ready evidence.
SESSION 2: Use of pragmatic elements in clinical trials
- From a patient perspective, Gilliosa emphasized that treatments and trials should be pragmatic from all perspectives.
- ‘Don’t punish patients if they under-report side effects because they want to stay on the trials”
- From a trial design perspective, Shaun recommends to always start from the scientific question/ intention and what the trial aims to achieve.
- Streamlined safety data collection (ICH E19) should be considered based on the knowledge and trial population. Registry based trials can be used to incpor[orate pragmatic elements
- From a regulatory perspective there is interest to explore use of pragmatic trials
- From an EFPIA perspective, industry are interested to build confidence in the concept of pragmatic trials through case case studies and co-designing trials with all involved stakeholders, including patients and regulators