The second day of the CDDF Annual Conference on Challenges, Advances, and Open Questions in Global Cancer Drug Development and Clinical Trial brought together a diverse group of stakeholders into open, constructive discussions in order to improve cancer drug development. Here are the key takeaways from Day 2’s program:
SESSION 3: OPTIMIZE EVIDENCE GENERATION AND PATIENT ACCESS WITH INNOVATIVE CANCER TRIALS (METHODOLOGY)
[Key Takeaways]
🔶ACT EU aims to establish EU as attractive for clinical research, clinical trial methodology is a priority action area for ACT EU.
🔶RCTs remain the gold standard, preferred by decision-makers, even for small populations.
🔶External control data / borrowed data can be used when carefully planned and where RCTs cannot be performed for ethical reasons or where in rare conditions there are very few patients.
🔶Include the patient voice early in drug development/trial design.
🔶HTAs need to see data on diverse patient populations like those treated in clinical practice. The absence of these data will give rise to many questions.
[Next Steps]
🔶Sponsors, and the wider research community, need to plan prospectively to ensure the availability of suitable patient data of good quality to enable construction of external (concurrent) control arms.
🔶More work is needed to enable high quality use of external data, for instance in the context of the European Health Data Space (EHDS), there is a need for all patient data to be carefully recorded and curated (EHDS).
🔶Include patient perspective more systematically in JSC/HTA process.
🔶Advance understanding of “pragmatic trials” and how to do better trials that represent the actual patient populations, and preferences.
SESSION 4: NAVIGATING PRECISION ONCOLOGY DEVELOPMENT IN VIEW OF NEW REGULATIONS
[Key Takeaways]
🔶The IVDR and the FDA LDT regulations are welcomed by all stakeholders and will ensure quality diagnostics are available for patients.
🔶Alignment of IVDR requirements and streamlining of approval process within Europe is crucial for clinical research , especially at early stage of development, to ensure EU remains attractive.
🔶Combined studies drug/IVD face extended approval timelines and complex regulatory hurdles, which are impactful also for academic research, potentially leading inequities in access to clinical trials across Europe.
🔶COMBINE was a swift reaction from regulators, which brings all stakeholders together, with the promise of addressing the current issues in a timely manner.
[Next Steps]
🔶Ensure education of all stakeholders to ensure a productive and collaborative discussions moving forward.
🔶Implementation of the solutions identified by COMBINE.
🔶Continued monitoring effectiveness of the implemented solutions and dialogue with all stakeholders.