The first day of the CDDF workshop on Rare Cancers brought together a diverse group of stakeholders into open, constructive discussions in order to improve access to innovative therapies for patients with rare cancers. Here are the key takeaways from Day 1’s program:
SESSION 1: CHALLENGES, COLLABORATION AND NEEDS
[Key Takeaways]
🔶Regulators are open to considering the totality of data, including Real-World Data (RWD), when making approval decisions; especially when investigational medicinal products for rare cancers show high activity.
🔶Having incentives for drug development for rare diseases is very important; as is collaboration with patient advocacy groups.
🔶Data shows that treatment in Reference Centres is key to improving patient outcomes, especially for rare cancers. Collaboration to share data around the EU to build data cohorts is also vital.
[Next Steps]
🔶To overcome the challenges described in this session, improved collaboration and willingness for this to happen between industry, regulators, academia and patients will be key.
SESSION 2: INNOVATIVE TRIAL DESIGNS
[Key Takeaways]
🔶Drug development in children and in adolescent and young adult (AYA) patients (15-24 years) is much delayed versus adults. The ACCELERATE FAIR trial is a multistakeholder solution to enable early drug trials in children and AYAs. More collaboration between the pediatric and adult oncology is necessary.
🔶There are different aims for histology agnostic trials: learn, conclude, submit for regulators or implement in healthcare systems; the trial design should be related to the specific aims.
🔶There are many challenges for comparative effectiveness evaluation in rare cancers. Single arm studies form such challenges and maybe used too often. Randomizing, even in phase 2, generates more meaningful data, particularly when prospective Real World Data are collected simultaneously.
🔶While Overall Survival benefit is still what regulators/HTA are looking for, there may be other valuable endpoints for patients.