Activating the Patient Voice to Drive Clinical Development Success

 •Clinical validation of novel therapies for orphan/rare diseases remains challenging, especially when fast-tracked or granted one of the many accelerated approval designations by the EMA or FDA. Limited resources and aggressive timelines can often result in the patient perspective being overlooked. 

•Developers of rare disease therapies can harness and activate the patient voice in a manner that strategically informs the design and evolution of clinical development plans from the outset. Accounting for the patient voice in protocol development makes the study more relevant to them, and will go a long way towards sustaining maintaining for the duration of the study, especially in rare disease, where transitions can occur quickly.  

•Involving rare disease patient advocacy groups (PAGs) can facilitate trial candidate
identification and recruitment, contributing to rare disease clinical trial implementation success. PAGs can be a great source of non-dilutive financing, enabling bootstrapped startups and established rare disease players alike access to funding critical to early stage programs.  

•Join members of the Cello Health team as we discuss evidence-based strategies for
incorporating the patient voice early, often and in real-time during the development of rare disease therapies. We’ll address questions around the unmet needs industry may be overlooking and present illustrative examples where pairing the right technology with patient-centric perspectives enabled those needs to be addressed.