A recent panel debate moderated by Christian K. Schneider, head of Biopharma Excellence, explored emerging approaches to clinical research and data collection for therapies linked to rare diseases.
Key insights:
- The panel considered the potential for supplementary data sets – such as pharmacodynamic read-outs, histological evidence and historical controls - in enabling regulators to reach robust decisions.
- The panel also discussed on to the topic of reimbursement, which requires clarity about endpoints. Developers and regulators may be thinking about patient risk/benefit, while payers are more likely to be weighing up the cost/benefit.
- Establishing endpoints is critical when studying rare diseases for which there have previously been no real treatment options; and include the patient voice as early as possible.
The debate panel: