ISSUE:

The burden of rare neuromuscular disease such as Duchenne Muscular dystrophy (DMD) are typically measured using self-reported outcomes such as the EQ-5D. We will debate to what extent wearable technologies can replace subjective endpoints.

Andrew Lloyd will introduce the session and outline how patient outcomes data can support the work of decision makers. Jamie Marshall (Principal Scientist, Solid BioSciences) will describe recent research exploring the use of novel wearable technologies which for assessing motor function in DMD. Michelle Campbell (Reviewer, US Food & Drug Administration) will provide a regulatory perspective on the role of different clinical outcomes in the assessment of rare paediatric conditions. Lastly, Valeria Ricotti (UCL & DiNAQOR AG) will describe the role of novel and traditional outcomes in understanding outcomes in conditions like DMD to understand the value of new treatments. The discussion will delve into the benefits and importance of different approaches for regulation and for HTA, and summarise issues to consider in trial design and interpretation.

OVERVIEW:

Challenges with measuring treatment outcomes in rare neuromuscular disease means that novel treatments may not reach patients because the available data don’t reflect their true value. Project HERCULES was partly established to address perceived limitations in outcome measurement by developing the bespoke DMD-QoL; a measure designed for regulators and HTA. However even disease specific measure may be improved upon substantially through the use of video capture and wearable technologies. But novel approaches may also lead to novel data problems.