We aim to understand and influence the process of “early access/compassionate use” schemes and to interact with sponsors and companies who are developing treatments.

We aim to understand the capacity of the network to deliver intrathecal treatment, to take steps to improve this, to provide training and good practice guidelines.

We aim to guide the process of HTA (Health Technology Assessment), which includes analysis of existing data and steering the current round of research concerning burden of care/cost/QoL/function.

We aim to develop a communication strategy to provide the HD community with a consistent message about access to effective treatments.


  1. Finding out which data currently exist relating to Burden of care/QoL/function/cost (from observational and intervention studies)
  2. Creating a registry of ongoing projects in relation to Burden of care/QoL/function/cost
  3. Establishing a network of experts and advisors from other fields.

WG documents


Lead Facilitator:
Associated EHDN Language Area Coordinator:
Tomas Bernard, EHDN Lanco, Czech Republic