Value of Treatment (VoT) project

• Identify treatment gaps (or barriers to care) and causing factors along the care pathway and propose solutions to address them
• Assess health gains and socio-economic impacts resulting from best practice healthcare interventions, in comparison with current care or no treatment
• Converge evidence to policy

There are two VoT project rounds:

→ First round 2015-2017, Early intervention: bridging the early diagnosis and treatment gap
→ Second round 2018-2021, Bridging the gaps & achieving seamless coordinated care (VoT2)

ERN-RND is involved in the second round of the Value of Treatment project called VoT2 and launched on March 26th, May 23rd and September 24th 2018. VoT2 includes case studies on Rare Neurological Disorders (RNDs): Ataxia, Dystonia, Phenylketonuria. It will run between 2018 and 2020.

The study will look at value, early intervention and will explore the potential benefits and consequences of coordinated care through the examination of health services, patient outcomes and patterns of multidisciplinary care. Case studies are conducted in collaboration with experts and with the support of Academic Partners (University College London and University of Zagreb Medical School), applying empirical evidence from different European countries.

The final aim is to look at health gains and socio-economic impacts resulting from best practice healthcare interventions in comparison with current care or no treatment. The study on Ataxia together with Dystonia and Phenylketonuria will provide insight on the value of specialist centres in terms of diagnosis, management of patients with rare conditions and its cost implications in several European countries.

• Ataxia & PKU: University College London Hospitals (ERN-RND member), University of Cambridge, University College Dublin
• Dystonia: University of Zagreb Medical School

• VoT2 poster
• Article “Toward earlier diagnosis and treatment of rare neurological disorders: the value of coordinated care and specialist centers”