UCL Queen Square Institute of Neurology


Value of Specialist Centres for the treatment and care of patients with ataxia – Value of Treatment

14 July 2021

Professor Paola Giunti (Head of London Ataxia Centre, UCL Queen Square Institute of Neurology) presented the preliminary results of a project on ataxia to the European Brain Council (EBC), as one of three rare diseases included in the ‘Value of Treatment’ (VOT) project.

dr julie vallortigara and_prof paola giunti

The Value of Treatment (VOT) project aims to examine health gains and the socio-economic impact of optimized healthcare interventions in comparison with current care and converge data evidence to policy recommendations on how to improve the care pathway(s). Motivated to address the existing gaps in the care pathways, the EBC selected three case studies on Ataxia, Dystonia and Phenylketonuria (PKU). The objective of the case studies was to assess the effect of early coordinated care and treatment, provision of multidisciplinary care and health care providers specialist knowledge on outcomes to patients and on the healthcare costs.

The project broadly analyses adult patient diagnosis and care pathways and satisfaction in the Specialist Ataxia Centres compared with general non-specialist settings. Importantly, the project explores the health economic effects of both settings; this was carried out by Prof. Steve Morris (UCL and Cambridge University).  A survey was designed and disseminated in the UK in order to capture patient data on diagnosis, care pathways, symptom management, health resource use, patients’ experience and satisfaction. Two other European countries were selected for the ataxia case study: Germany and Italy. Dissemination of the survey was done in collaboration with the patient groups in the three countries: Ataxia UK, German patient groups DHAG and Friedreich Ataxie Förderverein e.V., and Italian patient group AISA.

EBC held a synthesis meeting on the VOT project on 8th June 2021, which was the opportunity to share results on the ataxia case study and hear the progress made on the other case studies.

Data from all countries will be analysed and published later this year. This will form the basis of policy recommendations regarding Specialist Ataxia Centres and more generally rare disease patient pathways in Europe. This work is highly relevant in informing policy changes for healthcare pathways for patients with rare neurological diseases with the creation of a White Paper. The final work will be presented to the European Parliament.

Professor Paola Giunti (Head of London Ataxia Centre, UCL Queen Square Institute of Neurology: “From the results of this study, it is very clear that patients with rare neurological diseases have a better management and treatment of their condition in the specialist centres compared to the care they had previously in non-specialist settings. Moreover, patients have valued the translational research aspect of these centres, being at the forefront of new diagnostic tools and management and treatment options. “

Dr Julie Vallortigara (Project Coordinator and Researcher, Ataxia Centre, UCL Queen Square Institute of Neurology): “I am thrilled to participate in this important work, with the hope that this will result in recommendations and a White Paper so patients can have equality of access to high quality treatment and care, particularly for this group of patients with rare diseases. We also hope to highlight the relevance of the patient’s involvement in research. I thank the pharmaceutical companies, the funders Reata and Takeda, who have seen the value of this work.”

Dr Julie Greenfield (Head of Research Ataxia UK): “The preliminary results illustrating the benefit of Specialist Ataxia Centres are encouraging and emphasise the importance of the involvement of patients in establishing the centres and the continued involvement that allows the Centres to always remain in keeping with the patients’ needs. This includes the exposure and possibility to participate actively in research.”

Professor Steve Morris (RAND Professor of Health Services Research, University of Cambridge): “Preliminary results showed the value of the specialist centres compared to non-specialist clinics for patients with rare diseases. I am excited to analyse the new data for Germany and Italy to complete the patient care map of treatments for rare neurological diseases in Europe and evaluate its costings.”