The ‘Value of Treatment’ (VoT) project was a European Brain Council (EBC) project focused on evaluating coordinated care in three neurological diseases, one of these being ataxia. The EBC is a non-profit organisation which promotes brain research to improve the lives of Europeans with brain conditions. The ataxia study, funded by Takeda and Reata, commenced in 2018, was run by Prof Paola Giunti, lead of the London Ataxia Centre. The study analysed adult patient diagnosis and care pathways in Specialist Ataxia Centres (SACs) and non-specialist settings and explored the health economic effects of both settings by disseminating a survey that was designed with support from Costello Medical. A survey was distributed to people with ataxia in the UK, Germany and Italy, to gather information about diagnosis and management of their ataxias, healthcare resource use and costs per person in SACs or non-specialist settings. Dissemination of the survey was done via collaboration with patient groups in these countries, who are members of Euro-ataxia.
The project’s results highlight the value of coordinated care and specialist centres to address the challenges for people living with ataxia. You can find more details about the results below where summaries of the VoT project’s numerous publications are provided. Based on these results, the ataxia case study working group, together with the EBC, made several recommendations designed to increase access to, and raise awareness of, SACs, as well as improving education on management of the ataxias amongst primary care settings. The results of the project will be used to make policy recommendations which we hope will improve the healthcare of people with ataxia.
Summaries of publications from the VoT project:
1 – Patient pathways for rare diseases in Europe: ataxia as an example – PubMed
For the first part of the study, 552 people with a range of ataxias (or their parents/caregivers) in the UK (277), Germany (101) and Italy (174) shared their experience of the diagnosis and management of their ataxias in SACs or in non-specialist settings. Satisfaction was higher in the SAC group, with regards to understanding and managing their ataxia, delivering care adapted to needs, coordinating referrals to healthcare specialists, and offering opportunities to take part in research.
For those who had not been to an SAC, or stopped attending, there were two key barriers to attending in all three countries – difficulties travelling to a SAC and a lack of a re-referral system to a SAC.
In the UK, over half of the respondents said that healthcare professionals in primary care services neither had a good understanding of their ataxia nor had a good knowledge of treatments. Feedback was less negative for patients’ experiences with secondary healthcare practitioners (including neurologists) compared to primary care, in all three countries.
In the UK, 91% of the group attending a SAC gave positive feedback on the understanding of ataxia, and 85% gave positive feedback on healthcare practitioners’ knowledge about ataxia treatments.
Survey participants shared how they thought their treatment and care could be improved:
- Earlier specific diagnosis
- Improvement and sharing of information about the condition and available treatments
- Advice to feel in control of the condition, and practical advice on living with the condition
- Better access to therapies and better management of their symptoms
- Advice on adapting their home
- Advice on maintaining the level of healthcare when SAC visits are no longer possible
The results highlight the need to improve access to SACs, support SACs, and create new SACs as participants felt ataxia management was better overall in SACs compared to non-specialist secondary care settings.
The second part of the project compared average healthcare resource use and costs per person for those who attended SACs and those who did not in the previous 12 months. The experience of 320 people with ataxia within the same cohort (or their parents/caregivers) across the UK (181), Germany (43) and Italy (96) was collected with the same survey.
Healthcare costs per person were similar in both groups in all three countries. Importantly, although the costs were similar in both settings results highlighted in the paper above show that patients reported better healthcare in SAC compared with other settings. Within countries there was little variation in resource use for the SAC group compared to the non-SAC group, but there were variations between countries in some types of healthcare contact. The highest number of SAC visits occurred in Italy, then Germany and the UK. Physiotherapy visits were the most common type of healthcare contact in all three countries.
Following the project, the researchers set out key healthcare policy recommendations to support ataxia management and care:
-Provide whole genome sequencing to give a diagnosis sooner
-Increase the availability of multidisciplinary team clinics
-Improve rare disease training for healthcare professionals
-Ensure centres have a research focus as well as symptom management
-Implement telemedicine in existing SACs and increase the number of SACs
-Increase access to SACs by improving awareness of them and ensure referrals to SACs are part of national care pathways
This paper published the results of the second part of the VoT project, focusing on the UK only, using the same survey described in the publications above. In addition to focusing on the UK it describes more in-depth analysis of the data collected in the survey.
Participants who attended a SAC reported better management of their symptoms and more personalised care compared with participants who never attended a SAC.
Most participants had to wait to see a neurologist, with a delay from six months to over five years in referral after symptoms started – this delay is not acceptable. Only 29% of the survey participants attended a SAC. This low proportion, similar to the third of patients attending a SAC reported in a previous Ataxia UK survey, needs to be addressed.
Of those participants attending a SAC, 96.8% of those who attend or used to attend SACs gave positive feedback on the role of the SAC in understanding their condition, 86.6% gave positive feedback on the SAC’s role in coordinating referrals to other healthcare specialists and 85.2% gave positive feedback on the SAC’s role in offering opportunities to take part in research. 77% gave positive feedback on symptom management compared to 55% of those not attending a SAC. This significantly higher satisfaction with symptom management among those attending SACs was not seen for participants in Germany and Italy. This difference may be due to the unique involvement of Ataxia UK and UK patients in shaping the SAC services, including monitoring and accreditation of UK SACs by Ataxia UK.
Costs were not significantly different between those attending a SAC and those who did not.
There were some barriers for patients in accessing the SACs, and some gaps in the care provided, including long travel times to attend a SAC, lack of access to benefits and social workers, and financial difficulties.
Overall, the results showed significantly higher satisfaction with care in those who attended a SAC compared with those who did not, at similar costs, which further supports the recommendation to improve access to SACs.
This paper focuses on the survey results from Germany.
Specific areas where people provided positive feedback about SACs were in understanding their condition, giving practical advice to better manage their ataxia, helping people to cope with their condition, offering opportunities to take part in research, and providing medical advice on the management of ataxia symptoms.
In terms of unmet needs, people wanted to see improvement in information shared about their condition and potential treatment, wanting to feel more in control, receive better advice on how to cope, and gain better access to holistic treatments.
Barriers to accessing and staying under the care of a SAC included a lack of a systematic referral process and travel required to a SAC.
Interestingly, in Germany, the SAC focus is on research rather than the management of symptoms and care coordination which contrasts with the care model of SACs with MDT clinics which was first created in the UK. Among other recommendations, the paper recommends incorporating these focuses into German SACs as this has been highly valued by patients in the UK as demonstrated in the same survey. The results also suggest that SAC services that include patient feedback, which have been pioneered in the UK, could be implemented in Germany to further improve the management of people with ataxia. Higher patient satisfaction in SACs compared to non-SAC settings without a significant difference in costs again highlights the importance of access to SACs.
This paper focuses on the survey results from Italy, gathered between May and September 2021.
Notably, in Italy, the first referral rate to a SAC was 56.2% compared to in Germany (18%). In Germany and Italy, self-referral to a SAC was reported, a path which does not exist in the UK, where referral must be by a healthcare professional. It was felt that this may reflect a difference in the role of SACs in Italy and Germany compared with the UK.
The proportion of people accessing an MDT clinic is highest in Italy compared to the UK and Germany and people were generally satisfied with the care received in the MDT clinic.
49% of people who attended a non-SAC before attending a SAC reported that they received better care at the SAC which is proportionally less positive than in the other countries. When looking in more detail, however, people reported that the SACs provided a better service in most areas.
For those who stopped attending a SAC, the main barrier was travel, despite there being more SACs across Italy. The authors suggest a mix of telemedicine and in-person attendance may facilitate maintaining patients under SAC care.
In terms of resource use and costs, there was no difference in the total cost between non-SAC and SAC groups which leads the authors to suggest that cost of additional SAC visits was offset by savings from fewer non-specialist neurologist visits.
Overall, feedback was felt to be similar to that collected in Germany and the UK and led to numerous recommendations. These include developing better communication methods between SACs and primary and secondary care providers to improve monitoring and continuity of care and a multidisciplinary approach to deliver care with more impact on symptom management and quality of life. The study also identified the importance of coordination, continuity and flexibility of care, not solely geographical location of care.
6 – Frontiers | Friedreich’s ataxia patient pathway in Europe
Using the same survey, this publication focuses on data from 97 people living with Friedreich’s ataxia (FA) in the UK, Germany and Italy.
In the UK and Italy transport was the highest ranked reason why people stopped attending a SAC. In the UK this highlights a need for more SACs across the UK.
For the majority of participants in all countries, multidisciplinary team (MDT) care was effective.
People with FA who attended SACs reported that attending a SAC helped with taking part in research. In the UK, more so than in Germany and Italy, SACs helped with coordination of referrals to other healthcare professionals, reinforcing the need for more involvement of SAC services in disease management in Italy and Germany.
SACs, followed by secondary care, received the most positive feedback about the level of understanding of their condition, management, and treatment available. There was mixed feedback about primary care.
What mattered most to participants across all three countries was: getting information about available treatment, better access to therapies and better practical advice on living with FA.
This data reflected what had been concluded from the other publications, a higher satisfaction with SAC services compared to non-SAC without a significant increase in cost.
This publication defines the characteristics of survey participants and explores differences between SAC and non-SAC cohorts.
In the UK, 56.3% of participants were over 60 years of age, compared to Germany and Italy where there were more participants between 30 and 59 years.
40.3% of all responders reported they had an inherited cerebellar ataxia. There were a significantly higher proportion of people with idiopathic (unknown cause) cerebellar ataxia in the UK, and a significantly higher number of people with FA in Italy.
35.8% of the total cohort had never had a referral to a SAC. There were a higher proportion of participants in Italy (58.9%) and Germany (56.5%) who reported being under the care of a SAC or having been referred to a SAC than in the UK (27.2%). There were also higher proportions from Italy and Germany referred to SACs, compared with the UK.
People who reported fewer co-existing medical issues, being employed and having a shorter travel duration to the SACs, were associated with attending or being referral to a SAC. Having more issues with mobility was associated with discontinuing care under a SAC. These results help understand factors that might influence attendance or referral to a SAC and expose areas that could be focused on supporting access to SACs and may influence future study design.
For Information on UK Specialist Ataxia Centres and how to get a referral, visit this page on our website.
