HCD Economics, University of Chester and the Huntington’s Disease Association, are working together to design and launch a socioeconomic study, with an emphasis on informing and facilitating public and patient involvement and engagement (PPIE) in Huntington’s Disease.

The study seeks to address the immediate and long-term challenges for those impacted by Huntington’s Disease, including patients, their carers and families and the political and healthcare framework that makes decisions on treatment priorities and healthcare resource allocation.

An initial development and scoping research report developed by HCD Economics, has been undertaken to inform the work of the three study collaborators and the report will also support the process of setting up an Expert Review Group to oversee the study design, implementation and utilisation of results as an objective source of real world evidence.

The following is an extract from the report. All parties interested in investing in Huntington’s Disease socioeconomic research are invited to contact the study coordination leader at HCD Economics, Alison Rose for a face to face discussion.

To speak with Alison, please contact her on Alison.Rose@HCDEconomics.com.

Existing challenges:

  • The increasing prevalence of HD from a global perspective.
  • A substantial number of ongoing R&D works in the area of HD focusing on both curative, disease-modifying or symptomatic therapies.
  • To date, there is a lack of extensive real-world evidence documenting the economic and societal burden of HD at a large geographical scope.
  • The necessity to provide up-to-date and accurate data on the economic burden of HD (last available data from 2013).
  • Limited number of publications focusing on the real-world evidence of HD costs (especially from the societal perspective).
  • Limited real-world evidence on HD costs stratified by stages of the disease.  (It was shown that costs and resource utilization increases with disease progression. Analysing costs across all severities of the HD would bring important insight on possible savings associated with an introduction on new treatments.)
  • The most recent BoI studies have some methodological limitation which should be addressed by future research.

Future research can add to the current state of the knowledge on HD:

  • Develop patient-focused, Real World Data study in HD which is supported by both academia & patient advocacy groups in order to inform modelling and aid the development of evidence-based guidelines and clinical pathways.
  • Use a a comprehensive bottom-up approach to the societal cost of HD which accounts for productivity losses and overall costs of HD treatment.
  • Collect data on the QoL as stated by patients and carers.
  • Provide actual financial landscape across different counties – up-to-date published data usually refers to one country. Usage of universal methodology across countries allows for detailed comparison of cost drivers.
  • Conduct cost identification stratified by disease severities.
  • Learn about the disease porgression and treatment offered to the HD patients. (Symptoms experienced and employed treatment can substantially vary from one patient to another.
  • Investigate burden associated with caring for HD patients.