The Promise of Disease-Modifying Treatments (DMTs)
Emerging DMTs, including gene therapy, antisense oligonucleotides (ASOs), and small-molecule therapies, aim to slow, stop, or even reverse HD progression. These innovative treatments could revolutionise HD care, offering long-term health benefits and improved quality of life.
Our Study: A Health State Transition Model
To anticipate the impact of future DMTs, we developed a model that separately analysed 40-year-old individuals with prefunctional decline (PFD) and incorporating:
- Total Functional Capacity (TFC) score, a key measure of HD progression
- HD Integrated Staging System (HD-ISS), a framework to understand and predict the progression of HD, from early pathophysiological changes to functional decline
- Exploration of the Prognostic Index Normalised for HD (PINHD) as an alternative method to predict the onset of functional decline
Costs and quality-adjusted life years (QALYs) were estimated over a lifetime horizon by applying health state–specific costs and utilities derived from a related HD burden-of-illness study
Key Findings
Our model predicts that DMTs could:
- Reduce the economic burden of HD by delaying functional decline & slowing disease progression
- Extend patients’ ability to work, manage finances, live independently
- Improve quality of life
However, although the potential of DMTs to delay progression and improve quality of life is evident, the economic implications of uptake need careful consideration, given the costs associated with prolonged survival in advanced stages may offset some of these benefits.
Future Directions
As we look to the future, the development of effective DMTs could revolutionise HD treatment. While our current analysis is speculative, it underscores the commitment of Prime to global healthcare challenges, and the importance of continued health economic research and economic evaluation to guide healthcare decisions and improve patient outcomes.