AXIS Healthcare Consulting Ltd’s Post

Emma McGrath attended the short course Technology Assessment for Rare Diseases on Sunday as part of #ISPOREurope 2024. She captures her learnings below. Speakers: Hwee-Lin Wee -Lin Wee, PhD joshua byrnes , PhD Diana Beatriz Bayani, MSc, PhD Session 1: Overview of rare diseases Looking at the case study of Spinal Muscular Atrophy (SMA) Limited research and development to receiving treatments for rare diseases such as regulatory barriers, recruitment and unpredictability. The European Organisation for Rare Diseases (EURORDIS) and the Orphan Drug regulation initiatives to encourage R&D for rare diseases. Singapore’s approach to finance rare diseases addressing the need for an appropriate financing framework, that is both sustainable and will prevent a schism between those who can afford precision medicine and those who cannot. Session 2: Comparing the Experiences of England and Singapore. Key features of rare disease policies, HTA pathways and funding Case Studies: Comparing the experiences of England and Singapore Implications for future policy and practice Session 3: Economic Evaluation of rare diseases Types of economic evaluation Cost-effectiveness planes and quantifying the uncertainty Spotlight on extrapolations and their uncertainty over time Session 4: Multicriteria Analysis (MCDA) requires the aggregation of criteria into a single expression of value. MCDA has been used to organise data and questions relevant to the development of clinical practice guidelines (CPG) for a rare genetic condition, Prader-Willi syndrome (PWS) Pros of MCDA are how it Improves transparency, promotes consistency, Does not replace judgement Cons of MCDA: Requires careful design considerations and is time consuming. #shortcourse #ISPOR #learning #raredisease #economicevaluation #multicriteriaanalysis

  • chart, bubble chart

To view or add a comment, sign in

Explore topics