Scottish Medicines Consortium (SMC) have published assessment reports for two new treatments under their new process for treatment for rare disease

The SMC have recently approved Luxturna (a gene therapy for a rare type of inherited retinal dystrophy) and Crysvita (a treatment for X-linked hypophosphataemia which is a rare skeletal disorder). Both submissions highlight the challenges in understanding the impact of very rare conditions on patients’ health related quality of life (HRQL). Acaster Lloyd Consulting Ltd worked with both of these companies to capture data regarding the impact of the disease and treatment on HRQL; data which was used to inform cost effectiveness analyses. In their review of both treatments the SMC highlighted uncertainty regarding the HRQL data because the methods relied in part upon expert opinion. We are keen to highlight this in order to illustrate the challenges we face in capturing HRQL data in rare diseases. The treatments will be available for use in Scotland for 3 years while the companies collect more data on the impact of the treatments on patients and carers.

Decision making regarding the adoption of very high price, potentially very high value treatments is probably the most challenging area of Health Technology Assessment. Read more about the news from the SMC here.