Research recommendations coming out of this guidance In its evaluation of burosumab, the committee noted substantial uncertainty in several areas, and agreed that further research into the treatment benefit of burosumab in young people aged 13 years and over would relieve some of the clinical uncertainty in an age group covered by the marketing authorisation. The committee also noted that the additional evidence exploring the relationship between radiological measures of bone defects (that is, the Rickets Severity Score and the Radiographic Global Impression of Change) and health-related quality of life would help to address several key uncertainties in the economic analysis. Evidence on the progression of X-linked hypophosphataemia (XLH) over time and the long-term benefits of burosumab would also be valuable.
In its evaluation of burosumab, the committee noted substantial uncertainty in several areas, and agreed that further research into the treatment benefit of burosumab in young people aged 13 years and over would relieve some of the clinical uncertainty in an age group covered by the marketing authorisation.
The committee also noted that the additional evidence exploring the relationship between radiological measures of bone defects (that is, the Rickets Severity Score and the Radiographic Global Impression of Change) and health-related quality of life would help to address several key uncertainties in the economic analysis.
Evidence on the progression of X-linked hypophosphataemia (XLH) over time and the long-term benefits of burosumab would also be valuable.