What this study looked at
Alpha-mannosidosis is an inherited rare disorder affecting the breakdown of mannose-containing oligosaccharides, which can be managed with enzyme replacement therapy, bone marrow transplantation or supportive care. This study used a computer simulation model to compare how cost-effective these options are for mild to moderate disease, from the perspective of the Serbian health system and over a lifetime.
Key takeaways
- Enzyme replacement therapy with velmanase alfa was not cost-effective when compared with either supportive therapy or bone marrow transplantation.
- Bone marrow transplantation could be cost-effective compared with supportive care, but only if the amount society is willing to pay for a year of good-quality life were raised substantially.
- The authors argue that judging orphan drugs needs to look beyond a single cost figure and consider factors such as fairness, risk-sharing and dedicated funding.
Why it matters
The findings highlight the difficult trade-offs health systems face in funding rare-disease treatments, and make the case for broader criteria so that effective therapies can reach more patients.
