CHILDREN DIAGNOSED WITH ACHONDROPLASIA AND FUNDING ITS TREATMENT UNDER RARE DISEASE FUND

MP Dennis Tan

Mr Dennis Tan Lip Fong asked the Minister for Health (a) how many children in Singapore are diagnosed with Achondroplasia in the past 10 years; (b) what are the current prospects of funding the treatment of Achondroplasia under the Rare Disease Fund; and (c) whether the Ministry will engage the manufacturers of Voxzogo to achieve more affordable access to the drug.

Mr Ong Ye Kung: Achondroplasia is a genetic condition that causes abnormal bone and cartilage growth, leading to short stature and disproportionate growth. MOH does not collect data on the number of patients diagnosed with achondroplasia, but globally, the condition affects around one in every 30,000 newborns. 

The drug Vosoritide, sold under the brand name Voxzogo, is intended to increase linear growth in children with achondroplasia. Vosoritide is currently not registered in Singapore. MOH has therefore not assessed the drug for clinical and cost effectiveness, nor conducted pricing negotiations with the manufacturer.

Further, given the limited size of the Rare Disease Fund (RDF), funding support is focused on rare conditions that are life-threatening and where the treatment is effective in extending the patient’s lifespan.

Vosoritide is just one example. With medical advances, there will be novel and high-cost treatments for many medical conditions. Healthcare authorities in the world are finding themselves needing to review their healthcare financing policy, including healthcare insurance system, to ensure treatments remain cost effective and affordable.

Ministry of Health
10 January 2023

https://sprs.parl.gov.sg/search/#/sprs3topic?reportid=written-answer-12576

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