Inclusion of Risdiplam in Rare Diseases Fund to Treat Spinal Muscular Atrophy

Mr Ong Hua Han asked the Minister for Health in view of the Health Sciences Authority's assessment of risdiplam which cited data showing improvements in motor function following 12 months of risdiplam treatment as compared with placebo or historical data, whether the Rare Diseases Fund can include medications, such as risdiplam, to treat spinal muscular atrophy.

Mr Speaker, my answer will also address Question Nos 24 and 25 for written answer in today's Order Paper.

The Rare Disease Fund (RDF) is a charity fund for which the Government matches donations three-to-one, and it is managed by the RDF Committee (RDFC) comprising community representatives under the advice of a panel of medical experts.

The RDFC oversees the RDF's fundraising efforts and donor engagement. The expansion of RDF to cover Cell, Tissue and Gene Therapy Products (CTGTPs) and the ability to draw on the principal donation to spend on CTGTPs are fairly recent arrangements, and the RDFC will communicate with new donors.

On Mr Ong's suggestion to publish an annual report, the Ministry of Health (MOH) will relay this to the RDFC for its consideration.

Currently, the RDF has listed seven treatments for five conditions. This is based on the clinical effectiveness of these treatments and if the RDF is adequate to support all known patients requiring the same treatment. Since November 2022, the RDF has received and supported applications from eight patients.

We hope that donors will consider supporting the RDF so that more treatments and conditions can be listed, and more patients can be supported.

Separate from the RDF, MOH will review the clinical and cost-effectiveness of spinal muscular atrophy treatments for subsidies and mainstream financing. In the interim, subsidised patients who face concerns affording these treatments may approach Medical Social Workers to apply for MediFund.

Mr Ong.

Mr Ong Hua Han (Nominated Member): Thank you, Mr Speaker. I thank Senior Parliamentary Secretary for her reply. It is good to know that the RDF is continuously reviewed to improve access for rare disease patients and is also considering greater transparency of the fund. I just have one supplementary question.

While I understand that the RDF is ultimately limited by donor contributions, are there considerations to modify the medicine eligibility criteria of the RDF, such that all the medicines that clearly enhance quality of life can be included, even if extension of life span is not evidenced?

I thank the Member for the question. I appreciate his concern. The RDF is actually set up and gets advice from an expert panel of senior doctors with experience in rare disease management and decides on a list of medicines and conditions covered under the RDF. So, actually, there is expertise in that space and many of them would be able to guide as to what would then be included in that list. Medicines, as explained, can only be listed if there is sufficient investment income to support all known patients for that same medicine. So, these are some of the limitations of the RDF.