SINGAPORE — “There remains the broader question as to whether the Government should support treatments regardless of their cost, particularly if the efficacy is uncertain,” said Minister of Health, Ong Ye Kung.
Further noting that this position taken by the Ministry of Health (MOH) on supporting treatments for rare diseases, needs to be carefully reviewed and must not be taken lightly.
This was said in a written reply on 8 November to questions filed by Member of Parliament for Aljunied GRC, Mr Leon Perera who had asked about the number of applications that the RDF received since April 2021 and if it has available funds to support new applicants at the current time.
In his reply, Mr Ong affirmed that there are sufficient funds to support the six patients currently supported under the Rare Disease Fund (RDF).
According to Mr Ong, these six patients had filed applications to RDF as beneficiaries in the period between April last year till November.
According to RDF’s website, a rare disease is defined as one which affects fewer than one in 2,000 patients.
“The RDF Committee which oversees the fund adopts a ‘listing’ approach, which means that a medicine can only be listed for support after it assesses that the medicine is able to meaningfully extend life expectancy and improve patient’s quality of life.”
Currently, the RDF has listed 7 medicines for 5 conditions.
| Condition | Medicine(s) |
|---|---|
| Primary bile acid synthesis disorder | Cholic acid |
| Gaucher disease (Type 1 or 3) | •Imiglucerase (Cerezyme) •Velaglucerase alfa (VPRIV) •Taliglucerase alfa (Elelyso) |
| Hyperphenylalaninaemia due to tetrahydrobiopterin (BH4) deficiency | Sapropterin dihydrochloride |
| Pompe Disease | Alglucosidase alfa (Myozyme) |
| Mucopolysaccharidosis type VI (MPS VI) | Galsulfase (Naglazyme) |
Mr Ong notes that the support is reviewed annually for each beneficiary, to factor in changes in their clinical condition and financial situation.
“We recognise that there are other rare disease patients requiring non-listed medications, which the RDF currently does not support,” said Mr Ong in his reply.
“Medicines for rare disease patients can exceed $200,000/- per patient annually, with varying efficacies, and our healthcare financing system is not designed to support such high-cost treatments.”
“Instead, society and the Government collectively support the financial needs of Singapore citizens with rare diseases through the RDF, with the Government providing three dollars of matching grant for every dollar of donation.”
Mr Ong also points to examples of how some patients are able to obtain support from the community through crowd-sourcing or other charity funds and shared that the Ministry of Health (MOH) will continue to monitor the situation and work towards expanding the scope of RDF treatments that can be supported.
“We also encourage members of the public to support the RDF with donations so that more medicines and conditions can be listed, and more patients can be supported.”
Mr Perera also asked about the RDF’s ratio of applicants to grants but it was not answered in Mr Ong’s written reply.
Applications To Be Considered On A Case-To-Case Basis And Reviewed Annually
In February last year, Mr Perera also filed another parliamentary question for the number of applications under RDF received and the specific eligibility criteria or conditions that applicants must meet to receive support from the Fund.
In response, Mr Ong states that Singaporeans undergoing treatment at public healthcare institutions (PHI) for rare disease conditions covered by RDF can apply for support through the medical social workers.
Each RDF application is said to be carefully assessed by an independent RDF Committee, comprising representatives from NGOs, public healthcare institutes, and private corporations.
“As the Committee has to prioritise the allocation of limited funds, it takes into account various factors such as whether the patient is clinically eligible and likely to benefit from the treatment, their family’s ability to afford the treatment either in part or in whole, including any extenuating circumstances faced by the patient and family, and whether other financing support is available.”
According to Mr Ong, RDF funds were already fully allocated last February, and “new beneficiaries, conditions or treatments can only be approved when additional funds are raised.”
Mr Ong also shared that there are six beneficiaries supported by the RDF at that point in time.
It is, however, unknown if these six beneficiaries are the same patients in Mr Ong’s answer this month as the applications are reviewed annually for each beneficiary, to factor in changes in their clinical condition and financial situation.
Mr Perera had also asked for the specific eligibility criteria or conditions for the selection of rare diseases to be covered under the Fund and whether MOH can provide an update on the timeline of more rare diseases to be covered under the Fund.
These were not answered by the Minister.
Endowed Fund With $137.9 Million, Paid $1 Million Last Year In Grants
The RDF is managed by KK Women’s and Children’s Hospital (KKH) Health Fund (part of SingHealth Fund) with the support of MOH, operates as an endowed fund.
The fund is said to ensure beneficiaries are supported for life, as rare disease patients generally require high-cost medicines on a lifelong basis.
In response to a motion raised by Ms Cheryl Chan last August, asking for more rare diseases and medicines to be listed on the RDF, Minister of State for Health Koh Poh Koon said that “adding more medicines does not in itself enable more patients to be helped, especially if the RDF does not have sufficient funds to support more patients for the entire lifetime of their treatment costs,”
Dr Koh also shared that the RDF made grant payouts of around $1 million and is projecting to make grant payouts of around $1.5 million which is 50% more than the last financial year for the same group of patients.
It was revealed last month that RDF raised about $137.9 million from both public donations and the Government’s 3:1 matching contributions as of 31 July since its inception in 2019. This includes $37.9 million raised in FY2021, and $8.6 million raised in FY22 as of 31 July 2022.
State-funded local media, Straits Times earlier reported that the parents of a five-month-old boy who has a rare genetic disorder have just managed to raise $3 million through crowdfunding for his treatment.
Baby Zayn who suffers from Type 1 spinal muscular atrophy (SMA) which is also called Werdnig-Hoffmann disease – will be able to get the drug Zolgensma by early December, said his parents, Mr Nabeel Salim Abdat and Ms Syahirah Yakub.
Available in Singapore only via the Special Access Route – which allows for unregistered life-saving drugs to be imported into the country – it is often touted as the most expensive drug in the world, costing almost $3 million.
Just last year, another couple also successfully raised funds to save their 22-month-old son’s life for a one-time-only gene therapy treatment costing $2.868 million.
