Paul Tambyah: Not appropriate to fund treatment of rare diseases through crowd-funding or charity funds

SINGAPORE — Dr Paul Ananth Tambyah says that it is not appropriate for Singapore to rely on crowd-funding or charity-based funds to support treatments for patients suffering from rare diseases.

Responding to an email query from TOC, Dr Tambyah who is the Chairman of the Singapore Democratic Party (SDP) shared his views on how the party’s proposed healthcare plan would better support the treatment of rare illnesses compared to the current healthcare system.

On 8 November, Mr Ong Ye Kung in a written reply to parliamentary questions filed by Member of Parliament for Aljunied GRC, Leon Perera, stated that Singapore’s healthcare financing system is not designed to support such high-cost treatments for rare illnesses.

Mr Ong affirmed that there are sufficient funds to support the six patients currently supported under the Rare Disease Fund (RDF) but also noted that the support is reviewed annually for each beneficiary, to factor in changes in their clinical condition and financial situation.

The RDF is managed by KK Women’s and Children’s Hospital (KKH) Health Fund (part of SingHealth Fund) with the support of the Ministry of Health, which operates as an endowed fund.

Mr Ong in his written reply also noted that 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.

“We recognise that there are other rare disease patients requiring non-listed medications, which the RDF currently does not support,” said Mr Ong.

“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.” said Mr Ong.

According to RDF’s website, a rare disease is defined as one which affects fewer than one in 2,000 patients.

Negotiate With Drug Manufacturers To Bring Down Prices

When asked about how SDP’s healthcare policy can better manage the cost of treatment as compared to the current system in Singapore, Dr Tambyah noted that the policy’s approach is to negotiate intensely with the manufacturers of these drugs and devices to force lower prices.

According to Dr Tambyah who established the National University Hospital’s Division of Infectious Diseases in 2003, this has already worked even for a drug like zolgensma which is cheaper in Japan than in most other countries due to Japanese government negotiations.

“If this does not work, the SDP plan advocates compulsory licensing which is to enable parallel drug manufacturers to produce the drugs – this has been done for other drugs in Thailand, India and Brazil and there is a good chance that this will happen with these expensive gene therapies with time.”

“This is actually allowed under the WTO’s TRIPs but only in low and middle-income countries because the assumption is that high-income countries will pay for these drugs for their citizens.”

“Unfortunately for those in Singapore and the United States, that is not always true and children born with these conditions may have to depend on charity.”

“This is really unfair as it is no fault of the child and that is why we in the SDP believe in a universal insurance policy which has a reinsurance component. The whole concept of insurance is risk pooling against rare and unusual situations.” said Dr Tambyah.

Claim-Based Insurance Undermines The Principle Of Insurance

Dr Tambyah, who is currently the President of the Asia Pacific Society of Clinical Microbiology and Infection and President-elect of the International Society of Infectious Diseases, further pointed to a Facebook post published by Mimi Ho, where she expressed her dismay that “some insurance companies have also implemented the claim-based pricing (CBP) that the HITF has specifically discussed, rejected and NOT recommended”.

In her forum letter, Ms Ho, who was chairman of the Health Insurance Task Force (HITF) urged the Monetary Authority of Singapore (MAS) and the Ministry of Health (MOH) to review and stop this practice for the public interest.

In 2016, the task force produced a paper, “Managing the cost of Health Insurance in Singapore” which

Ms Ho said in her letter, “The CBP is an expedient solution because it only aims to alter consumer behavior, when in fact they may not have the necessary knowledge to exercise judgement, without addressing the root cause of rising medical cost. It is also destructive and not viable in the long run, because it undermines the fundamental principle of insurance.”

She goes on to state, “This is why insurance works on the principle of risk pooling: the insured loss is spread over a group of individuals so that the burden is more affordable to each individual. When the risk-pooling is removed, it is no longer insurance. It is self-reliance.”

Under the proposal of a Reserve Drugs List in the SDP Healthcare Plan, the policy will have prices of these drugs, including expensive life-saving proprietary drugs in emergencies, national health crises or epidemic situations, aggressively negotiated with manufacturers.

“Once the quantity of use of a drug on the reserve list exceeds a certain amount, it will trigger a larger scale government bid for the said drug. If bidding fails to bring down the price of the drug, compulsory licensing may be enacted to circumvent unfair patent laws in order to combat serious diseases and epidemics.” as written in the plan.

Not Appropriate To Support Treatment With Crowdfunding

Mr Ong in his written reply also noted how the financial needs of Singapore citizens with rare diseases are supported collectively through the RDF with the Government providing three dollars of matching grants for every dollar of donation.

He pointed to examples of how some patients are able to obtain support from the community through crowd-funding 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.”

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.

When asked about the view of having rare diseases covered through crowd-sourcing or charity funds, Dr Tambyah said that he does not think this is appropriate.

“There are many problematic issues with crowdfunding although it is widely practised all over the world for drugs like zolgensma — these include loss of privacy, overstating the benefits of the drugs and a host of other issues covered in this article.”

“The drug company which makes zolgensma has tried to respond to global criticism of its pricing by introducing a lottery in which 100 children get the drug free – that is even more problematic as it creates a kind of “hunger games” situation.”

“The SDP believes that healthcare is a basic human right and there should not be a price to that. There are alternative approaches which can and should be used.” said Dr Tambyah.

Listing Approach On Rare Diseases Not Sustainable

Dr Tambyah points out that the “listing approach” is limited by the capacity of the RDF to review the numerous “orphan drugs” which are in use right now and by the limited funds available.

Member of Parliament Cheryl Chan in a Parliamentary Motion last year, called for more support for those with rare diseases, noting that RDF has benefited only seven people — out of eight who applied — since it was launched two years ago.

In response to Ms Chan’s motion, 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.

Dr Tambyah noted that the disease that Baby Zayn is suffering from is not that rare. The incidence is 1 in 11,000 worldwide which means that there are three to four babies born every year with this condition.

“With a price tag of S$3 million per child, this would exceed the S$2.5 million paid out for all seven beneficiaries in the last two years.”

As such, this approach is not sustainable, said Dr Tambyah.

When asked about the funding for such coverage as proposed in the SDP Healthcare Plan, Dr Tambyah reaffirms the need to negotiate with manufacturers of drugs for rare diseases which have proven efficacy to bring the prices down and to mandate that the insurance provider — in this case the government, obtain reinsurance to cover for the high expenses associated with these drugs.

“The bottom line is that the SDP does not believe that having a child with a rare disease should force parents to decide between letting their child suffer and die or bankruptcy.”

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