Paul Tambyah: It is inappropriate to fund rare disease treatment through crowdfunding or charitable funds

Dr Paul Ananth Tambyah, Chairman of the Singapore Democratic Party. He is also currently president of the Asia Pacific Society of Clinical Microbiology and Infection and president-elect of the International Society of Infectious Diseases

SINGAPORE – Dr Paul Ananth Tambyah says it is inappropriate for Singapore to rely on crowdfunding or charitable funds to support treatment for rare disease patients.

Responding to an email question from the TOC, Dr Tambyah, Chairman of the Singapore Democratic Party (SDP), shared his views on how the party’s proposed health plan would better support rare disease treatment than the current health system .

On November 8, Mr.

Ong in its written reply to the parliamentary questions tabled by the Member of Parliament for Aljunied GRC, Leon Perera, said that there are enough funds to support the six patients currently cared for under the Rare Diseases Fund (RDF), but also noted that support was reviewed annually for each beneficiary, to take account of changes in their medical condition and financial situation.

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

Mr. Ong also noted that the RDF committee that oversees the fund takes a “list” approach, meaning a medicine can only be listed for support after it has assessed that the medicine is likely to significantly extend the life expectancy. of life and improve a patient’s quality of life.

Currently, the RDF has listed 7 drugs for 5 conditions.

“We recognize that there are other rare disease patients requiring medicines not listed, which the RDF does not currently support,” Ong said.

“Medications for rare disease patients can exceed $200,000/- per patient annually, with varying efficacy, and our health care funding system is not designed to support such expensive treatments.” Mr. Ong said.

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

Negotiate with drug manufacturers to lower prices

When asked how SDP’s health policy can better manage the cost of care than the current system in Singapore, Dr. Tambyah noted that the policy’s approach is to negotiate intensively with the manufacturers of these drugs and devices to force lower prices.

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

“If that doesn’t work, the SDP plan supports licensing mandates that allow parallel drug makers to make the drugs — this has been done for other drugs in Thailand, India and Brazil and there’s a good chance this will happen with these costly gene therapies over time.”

“This is indeed allowed under WTO 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, this is not always true and children born with these conditions may have to depend on charity.”

“This is really unfair as it is not the child’s fault which is why we at SDP believe in a universal insurance policy that has a reinsurance component. The whole concept of insurance is the pooling of risk against rare and unusual situations. Dr. Tambyah said.

Claims-based insurance undermines the principle of insurance

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

In her letter to the forum, Ms. Ho, who was chair 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 document, ‘Health Insurance Cost Management in Singapore’, which

Ms. Ho said in her letter, “CBP is a useful solution because it only aims to change consumer behavior, when in fact they may not have the knowledge to exercise judgment, without addressing the root cause of rising costs. doctors. It is also destructive and not viable in the long run, because it undermines the fundamental principle of insurance.’

He goes on to say: “This is why insurance works on the principle of risk sharing: the insured loss is distributed over a group of individuals so that the burden is more accessible to each individual. When the pooling of risks is removed, it is no longer insurance. It’s self-sufficiency.”

Under the proposed list of backup drugs in the SDP health plan, the policy requires that the prices of these drugs, including expensive life-saving proprietary drugs in emergencies, national health crises or epidemic situations, be aggressively negotiated with producers.

“Once the consumption amount of a reserve-listed drug exceeds a certain amount, it will trigger a larger-scale government bid for said drug. If the offer fails to bring down the price of the drug, compulsory licensing can be enacted to circumvent unfair patent laws in order to combat serious diseases and epidemics. as written in the plan.

Not appropriate to support crowdfunding treatment

Mr. Ong also noted in his written response how the financial needs of Singaporeans living with rare diseases are collectively supported 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 gain community support through crowdfunding or other charitable trusts and shared that the Ministry of Health (MOH) will continue to monitor the situation and work to broaden the reach of RDF treatments that can be supported.

“We also encourage members of the public to support RDF with donations so more drugs and conditions can be listed and more patients can be supported.”

The Straits Times previously reported that the parents of a five-month-old baby suffering from a rare genetic disease just managed to raise $3 million through crowdfunding for his treatment.

Baby Zayn, who suffers from spinal muscular atrophy type 1 (SMA), 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 through the Special Access Route – which allows unregistered life-saving drugs to be imported into the country – it is often touted as the world’s most expensive drug, costing nearly $3 million.

When asked about the idea of ​​covering rare diseases through crowdsourcing or charitable funds, Dr Tambyah said he did not think this was appropriate.

“There are many problems with crowdfunding, even though it is widely practiced around the world for drugs like zolgensma, including loss of privacy, exaggeration of the benefits of drugs, and a host of other issues covered in this article.”

“The pharmaceutical company that makes Zolgensma has tried to respond to global criticism of its pricing by introducing a lottery where 100 children get the drug for free – which is even more problematic as it creates something of a ‘hunger game’ situation. .

“The SDP believes that healthcare is a basic human right and there shouldn’t be a price for it. There are alternative approaches that can and should be used.” Dr. Tambyah said.

Unsustainable rare disease listing approach

Dr. Tambyah notes that the “list approach” is limited by the RDF’s ability to screen the many “orphan drugs” currently in use and by the limited funds available.

MP Cheryl Chan in a parliamentary motion last year called for more support for people living with rare diseases, noting that RDF has benefited just seven people – out of eight who applied – since it launched two years ago. does.

In response to Ms Chan’s motion, State Health Minister Koh Poh Koon said that “adding more medicines does not by itself help more patients, especially if the RDF lacks sufficient funds.” to support more patients over the lifetime of their treatment costs,

Dr. Koh also shared that the RDF made approximately $1 million in grant payments and expects to make approximately $1.5 million in grant payments, 50% more than in the last financial year for the same group. of patients.

Last month it was revealed that RDF has raised about $137.9 million from public donations and matching 3:1 government contributions as of July 31 since its inception in 2019. This includes $37.9 million raised in fiscal 2021 and $8.6 million raised in fiscal year 22 as of July 31, 2022.

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

“At a price tag of S$3 million per child, this would exceed the S$2.5 million paid for all seven beneficiaries over the past two years.”

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

When asked about funding such coverage as proposed in the SDP health plan, Dr. Tambyah reiterated the need to negotiate with manufacturers of rare disease drugs that have proven effective in lowering prices and to mandate that the insurance provider – in this case the government, get reinsurance to cover the high expenses associated with these drugs.

“The bottom line is that 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 fail.”

@paultambyah Replying to @laksjdg123456 yes they all pay the same! #tiktoksg #DidYouKnow #fyp #sgpolitics #xyzbca #teamSDP #singapore #politics #fypシ #sgtiktok #foryou #makeitviral #viral #healthcare #singaporetok #viralvideo ♬ original sound – Paul Tambyah
Paul Tambyah: It is inappropriate to fund rare disease treatment through crowdfunding or charitable funds

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