Navigating the high seas of rare disease rreatment in Singapore: A deep dive into Sherry Toh’s experience and the current health policies

SINGAPORE: Amidst the stunning skyline of one of the world’s leading economic powerhouses, there’s a less visible struggle playing out. Singapore’s renowned healthcare system is grappling with an intricate challenge – how to manage the astronomical cost of treating rare diseases.

An individual who stands at the heart of this struggle is Sherry Toh, a former administrative assistant, now advocate, who’s been battling Spinal Muscular Atrophy (SMA) since her childhood.

Sherry’s story shared in a write-up on “We, the Citizen”, filled with grit, hope, and an ardent call for change, casts a spotlight on the lived experiences of rare disease patients in Singapore. It elucidates the harsh reality of these patients who are often caught between the promise of revolutionary medical treatments and the staggering price tags that accompany them.

A glimpse into Sherry’s life journey reveals a story of resilience. Diagnosed with SMA, a rare genetic disorder that progressively weakens skeletal muscles, her life has largely unfolded in the confines of a wheelchair.

Childhood memories are woven with the narrative of physical limitations, the ceaseless reliance on others, and the enduring spirit to embrace life nonetheless.

“Growing up with SMA was certainly challenging. Mundane activities that most people take for granted, like eating, dressing, or even moving about freely, posed as daunting challenges for me,” shared Sherry .

A written reply from Singapore Health Minister Ong Ye Kung in March 2023 to a Parliamentary question filed by Louis Chua, Member of Parliament for Sengkang GRC, revealed that about 40 to 50 persons in Singapore are currently diagnosed with SMA.

Life, however, had a dramatic turn for Sherry when she was granted access to Risdiplam, an SMA drug, through a clinical trial.

The drug Risdiplam, sold under the brand name Evrysdi, is an oral medicine and the only treatment option registered with and approved by Health Science Authority (HSA), and It costs around S$375,000 a year in Singapore’s public healthcare institutions.

This medicine, she recounts, redefined her life’s trajectory and offered her a glimpse into a world less defined by her debilitating condition.

“Risdiplam has undeniably been a game-changer for me,” Sherry expressed, her voice brimming with gratitude. “It’s given me the ability to breathe without support, to project my voice louder, and to move with an ease I’d almost forgotten. It’s been truly transformative and offered me a semblance of a life that I’d stopped dreaming about.”

To facilitate Sherry’s treatment, payments will be made directly to the hospital in two installments: S$187,500, which constitutes 50% of the campaign target, and the remaining amount of the campaign, also S$187,500.

However, since Ms Toh’s crowdfunding campaign started in March, it is still 95% away from reaching the goal of S$375,000.

Despite Risdiplam’s life-altering effects, Sherry’s narrative illuminates a less celebrated aspect of rare disease treatments – their exorbitant cost.

These costs often escalate to hundreds of thousands of dollars annually, making these potentially life-saving treatments a luxury, inaccessible to many grappling with rare diseases and their families.

In an attempt to address this financial challenge, the Singapore government established the Rare Disease Fund (RDF). The RDF is designed to subsidize the cost of certain life-saving drugs for rare diseases.

However, this model has not been immune to criticism, with some arguing it only provides a band-aid solution to a more profound issue.

Leading the critique is Dr Paul Ananth Tambyah from the Singapore Democratic Party (SDP), who questions the sustainability of the RDF’s ‘listing approach.’

Under this scheme, only a select few treatments for specific rare diseases are covered, and this, Dr Tambyah points out, is far from a comprehensive solution.

“With its current structure, the RDF has a significant limitation,” explained Dr Tambyah.

Since its inception in 2019, it has made grant payouts of around $1 million. It’s projecting to disburse about $1.5 million this financial year for the same group of patients.

Now consider this against the cost of treating just one rare disease, such as SMA that Sherry battles. The expense involved would far exceed the entirety of the RDF’s disbursements in the past two years.

Dr Tambyah suggests that the current approach is not equipped to meet the high-cost demands associated with rare diseases. According to him, the inadequacy of the funds available in comparison to the rising costs of treatments poses an issue that’s bound to escalate.

To address this, Dr Tambyah proposes a radically different solution. As an alternative to the current framework, he suggests that the government, as the insurance provider, should engage in aggressive price negotiations with drug manufacturers.

In instances where these negotiations do not lead to substantial price reductions, he proposes enacting compulsory licensing to locally produce these drugs, thus bypassing the crippling costs.

“The bottom line is that having a child with a rare disease shouldn’t force parents into making an unthinkable choice – between letting their child suffer and die or confronting bankruptcy,” stated Dr Tambyah emphatically.

His statement not only echoes the sentiments of many rare disease patients but also offers a glimpse into the tangible fears and heart-wrenching decisions families like Sherry’s grapple with regularly.

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.

In the grand tapestry of Singapore’s healthcare system, stories like Sherry’s and the vocal critique of policy-makers like Dr Tambyah serve as potent reminders.

They underscore the urgent need to reevaluate and reform existing structures. They challenge the city-state to reconfigure its approach to ensure it continues to lead as a nation that upholds the health of all its citizens – not just those with common diseases, but those confronting the rare and the complex.

The crux remains clear – sustainable, equitable, and comprehensive solutions are not a mere option, but an absolute necessity for the sake of the patients who are at the heart of this healthcare narrative.