Who deserves healthcare?

Are Singaporeans generous when giving to complete strangers? Without a doubt, going by recent crowdfunding campaigns for those afflicted by spinal muscular atrophy (SMA), a rare, genetic neuromuscular disease. SMA affects motor neurons, the nerve cells in the spinal cord that control voluntary muscle movement. Since the muscles cannot respond to signals from the nerves, they atrophy—weaken and shrink—from inactivity. Many born with SMA are unable to sit independently, and die young from respiratory failure. 

The most common form of SMA is caused by a defective or missing gene known as survival motor neuron gene 1 (SMN1). There are five types of this (0-4), with the first two being the most severe.

In 2019, the US Food and Drug Administration (FDA) approved a seeming miracle cure. Zolgensma, a revolutionary therapy for children under two years with SMA, replaces the missing or mutated SMN1 with a one-time intravenous infusion, providing the cells with new DNA to produce the missing protein. It was believed then to be the “most expensive drug” on the market—a jaw-dropping S$3m. How could the child Singaporean sufferers of SMA ever afford it?

By asking the public. In 2021, about 30,000 people donated almost S$2.9m in 10 days to 22-month-old Devdan Devaraj, who was diagnosed with SMA type 2, or Dubowiz disease. 

Then in 2022, Nabeel Salim Abdat and Syahirah Yakub, first-time parents, received devastating news that their son, Zayn, was diagnosed with the most common type of SMA, type 1, or Werdnig-Hoffmann disease, at about five weeks’ old. If he was to have a fighting chance of living past two years—the average prognosis for this form of SMA—he would also need the same drug as Devdan. Despite a sluggish start, Nabeel and Syahirah succeeded in raising the amount they needed, hitting their S$3.01m goal after an anonymous person donated the remaining S$1.5m.

On December 1st the same year, Sri Lankan natives Dunlanjali Wakwella and Achintha Pilapitiya, came forward to raise funds for their first-born, Shamel, diagnosed with SMA type 2. It took five nail-biting months for Wakwella and Pilapitiya to raise S$2.3m. Thankfully, Zolgensma was finally registered on April 21st 2023 in Singapore, which reduced its price to S$2.4m, and allowed Shamel’s parents to use their insurance to cover the remaining S$100,000.

Which brings us to Lucas Wang’s campaign, the latest [as of writing] on Ray of Hope—a local fundraising platform. At two months’ old, he was diagnosed with SMA type 1. Some three weeks after the campaign started on July 7th, donations exceeded the target of S$2.409m, allowing Lucas to receive his life-saving dose of Zolgensma on August 2nd. 

Globally, crowdfunding for medical treatment has grown significantly in the last decade, stemming from families’ inability to access desired treatment. For example, GoFundMe, the leader in online medical crowdfunding, raised more than US$2bn (S$2.7bn) from 21.7m donations between 2016 and 2020. The above cases might indicate that Singaporeans are willing to offer financial assistance to those who have gotten the worst possible draw in the birth lottery. However, it appears that we are less eager to give to every Singaporean sufferer of SMA. 

Some 10 months into her campaign, Sherry Toh, the sole adult in the mix, has only raised 10 percent of her S$375,000 target. Was this due to donor fatigue or discrimination? Unlike SMA treatment for children, adults with SMA cannot be cured by therapy, although it allows them to lead better lives. Sherry is raising a much lower amount, because it’s only for a one-year supply of the drug she needs, Evrysdi (risdiplam). Perhaps the sluggish response to Sherry’s crowdfunding effort reflects a societal bias, which considers the life of infants with a rare disease to be of greater value and thus more worthy of saving, than that of adults like Sherry. 

“What would the government think? That the public supported infants and toddlers, who could become closer to non-disabled, and not adults, who can’t,” she asked. “Would it [become] like a referendum that could affect healthcare policy?”

Why do we give to some but not others? Sherry’s experience forces us to confront deep ethical dilemmas—the valuing of life based on age, and able-bodiedness—that have never been sufficiently explored in Singapore.

SMA is a genetic, progressive disease that affects the nervous system and muscles, but doesn’t affect sensory nerves or intellect. The defective or missing SMN1 gene is usually responsible for producing a protein essential to motor neurons, which in turn control movement in the arms, legs, face, chest, throat and tongue, as well as skeletal muscle activity, such as speaking, walking, swallowing and breathing. 

SMA type 2 has left Sherry unable to stand or walk without assistance; as well as with a weak respiratory system that’s significantly prone and vulnerable to respiratory complications; and a loss of muscle control, movement and strength, affecting her ability to speak and to swallow. 

Despite growing up with SMA, Sherry remembers her childhood as a “pretty happy one”. She could have felt isolated outside the formal education system, but didn’t, because she met other kids with neuromuscular diseases at programmes run by the Muscular Dystrophy Association. There is also a younger brother, Gabriel—five years her junior—whom, she said jokingly, she “had to learn to love.” When we met on Zoom for the first time, it’s quite clear how close they both are, their easy rapport peppered with wisecracks. It’s also evident how much Sherry delights in being the proud big sis. There was the time she insisted on attending Gabriel’s graduation, even though she was hospitalised. And the many instances when she helped him with his homework. Financially, however, things were far from optimal. She was always aware of the financial burden the disease placed on her parents: struggling with medical bills; “months in and out of the hospital”, which forced them to borrow from family, friends and her father’s business partner, thus incurring more debt.

If there was any chance of her accessing treatment that could improve her quality of life and extend her life expectancy beyond 25 years, there was no alternative but to turn to the kindness and generosity of strangers.

Choosing to crowdfund was ultimately a decision born of desperation and dead ends. “Asking for money is generally frowned upon in Singaporean society, where ‘face’ is used as social currency,” explained Sherry who’s worried that the loss of privacy could stigmatise her family.

But all other doors to financial assistance were shut. Her appeals for government aid had been denied “over and over again” at every turn. The reasons given: her age (24) and state of disease progression, as well as being told that there was a lack of evidence of significant quality-of-life improvement for adult patients and/or patients on a respiratory ventilator.

So why wait until now to crowdfund? Risdiplam, the therapy that Sherry’s raising funds to purchase, was only approved by the FDA in 2020. Singapore’s Health Sciences Authority gave the green light for its use in October 2021. Thereafter, Sherry said that it took some six months to confirm a prescription, as there wasn’t any infrastructure in place for adults with SMA to pursue medication. 

By the time her online campaign began on March 17th, Sherry’s condition had deteriorated to the point where she could no longer lift her head, or feed herself. She was also suicidal. Years of hospital visits, several near-death emergency room episodes, and many failed attempts to get potentially life-altering, but expensive, medication had chipped away at her resolve, weakening her will to live. “It felt to me that I had become expendable and not worth saving just because I could no longer become non-disabled,” Sherry said. “The argument about ‘limited resources’ made by the denials [for financial aid] caused me to think, ‘Oh, well, if this is about money and resources and therefore about how selfish I want to be…I might as well drop dead.’”

Should the state pay? The ruling People’s Action Party (PAP), led by its core beliefs of individual responsibility and targeted state intervention, drives Singapore’s approach to healthcare. While promoting “personal responsibility for one’s health”, the PAP claims to have achieved universal health coverage through a mixed financing system that incorporates direct government subsidies, compulsory comprehensive savings (Medisave), national healthcare insurance (MediShield and MediFund) and cost-sharing.

Singapore has consistently delivered high health outcomes, despite a relatively low expenditure on healthcare—four percent of gross domestic product (GDP) each year, a fairly conservative figure compared to members of the Organisation for Economic Co-operation and Development (OECD). 

Free healthcare is frowned upon by the city-state’s leaders. Ong Ye Kung, health minister, warned in Parliament on May 10th that Singapore needs to make sure that healthcare spending as a proportion of GDP doesn’t increase too much: “In the coming years, our challenge is not to spend more, but to ensure we do not go the way of many OECD countries, with the healthcare fiscal burden spiralling and escalating out of control.” 

In order to keep drugs affordable to the majority of Singaporeans, the government manages the market in various ways: it achieves economies of scale by aggregating demand and integrating supply chain management; ensures the appropriate use of drugs, by getting doctors to prescribe what’s proven to be effective; and adopts value-based pricing approaches through the Agency for Care Effectiveness. 

Approved drugs are published online in the standard drugs list (SDL), and citizens can access these medications at a subsidised rate of between 50 and 75 percent. If eligible, they can also use their Medisave funds to purchase government-approved drugs. Drugs that don’t make this list—like risdiplam, which Sherry needs—are still available in hospitals, but at prohibitive prices. 

To further help eligible patients pay for high-cost drugs that are not on the SDL but have been assessed to be clinically efficacious and cost-effective, they can turn to the Medication Assistance Fund. Drugs are subsidised based on their ability to fill a therapeutic gap, and if they’re expected to be of “significant benefit” to patients in terms of clinical efficacy or improved side-effect profile, and there is enough evidence to support an evaluation. Zolgensma and risdiplam aren’t on the SDL and aren’t subsidised.

“I was on Cloud 9 — then Cloud 10, 11, 12 when I started seeing improvements in my strength and stamina,” she wrote in her journal. Day six: “I practically gulped down my winter melon soup with mee sua. I hadn’t done that in years.” Her voice was also louder—20 percent her mother told her. In all the excitement, however, reality wasn’t far from their minds. In a text to Brianna, or “Brie”, she noted that her mother had gone from being afraid of what risdiplam might do to her and whether the “fight was worth it”, to being afraid of what they were going to do when the supply ran out. In another message to Brie on day 17: “I’ve been scaring myself with my voice lmao. I’ll forget it doesn’t take as much energy to project my voice as loud as is appropriate and then end up being much louder than intended.” Her health kept improving. On day 18, she felt her back muscles “come back online”, even though they were still very weak. “My neck muscles weren’t cooperating, and I was tied to my chair, but I managed to sit upright without someone holding me for a few seconds.” 

On day 19, she texted: “When my mum was at work and she was in a fairly noisy environment, she said she could hear me much better over the phone and didn’t need me to repeat myself anymore 🥺…and I realised while I was playing Cyberpunk 2077 today that I was going between my mouse and keypad swiftly without getting fatigued. I needed help doing so before I started the drug.” And by June 21st, she wondered if her seemingly small improvements might add up to the “significant improvements in quality of life” clause that would make her eligible to receive public aid for rare disease medication. “Maybe I can say it’ll help me become a significantly more productive corporate worker if I land a job soon. That should intrigue Singapore as a meritocracy and corporate paradise,” she envisaged in a journal entry.

When Jom visited her at home on June 29th, it was confronting to meet Sherry in person. The fragility of her skeletal legs and hunched torso were in such sharp contrast to the force of her personality—bold, articulate, smart and quick-witted. But the medication was obviously working. Not only did Sherry look better than she did on our Zoom call, but her energy levels had improved by leaps and bounds—the most concrete evidence she said was shaving off more than half the amount of time on her swallowing test; taking just 20 seconds to finish a certain amount of water, without choking or pauses. The Sherry I had first met online in May was but a pale shadow of the sprightly, cheerful and chatty young adult she now was. She was speaking with a more assured tone, her gestures came across deliberate; and her body was upright. Seeing this transformation was a powerful reminder that Sherry, with SMA or not, was essentially a young adult trying to live her best life. The one major difference is that she has defied all the limitations that were foisted on her because of SMA. 

It’s been 16 weeks since she took her last dose of risdiplam, and she’s feeling her strength wane a little more each day. It seems almost cruel to have tasted what life could be and then have to watch one’s body deteriorate again. Any hope of returning to her daily dose of risdiplam lies again in her crowdfunding campaign. But her chances are looking ever more tenuous as the campaign’s deadline nears (it’s already been extended once).

In 2019, the government set up a Rare Disease Fund (RDF), which matches donations 3-to-1 and offers a 2.5 times tax deduction. The RDF is predominantly an endowed fund that relies on investment income to support beneficiaries on lifelong, high-cost treatments. According to the Ministry of Health (MOH), at the end of FY22, the RDF’s size was S$143m. For this financial year, the RDF’s projected investment income and expenditure for existing RDF beneficiaries are S$4.8m and S$2.4m respectively. Expenditure is often lower than income, MOH noted, because new treatments are only listed for RDF coverage if the projected investment income is adequate to support all known patients requiring that treatment. It currently lists seven treatments for five conditions. Coverage was recently extended to include Cell, Tissue and Gene Therapy Products that are higher cost, one-off treatments. Since November 2022, the RDF committee has received and supported applications from eight patients. But not from anyone suffering from SMA. Zolgensma and risdiplam remain uncovered. 

In a parliamentary reply on March 22nd to Louis Chua, a Workers’ Party member of Parliament, Ong, the health minister, said that most patients with rare diseases are able to get financial help from schemes such as MediSave and MediShield Life and subsidies. He added that the RDF specifically supports high-cost medications for a few patients who may need additional financial assistance. “Given the limited size of the RDF, funding support is focused on treatments for rare conditions that are life-threatening, and where the treatment is effective in extending the patient’s lifespan.” 

More recently on November 22nd, Rahayu Mahzam, senior parliamentary secretary for health, in reply to a parliamentary question from Ong Hua Han, nominated member of Parliament, said that MOH would review the clinical and cost-effectiveness of SMA treatments for subsidies and mainstream financing. And suggested that in the interim, subsidised patients who face concerns affording these treatments may approach medical social workers to apply for MediFund. 

Sherry has thus far been unable to receive any financial assistance from the RDF to pay for treatments that could significantly improve her quality of life. And while Rahayu’s response offers her hope that the state might finally add SMA treatments to the Fund, the wording of the statement worries her. “What does reviewing ‘the clinical and cost-effectiveness’ mean?” she asked. She wonders if child SMA sufferers will be prioritised over adults because in the cold calculus of the state, they are more likely to become more able-bodied, productive members of society. “If the monetary cost is cheaper to let us waste away, is that going to be their [the government’s] justification to not help adult patients?”  

Now that Sherry has been given a taste of what the medication can do to improve her quality of life, is it any wonder that she’s anxious for progress, her patience worn thin. The answers to these questions can perhaps lead the public and other stakeholders—including government; the medical fraternity; industry like big pharma and insurers—towards re-examining their roles in ensuring health equity for all; and not just the most vulnerable or those who are most able to foot the bill. 

It’s expected that with advances in medical innovation, newer and more effective cell and gene therapies (CGT) will become available soon. Last year, there were over 2,000 in development, with some 2,200 clinical trials underway around the world, and 23 CGTs are expected to be approved in the US and Europe soon. The hope is that when more options enter the market for the same rare diseases, prices will eventually fall, although this is unlikely to happen in the foreseeable future. What all this means is that countries around the world, including Singapore, are grappling with the dilemma of how to pay for these treatments. Are our healthcare systems adequately prepared to integrate and deliver this wave of transformative treatments?

Gene therapies appear to present a financial conundrum for health systems like Singapore’s, which are oriented toward chronic diseases. In a summary of a panel discussion titled, “Gene Therapies: A Wave is Coming - Are Health Systems ready?” hosted by the London School of Economics (LSE), it was noted that existing financing models and annual budget cycles were ill-suited to account for gene therapies; despite a high initial outlay, such therapies may be relatively cost-effective over the long term. Speaking at the event, Rifat Atun, professor of global health systems at Harvard University, said: “There is a complete misalignment between the budget impact of the intervention, the costs that are accrued without the intervention, and the benefits that can be realised with an intervention.” Hence, adequate funding and financing models that recognise the specific characteristics of gene therapies are critical. 

Governments must prepare for this “tsunami” urged Dr Jeremy Lim, associate professor (adjunct) at the National University of Singapore’s Saw Swee Hock School of Public Health. “[It’s] a happy problem, because we can now treat diseases that were previously untreatable.” But he also warned, in an interview with Jom: “If you don't plan for accessibility and inclusion, then the availability of these therapies will only worsen the already rather fragile state of equilibrium.”

In a commentary for CNA, Drs Lim, Chan Hwei Wuen and Ng Qin Xiang said that “[b]etween funding nothing and funding everything, the right balance has to be struck.” They called for a rethinking of the financing approach for Zolgensma, where the government found a way between hard-headed pragmatism and compassion. 

Despite its successes, crowdfunding is considered by many experts as unsustainable. Dr Lim dislikes the practice, because while it benefits the patient, he said that crowdfunding can have serious system implications. If someone has to crowdfund every week, he asked, what would it say about the adequacy of Singapore’s social security systems and health safety nets? “When this erodes confidence in the system…people then say, ‘Well, why the heck do I need to pay tax, it’s not doing anything.’...This starts a very negative spiral,” he told Jom.

At the LSE panel, Atun suggested some solutions: performance or outcome-based schemes that share risk between the payer, provider, and manufacturer; subscription models that spread payments over time; expanded risk pools; and reinsurance models. Other than hefty price discounts from manufacturers, Drs Cham, Lim and Ng proposed similar approaches, including “value-based contracting”, where prices are linked to how well the drugs perform. “It helps to avoid wasting taxpayers’ money on therapies that may ultimately prove ineffective,” they explained. 

Herein lies the many important ethical considerations that accompany the dealing of rare diseases and orphan drugs. At their core is the “clash between the moral duty of non-abandonment and the requirement of distributive justice.” Distributive justice deals with the fair allocation of goods, resources and opportunity among members of society. Considering age as a criterion to allocate health resources expands its ethical dimension. One aspect of this, “microageism”, considers that between extending the life of either a “young” or “old” patient, the former should be prioritised. In “Healthcare Priorities: The “Young” and the “Old’”, the author highlighted two justifications for using age to distribute limited resources. One relates to maximising the health benefit produced by a set of resources. The other appeals to equity, or “egalitarian ageism”, in which it’s fair to prioritise extending the life of the young, since they have lived less time than the old. 

The often competing approaches of the individual and society make it especially complex. Picavet et al (2013) noted that the principles of equity, entitlement and non-abandonment favoured individuals, whereas society may strive to maximise the health of the whole population. That said, it has been argued that society has a moral obligation to protect and assist its vulnerable members. In doing so, it might help to consider Van der Burg & Oerlemans (2018), who called for a clarification of values and distinguished hard impacts, like health, life expectancy, absence of suffering, and soft impacts, like well-being, good care, and a supportive environment, with the former to be managed by policymakers, and the latter by patients and their families.

To Sherry, though, the ethical conundrums that surround her experience as an adult with SMA ring personal. It’s been a constant moral tussle between self-interest and advocacy for the greater good. “While I was trying to appeal for funding in 2022,” she said, “I came across Devdan’s story and thought how unfair it was that he got funding so easily, while I was told I was too old.” But after her campaign launched, she noticed how many people needed help on the same crowdfunding website and thought, “If people donated to me, and not them, how is that fair?”

In the systematic review examining “Ethical Questions Linked to Rare Diseases and Orphan Drugs”, the paper’s authors concluded that an ideal way of dealing with rare diseases would be through global research, development, and risk sharing. But such an approach would require cooperation, not only international and supranational, but across different stakeholders, including medical staff; the pharmaceutical industry; various experts; political bodies; patients and their families. There is currently, however, no internationally accepted model to evaluate and fund rare disease therapies. And this, the authors rationalised might be due to the lack of a globally accepted set of values and rules.

At his May Day Rally speech, Wong warned against succumbing to the harsh inequality in many other countries, and assured Singaporeans that no matter the “treacherous” terrain ahead, if the country continues to progress, then all Singaporeans must do the same. “No one must be left behind,” he said. Singapore ratified the UN Convention on the Rights of Disabilities in 2013, which promotes respect for the inherent dignity of people with disabilities (PWD). In article 10, it also affirms that every human being has the inherent right to life and that all parties should take all necessary measures to ensure its “effective enjoyment” by PWDs on an “equal basis with others”. It might be time to re-examine our commitment to fulfilling this right. In doing so, we might ask, “What is health”, and “Who deserves healthcare?” 

“We should all pay more, either in tax[es] or in premiums, so that the poor child can avail a particular therapy,” declared Dr Lim. “If a child whose parents cannot afford a S$1m therapy dies, but the child of a wealthy family can and this is played out not just for SMA but for 25 other diseases, the social compact that holds Singapore together is going to be very challenged,” warned Dr Lim. But he has high hopes for this affluent country: “As we build a nation, we have gone past survivalist instincts. Now, we can start to look at what truly builds community.”

Sherry turns 25 tomorrow, December 16th. It’s the year that people with SMA type 2 have typically been prognosed to live until. Youth—it’s an age, a quarter of a century in, that most of us might take for granted. But Sherry takes nothing for granted. It’s sobering to think that her life, without treatment, might be extinguished at any time. Her birthday wish: “Perhaps predictable, my biggest wish for my 25th year of survival is to see risdiplam, Spinraza and Zolgensma become financially accessible to SMA patients of all ages in Singapore.” No matter the uncertainties, she remains an advocate for health rights.

Whether she’ll live past 25 isn’t stopping Sherry from imagining a future that includes all the things that she loves—like attending the musical “Hamilton” and Taylor Swift’s “Eras Tour” with friends and family. “But all this will take time and strength,” she said, “which I’m not sure I’ll have without risdiplam once again in my hands.”

Give Sherry a future, donate to her crowdfunding campaign here.

Tsen-Waye Tay is Jom’s head of content. For this piece she's also indebted to Dunlanjali Wakwella, Achintha Pilapitiya, Nabeel Salim Abdat and Syahirah Yakub.

Letters in response to this piece can be sent to sudhir@jom.media. All will be considered for publication on our “Letters to the editor” page.