Helping Children with Rare Diseases

Mr Deputy Speaker, Sir, I beg to move, "That Parliament do now adjourn."

Question proposed.

Mr Deputy Speaker, I declare that I am a medical doctor in private practice.

In a fair and just society, we recognise that people do not get to choose where or when they will be born; that people do not get to choose their parents; that children do not get to choose their genetic background. And today, I am speaking about the challenges faced by children with rare diseases.

Each of us inherits half our genes from our father, one half from our mother. Sometimes, a gene is inherited which does not work the way it is expected to. If there is a copy from the other parent which is working all right, the child may still be fine. But in some cases, a child is doubly unlucky and inherits two copies of a flawed gene. These diseases are rare. Very rare. Not much published literature is available on the Singapore experience, but a 2006 paper in the Annals of the Academy of Medicine (Singapore) reported a 13-year experience at the Singapore General Hospital reference laboratory. From 1992 to 2005, a total of 127 patients were diagnosed, although the tests offered did not encompass all known rare diseases. So, even with better technology, better testing and better awareness, the number would still be small.

One estimate by patient advocacy group Rainbow Across Borders reported in The Straits Times suggests there could be 2,000 to 3,000 rare disease patients in Singapore. However, even if rare, they can be devastating for a child and worrying for the parents. And even if the disease is so rare that the odds of getting it are 0.001%, for the child who gets the disease, the impact is 100%. Let me illustrate with a few examples.

Pompe disease is caused by the lack of an enzyme called acid alpha-glucosidase. It is a rare disease; some estimate it affects between one in 40,000 or as few as one in 138,000. It affects the muscles, including the muscles of the heart. So, children with Pompe disease have weak hearts, get heart failure and can have difficulty moving or breathing.

Without treatment, the classic severe early-onset type of Pompe disease is devastating. The median age at death for children with the severe form of the disease is nine months. Yet, if patients are given enzyme replacement therapy with alglucosidase alpha, a treatment approved by the Food and Drug Administration (FDA) in the United States (US) since 2006, there is a significant reduction in the risk of death of such children of between 79% and 95% reduction in the risk of death. But this treatment is expensive. It has been reported that it can cost as much as $10,000 a week to treat a young child.

Deputy Speaker, Gaucher disease is another rare disease, caused by the lack of an enzyme in the body. In children with Gaucher disease, the liver and spleen will swell and swell. The brain and other organs can be affected, too. In the more serious cases, the children can die before the age of two years. There are treatments for Gaucher disease, which work by replacing the missing enzyme in the body. But again, the treatment can be very expensive, with some estimate at over $20,000 a month.

Another rare disease is bile acid synthesis disorder and it is very rare, even among rare diseases. Fewer than 50 reported cases around the world, in the international medical literature. But we also have a little boy in Singapore who, unfortunately, has this rare disease. There is a drug available, called cholic acid. But the cost was estimated at over $25,000 per month. Even with parallel imports from other markets, the drug cost was reported as about $5,000 per month.

Deputy Speaker, what these rare genetic diseases, and similar diseases, have in common are: (a) the condition is rare; (b) the impact is catastrophic; and (c) the need for treatment is not elective. Indeed, treatment is necessary for the child to continue living a normal life. Or in some cases, to carry on continue living.

Another recurring theme is that the drugs are very expensive and can be very hard to get hold of. The market is small. So, there are no economies of scale for the producer. The costs of research and development can be very high. There are administrative overheads with registering such “orphan drugs” in Singapore, as they call it, and there is little commercial interest in developing such drugs or making them widely available at a good price even if development is successful by the drug company.

Mr Deputy Speaker, the affected children may be few. The affected families may be small in number. But we must not leave behind any Singaporean child. And over the past decade, since the mid-2000s, it is clear that our Government thinking has shifted towards a more inclusive society. Our social policies today include the Workfare Income Supplement, ComCare, Edusave Bursaries and many, many more. Part of a progressive fiscal system where the fruits of progress are shared, especially with the less well-off and the less fortunate.

And we can see that the Ministry of Health (MOH) has shifted, too, over the years, as part of what some have called an evolving New Way Forward. MediFund was set up in 1993 as an endowment fund to help needy Singaporeans with medical bills. MediShield Life was implemented in 2015. It is not just an insurance policy. It is a social policy to help Singaporeans from all walks of life, in case of serious illness with catastrophic medical bills, serious illness. Importantly, MediShield Life helps, regardless of whether one is very old or whether one has a pre-existing condition from birth. There is also the Medication Assistance Fund (MAF) and MAF Plus to help patients access new medications which may be beyond their means but which could still benefit the patients.

Deputy Speaker, at the same time, no policy is perfect. And when a condition is very unusual, like a rare disease, it sometimes does not fit easily into existing policies.

So, what I propose, Mr Deputy Speaker, is for MOH to look at how the Government can extend more support to children with rare diseases. It is a commonsense move and it is not a radical policy shift either. In fact, it is very much aligned with the deeper philosophy that already underpins our current social and healthcare policies.

For example, let us look at risk pooling. MediShield Life and other policies recognised the role of pooling risk, of sharing risk within the cohort or the population. It is part of a broader social policy message that when we share risk, when we pool risk, it means no Singaporean has to walk alone.

And just as MOH has, over the years, improved coverage for rare, catastrophic illnesses, we can and should find ways to better support children with rare diseases – diseases which are also rare and catastrophic. Because the condition is genetic, because it is inborn, there is no direct moral hazard from providing that extra coverage and support.

Furthermore, Deputy Speaker, the private sector insurance market cannot adequately address this issue. Rare childhood genetic diseases are inborn. And insurance companies, being profit maximisers, will act in accordance with their business interests and this means that insurers will cherry pick who they want to cover. In practice, this means private insurance companies limiting or even denying coverage to people with pre-existing conditions, especially rare diseases which are present from birth.

Again, Mr Deputy Speaker, we see a role for the Government stepping in where the free market is inadequate, or where no private sector player can find a compelling, commercial case to support such patients.

Mr Deputy Speaker, as a people, we decide what kind of society we want. In particular, would we want a child with rare diseases to be supported, if that child were our son or our daughter? Would we want the parents to have peace of mind, if the parents were our friends or family? And especially when doing so is a natural progression and harmonisation of existing policies and existing policy philosophy.

Mr Deputy Speaker, I was thinking back to my maiden speech in this House in January 2016. I said back then, “We do not choose our parents or when we will be born. So, we must always be a fair and just society.”

Indeed, Mr Deputy Speaker, we do not choose our parents. We do not choose the genes we inherit from our parents. And so, we must continue to be a fair and just society – for healthy children, as well as children born with rare diseases.

Mr Deputy Speaker: Senior Minister of State Chee Hong Tat.

Mr Deputy Speaker, I thank Dr Tan Wu Meng for highlighting the challenges faced by families with children suffering from rare genetic diseases. As a parent, I empathise with the challenges all parents encounter and the daily struggles we have to go through to care for our children. Parents of children with rare diseases will undoubtedly face even greater challenges. I agree with Dr Tan that as a caring and inclusive society, we should pool our resources together and do what we can to support these families. It is the right thing to do.

Singaporeans have access to good quality subsidised healthcare at public hospitals, specialist outpatient clinics and polyclinics. Many of our subsidies are means-tested to provide more support for Singaporeans with lower incomes. And this is part of our progressive social policy and safety net.

On top of Government subsidies, we have MediShield Life, which provides universal protection for all Singaporeans against large hospital bills and costly outpatient treatments through risk-pooling. The Government provides means-tested subsidies to help Singaporeans pay for their MediShield Life premiums. We also offer financial assistance for low-income Singaporeans through Additional Premium Support to ensure that no Singaporean will lose coverage because he is unable to afford his premiums. So, MediShield Life, as Dr Tan pointed out, is not just an insurance scheme, it is a social policy which reflects our values as a community to build a fair and just society.

For needy families who still require financial assistance after Government subsidies and MediShield Life payouts, and using their MediSave and other personal savings, we have MediFund as a further safety net to ensure that no Singaporeans will be denied access to appropriate care due to their financial situation.

These social policies and assistance schemes are possible because we have grown our economy over the years, we have generated the required resources and we have managed our system efficiently. Now, as we face an ageing population in Singapore, we must continue to pay careful attention to these important areas.

With these policies in place, the vast majority of Singaporeans are able to pay for their medical treatments and drugs. Yet, as Dr Tan has pointed out, even the best policies cannot address all circumstances. There will be rare diseases where treatments and drugs are very expensive, and our insurance schemes, our subsidies will not provide adequate coverage for the patients and their families.

When we encounter such cases, our public healthcare institutions will do their best to help. There was a recent example in one of our public hospitals – a patient by the name of Emily who was diagnosed with Pompe disease since she was a teenager. Emily needs to visit Changi General Hospital (CGH) every fortnight to receive an infusion of her missing enzymes, which is a costly drug. Combined with other aspects of her treatment, such as consultations and tests, the monthly cost adds up to more than $50,000. Emily's father is a technician and her mother a homemaker and she has a sister who is still schooling. Financially, it is very challenging for the family.

CGH reached out to the family and brought in other parties to help. Emily received subsidies for consultations and investigations, and the hospital helped her apply for funding under the Medication Assistance Fund. These covered a significant proportion of the infusion cost. The hospital also engaged a pharmaceutical company, which kindly agreed to extend financial assistance to further defray the cost of the medication she needs. More recently, after discussing her situation with the hospital, her employer helped her to claim part of her treatment cost from their group insurance policy. So, through the combined efforts by CGH, the pharmaceutical company and her employer, Emily’s monthly healthcare expenses were reduced from more than $50,000 to less than $500. I am heartened to know that Emily is coping well with her treatment.

Another example is Chloe, a little girl with the same condition as Emily. Like Emily, she needs to visit the hospital regularly to receive the infusion of enzymes. Her monthly treatment cost is about $40,000. This is another example of how multiple parties have come together to support the family. Government subsidies were provided for her treatment, the family’s insurance policy covered the post-subsidy costs of Chloe’s inpatient treatment, and additional Government and hospital assistance supported the other aspects of her treatment, such as mobility devices and consumables. Together, these helped to reduce Chloe’s healthcare expenses from $40,000 per month to less than $500 a month.

Dr Tan also mentioned in his speech a young boy, the name of this boy is Christopher, who was diagnosed with a rare disease called bile acid synthesis disorder. We want to help the family. The public hospital which is looking after Christopher is KK Women’s and Children’s Hospital (KKH) and they are working with the manufacturer to try and bring down the cost of the drugs for the family. I have also asked my colleagues in MOH and KKH to assist Christopher’s family and find a way to help them to reduce their out-of-pocket expenses.

Mr Deputy Speaker, while we have many positive examples of how dedicated healthcare workers and caring individuals in our society are helping patients who are facing difficulties, we recognise that there are still families with children suffering from rare diseases who are struggling and are worried how they can cope with their healthcare expenses. This is not an easy challenge to solve because many of the cases have unique circumstances. The needs are diverse. What is clear is that, as a caring and inclusive society, we must do our best to find practical ways to help our fellow Singaporeans who are in these difficult situations. This requires a collective effort from the public, private and people sectors.

MOH will review how we can better support children with rare diseases and their families. We are studying different options, including the use of insurance and discretionary funds, like MediFund and Medication Assistance Fund, to reduce their out-of-pocket expenses. Another possibility is to set up a separate fund which our public healthcare institutions can use on a discretionary basis to help children with rare diseases whose treatment costs remain unaffordable in spite of subsidies, insurance and financial assistance. Contributions to the fund can be made by the Government but also by companies, charities, community groups and individual donors, to encourage different stakeholders in society to chip in and help these families and help these patients.

The Ministry will study the different options carefully and we will come up with a proposal later this year. By harnessing the collective giving from all parts of our community, we can help these families and create opportunities to encourage philanthropy and build a more caring society.

Question put, and agreed to.

Resolved, "That Parliament do now adjourn."

Adjourned accordingly at 7.07 pm.