Improving Access to and Sustainability of the Rare Disease Fund

ADJOURNMENT MOTION

The Leader of the House (Ms Indranee Rajah): Mr Speaker, Sir, I beg to move, "That Parliament do now adjourn."

Question proposed.

Mr Deputy Speaker: Miss Cheryl Chan.

Improving Access to and Sustainability of the Rare Disease Fund

7.16 pm

Miss Cheryl Chan Wei Ling (East Coast): Mr Deputy Speaker, yesterday was Nurses Day. I wish to take this opportunity to express my heartfelt thanks to all our healthcare workers for the tireless work they have been doing for us all these years and, in particular, during this pandemic. It has not been easy, but we have been very appreciative of all that they have done.

Mr Deputy Speaker, thank you for allowing me to speak on the Rare Disease Fund (RDF).

Jointly established by the Ministry of Health (MOH) and SingHealth Fund in July 2019, RDF aims to ensure affordability in treatment costs for Singapore Citizens with rare diseases through providing supplemental financial assistance for those who still require it after Government subsidies, insurance and financial assistance, with the premise that treatment is sought at public healthcare institutions.

According to the Agency for Care Effectiveness (ACE), there are over 2,000 Singaporeans with rare diseases and 700 of them being children. Treatment and medications for rare diseases are for life, with expenses ranging from tens of thousands per month to over half a million per year. Hence, any help for these families is welcomed and much appreciated.

Through a $3-to-$1 matching donation from the Government to the RDF, it is with the hope that this would encourage the larger community including philanthropists, corporates, community groups and individuals to join in to support these patients in fostering a more caring and inclusive society. In just five months after the introduction of RDF, the initial fund of $70 million has increased to $90 million. However, a year since November 2019, it was reported by MOH that the value of the RDF had only increased from $90 million to $93 million, with one's hypothesis that a comparatively slower increase could be due to the economic fall-out from COVID-19, which saw a corresponding decrease in public donations.

While I understand that the objective of the RDF is to sustainably support beneficiaries, giving them the assurance that financial support will be available over their lifetime, but the number of beneficiaries since the introduction of the RDF appears to be small.

Today's Motion seeks to explore ways to increase the uptake of the RDF through more awareness, expanding the scope of eligible conditions and drugs under the RDF. Further, what can be done to ensure sustainability of the RDF through continued encouragement of donation and the involvement of medical insurers and drug manufacturers?

In response to a Parliamentary Question by Dr Tan Wu Meng, MOH replied that, currently, only six medicines that treat the four rare conditions are eligible for the RDF, with a few more drugs under consideration. Six beneficiaries are supported with a total of $2 million and, by April 2021, the number of beneficiaries has increased to seven.

While I can understand the RDF had, indeed, managed to assist seven beneficiaries, I cannot help but worry about the other 2,000 Singaporeans with rare diseases and how they are being supported. Thus, I would like to raise some points for MOH to consider.

First, potential to increase the uptake of the RDF. With only eight applications received since the introduction of the RDF, it begs the question, are we doing enough through the RDF? There are possibly reasons why people do not apply: from not fulfilling the eligibility requirements, to other funding sources available, lack of awareness of the existence of the RDF, or some reasons we may not even be aware of.

Regarding the eligibility requirements, I would call for an expansion in scope in the list of drugs and conditions eligible for the RDF. But before that, the question is, are there sufficient awareness on the existence of the RDF amongst those who are suffering from rare diseases? I understand that groups like the Rare Disorders Society (Singapore) and Rainbow Across Borders help in raising awareness and providing support to patients with rare diseases. But could there be further touchpoints that could allow those with rare diseases to know more about and benefit from the RDF? For example, this could be more widely shared through the community level and family touchpoints like the general practitioners, pediatricians and polyclinics.

Second, the list of conditions that are eligible for the RDF. For the current four eligible conditions for the RDF, there is a strict process in assessing eligibility. Understandably so, as we want to ensure that those who really need the funds can obtain the funds and on a long-term basis. However, for those with rare diseases beyond these four eligible conditions, they face the strictest of processes and a flat rejection. One example would be the Ding family whose young daughter suffers from Turner Syndrome, a condition, currently, that is not eligible and, despite their best efforts in appealing for coverage, they were rejected.

There are, presently, over 6,000 types of rare diseases in the world and, yet, only four types are eligible for the RDF. While I can understand that it is not feasible to be comprehensive and exhaustive to cover all rare diseases for eligibility, could we do more to expand the current scope of conditions?

The sad truth is that some of these rare diseases that affect young children currently do not have any treatments that are readily available and, even if they do have some form of preventive measures, the measures would, usually, result in death. Hence, it is very important that for conditions with appropriate treatments and medications, we provide sufficient resources and support to ensure that these young children who need it, get it. With that, I would like to propose to include coverage of the following two conditions: neuroblastoma and Krabbe Disease. These two conditions have been listed under the 10 most common rare diseases among young children in the world today.

Neuroblastoma is one of the most common childhood cancers found in children under the age of five. Neuroblastoma accounts for about 5.3% of all childhood cancers diagnosed in Singapore annually, equivalent to about 15 children from March 2017 to February 2019, and with the cost of treatment going upwards of $1 million and some of the specialist treatments not available in Singapore.

Meanwhile, Krabbe Disease is a hereditary genetic disorder and 43 Singaporeans are known to have this. The younger the disease develops in the individual, the more severe it would be as they grow to adulthood.

Third, the list of drugs that are eligible for the RDF. With reference to the United States, there are close to 30 million people who suffer from some form of rare disease. The US Food and Drug Administration (FDA) has approved 22 novel drugs in 2019 alone just to combat against a variety of rare diseases.

While it is understood that the RDF is currently considering more drugs for eligibility, could the Minister shed more light on the process and timeline of the type of drugs under evaluation? In order to expedite approval and eligibility to allow more beneficiaries for this fund, could there be an opportunity for the RDF to collaborate with overseas governments and institutions to exchange research and information? This will better accelerate local understanding of different rare diseases and the corresponding drugs and treatments here.

Current eligibility for drugs under the RDF follows a rigorous process. I am not against a rigorous process, as, rightfully so, this matters lives. But could some relaxation on the criteria for drug approval be exercised to include palliative care as well? For the Krabbe Disease, there are, currently, no cures with the existing treatments. What is available, usually, involves the relief of the severity of the symptoms as well as supportive care. Only a limited number of success in infants who were given stem cell transplants at an early age had been known at this stage.

For the 43 Singaporeans with Krabbe Disease, they have to shoulder the high medical costs year after year. If the drug eligibility could be relaxed, I believe this would bring some respite and cheer to them and help them to continue life with zest despite the perennial looming thought that the disease has no cure and the treatment can only allay their symptoms.

Looking at our nation with an ageing population and the likely exponential budgets we are projecting for future healthcare costs, I can only imagine the treatment costs for rare diseases and, consequently, the RDF as no exception. The following two points would explore how to ensure the sustainability of the RDF and the role of medical insurance and drug manufacturers in achieving this goal together.

So, first, the efficacy and sustainability of the RDF. Apart from the seed fund, the RDF is heavily reliant on community donations matched by the Government grant, with eventual reliance on investment returns. However, with the call to increase the scope of conditions and drugs included, it is inevitable this will lead to more funds required if the number of beneficiaries grows. Herein lies the question: how do we ensure sustainability of the RDF, so that the RDF would be able to last the beneficiaries a lifetime and also account for an increase in the number of recipients?

As last year has shown, community donations are very much dependent on economic conditions. However, those suffering from rare diseases cannot stop treatment and wait for the conditions to improve, regardless of economic conditions. So, how can we encourage a more sustainable and recurring model of community donations?

Besides raising awareness on the RDF for more fundraising efforts, can corporate donations that span over a period of five to 10 years be explored with differentiated tax treatment? In so doing, it could increase donations from corporates or family offices. With some certainty in donations, it could help to act as a bulwark against the smaller community donations that are more dependent and fluctuate according to economic conditions.

Given the limited drugs that are available for each rare disease, the drug manufacturers can consider, as their social effort, to pay it forward and balancing the cost offset with other commercial drugs in their portfolio by subsidising the cost of trial or unique medication, or what they call "novel drugs" for patients. This will greatly assist the patients as they are restricted by options, to begin with, and unable to bear the cost of standard commercial models of drugs and treatment. So, this is probably a critical way forward for a sustainable outcome.

Finally, on medical insurance for those with rare diseases. Many of those with rare diseases do heavily rely on insurance to manage medical costs. However, the knotty situation is that many insurance products excludes pre-existing conditions, and even if the insurance does allow for some pre-existing conditions, it comes at a very high premium.

The pricing of insurance premiums is a delicate one. Through a risk-pooling mechanism, insurers seek to diversify the risk of payout. Generally, in recent times, insurance premiums have been increasing, with increased consumption of medical services as an oft-cited reason. While active measures have been taken to keep the insurance premiums in check, this will place an even heavier toll on those with rare diseases; if only they are covered in the first place.

In order to allow for better access to medical insurance and a more manageable and sustainable insurance premiums for those with rare diseases, can insurance companies consider providing coverage under a higher co-payment condition for those with rare diseases? Alternatively, could the RDF be explored to partially offset the cost for the insurance premium in order to assist them?

Having medical insurance does, indeed, help take some financial and mental pressure off the individual and their family. This may allow them to better focus on improving and have better health outcomes. But as a country, it is pivotal that we try our utmost to support them through the RDF and other means and jointly work towards our vision of creating a more caring and inclusive society.

To conclude, Mr Deputy Speaker, the RDF is indeed necessary and very helpful for those with rare diseases. After all, every bit counts. However, it is timely that we ask whether RDF has achieved its initial objectives and what more can we do to make it more inclusive. I look forward to the Senior Minister of State's reply.

Mr Deputy Speaker: Senior Minister of State Koh Poh Koon.

7.30 pm

The Senior Minister of State for Health (Dr Koh Poh Koon): Mr Deputy Speaker, Sir, having a child born with a rare disease can be a life-changing event for an entire family. In addition to the suffering that the child goes through, it also creates a lot of emotional distress, uncertainties and anxieties for the parents and can impose greater financial as well as physical demands in caring for the needs of the child. The high cost of treatment adds to the overall burden.

As a doctor, I have walked this difficult journey with many patients and their families before, especially when they had an unexpected diagnosis being revealed to them.

So, I would like to thank Miss Cheryl Chan for her passionate speech and proposals relating to the Rare Disease Fund (RDF) in support of these families. The Government shares her concern for individuals with rare diseases where medication is very costly and required for life. This was why, in 2019, the Government provided a generous matching of $3 for every $1 of public donation to establish the RDF as a charity fund to support Singapore Citizens diagnosed with rare genetic diseases.

The RDF supports high-cost, life-saving treatments for very rare genetic conditions where the medicines, firstly, have a proven therapeutic efficacy; secondly, are able to substantially extend a patient's lifespan and maintain a quality of life as a direct consequence of its use and; thirdly, be reasonably priced as when compared to its price in other countries.

Rare diseases with onset in childhood are prioritised initially as these patients with conditions, such as rare diseases, can often go on to lead full and meaningful lives if they are diagnosed early in the course of their disease and receive the required treatment promptly.

Because these treatments tend to be lifelong, the RDF is structured as an endowment fund to ensure that only the interest income generated is used to fund the treatment and the capital sum continues to generate a sustainable stream of income to ensure patients enrolled into the programme will have assured support.

While welcoming the set-up of the RDF, Miss Cheryl Chan has highlighted that the number of patients, conditions and medicines covered by the RDF is low, potentially leaving some rare diseases patients unassisted.

Let me, first, clarify three common misconceptions.

First, this is not a situation in which eligible patients are not applying for the RDF due to a lack of awareness. In fact, as the costs are high, patients with rare diseases, generally, seek care in the public sector. We can, therefore, easily identify patients with rare diseases covered by the RDF and invite those who require financial assistance to apply. Furthermore, many of the public sector specialists who treat genetic disorders are on the expert panel that advises the RDF Committee on the rare disease medicines that should be considered for coverage by the RDF. Some patients with rare diseases have insurance coverage or alternative financial means and need not apply for RDF grants. For those who are in financial need, medical social workers in the public healthcare institution and the patient’s specialist will assist the patient to apply for RDF assistance.

Second, when we first set up the RDF, it was established that there were up to 2,000 patients with rare diseases in Singapore. However, the majority of these patients have conditions where no treatments are available to correct the genetic deficiency and life expectancy may be limited with poor quality of life; or where treatments were relatively low cost and, hence, can already be supported under current funding sources.

We had, therefore, anticipated there could be up to 15 medicines that needed RDF support, based on experiences of equivalent rare disease funds from overseas. In two years, we have covered six medicines. We need to do more, but it is tangible progress in a short time of two years. I would like to thank the RDF Committee for what has been achieved and for the work that is to come to bring support to more patients.

Third, Miss Cheryl Chan also proposed to list more rare diseases and medicines on the RDF. We should only do so if the rare diseases have effective treatments that can meaningfully extend the life expectancy. But 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.

In the last financial year alone, the RDF made grant payouts of around $1 million. Many of the beneficiaries are children. They will grow and increase in body weight, which means that the medication dosage required, along with the cost of treatment, will go up commensurately. For example, in this financial year, RDF 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.

In the coming years, the annual sum in grant payouts required would increase significantly just to support these same patients. We have to cater for these increases with age when we decide how many patients the RDF can sustain, in order that the supported patients can be assured that they can continue to afford their medications once they are started on treatment.

The reality is that this is a large sum for a very small number of patients. But that is how expensive rare diseases medicine costs and why we need all of us – the Government, the community – to work together to help this group. Without more donations, it will be very difficult for the RDF to list more medicines or to support new patients.

This, therefore, brings me to the crux of what will allow RDF to help more patients with rare diseases – raising more donations. Miss Cheryl Chan has also recognised this in her speech. It is not easy raising funds for patients with rare diseases. It was in recognition of this difficulty that the Government decided to match donations $3-to-$1, so that every donation dollar is stretched further and also to help encourage donors to come forward. In addition, all donations to the RDF are also eligible for a generous level of tax deduction at 250% and the Government also funds all administrative costs of the RDF so that donors are assured that every single cent of their donation goes to the patients. This also applies to all donations made by corporations and family offices.

Fully aware of the importance of raising more funds, the RDF Committee, together with its secretariat at KK Hospital, is doubling up their efforts to raise funds. It has and will continue to approach high networth individuals, foundations as well as corporate sponsors, as suggested by Miss Cheryl Chan. It also stands ready to collaborate with organisations to help raise funds for the RDF, as it has done so on a few occasions in the past. It is also exploring how it can develop a base of supporters who can reach out to their networks to fundraise.

The RDF Committee will appreciate all the help that it can get. If Members of this House would like to help raise funds for the RDF, please let me know and I will gladly put you in touch with the RDF Committee.

Let me now turn to a few other proposals made in Miss Cheryl Chan’s speech.

First, Miss Cheryl Chan proposed to include two specific rare diseases: neuroblastoma and Krabbe Disease. Neuroblastoma is a form of cancer. Treatments for cancer, including neuroblastoma, are not covered under the RDF as mainstream financing is available through MediShield Life currently. Those who need further assistance can apply for MediFund. As for Krabbe Disease, there are, unfortunately, no treatments available to slow disease progression today, with most patients succumbing by age two. The current available medicines are for palliative and supportive care only, which would not fulfil the inclusion criteria for RDF of being able to substantially extend a patient’s lifespan. Patients who require additional assistance for palliative care are already supported via mainstream financing schemes, such as subsidies and our 3M of MediSave, MediShield Life and MediFund.

I would like to assure Miss Cheryl Chan that the RDF Committee is well advised by the Rare Disease Expert Group on the rare disease drugs that should be prioritised for coverage. Currently, the RDF Committee has a pipeline of five other medicines for three more rare diseases that stand ready to be added to the RDF list once sufficient funds are raised.

Miss Cheryl Chan also suggested going beyond the RDF and leveraging more on insurance. I note that Miss Cheryl Chan is familiar with the practice among private insurers of not covering pre-existing illnesses.

Where coverage of pre-existing illnesses is provided, it comes with the loading of the insurance premium, which can be significant. The benefits and premiums of private insurance products, such as Integrated Shield Plans (IPs), are determined by insurers, based on their own commercial and actuarial considerations.

In doing so, insurers have to strike a balance between providing more comprehensive coverage and ensuring the affordability of premiums, as well as the sustainability of their insurance fund. As private insurance is optional, the insurers also need to guard against adverse selection that can affect sustainability.

Miss Cheryl Chan may, however, be pleased to know that some insurance products, such as IPs, do not set exclusions for rare diseases, unless these are pre-existing conditions. In addition, our universal national medical insurance, MediShield Life, covers all Singaporeans from birth, with no disease exclusions, including all rare diseases, subject to the relevant claims limit. Where there are claims limits to payouts such that there remains an out-of-pocket payment for the patient, the RDF would consider an application to support this remaining out-of-pocket payment.

In conclusion, establishing the RDF was but a first step in assisting patients with rare diseases. The task remains ahead of us, as a community, to muster all the necessary resources and assistance that we all agree are needed for this group of fellow Singaporeans.

I would like to thank Miss Cheryl Chan for calling on both the community and the Government and even the pharmaceutical companies to provide stronger support towards patients with rare diseases. I would like to reiterate the Government’s commitment towards supporting these patients with rare diseases. I encourage everyone to donate generously, so that more can benefit from the RDF.

Question put, and agreed to.

Resolved, "That Parliament do now adjourn."

Adjourned accordingly at 7.41 pm.