What are
Rare Diseases?

Donate today

A rare disease is one which affects fewer than one in 2,000 patients. Many rare diseases are fatal. Most rare diseases are genetic, directly caused by changes in genes or chromosomes that affect the body's ability to produce enzymes or biochemicals needed for the body to function normally.

Rare diseases can affect people of all ages but they are often diagnosed at birth or during early childhood. Without treatment, most conditions can be life-threatening. About one-third of patients with rare diseases globally do not live beyond five years old.

There are no treatments available for most rare diseases. But for a few, effective medicines are available which can replace the missing enzymes or biochemicals. In such cases, with early treatment, patients' lifespans and quality of life will be greatly improved, allowing them to live relatively normal lives.

The medicines usually have to be taken for the patients' entire lives and can be very expensive. This places a heavy financial burden on the patients and their families.

Working together to support
patients with Rare Diseases


About Rare Diseases

More than 7,000 rare diseases globally

of rare diseases have genetic origins and are present for the patient's entire life

Symptoms begin in infancy or childhood in 50% of rare diseases

Effective treatments are only available for about 5% of rare diseases

30% of children will die before their fifth birthday unless early treatment is given

Rare Diseases in Singapore

2,000-3,000 people are estimated to have a rare disease in Singapore

Approximately 700 of them are children

Why financial assistance is needed

Some medicines can cost each patient more than S$200,000 a year

Patients will need these medicines for life which is a significant financial burden

Funding support will ease financial burden and stress on patients and their families

About the Rare Disease Fund

Just as every rare disease is unique, so is the patient. Patients with rare diseases can have a wide range of care needs, from regular medical consultations to home care support. While there are existing financial schemes available to help patients with some of these needs, more support is needed to help with the cost of their expensive medicines.

WHAT IS THE RARE DISEASE FUND?
The Rare Disease Fund (RDF) is a charity fund that seeks to provide long-term financial support for patients with rare diseases requiring treatment with high cost medicines. The RDF is managed by KK Women's and Children's Hospital (KKH) Health Fund (part of SingHealth Fund). It has the support of the Ministry of Health and provides a platform for the community and Government to come together to support patients with rare diseases over their lifetime.

HOW DOES THE RDF SUPPORT PATIENTS?
The RDF provides long-term financial support for patients with rare diseases requiring treatment with high cost medicines. It is a last line of support for patients after other financial schemes.

WHICH MEDICINES FOR RARE DISEASES ARE COVERED BY THE RDF?
The RDF provides funding support for patients with very rare conditions who require medicines which are proven to be clinically effective and can extend a patient's lifespan, but are high cost and pose a financial burden on them. See here for details of the criteria.

Considering funds raised to date, the RDF Committee has approved the following medicines for inclusion under the RDF. These medicines are used to treat conditions that generally have symptom onset during childhood.

Condition Medicine(s)
Primary bile acid synthesis disorder Cholic acid
Gaucher disease (Type 1 or 3) Imiglucerase (Cerezyme)
Velaglucerase alfa (VPRIV)
Taliglucerase alfa (Elelyso)
Hyperphenylalaninaemia due to tetrahydrobiopterin (BH4) deficiency Sapropterin dihydrochloride (Kuvan)

Over time as more funds are raised, the RDF can be expanded to cover more medicines for other rare conditions.

WHO MAKES DECISIONS ABOUT THE RDF?
A voluntary RDF Committee, comprising members from the community, has been set up to approve the list of medicines covered under the RDF, assess patient applications for financial support and exercise stewardship over the Fund.

The RDF Committee is advised by a panel of local clinical specialists with experience in the treatment of rare genetic diseases, who provide expert opinion about which medicines meet the eligibility criteria for inclusion in the RDF.

WHO ARE ELIGIBLE?
Singapore citizens seeking treatment for their rare disease with any of the medicines covered under the RDF at any Public Healthcare Institution (PHI) may apply for RDF support through the medical social workers.

Each application will be considered by the RDF Committee on a case-by-case basis, taking into consideration the financial means of families and whether other financing support is available. The level of co-payment by the patient will be commensurate with the family's means. Patients will have to meet specific clinical criteria for initial or continuing treatment with RDF medicines to qualify for financial assistance from the RDF.

Which medicines will be supported by the RDF?

Medicines treating rare diseases must meet specific criteria to be eligible for inclusion in the RDF including all of the following:

  1. Medicine is registered by the Health Sciences Authority (HSA) or a reputed international regulatory authority (Food and Drug Administration (FDA, USA) and/or European Medicines Agency (EMA)) for the condition assessed (i.e. medicine has proven therapeutic modality).
  2. Medicine treats a rare but clinically defined genetic condition that is chronically debilitating or life-threatening.
    1. There is acceptable evidence that the condition causes a significant reduction in either absolute or relative age-specific life expectancy or quality of life for those suffering from the condition.
  3. There is acceptable evidence that the medicine is likely to substantially extend a patient's lifespan and quality of life as a direct consequence of its use.
  1. There is no cheaper alternative option (including non-drug therapy) for the condition.
  2. The medicine is not indicated for the treatment of other conditions, or if it is, the cumulative prevalence across all indications still falls within the definition of rare (<1,600 patients across all indications).
  3. The annual cost of the medicine would constitute an unreasonable financial burden on the patient and/or their family or carer.

Living With Rare Diseases

MEDICINE COSTS MORE THAN S$7,000 A MONTH

Christopher has an extremely rare condition called bile acid synthesis disorder. There are fewer than 50 reported cases around the world. His condition was discovered when he was four months old when his parents noticed that a scratch on his nose and a prick from a blood test left his nose and finger bleeding for more than 30 hours. Christopher's eyes and skin were also jaundiced, and he was slow to gain weight, leaving him on the bottom of the growth chart.

For his parents, the pain was not just seeing their first born unwell, but in watching Christopher struggle and being bruised from blood tests and getting so hungry he reached for the pacifier for the first time. They were told then that his liver was enlarged and had some scarring.

This rare disease Christopher has interferes with the production of bile acids such as cholic acid, which help the flow of excretion of bile from the liver and assist in the absorption of fat and fat-soluble vitamins from food. Without cholic acid, toxic bile acids will build up and damage the liver, ultimately causing it to fail, and requiring early liver transplantation.

The medicine Christopher needs costs at least S$7,000 a month. As he will require a higher dose of medicine as he grows older and gains weight, the family worries each day about their ability to continue to afford Christopher's treatment, and for his financial means in the future, as he has to be on medication for life.

Thankfully, the medicine has worked. Today, Christopher is a bundle of joy. He squeals, laughs, smiles and has a lot of personality. He is a very special mischievous two-year old who loves to have fun, loves music and big open spaces.

He has a long road ahead, but for Christopher and his parents, each day he is alive is a blessing as they celebrate every small milestone.

FORTNIGHTLY TREATMENT FOR LIFE AT S$24,000 A MONTH

Eleven year old Zecia has Gaucher disease, a rare neurodegenerative condition where her body does not produce an enzyme to get rid of a specific type of fat. This fatty substance can then build up in various organs causing damage. Only a handful of children in Singapore have this rare ailment.

When Zecia was first diagnosed, her liver and spleen were extremely enlarged and hence her abdomen was severely distended. This affected her ability to eat and her growth was poor. Her bone marrow was also not functioning well, leading to easy bleeding and a low blood count. Her father recalled how the disease caused her stomach to become so bloated as a toddler that she couldn't balance well and she kept falling down.

It was a difficult journey for the family. As Gaucher disease was very rare, it took more time to arrive at a definitive diagnosis. The family found the condition highly unfamiliar and had to contend with the high cost of the treatment. To manage her condition, Zecia requires fortnightly enzyme replacement therapy for life, which costs her family more than S$24,000 a month. Without the treatment, her liver and spleen will swell progressively and affect her health and life. Further accumulation of the fatty substance in her bone marrow and brain can eventually prove fatal.

With treatment, Zecia's liver and spleen have reduced in size and her abdomen is no longer distended. Her bone marrow is now functioning normally. She is now able to attend school like any other child.

As the medicine is expensive, the family is unable to afford the treatment on their own. Futhermore, Zecia has two siblings, and a grandfather who is receiving treatment for cancer. Fortunately, the hospital managed to source financial assistance for the family to reduce the burden.

However, Zecia will have to receive treatment for the rest of her life. As she grows bigger and gains weight, she will need a higher dose of medicine. That means that her monthly medical bill will grow over time. Her family worries whether she will be able to continue to receive treatment in the long term, without a stable source of financial support. But left untreated, she may not survive.

For now, Zecia is enjoying every moment of her childhood. Her father says she loves to draw and enjoys doing paper craft, and hopes to be an artist when she grows up.

Every Dollar Counts

Your gift to the RDF is life-giving. You will be providing financial assistance for the expensive medicines that patients need to allow them the opportunity to live a relatively normal life.

All donations are welcome, and no donation is too small for the RDF. Your kind contribution will be a great source of help and support to the patients and their families as they battle these diseases.

For every dollar of public donation, the Government will match $3, boosting the impact of the fund.

Tax deduction for all eligible donations made from 1 January 2016 to 31 December 2021. For more information, please refer to www.iras.gov.sg

Donate today

RDF Donor Wall

We are honoured to recognise the following donors and partners of the Rare Disease Fund for their support. On behalf of the beneficiaries, thank you for giving them the Hope for a Lifetime.

$5,000,000 and above Far East Organization
$1,000,000 to $4,999,999 Ho Bee Foundation
National Healthcare Group Fund
National University Health System Pte Ltd
Singapore Health Services Pte Ltd
$100,000 to $999,999 The Grace, Shua, and Jacob Ballas II Charitable Trust
$10,000 to $99,999 AIA Singapore Private Limited
AXA Insurance Pte Ltd
Mount Alvernia Hospital
One-MOH Challenge
Raffles Medical Group
$1,000 to $9,999 Benjamin Koh
Chan Beng Seng
Derrick Heng
Edward Teo
Eu Oy Chu
Kelly Wong
Lim Siew Wah Grace
Mak Seck Wai, Kenneth
Norman Chong
Viking Open Scout Group
With special thanks to Balaji Sadasivan Endowment
Managed by Temasek Foundation
Community Chest
President's Challenge
Tote Board

*The list is updated as of 27 June 2019.
We are grateful to everyone and we apologise if we are unable to include every individual's / company's name in time.