3 November 2019, Singapore – The Rare Disease Fund (RDF) Committee has approved the expansion of the coverage of the RDF to include a sixth life-saving medicine, Alglucosidase alfa (Myozyme), which treats Pompe disease. Applications for financial assistance from the RDF is now open for patients with Pompe disease.

The RDF was launched in July 2019 by the Ministry of Health and SingHealth Fund as a charity fund that combines community donations and 3-to-1 government matching contribution to support Singapore citizens with specific rare diseases who require treatment with high-cost medicines. Please find details of the RDF in Annex A.

Pompe disease (also known as acid maltase deficiency or glycogen storage disease type II) is an inherited disorder which is caused by mutations in a gene that makes an enzyme called alpha-glucosidase (GAA). Normally the body uses GAA to break down glycogen (sugars). Deficiency of the enzyme leads to the accumulation of glycogen within certain organs and tissues, especially cardiac and respiratory muscles, which impairs their ability to function normally. The incidence of Pompe disease is approximately one in 40,000 births, and there are estimated to be up to 10,000 people worldwide with the condition.

Mrs Laura Hwang, chairperson of the RDF Committee said, "We are grateful for the strong community support in aid of patients with rare diseases. Through the generous donations to the RDF, we are able to expand the RDF to include a sixth life-changing medicine to treat patients with Pompe disease. This is only made possible through the generosity and support of the community. We hope to receive further contributions to expand even further the conditions covered by the Fund to support more patients".

With this latest expansion, the RDF now covers six life-saving medicines which treat four rare disease conditions. These are:

Singaporeans undergoing treatment at public healthcare institutions for these rare diseases who require additional financial assistance may apply through the medical social workers at their public healthcare institution. To date, two individuals are receiving RDF funding for their medication.

The inclusion of Alglucosidase alfa (Myozyme) was made possible following public support and donations to the RDF since its launch in July 2019. About $20 million have been raised to date. Together with the government seed funding and matching of 3-to-1 for public donations, the RDF currently has close to $90 million. The list of major donors who supported the RDF is listed in Annex B.

Donations may be made through www.giving.sg/rare-disease-fund. Donations will be eligible for a 250% tax deduction. More information on the RDF is available on www.kkh.com.sg/rarediseasefund.

ANNEX A

The RDF seeks to provide long-term financial support for patients with rare diseases requiring treatment with high cost medicines. The Fund aims to encourage community donations towards supporting Singapore citizens with rare diseases who are treated in our public healthcare institutions. This will give support to patients whose treatment costs remain unaffordable even after government subsidies, insurance and financial assistance (such as Medication Assistance Fund, MediFund) have been provided, due to the high-cost nature of the treatments. The RDF is managed by KK Women's and Children's Hospital (KKH) Health Fund (part of SingHealth Fund).

A RDF Committee has been set up to exercise stewardship over the Fund, determine which medicines are eligible for RDF support and assess patient applications in consultation with clinical experts. Applications for financial assistance from the RDF has commenced for patients for these four conditions. Singaporeans undergoing treatment at public healthcare institutions for these rare diseases and require additional financial assistance may apply through the medical social workers at their public healthcare institution. Each application will be considered by the RDF Committee on a case-by-case basis. To date, the RDF Committee has evaluated and approved applications from two patients.

More information on the RDF is available on www.kkh.com.sg/rarediseasefund.

ANNEX B

Founding Donors to the RDF

Balaji Sadasivan Endowment (Managed by Temasek Foundation)

Far East Organization

Ho Bee Foundation

National Healthcare Group (NHG)

National University Health System (NUHS)

Singapore Health Services (SingHealth)

The Grace, Shua, Jacob Ballas II Charitable Trust

T-Touch

Major Donors to the RDF

Temasek

Tsao Family Fund

Partners Supporting the RDF

Community Chest

President's Challenge 2018

Singapore Totalisator Board