Many rare diseases—defined as one that affects fewer than 1 in 2,000—are fatal. Often, they don’t even have a viable means of treatment, which means that a third of patients with rare diseases don’t live past the age of five.
For those that do have medicines available, they can cost hundreds of thousands of dollars—and must be taken for life. It exacts a heavy toll on patients that’s equal parts financial, emotional, and physical.
But these treatments also give them a shot at a normal life.
On July 2, the Rare Disease Fund was launched to alleviate the financial burden that many patients with rare diseases face. It is jointly formed by the Ministry of Health and SingHealth Fund.
The RDF currently supports the funding of five different medicines that treat three diseases.
One of these diseases is bile acid synthesis disorder, or BAS. There are fewer than 50 reported cases worldwide, and little is known about its prognosis, other than the fact that the disease, if left untreated, is fatal.
Other illnesses that the RDF supports include tetrahydrobiopterin (BH4) deficiency, and Gaucher disease. Of the 2,000—3,000 people that have rare diseases in Singapore, 700 are children.
Such illnesses often occur during childhood and will be present throughout the patient’s entire life. And since many of these conditions occur due to a deficiency in biologically-occurring enzymes or chemicals, the dosage and cost of treatment often grows along with the child.
The Fund hopes to expand the number of medicines and the range of illnesses it can support as it matures.
To help bolster the fund, the Singapore government has pledged to contribute $3 for every $1 that the public donates. It will also cover the RDF’s operating expenses to ensure that all donations received through the fund go straight to the patients.
It puts the RDF’s current amount raised to around S$70 million.
To support the cause, A will donate all subscription dollars generated between July 2019 and June 2020 to the Rare Disease Fund.
Learn more about the Rare Disease Fund here.