PETALING JAYA: The Health Ministry should approve a drug named Spinraza that can help people with spinal muscular atrophy (SMA), said WeCare Journey co-founder Yap Sook Yee.
"On December 23, 2016, the United States Food and Drug Administration approved the first-ever drug therapy for spinal muscular atrophy or SMA, called Spinraza (Nusinersen)," said Yap.
Yap - the mother of eight-year-old Branden who lives with SMA - said that in Malaysia, SMA families are still clinging on to the hope for similar approval to happen soon.
"The SMA community in Malaysia has watched as country after country approves use of this drug, bringing heartening joy to the SMA families there," said Yap on Sunday (June 10) in a letter to Health Minister Dr Dzulkefly Ahmad.
The Malaysian Rare Disorders Society vice-president said that the disorder affects 1 in every 11,000 live births, adding that this means that on average one baby is born with SMA almost every week in Malaysia
"About 1 in 50 people (globally) are carriers of the defective gene that causes SMA, or about 640,000 Malaysians. SMA is the number one genetic cause of death for infants and affects the motor nerves, resulting in muscle wasting and weakness. It is a progressive disease, robbing patients of their ability to sit, walk, run, and later to even breathe and eat," she said in the letter.
She added that the SMA community and those with other rare disorders are often marginalized and burdened due to absent and poor implementation of policies relating to health, disability and insurance.
"Whilst patient groups strive to cover many gaps in required care and support, we face obvious limitations; for example in getting funding and manpower, our reach, and involvement in person-centred care," she said.
Yap also said that while many Malaysians still cannot afford to buy health insurance that they require, SMA patients are denied the right to buy any insurance coverage.
"Although there is a rare disease fund as in the 2018 Budget, it must be made more inclusive and expanded to include Spinraza," she said.
Yap pointed out that on May 21 at the World Health Assembly in Geneva, Malaysia reiterated its commitment to universal health coverage that leaves no-one behind.
"We urge the government to follow the World Health Organization declaration made on 28 February 2018 which stated specifically that ‘leaving no one behind’ would also include people who suffer from rare disorders, also known as orphan diseases," said Yap.
Yap also said that as there are over 7,000 types of rare disorders affecting one in 10 people worldwide, rare disorders are a public health priority that needs a National Rare Disorder Plan.
"The plan will require a multi-stakeholder approach and the government should consider supporting initiatives for translational research towards improving patient health outcomes. We advocate for the establishment of a Rare Disease and Orphan Drug Act, which will be beneficial to families with SMA as well as other rare disorders," said Yap.
She urged the Health Ministry to work with all relevant ministries, stakeholders and patient groups to improve the well-being of families affected by rare disorders.
"Together we can press for and ensure there is proper access to information and training, services and equipment, and medicines and treatments – all of which are needed to better prevent, detect and respond to SMA and rare disorders," said Yap.