Treatments for SMA are available, but unaffordable for most Malaysians

WeCareJourney co-founder and Lim's wife Yap Sook Yee prepares the daily dose of risdiplam for their son Branden. This drug, which is based on patient weight, is estimated to cost a maximum of US$340,000 (RM1.5mil) a year.

In the past six years or so, patients with spinal muscular atrophy (SMA) and their families were filled with hope as treatments for this rare disease were confirmed to be effective and approved by regulatory agencies around the world.

Previously without treatment and with a limited lifespan for the majority of patients, this progressive disease now has three available drugs that tackle the condition directly.

While getting hold of these drugs was previously a problem for Malaysians, the last year or so has seen the approval of two of these treatments by the National Pharmaceutical Regulatory Agency (NPRA).

They are nusinersen and risdiplam.

The other treatment, the genetic therapy onasemnogene abeparvovec-xioi, is not yet approved in Malaysia, but is possible to access via a special pathway on an individual patient basis (see this Sunday’s article in StarHealth for more information).

Although this is one major barrier gone, there is another one preventing patients and their families from getting the life-saving treatment that they need: that of cost.

These are expensive treatments:

• Onasemnogene abeparvovec-xioi – which until recently held the title as the world’s most expensive drug – costs about US$2.1 million (RM9.29 million) per shot.
• Nusinersen costs between US$625,000 (RM2.76mil) to US$750,000 (RM3.32mil) the first year, then about US$375,000 (RM1.66mil) annually subsequently, and
• Risdiplam, which is based on patient weight, costs a maximum of US$340,000 (RM1.5mil) a year.

Onasemnogene abeparvovec-xioi only needs to be taken once, but both risdiplam and nusinersen are lifelong treatments.

In Malaysia, none of these drugs are covered in public hospitals under the regular Health Ministry budget, i.e. you cannot get them for the heavily-subsidised fee of RM5 as you can for many other more common medicines.

Instead, there have been specific allocations for rare diseases, dating back to 2008 when Hospital Kuala Lumpur received RM250,000 to send patient samples overseas to test for rare diseases.

This allocation has been increased over the years, culminating in RM25mil in the 2023 Budget.

However, the Budget, which was not passed by Parliament due to the calling of the 15th General Election, may be revised when it is tabled in Parliament next month (February 2023).

Even so, WeCareJourney co-founder and president Edmund Lim notes that no SMA patient has benefited from this annual allocation for their treatment.

WeCareJourney is a non-profit organisation that helps provide resources and support for Malaysian patients with SMA and their families, raises awareness about the rare disease, and engages with various stakeholders to help facilitate access to treatments for both Malaysian and Asean SMA patients.

“We would like to see a transparent, equitable way how funding can be allocated between drug therapies based on unmet needs.

“It’s just not clear to us,” says the father of Branden Lim, 12, who has SMA.

“The clarity of the process and the timeline would be nice to know,” he adds, saying that then-Deputy Health Minister Dr Lee Boon Chye had indeed clarified that the allocation was for all rare diseases in Malaysia.

“Without government reimbursement, you will be seeing more and more cases of parents going out and asking for public help to pay for medication for their children,” he says.

The most recent successful case of such crowdfunding was for 17-month-old Reese Tan Rui Xin.

Her parents, accountant Rachel Chung and car engine oil and spare parts salesman Jason Tan, had appealed to the non-profit organisation One Hope Charity and Welfare to help raise RM9mil to pay for onasemnogene abeparvovec-xioi.

As the therapy is only approved for children under the age of 24 months, it was also a race against time for Reese to get the drug.

The huge sum was successfully raised within a week, with a final sum of RM9,002,230 from 26,425 donors.

But with an estimated 50 babies born with SMA in Malaysia every year, this is certainly not a sustainable method of financing

And indeed, not all such crowdfunding efforts are successful.

Lim also notes that it can be difficult to account for how such donations are spent, or what happens to the donated monies that don’t reach the target.

However, as an alternate source of funding for rare diseases, the Health Ministry is also hoping to tap into the generosity of Malaysians with its Rare Diseases Trust Fund established last July (2022), to which the public can donate.

Tax exemption status was given to such donations in the previously-presented Budget 2023, but it remains to be seen if this is still the case in the upcoming Budget.

No insurance plan covers rare diseases in Malaysia either, so parents have no options other than to hope to get into a clinical trial or compassionate use programme that will enable their child to get the treatment for free, or to pay out-of-pocket, which is unaffordable for most families.

As US-based EveryLife Foundation for Rare Diseases Chief of Policy and Advocacy Annie Kennedy shared during the 2022 US National Press Foundation Covering Rare Diseases online conference, a mother of a child with a rare disease once told her: Having no treatment is bad, but what’s worse is having a treatment that is unaffordable.

This article is part of the second package of a short series written as part of the US National Press Foundation’s 2022 Covering Rare Diseases: Journalism Fellowship & Global Reporting Grant. The next one will be published this Sunday (Jan 8, 2023) in StarHealth.