Korea expands Evrysdi prescription limit for SMA but experts urge further access improvements

The long-term prescription threshold for the oral treatment of spinal muscular atrophy (SMA), Evrysdi (risdiplam), will be expanded from a maximum of two bottles to three per month.

Experts have noted that this is a step forward in improving patient access to treatment, but there is still a long way to go.

Last Friday, the Health Insurance Review and Assessment Service (HIRA) issued an administrative notice on the “Partial Amendment to the Details of the Application Criteria and Methods of Insurance Benefits,” expanding the long-term prescription criteria for Evrysdi.

According to the amendment, the maximum prescription amount for long-term Evrysdi use will increase from two to three bottles. However, the criteria for applying for insurance benefits and the principle of in-house prescription remain the same within the permission.

“Based on a comprehensive review of textbooks, guidelines, and scientific opinions, we decided to increase the prescription dose to improve the continuity of treatment for patients,” HIRA said.

SMA is an inherited disease that causes the muscles of the limbs to gradually atrophy and lose strength due to the degeneration of motor nerve cells. The lower limbs are more affected than the upper limbs, and this severely limits daily mobility and physical activity.

Patients with SMA have pointed out that it is difficult for them to receive sufficient treatment due to the maximum prescription of two vials of Evrysdi per reimbursed prescription. This issue was also discussed in the parliament’s audit of the Ministry of Health and Welfare.

“It is difficult for patients to continue treatment stably because the prescription amount of Evrysdi is limited to two bottles,” said Rep. Seo Mi-hwa of the Democratic Party of Korea (DPK) on Oct. 16. “Since the drug can be used for up to two months after dispensing, the prescription amount should be expanded to five bottles based on two months.”

As the revision is limited to three bottles, experts said it is still insufficient to reflect patients' realistic needs.

“Even though Evrysdi has a two-month expiration date, patients in Korea have to get a prescription every three weeks,” said Moon Jong-min, head of the Korean Association of SMA Patients, pointing to the inconvenience of the current prescription system and the need for improvement. “Increasing the prescription to three bottles would only increase the visit cycle to four weeks. A two-month supply should be allowed to be prescribed, which will practically help patients.”

Moon continued, “We understand the government's position, as the drugs are expensive, and there may be storage issues. However, it is frustrating for patients to have to visit hospitals frequently. If patients are allowed to prescribe a two-month supply, it will greatly improve their convenience by reducing the need for three visits to the hospital to one.”

There is also a need to relax the prescription threshold for SMA treatments

Calls are also mounting to improve reimbursement criteria so SMA patients receive more reliable and consistent treatment. Many SMA treatments, including Evrysdi, are now reimbursed only when they are on the high-cost drug list and receive prior authorization, forcing many new patients to struggle to get past the prior authorization threshold.

Since October of last year, when new and expanded coverage criteria for Evrysdi and Spinraza (nusinersen) were established, 24 out of 51 new patient applications have been denied based on a physician's determination of treatment need. More than half (13) of those denials were for failure to demonstrate symptom onset. Specifically, six of the 12 rejections for Evrysdi and seven of the 12 rejections for intrathecal injections were due to failure to demonstrate the onset of symptoms and signs.

In another parliamentary audit session, SMA patient Lee Seung-joon testified as a witness and presented cases of pre-screening rejections. Lee complained that “adult patients who have difficulty submitting past diagnostic data are losing treatment opportunities because they do not meet the criteria for payment.”

Rep. Kang Sun-woo of the DPK also pointed out that the Medical Service Act limits the period for keeping medical records to 10 years, making it almost impossible for patients born before 1994 to find their records, calling for relaxing the criteria and alternative measures.

Currently, patients are also required to make additional visits for motor function evaluation to continue receiving the drug, which adds to the burden on patients and their guardians.

In this regard, Rep. Seo stressed the need to relax the monitoring criteria for motor function assessment.

“If a patient fails to meet the baseline score for two consecutive four-monthly monitoring visits, the drug is discontinued, which is too short a period to assess the effectiveness of the drug. As Japan conducts a review every 10 years, it is also necessary to review domestic standards,” the DPK lawmaker added.