Drug price negotiations for two spinal muscular atrophy (SMA) drugs --Spinraza (nusinersen) and Evrysdi (risdiplam) – have reportedly failed to settle by the deadline, deepening the concerns of Korean patients, according to sources Monday.
The National Health Insurance Service (NHIS) failed to narrow differences in setting drug prices for Spinraza and Evrysdi by Friday of last week, the deadline for negotiations with the companies, the sources said.
Considering that Evrysdi’s application for reimbursement was filed in July 2021 and Spinraza's was filed in late 2021, patients have been waiting for these treatment options for nearly two years.
After receiving an application for Evrysdi, the Health Insurance Review and Assessment Service (HIRA) suspended its review, faced with the back-to-back application of Spinraza. At the time, HIRA intended to expand the benefits of Spinraza in keeping with the expanded indications of Evrysdi.
However, the government, considering that Spinraza’s treatment costs nearly 300 million won ($223,300) per patient per year, thought it was necessary to prepare a follow-up plan to secure the insurance’s financial soundness before expanding the benefit, postponing the benefits review of the two drugs and establishing dosing guidelines for SMA treatments first.
As a result, the two drugs could enter into drug price negotiations with the public corporation no earlier than May last year. However, the drug price negotiations were not completed within the deadline, and the two drugs entered into extended negotiations.
The problem is that SMA is a progressive disease. The government's dosing guidelines for SMA drugs include conditions that patients must meet with types 1-3 at initiation. For instance, patients older than 24 months of age must meet a specific assessment, such as Hammersmith Functional Motor Scale Extended (HFMSE) score, at age 18.
Some patients will have lost access to treatment in the nearly two years that reimbursement for the two drugs has been delayed. Even those currently near that cutoff are in danger of losing access forever by falling short of just one point.
Worse yet, considering that Spinraza and Evrysdi have the same mechanism of action but different administration methods, patients' treatment options have been compromised due to the delayed process.
Unlike Spinraza, which requires intrathecal injections, Evrysdi is taken orally and is the only treatment option for patients with scoliosis, a common condition in SMA.
Evrysdi has reportedly been reimbursed in all A8 countries -- the U.S., Germany, Italy, France, the U.K., Switzerland, Canada, and Japan -- to which Korea refers for setting drug prices within one year of approval.