Novartis' rare disease drug Ilaris passes health insurance review panel

Novartis' rare disease treatment Ilaris (canakinumab) has crossed the threshold of the Health Insurance Review and Assessment Service (HIRA)'s Pharmaceutical Reimbursement Evaluation Committee (PREC), which reexamined its “conditional” approval application.

At the same meeting, the panel also recognized Kyowa Kirin's thyroid drug Orkedia (evocalcet) and Santen Pharmaceutical's glaucoma drug Rhopressa (netarsudil mesylate) as appropriate for reimbursement.

HIRA released the results of its deliberations at the fourth PREC meeting this year on Thursday.

Ilaris, a treatment for hereditary recurrent fever syndrome, an extremely rare autoimmune disease, was tabled for re-deliberation and passed.

In February, PREC approved the appropriateness of Ilaris for some indications on the exceptional condition that the drugmaker submits additional evidence. However, Novartis failed to accept the condition, threatening to derail the approval for insurance benefits.

Novartis requested that HIRA reconsider the submission requirements so the company can meet them. However, the request was not accepted at the latest meeting, either.

Ilaris is indicated for treating Cryopyrin-Associated Periodic Syndromes (CAPS), Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS), and Hyperimmune Syndrome in children (two years and older) and adults), Hyperimmunoglobulin D Syndrome/Mevalonate Kinase Deficiency (HIDS/MKD), Familial Mediterranean Fever (FMF), and active Systemic Juvenile Idiopathic Arthritis (SJIA).

Among them, the PREC recognized the appropriateness of Ilaris' reimbursement for CAPS, TRAPS, and FMF indications with the condition that the drugmaker submit evidence in the future.

Novartis won approval for Ilaris in 2015 and applied for reimbursement in 2017 and 2022, but both applications were unsuccessful.

Only Ilaris and Kineret (anakinra) are available for hereditary recurrent fever syndrome. However, Kineret has been withdrawn from the Korean market, forcing patients to import it through the Korea Orphan and Essential Drug Center.

Moreover, the only drug available to patients who failed with Kineret is Ilaris, which is expensive, costing more than 20 million won ($14,783) for a single treatment.

Unable to afford the bi-monthly treatment, patients and families began to file a petition, prompting the government to consider adding drugs that have significantly improved the quality of life in children, including Ilaris, to the list of drugs that can be exempted from economic evaluation.

However, as the development of treatments for serious diseases, such as rare diseases and cancer, is increasing rapidly, the financial burden for drugs skipped for economic viability is also increasing, making it necessary for the government to strengthen the follow-up management of these drugs.

Therefore, rather than skipping the economic evaluation altogether, the government sought a new approach that effectively suspended it, with the condition that the company produce and submit additional evidence of cost-effectiveness after the payment.

Ilaris was the first such case.

In the end, Ilaris has crossed eight out of 10 hurdles for reimbursement ten years after getting approval. However, Ilaris is still subject to drug price negotiations with the National Health Insurance Service and a decision by the Health Policy Review Committee of the Ministry of Health and Welfare.

Orkedia and Rhopressa, who were tabled at the meeting for new reimbursement, also passed the review.

Orkedia was approved for treating secondary hyperparathyroidism, and Rhopressa was approved for treating open-angle glaucoma and ocular hypertension, with the condition that the drugmaker accept a drug price below the government's assessment.

In contrast, Takeda Korea's application to expand Zejula's coverage in unresectable ovarian cancer failed to pass the PREC.