High-Cost Treatment 'Vindamax' Without Alternatives Fails to Gain Health Insurance Coverage

Non-reimbursed Decision After 2020 Approval
Annual Drug Cost of 300 Million USD Based on US Standards
Concerns Over Health Insurance Financial Deterioration and Patient Surge Are Obstacles

The ultra-expensive rare disease treatment drug 'Vyndamax' has failed to enter the National Health Insurance (NHI) reimbursement list.

Pfizer's transthyretin amyloid cardiomyopathy (ATTR-CM) treatment 'Vyndamax'

The Health Insurance Review and Assessment Service (HIRA) announced on the 6th that the 4th Drug Reimbursement Evaluation Committee of 2023 reviewed Vyndamax (active ingredient tafamidis) by Pfizer Korea, a treatment for transthyretin amyloid cardiomyopathy (ATTR-CM), and decided it was not appropriate for reimbursement. After receiving approval from the Ministry of Food and Drug Safety in August 2020 and attempting to enter the health insurance system, Vyndamax passed the Drug Reimbursement Criteria Subcommittee after four attempts but ultimately failed to clear the final hurdle.

Vyndamax is virtually the only treatment option for ATTR-CM. Without proper treatment, the survival period for ATTR-CM patients is only 2 to 3 years and 6 months. However, no other suitable treatments besides Vyndamax have been developed. According to Pfizer, clinical phase 3 trials showed significant effects, including reduced risk of all-cause mortality and cardiovascular-related hospitalizations in the Vyndamax group compared to the placebo group. Long-term follow-up studies showed a 30% reduction in mortality risk.

Despite these benefits, Vyndamax has not even passed the first reimbursement hurdle in Korea for two years, largely due to pricing issues. The annual cost of Vyndamax treatment is approximately $225,000 (about 300 million KRW) in the U.S. and about 150 million KRW annually (around 410,000 KRW per tablet) in Korea under non-reimbursed conditions.

Recently, expectations rose as ultra-expensive treatments like 'Kymriah' and 'Zolgensma' successfully gained NHI coverage. However, unlike these ‘one-shot’ treatments that provide near-complete cures with a single dose, Vyndamax is not a fundamental cure and must be taken once daily for life, which appears to be a significant hurdle. If reimbursed, the NHI would have to cover over 100 million KRW per patient annually for life, imposing a substantial financial burden on the health insurance system.

The unpredictable patient population size is also a stumbling block. ATTR-CM is divided into ‘wild-type’ caused by aging and ‘hereditary’ types depending on the cause of onset. Although hereditary cases are currently more common in Korea, the number of wild-type patients is expected to increase rapidly with accelerated aging. ATTR-CM is sometimes called the ‘Alzheimer’s of the heart.’ With prevalence statistics still unclear and patient numbers rising, there are even calls to remove ATTR-CM from the rare disease category, raising concerns about a sharp increase in health insurance costs, which is another factor complicating Vyndamax’s inclusion in NHI coverage.

Health Insurance Review & Assessment Service CI
Photo by Health Insurance Review & Assessment Service

Meanwhile, the Drug Reimbursement Evaluation Committee judged that the following drugs are appropriate for reimbursement: 'Semblix tablets' (active ingredient asciminib hydrochloride) by Novartis Korea for chronic-phase Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML); 'Onureg tablets' (active ingredient azacitidine) by BMS Korea for maintenance therapy after induction therapy in adult patients with acute myeloid leukemia; and 'Nepoxil capsules' (active ingredient ferric citrate hydrate) by Kyowa Kirin Korea for hyperphosphatemia in chronic kidney disease patients undergoing hemodialysis, provided the price is accepted below the evaluation amount.

On the other hand, 'Tabrecta tablets' (active ingredient capmatinib hydrochloride monohydrate) by Novartis Korea for MET exon 14 skipping mutation-positive locally advanced or metastatic non-small cell lung cancer was decided as non-reimbursable, and 'Retevmo capsules' (active ingredient selpercatinib) by Lilly Korea for RET fusion-positive non-small cell lung cancer and other indications will be reconsidered.

This content was produced with the assistance of AI translation services.