Will the High-Cost Treatment 'Vindamax' Without Alternatives Be Covered by Health Insurance?

First Milestone Passed Two Years After 2020 Approval
Annual Drug Cost About $300 Million by US Standards
Concerns Over Health Insurance Financial Deterioration Pose Obstacle

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

[Asia Economy Reporter Chunhee Lee] Following the successful inclusion of ultra-high-cost rare disease treatments such as ‘Kymriah’ and ‘Zolgensma’ under the National Health Insurance (NHI), Pfizer’s ‘Vyndamax’ (active ingredient tafamidis), a treatment for transthyretin amyloid cardiomyopathy (ATTR-CM), a rare disease, is now seeking NHI coverage. As the only treatment available for ATTR-CM, the necessity for coverage is considered significant; however, concerns have been raised about the potential deterioration of health insurance finances due to the annual drug cost exceeding 300 million KRW based on U.S. prices.

According to industry sources on the 7th, Vyndamax recently passed the Drug Benefit Criteria Subcommittee of the Health Insurance Review and Assessment Service. Since receiving approval from the Ministry of Food and Drug Safety in August 2020, it has continuously attempted to enter the health insurance system but faced repeated setbacks, finally overcoming the first hurdle after two years.

Vyndamax is currently the only developed treatment option for ATTR-CM. Without appropriate treatment, the survival period for ATTR-CM patients is limited to 2 to 3 years and 6 months. However, no other suitable treatments have been developed besides Vyndamax. 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 indicated a 30% reduction in mortality risk.

Despite these benefits, Vyndamax has not passed the first hurdle for insurance coverage in Korea for two years, mainly due to pricing issues. The annual cost of Vyndamax treatment in the U.S. is $225,000 (approximately 310 million KRW). Unlike ‘one-shot’ treatments like Kymriah or Zolgensma, which provide near-curative effects with a single administration, Vyndamax is not a fundamental cure and must be taken once daily for life. If covered by NHI, approximately 300 million KRW per patient per year would need to be reimbursed. Over seven years, the financial support for Zolgensma administration exceeds 2 billion KRW.

The unpredictable patient population size is also an obstacle. ATTR-CM is divided into ‘wild-type’ caused by aging and ‘hereditary’ types depending on the cause of onset. Although hereditary cases are currently known to be more prevalent in Korea, the number of wild-type patients is expected to increase rapidly with accelerated aging. This is why ATTR-CM is sometimes called the ‘Alzheimer’s of the heart.’ Even now, with prevalence statistics not accurately established and patient numbers increasing, there are voices suggesting that ATTR-CM should be excluded from the rare disease category.

An industry official stated, "Since the government has continuously expressed its intention to expand coverage for ultra-high-cost treatments for severe diseases, although it may take some time, it is highly likely that Vyndamax will be covered. Because this concerns life, coverage should be implemented quickly, but given the significant concerns about cost-effectiveness, various insurance mechanisms such as risk-sharing schemes should be introduced."

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