"Multi-Million-Won Drugs Fail in 6 Out of 10 Patients... Call to Reconsider Expedited Reimbursement"

CCEJ Announces Findings on Therapeutic Effectiveness of Ultra-High-Priced New Drugs
Kymriah, Zolgensma, Luxturna and Others Fall Short of Expected Benefits
Claim That National Health Insurance Funds Should Not Be Wasted on Unproven New Drugs

A criticism has been raised that many ultra-high-priced new drugs, often referred to as "miracle cancer drugs," are increasing the financial burden on the National Health Insurance system without sufficiently proving their actual therapeutic effectiveness.

On the 9th, participants at a press conference held by the Citizens' Coalition for Economic Justice in Jongno-gu, Seoul, to present findings on the therapeutic effects of ultra-high-priced new drugs and to urge a review of expedited listing are chanting slogans. Yonhap News

On the 9th, the Citizens' Coalition for Economic Justice, the Association of Physicians for a Healthy Society, and the Korea Alliance of Patients Organization for Rare and Incurable Diseases held a press conference at the Citizens' Coalition for Economic Justice in Jongno-gu, Seoul, stating, "It has been found that the effectiveness of an ultra-high-priced new drug, which costs 360 million won per dose, is only 40%," and urging, "The government must reconsider its plan for expedited listing of new drugs for reimbursement."

According to data these organizations received from the National Health Insurance Service, annual pharmaceutical expenditures in Korea from 2020 to 2024 increased by an average of 7.7% per year, reaching 26.8 trillion won in 2024. In particular, among this, expenditures on new drugs whose prices were negotiated by the National Health Insurance Service increased more steeply, by an annual average of 13.1%.

This is eight times the rate of increase in health insurance premiums. There is particular criticism that, as the system exempting anticancer drugs and orphan drugs from health economic evaluation has been expanded, there are growing numbers of cases in which high-priced new drugs are included in the reimbursement list without prior verification.

An analysis by the Citizens' Coalition for Economic Justice of performance evaluation data on ultra-high-priced new drugs from the Health Insurance Review and Assessment Service showed that 59.1% of patients who received the immuno-oncology drug Kymriah (tisagenlecleucel), which costs 360 million won per dose, did not experience any therapeutic effect. Of the 129.6 billion won in reimbursement spending on Kymriah between 2022 and 2024, it is estimated that around 76.6 billion won produced no benefit relative to the amount spent.

For Luxturna (voretigene neparvovec), a treatment for hereditary retinal dystrophy, and Spinraza (nusinersen sodium), a treatment for spinal muscular atrophy, only 50% of patients showed improvement in motor function assessments. The upper price limits for the two drugs are 330 million won and 92 million won, respectively.

However, most post-marketing evaluation data related to the actual therapeutic effectiveness of these new drugs are managed as non-public information. The Citizens' Coalition for Economic Justice stated, "We requested disclosure of information on the therapeutic effectiveness and performance data of ultra-high-priced new drugs from the Health Insurance Review and Assessment Service and the National Health Insurance Service, but were notified that the data would not be disclosed on the grounds that they are part of contracts with pharmaceutical companies." This contrasts with the situation overseas, where performance data on new drugs are made relatively transparent to the public.

In November last year, the Ministry of Health and Welfare significantly streamlined the appropriateness assessment and price negotiation procedures for including treatments for rare diseases under National Health Insurance coverage, and is pushing a policy to shorten the reimbursement listing period, currently up to 240 days, to within a maximum of 100 days. The plan calls for largely omitting clinical usefulness and pharmacoeconomic evaluations, deciding on reimbursement eligibility within 30 days, and setting drug prices based on the average prices in eight foreign countries.

Over the past seven years, there have been 77 orphan drugs (comprising 60 active ingredients) approved by the Ministry of Food and Drug Safety that have not yet been listed for reimbursement, and the average treatment cost per drug is about 290 million won. If, going forward, the prices proposed by pharmaceutical companies are accepted as is and it is assumed that 100 patients per year will use each treatment, it is estimated that pharmaceutical expenditures for listed orphan drugs will increase by at least an additional 1.5 trillion won.

The Citizens' Coalition for Economic Justice argued that such a reform of the drug pricing system could instead weaken verification and even threaten the sustainability of National Health Insurance finances. Kim Seongju, head of the Korea Alliance of Patients Organization for Rare and Incurable Diseases, said, "The government is focusing only on expedited listing and is shifting uncertain risks and financial burdens onto patients and the public," and added, "We have to ask why unproven new drugs are used on patients first and why the results are not disclosed."

Kwon Hyeyoung, a member of the Health and Medical Committee of the Citizens' Coalition for Economic Justice and a professor at Mokwon University, stressed, "There is a high possibility that overseas prices are inflated prices that already reflect rebates, yet they are being imported as they are without verification," and added, "For ultra-high-priced new drugs with uncertain effectiveness, the prior authorization system and post-marketing evaluation should instead be strengthened."

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