[Reporter's View] Improving rare disease care environment

Demand for the improvement of the rare disease treatment environment rises every year

A few patients can only make a small noise.

 

Despite the need for the improvement of the rare disease treatment environment, which has risen year after year, the voice of its patients that implore difficulties has never ceased to exist.

 

In particular, there are cases where a drug is available, but due to the small number of eligible patients, it is difficult to prove the drug’s cost-effectiveness and predict the potential financial expenditures, rendering the drug’s reimbursement listing difficult in Korea.

 

According to the 'Status of Severe Diseases that were applied Special Calculation' released by the Health Insurance Review and Assessment Service, rare and incurable diseases accounted for 37%, 32%, and 33% of the distribution of the doctors’ office personnel, medical expenses, and reimbursement expenses per severe disease in 2022, respectively.

 

From 2012 to 2019, the relative proportion of reimbursement paid for rare and incurable diseases among the severe diseases that were applied special calculation was around 33%.

 

However, the government has a different story.

 

The average reimbursement rate for rare disease drugs was 85.3% (2016-2020) and 100% in 2020.

 

This would seem to indicate that patient access to rare disease drugs seems picture-perfect.

 

However, why isn’t it so in reality?

 

Results published by HIRA are based on the reimbursement rates for drugs that have undergone the review and evaluation process, which are different from the reimbursement rates of approved rare disease drugs.

 

In other words, they exclude various factors such as applications of drugs that dropped out or made voluntary withdrawals.

 

In order to increase the true reimbursement rate of rare disease drugs, the utilization of risk-sharing agreements and the special economic evaluation exemption system must increase.

 

Rare diseases are those with a prevalence of 20,000 or fewer people, or where the number of patients is unknown due to difficulty in diagnosis.

 

It is often difficult to conduct clinical trials due to the small number of patients.

 

Due to the small number of patients, it is difficult to expect profitability in the market, making it difficult to actively develop new drugs, and even if a new drug is successfully developed, it is difficult to prove its cost-effectiveness through cost-effectiveness analysis.

 

As a solution to this, the industry has been advocating the expansion of the pharmacoeconomic evaluation exemption system.

 

The industry has been advocating for the expansion of the exemption system, such as applying the exemption system even if the drug is approved with placebo-controlled trial data if there is no alternative drug or applying the number of patient requirements same as those used for special calculation.

 

However, it is also true that the government needs to worry about its limited finances.

 

The government is even considering introducing a system to further control the listed drugs due to the increased number of drugs covered by the system.

 

This means that the price of some drugs may be reduced even further from the current price.

 

If the government wishes to reduce the risk, they also need to tend to the blind spots.

 

As these are areas where there are few patients and no available medicines.

 

It is important to hold a closer year to the small but desperate voices.