[Reporter's view] Despite ↑reimbursed drugs for rare dz

The need for improvement emerges every year, but patients with rare diseases are still struggling.

 

In particular, even if there are drugs, the number of patients is so small that it is difficult to prove the cost effectiveness and predict financial consumption, making it difficult to register insurance benefits.

 

There were many opinions this year.

 

Kang Sun-woo, a member of the National Assembly Health and Welfare Committee, began holding a parliamentary debate in May to strengthen access to new drugs for rare genetic diseases, and in August, Kang Byeongwon, Kim Won-i, Seo Young-seok, and Shin Hyun-young held a public hearing to discuss ways to improve the treatment environment for rare diseases.

 

But the HIRA reported that the average benefit rate for treatments for rare diseases was 85.3% (2016-2020) and 100% in 2020.

 

At this rate, it is thought that the patient's access to the treatment for rare diseases was perfect.

 

Then, why were there still many opinions on expanding benefits for treatments for rare diseases?

 

The results announced by the HIRA differed from the benefit rate of drugs with rare diseases actually licensed as the benefit rate for drugs that went through the screening and evaluation process.

 

Various factors such as dropout and voluntary withdrawal were excluded.

 

According to data surveyed by the KRPIA and the KPBMA, only about 50% of the items designated as rare drugs over the past decade have been listed on the insurance benefit list for rare diseases.

 

In order for the benefit rate of treatments for rare diseases to rise, the use of RSA and PE systems must eventually increase.

 

Rare diseases are diseases in which the number of patients is less than 20,000 or difficult to diagnose, making it difficult to know the number of patients.

 

In many cases, clinical trials themselves are difficult due to the small number of patients.

 

Due to the small number of patients, it is difficult to actively develop new drugs because it is difficult to expect profitability in the market, and even if new drugs are developed with difficulty, it is difficult to prove cost effectiveness through PE.

 

As a solution to this, the industry has insisted on expanding the PE exemption system.

 

If there is no alternative drug, the PE exemption system should be applied even if it is approved for placebo control data, or the number of patients should be applied in accordance with the special calculation criteria.

 

However, drug price adjustment for drugs subject to PE exemption has recently been predicted.

 

The figure of 80% of the lowest A7 adjustment price has made the industry difficult for a while.

 

In this regard, it is said that the HIRA and KRPIA meetings have recently clarified that they are at the "reference" level.

 

The point is that PE exemption drugs increased and the government thought additional management measures were needed.

 

The point is that PE exemption drugs increased and the government thought additional management measures were needed.

 

If the government has reduced the risk factors, it should look at blind spots.This is because it is an area with few patients and no medicine.