Rare diseases still left neglected in Korea’s healthcare

Some diseases remain ineligible for RSA or PE exemptions benefits

Only 2 items receive reimbursement as rare disease treatment among drugs ineligible for special calculation exemptions

The limited scope of application of the special calculation exemptions remain a barrier... access improvement required

Patients with rare diseases suffer due to the rarity of their disease.

 

Even when treatments are available, the drugs have difficulty receiving reimbursement as it is difficult to prove cost-effectiveness and predict financial expenditures for the drugs due to the small number of patients.

 

The government is well aware of such difficulties.

 

This is why it has attempted to improve access to rare disease treatments through various systemic improvements.

 

However, the situation has not improved much.

 

Among the 6,000 to 7,000 rare diseases known worldwide, only about 6% have available treatments developed.

 

This is because it is difficult to secure statistical significance of the data due to the small number of patients and limited clinical data.

 

This is why even the small number of rare diseases for which treatments have been developed is a pie in the sky in terms of reimbursement.

 

In this sense, the ‘Study on the current status of access to rare disease treatments in Korea and measures to strengthen their coverage' that was recently announced by Professor Jong-Hyeok Lee of Chung-Ang University College of Pharmacy is attracting attention.

 

Results of the study suggest that there are still many blind spots that remain unattended for rare disease treatments.

 

Reimbursement rate remains at 33% for orphan drugs ineligible for special calculation exemptions

First, when analyzing the reimbursement status of the total of 136 orphan drugs that were approved over the past 10 years (2012-2021), the reimbursement rate for anticancer drugs rose to 58% after measures were implemented to strengthen coverage for 4 major severe diseases that began in 2013.

 

In the case of rare disease treatments, the rate is only 51%.

 

In particular, the rate for orphan drugs that are not subject to special calculation exemptions is only 33.3%, indicating the existence of the neglected areas, or blind spots that remain, in the area of rare diseases.

 

In addition, the period required for reimbursement was about 22 months for drugs subject to special calculation exemptions, while it was 34 months for those ineligible for special calculation exemptions, showing the significant difference.

 

Such results show that Korean patients' access to rare disease drugs is still limited, and the process is time-consuming.

 

◆Lacks application of RSA and PE exemptions

The RSA (Risk Sharing Agreement) and the pharmacoeconomic evaluation exemption (PE exemption) system, which are special systems established for the reimbursement of new drugs, were also found to be rarely applied to rare diseases.

 

Reimbursement evaluation results of the drugs analyzed in the study showed that the special systems were mainly applied to anticancer drugs.

 

The RSA and PE exemptions were only applied to 30% and 22% of the rare disease drugs, respectively.

 

All in all, the limited scope of application of the special systems was affecting access to treatments for patients with rare diseases.

 

Supported by such study results, the industry has been requesting that the government allow individual deliberations to be made for drugs that are not subject to the special calculation exemption but are chronically debilitating diseases as orphan drugs not subject to the special calculation exemption system cannot receive the systemic benefits for reimbursement (only 2 products have received benefits from the system).

 

Meanwhile, the Ministry of Health and Welfare held public-private consultative body meetings to improve the drug pricing system with the National Health Insurance Service, Health Insurance Review and Assessment Service, and three pharmaceutical organizations.

 

After 5 meetings, the MOHW is known to be preparing to announce the final measure to improve the drug pricing system.

 

The ultimate purpose of preparing the 'Innovative New Drug Compensation Plan' by the 2nd Vice Minister Min-soo Park of the MOHW for the implementation of ‘Yoon Administration’s National Policy Tasts’ is not on increasing the value of drugs that are already reimbursed, but to fulfilling the fundamental purpose of allowing drugs that have not yet entered the system (received reimbursement benefits) to enter the system.

 

Therefore, to expand access to rare disease treatment as listed as a national task by the Yoon administration, it is hoped that a practical and effective systemic improvement will be made rather than superficial attempts.