Non-reimbursed approvals of off-label drugs

10 times more than rejected approvals over 6 years

Off-label approval data, including OTC medicines, have been disclosed for the first time

2186 approved between 2020-2025…208 applications were rejected

The number of non-reimbursed approvals of off-label drugs: while 208 applications for off-label use were rejected, 2,186 were approved between 2020-2025.

The number of approvals for non-reimbursed use of drugs beyond their authorized indications (off-label) has been more than 10 times the number of rejections over the past six years.

 

While 208 applications for off-label use were rejected, 2,186 were approved, often because the benefits outweighed the risks.

 

On October 27, the Health Insurance Review & Assessment Service (HIRA) publicly disclosed, for the first time, the number of non-reimbursed approvals for off-label drugs, including over-the-counter (OTC) medicines.

 

Applications for non-reimbursed off-label drug use must first be reviewed by an IRB (Institutional Review Board) established in a hospital or by an academic society.

 

For OTC medicines, HIRA commissions the Ministry of Food and Drug Safety (MFDS) to review safety and efficacy, while anti-cancer drugs are approved by the Cancer Disease Review Committee (CDRC), which holds monthly.

 

Unlike anti-cancer drugs, only the records of rejections for OTC medicines had been disclosed until now.

 

This had been criticized as a problem, making it difficult for healthcare institutions to file applications for off-label non-reimbursed use.

 

There was also a persistent request to disclose approval records for OTC medicines to ensure fairness.

 

HIRA pre-announced a partial amendment to the 'Partial Revision to Operational Regulations for Approval of Non-Reimbursed Off-Label Drug Use' in September and proceeded with public consultation.

 

The newly disclosed approval cases totaled 2,186 from 2020 to the present, broken down as follows: 472 cases in 2020, 572 in 2021, 266 in 2022, 485 in 2023, 276 in 2024, and 115 this year.

 

Meanwhile, a total of 377 rejection cases were disclosed over 13 years, from 2013 to 2025.

 

Specifically, from 2020, when approval cases began to be disclosed, 208 cases were rejected.

 

Examining the trend of rejections by year shows: 33 rejections in 2020, 86 in 2021, 38 in 2022, 44 in 2023, 6 in 2024, and 1 this year.

 

This data indicates a significant reduction in rejection cases over the last two years.

 

The disclosed rejection data for this year includes one additional rejection for Mabthera (rituximab).

 

The application was filed citing expectations that the drug would be more cost-effective, have fewer side effects, and offer higher therapeutic efficacy than alternative drugs.

 

Yet, it was denied because the submitted evidence did not substantiate the claims.