HIRA, "Will enhance the monitoring of rare disease drugs"

Lee So-young, Director of HIRA's Pharmaceutical Performance Assessment Division, emphasized the need for establishing a sustainable system

"More than half of the newly approved drugs are rare disease drugs…need to improve the management of uncertain evidence monitoring"

With the rapid increase in newly emerging treatments for rare and severe diseases, there have been suggestions for establishing sustainable systems, such as strengthening post-market management and creating a separate dedicated fund.

 

It was explained that monitoring uncertain evidence resulting from expedited approvals and improving patient access through a separate fund, similar to overseas models, are necessary steps.

 

On September 25, the Health Insurance Review & Assessment Service (HIRA) hosted a symposium at the St.

 

Mary's Hospital Seong-ui Hall in Seoul on the future direction and social ethics of rare and severe disease treatment.

 

Lee So-young, Director of HIRA

During the symposium Lee So-young, Director of HIRA's Pharmaceutical Performance Assessment Division, said that as rare disease treatments rapidly increase, not only Korea but also other countries are deliberating on management strategies.

 

In the United States, 52% of new drug approvals are for rare disease treatments, and the FDA operates four expedited tracks to address unmet medical needs.

 

However, Lee pointed out that problems are arising where expedited approval, often based on surrogate endpoints, does not ultimately improve patient access or where research finds the drug provides no actual benefit.

 

Lee explained, "The FDA is also aware of the problem.

 

Since the year before last, they have required that when approval is based on surrogate index, a confirmatory clinical trial must be initiated or clearly planned," and added, "They have also reinforced measures to allow for rapid withdrawal of approval if the requirements are not met or if the anticipated clinical benefit is not realized." Lee emphasized that it is time for Korea to move beyond viewing rare disease treatment as an exceptional case and start considering a sustainable system.

 

Lee proposed three strategies: ▲Conditional reimbursement based on evidence generation ▲Establishing a separate dedicated fund ▲Improving reimbursement criteria and pre-and post-market management.

 

Regarding conditional reimbursement, Lee said, "The legal basis is established, and the guidelines will be released in November this year.

 

This is a structure where a drug is listed and covered on the condition that post-market evaluation is conducted, and the post-market management tracks the extent of evidence uncertainty and whether that uncertainty can be eliminated." Lee added, "The UK classifies drugs into those that should or should not be listed through the Cancer Drug Fund.

 

They have also established an Innovative Medicines Fund.

 

Taiwan and Australia operate similar funds," proposing the establishment of a separate fund in Korea.

 

Lee concluded, "Currently, reimbursement management is conducted at the patient level.

 

There is a need for monitoring at the drug and disease level, with performance management based on those results,"and added, "It's difficult for pharmaceutical companies alone to gather and compile all the data.

 

The EU also operates its system through transnational cooperation," and stressed the importance of a cooperative framework for strengthening post-market management.