Accelerated listing reshapes RWD evaluation

HIRA holds symposium on RWD on June 19

Kymriah and Zolgensma to undergo post-listing evaluations next year upon expiration of Risk-Sharing Agreements

Ra-won Kang, “Full-cycle management must be strengthened through greater collaboration among stakeholders”

Regulations governing full-cycle management using real-world data (RWD) are expected to be strengthened as part of the pilot program for expedited listing of rare disease treatments.

Because a new framework linking evaluation outcomes to reimbursement adjustments will be established, the Health Insurance Review and Assessment Service (HIRA) plans to gather stakeholder feedback and incorporate related standards and procedures into its internal regulations.

On June 19, HIRA held a symposium on real-world evidence (RWE) at the Yangjae AT Center, presenting a roadmap for enhancing the substance of pharmaceutical benefit evaluations.

During the event, Ra-won Kang, Head of Health Insurance Reimbursement and Assessment Service’s Rare and Severe Disease Performance Assessment Department, reviewed the current status of pharmaceutical performance assessment and emphasized the need for changes resulting from the accelerated-listing/post-evaluation pilot program.

At present, pharmaceutical performance assessments are conducted for risk-sharing agreement (RSA) drugs that lack evidence and require RWD collection.

There are two main types of pharmaceutical performance assessments. One is the ‘performance-based refund type,’ under which a manufacturer provides refunds at a predetermined rate if treatment outcomes fail to meet agreed targets for individual patients. The other is the ‘drug-level outcomes evaluation type,’ in which reimbursement is granted on the condition that additional evidence is generated. From 2022 through June 2026, HIRA conducted performance evaluations on a total of 32 drugs.

However, because the basis and mechanisms for reimbursement adjustments following data collection have remained unclear, there have been limitations in translating evaluation results into actual reimbursement adjustments.

Kang explained, “Performance-based risk-sharing arrangements are reaching a turning point. The 5-year risk-sharing agreements for Kymriah and Zolgensma will expire next year, meaning their re-evaluation period is approaching. At the same time, the expedited listing for rare disease treatments will be implemented in earnest. A system is being established to reflect accumulated evidence in post-listing evaluations, and registries centered on eligible drugs will be prepared.”

With the need for pharmaceutical benefit assessment using Real-World Data (RWD) rising, Kang explained that the system itself must be strengthened. This includes establishing plans in advance for how evaluation results will be utilized, disclosing evaluation outcomes, and clearly defining standards and procedures for linking those results to reimbursement adjustments.

Furthermore, regarding performance assessment, Kang noted that areas including ▲insufficient feedback following post-listing evaluations, ▲increased burden of generating evidence, ▲patient access to treatment, and ▲governance and decision-making required improvement.

Kang said, 'Management is necessary to prevent delays in new drug listing, and safeguard plans must be established so that patients already receiving treatment do not face discontinuation, even when the assessment results are unfavorable.'

"Full-cycle management is necessary. We are reviewing tasks to standardize the methodology for listing, data collection, monitoring, assessment, disclosure of results, and feedback. Once a draft is prepared, we will seek stakeholder input and refine it into a reasonable framework.”

Kang also emphasized, 'If the public sector takes the lead in building a platform with the participation of the medical and industrial sectors, data can be utilized in various ways to generate value. For the generation and utilization of RWD, collaboration among institutions and governance structures involving relevant stakeholders will be necessary.”