Will RWD drive accessibility to ultra-high-priced drugs?

by helping overcome the limitations of pharmacoeconomic evaluations

HIRA holds symposium on ‘Establishing measures for RWD collection to manage pharmaceutical reimbursement management’

Advanced countries in insurance and pharmaceuticals such as the UK and France are working to establish RWD management system

Need to diversify the system with measures such as performance-based RSA, adjustment of reimbursement standards, safe use, policy support, etc.

Korea’s RSA system is moving to become more of a performance-based mechanism rather than a finance-based system.

 

Performance-based RSA means that the system will be using real-world data (RWD) from clinical practice as real-world evidence (RWE).

 

Ultra-high-priced drugs like Kymriah inj.

 

(tisagenlecleucel) have had difficulties in reimbursement, and the government's RSA expansion attempt may be an important tool that can increase patient accessibility.

 

Ji-Hye Byun (Ph.D.

 

in Social Pharmacy), Head of Evidence-based research at the Health Insurance Review & Assessment Service, presented on ‘Plans for reimbursement management in Korea, including pharmaceuticals using RWD, etc’ at the Innovation Research Symposium, ‘Establishing measures for RWD collection to manage pharmaceutical reimbursement management,' that was hosted by HIRA today (4th).

 

Innovative anticancer therapies like Kymriah are ultra-high-priced drugs with much demand for prompt listing from patients and requests for high prices from pharmaceutical companies.

 

The insurance authorities are having difficulty allowing reimbursement for these innovative drugs that have an ultra-high price despite uncertainties in their effectiveness, safety, and cost-effectiveness.

 

This issue is not just a concern for Korea alone, as the trend in new drug development now turned to focus on high-priced drugs that innovatively treat rare diseases.

 

Advanced countries in insurance or new drug development have also been facing the same issue and pursuing the use of RWD to suit each country.

 

According to ‘The UK’s integrated management and use of RWD (Current and future role of RWE in NICE)' that was presented by the copresenter, Jihyung Hong, Professor of College of Social Science at Gacheon University, the UK has been rapidly reinforcing the state-led data integrated system.

 

Patient accessibility to anticancer drugs or rare disease drugs that have a high uncertainty has been reinforced through the "Managed Access Scheme (MAA)" during which additional data (including RWE) are collected.

 

Under the Cancer Drug Fund (CDF), a separate fund for high-priced anticancer drugs, NHS England and pharmaceutical companies collect additional data to agree upon the details on reimbursement.

 

The reimbursement level and degree of uncertainty collected in over 2 years of managed access are used as a basis to conduct further reassessment, based on which NICE will decide on whether to make a final recommendation.

 

In France, reimbursement for rare disease drugs that have uncertain clinical grounds is not deliberated immediately, but applied a temporary use system ‘ATU,’ and allowed conditional use upon reassessment.

 

The UK, France, Germany, Italy, and Spain are all using RWE to resolve the uncertainty in the decision-making process for Kymriah.

 

Spain uses an RWD (patient registration) system that it established that considers the ‘post-marketing safety-economic evaluation analysis.’ In Korea, HIRA has also been operating an expert advisory council since 2019 and had studied RWD cases in partnership with the Korean Cancer Study Group in the process.

 

Another co-presenter and KCSG president, Professor Dae Young Zang (Division of Hematology-Oncology, Hallym University), presented on the ‘Study on efficacy and safety of gastric cancer drugs using electronic medical records (EMR),’ on the RWD items of ramucirumab (Cyrmanza).

 

As such, Korea is also seeking to manage the whole lifecycle of reimbursed drugs using RWE.

 

RWE is a viable alternative that can overcome the limitations of the current system, including the fact that there is no cost-effectiveness benefit management mechanism for applying results that have changed since reimbursement, lacks data collection suited to the purpose of collecting clinical data for HTA (economic feasibility assessment), and the fact that reassessment will only take place after a certain period passes after the submission due to prospective data collection based on reimbursement notices.

 

During the presentation, Byun proposed several measures for systematically managing reimbursed drugs using RWD.

 

One key example was using an RSA system based on patient unit performance.

 

Korea is currently using an RSA mechanism that is overwhelmingly focused on finances when reimbursing high-priced drugs.

 

The main point of a performance-based RSA that uses RWD is in that the pharmaceutical company's responsibility increases with the high price of its drugs in order for the authorities to meet the continued challenge of reimbursing ultra-high-priced drugs with limited finances.