Only half of the multiple myeloma drugs reimb in KOR

Difficult to prove cost-effectiveness due to the rare cancer characteristic... requires improvements

Non-reimbursed bispecific antibodies Elrexfio and Tecvayli remain non-reimbursed

Despite the increased number of treatment options, patient access to those multiple myeloma drug options has not changed much.

 

Multiple myeloma remains an incurable disease, but in the past, survival rates were very low due to limited treatment options.

 

In recent years, however, innovative treatment options such as monoclonal antibodies, CAR-T therapies, and bispecific antibodies have diversified the treatment options, improving survival.

 

In fact, over the past 20 years, the five-year survival rate for multiple myeloma patients has increased from 29.8% in 2001-2005 to about 50.1% in 2017-2021.

 

However, this is still less than the 60% survival rate found in developed countries such as the United States, and limitation in access to care is regarded as the major contributing factor.

 

Only half of the guideline-recommended drugs are reimbursed in Korea In Korea, only 13 (52%) of the 22 drugs recommended in the NCCN guidelines for multiple myeloma are covered by reimbursement (based on the NCCN guidelines 2024 v2).

 

For example, Darzalex (daratumumab) was approved in 2019 as a first-line combination therapy for multiple myeloma but was only granted reimbursement as a fourth-line monotherapy in Korea.

 

In October last year, 5 years since the Drug Reimbursement Evaluation Committee recognized the appropriateness of expanding Darzalex’s reimbursement coverage under the Risk Sharing Agreement (RSA).

 

Also, Xpovio (selinexor) was granted reimbursement in July 2024, after 4 reimbursement attempts after its approval in 2021.

 

Burden of proving cost-effectiveness for rare cancers such as multiple myeloma One of the reasons why it takes longer to reimburse the crucial multiple myeloma drugs and hinders access is that multiple myeloma is a rare cancer, which renders it more difficult to prove cost-effectiveness than other cancer drugs.

 

In order to apply for reimbursement of high-priced anticancer drugs, the companies must submit data that demonstrates the drug’s cost-effectiveness, as per the guidelines for pharmacoeconomic evaluations.

 

In particular, due to the rising financial expense spent on anticancer drugs in recent years, the government has been setting higher standards for the submitted data to demonstrate the improvement in the effectiveness of new drugs over existing drugs.

 

However, multiple myeloma is similar to rare diseases in that it has a limited number of patients that can enroll in clinical trials, and it is difficult to set a comparator drug.

 

In the case of anticancer and rare disease drugs, clinical trials often have a single-arm design or are limited to Phase II studies, which introduces uncertainties and challenges in the reimbursement review process.

 

Add to this, the number of new treatment options has been growing.

 

Recently, bispecific antibodies, which are regarded as the next-generation biotechnology, have been approved and released for multiple myeloma.

 

Bispecific antibody treatments are immune cell therapies that consist of two monoclonal antibodies that recognize the target antigens of multiple myeloma and T cells.

 

Bispecific IgG2 kappa antibodies, which are composed of two monoclonal antibodies that recognize the target antigens of multiple myeloma, B-cell maturation antigen (BCMA) and CD3 antigen, respectively, are common and are a novel treatment that directly targets cytotoxic T cells to BCMA-expressing multiple myeloma cells.

 

Despite their high clinical efficacy, the bispecific antibody therapies currently approved in Korea, including Pfizer's Elrexfio (elranatamab) and Janssen's Tecvayli (teclistamab) and Talvey (talquetamab), remain non-reimbursed.

 

Suk Jin Kim, Professor of Hematology-Oncology at Samsung Medical Center and the President of the Society of Hematology said, “Although treatment outcomes have improved significantly with the active development of new drugs, the limited access to high-priced anticancer drugs have been preventing patients from receiving optimal treatment as needed.” Kim added, “Policy changes are needed, including flexibility in Korea’s reimbursement standards, to ensure that patients in Korea have access to treatment that meets global standards.

 

We must urgently expand patient access to treatment through early adoption of innovative therapies to close Korea’s survival gap compared with other countries.”