"Introduction of Imfinzi in cholangiocarcinoma treatment can be seen as a critical advance. That a new therapy offering the possibility of long-term survival has appeared after 12 years is highly encouraging."

As new treatment options for cholangiocarcinoma are introduced, a paradigm shift is said to be brought to this area, which was previously neglected and poorly developed.

Although it is too early to be certain of long-term survival in South Korea, Dr. Yoo says that it could be a game-changer since new treatment options can benefit patient.

Dr. Changhoon Yoo, Professor in the Department of Oncology at Asan Medical Center in Seoul, who has expertise in this field, shared limitations in the treatment setting of cholangiocarcinoma and possible improvements.

The prevalence of cholangiocarcinoma is known to be higher in Asia regions, including South Korea, China, and Taiwan, compared to Western countries. However, patients are often in advanced stages when diagnosed due to the challenging early diagnosis. It is one of the cancers that is difficult to reach a curative intent.

Dr. Yoo explained, "Cholangiocarcinoma has a low prevalence due to its high mortality, resulting in a low cumulative patient number relative to its incidence. Currently, only about 20–30% of cholangiocarcinoma patients are eligible for surgery, and the remaining approximately 70% must rely on drug treatments such as chemotherapy or immunotherapy."

While liver fluke infection was the leading cause of cholangiocarcinoma in the past, new factors such as fatty liver have emerged due to the westernization of dietary habits.

임핀지(더발루맙)After Imfinzi (durvalumab) received approval from the Ministry of Food and Drug Safety in November 2022, it is continuously expanding its influence.

Dr. Yoo said, "Although less than three years have passed since the indication approval and it is therefore difficult to confirm long-term survival rates, in clinical practice the proportion of patients showing improvement has increased compared with before," adding, "Considering that patients who received Imfinzi combination therapy early in the 2021 clinical study still have favorable outcomes, Imfinzi can be seen as providing benefits to patients."

In particular, Dr. Yoo focused on Imfinzi's side effects and safety.

Dr. Yoo explained, "Most side effects are caused by the cytotoxic chemotherapy agents used in combination, and there are almost no issues attributable to Imfinzi. It rarely causes patients to struggle or reduces clinical efficacy, making it a medication that is of considerable help not only to patients but also to healthcare providers."

"Korean subgroup analysis data on Imfinzi demonstrates long-term survival effects"

Another reason why Imfinzi combination therapy is gaining attention in cholangiocarcinoma is that overall survival (OS) was shown to be higher in Korean patients.

According to the study results, the two-year survival rate in the Korean patient group receiving the Imfinzi combination therapy was 38.5%, more than twice the 14.1% observed in the group that received chemotherapy.

Furthermore, the survival rate at 36 months was 21.0% in the Imfinzi combination group, more than double the 8.8% in the chemotherapy group.

Dr. Yoo analyzed, "In my opinion, I consider the Korean subgroup analysis data from the TOPAZ-1 study very encouraging. These results reflect the rapid accessibility and thorough patient management within the healthcare system."

In cholangiocarcinoma, where inflammation or adverse reactions often occur during anticancer treatment, leading to treatment interruptions and repeated hospital admissions and discharges, continuous cancer treatment itself is challenging.

According to Dr. Yoo, it is particularly common for treatment to be paused for a month or two due to inflammation, worsening the disease, and cholangitis can occur even when the cancer itself is not progressing. Therefore, high accessibility to treatment is critical in cholangiocarcinoma.

Dr. Yoo said, "In Korea, if inflammation or jaundice occurs, patients can quickly visit a hospital, receive a procedure, and recover, thereby immune checkpoint inhibitors can be administered continuously," and added, "Compared to countries with less-established healthcare systems, our accessibility and level of care are higher, so I believe the effectiveness of immune checkpoint inhibitors can be more pronounced."

Dr. Yoo also said, "Cholangiocarcinoma is indeed a challenging disease, but prognosis has improved recently and long-term survival cases are increasingly common," and added, "To secure approval or reimbursement for new drugs, one side's opinion is not enough. It is also necessary for patients and healthcare professionals to raise their voices together."

Reimbursement discussions remaining for Imfinzi combination Therapy… "The standard criteria application should be avoided"

However, the Imfinzi combination therapy is only reimbursed for the chemotherapy, and the cost barrier remains high.

Currently, Imfinzi's reimbursement criteria were established in November of last year. Following AstraZeneca Korea's application for the cost‐effectiveness track, the Health Insurance Review and Assessment Service (HIRA)'s Economic Evaluation Committee is expected to discuss Imfinzi's cost‐effectiveness this month.

According to industry sources, this month's Economic Evaluation Committee will review the cost‐effectiveness of Imfinzi+gem-cis combination therapy as a first‐line treatment for locally advanced or metastatic cholangiocarcinoma. It will be forwarded to the Drug Reimbursement Evaluation Committee if it passes the Economic Evaluation Committee.

In this regard, Dr. Yoo noted that, for the sake of patient access, the standard criteria should be avoided.

For example, in hepatocellular carcinoma, the standard treatment, sorafenib, is not particularly low‐cost, so its price could not be matched when a new drug emerged. Still, it is disadvantageous for a new drug to meet such price benchmarks for rare diseases or those where drug development has lagged.

Dr. Yoo said, "When nanoliposomal irinotecan was introduced as a second‐line treatment for pancreatic cancer, it also faced challenges in economic evaluation when compared with 5‐FU." And added, "Likewise, I do not think conducting a straightforward economic comparison between existing cholangiocarcinoma drugs, whose patents have expired and thus are inexpensive, and an innovative new drug developed after a decade is appropriate."

Dr. Yoo also described cholangiocarcinoma as 'the lung cancer of the gastrointestinal cancer family,' emphasizing the importance of precision medicine in new drug development.

"Although cholangiocarcinoma has one of the poorest prognoses among gastrointestinal cancers, I am interested in the possibility of developing targeted therapies based on genetic analysis of specific biomarkers," Dr. Yoo added, "Approximately 4–5% of cholangiocarcinoma patients carry specific gene mutations, making this a cancer type with high potential for precision‐medicine application, and research is underway."

Finally, Dr. Yoo urged, "Support for precision medicine and targeted‐therapy development is urgently needed to broaden patient treatment opportunities." And, "I hope that cholangiocarcinoma patients will not lose hope and will actively pursue their treatments."