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- 2026-06-06 10:09:54
- InterView
- [Desk View] Is all anticancer adjuvant therapy on a losing streak?
- by Eo, Yun-Ho Feb 02, 2026 02:17pm
- Anticancer drugs are on a losing streak in their quest for insurance reimbursement in the adjuvant therapy category. Reimbursement is deemed necessary, but meeting the agreement is not easy.The concept of continued medication administration as 'prophylaxis' is not new. In the field of chronic disorders, medicines have been used as part of a 'management' regimen rather than as a treatment. For example, anticoagulants have been designed as a prophylaxis.The issue emerged as prophylaxis field expanded to include high-cost, cutting-edge anticancer drugs. Various new anticancer drugs are securing peri-operative prophylaxis indications and being researched. Adjuvant therapies for numerous numbers of cutting-edge new drugs, including immune checkpoint anticancer agents, targeted anticancer drugs, and antibody-drug conjugates (ADC), are now one of the expanding indications. We are seeing flood of indications. The subject of adjuvant therapy raises concerns. The cost issue is one of them. While it is well known, recurrence of cancer is terrifying even after remission. AlthoughHowever, prescribing anticancer drugs as adjuvant therapy and applying insurance reimbursement is posing a burden on the Health Ministry. In fact, there have been only a few cases in Korea where adjuvant therapies have received reimbursement expansion.CDK4/6 inhibitors, for instance. Both Eli Lilly’s 'Verzenio (abemaciclib)' and Novartis's 'Kisqali (ribociclib)' have obtained indications in Korea for adjuvant therapy in early-stage breast cancer.Verzenio has been at the forefront of the reimbursement challenge, yet the result has been three rejections by the Cancer Disease Review Committee (CDRC). Verzenio struggled to even get on the CDRC list from its very first attempt. After a long six-month wait following the initial submission, it was finally tabled in May 2023, only to result in "failure to establish reimbursement criteria." Five months later, Lilly resubmitted the application to the Health Insurance Review and Assessment Service (HIRA) in October. While it was considered at the CDRC in March and July of last year, the outcome remained unchanged.Recently, the company reapplied for Kisqali. As it awaits its turn on the CDRC agenda, attention is focused on whether it can achieve a different result.What remains clear is that the benefits of adjuvant therapy are garnering significant attention from the academic community. Adjuvant therapies are already appearing in the guidelines of leading global societies, often receiving high recommendation grades.It is time for full consideration. We must meticulously weigh the necessity of oncology adjuvant therapies for each medication and prioritize practical benefits over vague "financial burdens." Administering treatment only after a patient has relapsed may actually prove to be less cost-effective. Recurrence and metastasis are fatal factors that increase cancer mortality rates. Since there is no single correct answer, the pros and cons must be carefully balanced. We cannot neglect the growing list of adjuvant therapies.
- InterView
- "Generic price cuts to signal pharma industry transition"
- by Lee, Jeong-Hwan Jan 26, 2026 01:19pm
- Jeong Kyung-sil, Head of the Office for Healthcare Policy under the Ministry of Health and Welfare (MOHW)"It is true that Korea's generic prices are over-valued compared to other countries. To ensure a stable supply of short drugs, we cannot rely on a generic-centric industry structure. Criteria must be established to drive the transition from generic production to new drug development. The government's drug price policy aims to improve the industry structure, rather than a matter of National Health Insurance funding, by funding pharmaceutical companies that contributed to innovation."Jeong Kyung-sil, Head of the Office for Healthcare Policy under the Ministry of Health and Welfare (MOHW) explained, related to the aim of the drug price reform plan, "The administrative direction of the new drug pricing reform is to improve the direction of the domestic pharmaceutical industry by adjusting generic prices that are high-priced compared to other nations."The MOHW notes that, since the 2012 drug price cuts, the domestic industry has failed to deliver sufficient innovation or new drugs as of 2026. Consequently, there is a significant need to send a clear message to domestic pharmaceutical companies toward innovation through this reform.Regarding the review of "ingredient-name-prescribing" for National Essential Medicines, the MOHW plans to implement a policy related to simplifying post-notification for substitution drugs. Then, it will be reviewed once society-wide needs about supply instability grow.Addressing the legislative delay of the bill prohibiting non-face-to-face treatment platforms like Doctor Now from operating as drug wholesalers, Jeong said, "The law has been incorrectly framed. Jeong stated the law does not ban the platform business itself but prohibits unfair trade practices with potential conflicts of interest. Furthermore, it should be discussed at the National Assembly."During a recent meeting with the Korea Special Press Association, Jeong stated MOHW's views on drug price reform plan, limited ingredient-name-prescribing, and the wholesale platform ban."Regarding drug price cuts, opinions on ways to relieve the burden on the pharmaceutical industry will be gathered"Jeong said that the reform is inevitable because today's Korean pharmaceutical industry remains preoccupied with generic manufacturing and sales.Even among 40 designated "Innovative Pharmaceutical Companies," there are cases where generic sales account for 90% of revenue, proving that high generic pricing does not lead to innovation.The MOHW intends to adjust generic prices set above overseas levels and use those resources to support pharmaceutical companies that contribute to new drug innovation and the resolution of supply instability.However, acknowledging industry backlash and demands for predictability, the MOHW plans to continue collecting opinions to reduce the impact on companies.Jeong said, The goals of reducing drug pricing are intended to reduce unnecessary drug expenditures and evolve the pharmaceutical ecosystem," and added, "Both the Office for Healthcare Policy and the industry agree on." "It is true that Korea's generic prices are over-valued compared to other countries. The Ministry decided that the current drug pricing system is designed to favor a generic-centric market. Therefore, the reform is intended to lower the drug price and encourage innovations," and added, "For example, the industry will advance if fostering innovative pharmaceutical companies and supporting R&D are accompanied."Jeong said, "During the drug price reform 13 to 14 years ago (2012 general price cuts), generic prices were maintained at a higher level than those in developed countries. While some pharmaceutical companies have successfully improved their fundamental competitiveness since then, others have shown no improvement," and added, "This is an evidence that maintaining high generic prices does not inherently lead to innovation. While the framework of the announced reform plan is unlikely to change significantly, the MOHW is currently reviewing ways to mitigate the impact on the industry by actively soliciting and considering the opinions of pharmaceutical companies.""Ingredient-name-prescription, first to simplifying substitution drug prescription…whole-sale ban has been incorrectly framed"Regarding the Medical Service Act's inclusion of non-face-to-face treatment effective December 24, Jeong assessed that the Pharmacy Act amendment, including coverage of wholesale bans and mandatory DUR checks, has been put in the wrong frame.Jeong explained that while traditional players operate within an institutional framework, new platforms have entered a regulatory vacuum. The law is intended to fill this legal gap rather than specifically target platforms.The MOHW plans to coordinate specific adjustments to the non-face-to-face treatment pilot program in sync with the upcoming revisions to an article of the Medical Service Act.Regarding the President Lee Jae Myung administration's national task of limited ingredient-name-prescribing, the MOHW said that this agenda will be discussed only after the administration of simplified post-notification for substitution drugs for pharmacists, which is set to begin on the 2nd of next month."While the pilot program has been ongoing, revised measures could be implemented early, ahead of the official December enforcement, as we are working on articles and gathering feedback from expert advisory groups," Jeong added, "It depends on the speed of drafting the article of the Act."Jeong also explained, "The bill has been incorrectly framed as similarity to Doctor Now or Tada Ban," and added, "While traditional healthcare entities operate within a strict institutional framework, the emergence of telemedicine platforms created a regulatory vacuum. She emphasized that the law is not intended to prohibit the platform industry itself, but rather to fill a legal gap and prevent potential unfair trade practices or conflicts of interest.Jeong emphasized, "While other existing players (doctors, pharmacists, etc.) were operating within an institutional framework, a new player (platform) entered, and the platform alone became able to do anything without any regulation," and added, "It is a concept of creating an overall system for the legal and institutional gap that has arisen, not a concept of only stopping platforms."Jeong said, "It has currently passed the standing committee and the Legislation and Judiciary Committee in the National Assembly, and the lawmakers who passed the bill have not significantly changed their positions to date. However, the MOHW has no authority to step forward. Ultimately, it is a structure that can only be discussed at the National Assembly level. It is difficult to call the wholesale operation of non-face-to-face treatment platforms innovation. It is a law to prevent unfairness, not to stop the platform business itself."Jeong concluded by stating, "Regarding the wider use of generic substitution, we will create tools to make it more convenient to utilize, and whether to mandate ingredient-name-prescribing requires social consensus," and added, "If we were to discuss mandating ingredient-name-prescribing, we should start by reviewing drugs with unstable supply. Nowadays, generic substitution can be used much more flexibly than before. Since the problem of supply-unstable drugs is linked from production to supply, whether to implement ingredient-name-prescribing should be discussed later."
- InterView
- "Leclaza+Rybrevant shifts the EGFR lung cancer trt paradigm"
- by Son, Hyung Min Jan 21, 2026 09:04am
- "The Leclaza + Rybrevant combination therapy confirmed improvement to survival in high-risk patient group with EGFR-mutant lung cancer. The result shifted the existing treatment strategy centered around monotherapy to combination therapy."During a recent meeting with DailyPharm, Professor Ji-Youn Han from the Division of Hematology-Oncology at the National Cancer Center explained the clinical significance of the Leclaza + Rybrevant combination therapy and the changes in EGFR-mutant non-small-cell lung cancer (NSCLC).Professor Ji-Youn Han from the Division of Hematology-Oncology at the National Cancer Center 'Leclaza (lazertinib)' is an EGFR-mutant NSCLC treatment developed by Yuhan Corp. It is a third-generation tyrosine kinase inhibitor (TKI) that targets exon 19 deletions and the exon 21 (L858R) mutation. Johnson & Johnson secured the global rights to Leclaza and has been conducting clinical research on its combination therapy with 'Rybrevant (amivantamab).'Rybrevant is a fully human bispecific antibody that fundamentally blocks tumor growth pathways by simultaneously inhibiting active EGFR mutations and MET mutations·amplification. This drug inhibits various resistance pathways observed in EGFR-mutated lung cancer by blocking ligand binding and promoting receptor degradation.The Leclaza + Rybrevant combination therapy demonstrated improvement in overall survival (OS) in the global Phase 3 MARIPOSA study. In an Asian sub-analysis presented at the European Society for Medical Oncology Asia Congress (ESMO Asia 2025) this year, the combination therapy reaffirmed an OS effect consistent with the global clinical data.A total of 858 participants were enrolled in the MARIPOSA study, including 501 Asian patients. In an analysis at a median follow-up of 38.7 months, the combination therapy reduced the risk of death in Asian patients by 26%. While the median OS for the combination group was not reached, it was 38.4 months for the control group receiving 'Tagrisso (osimertinib)' monotherapy, suggesting the survival benefit of the combination could exceed one year. The 36-month survival rate also remained higher for the combination group at 61% compared to the Tagrisso group.Since Asian patients have a higher prevalence of EGFR mutations and different disease characteristics compared to Western populations, shifts in treatment strategies have a more significant impact on clinical practice. This analysis, which confirmed survival-improvement effects identical to those in the global data, once again demonstrated that Leclaza + Rybrevant has sufficient efficacy in Asian patients.Currently, Leclaza + Rybrevant is approved as a first-line treatment in major countries, including South Korea, the United States, Europe, Japan, and China. Given the high prevalence of EGFR mutations among Korean patients, this Asian analysis is expected to significantly influence clinical guidelines and first-line treatment strategies. Furthermore, the fact that major adverse events (paronychia and rash) of the combination therapy can be managed preventively through the COCOON study is emerging as a competitive advantage.Professor Han assessed, "Leclaza + Rybrevant combination therapy clearly demonstrated consistency in therapeutic effect by showing results in the Asian patient group identical to those of the global clinical trial," adding, "I believe the paradigm shift toward combination therapy-centered treatment has begun."Q. Sub-analysis data from MARIPOSA were recently released. What is your evaluation of the results?Research on combination therapy for the treatment of EGFR-mutated NSCLC has shown a complete shift in the first-line treatment paradigm. Clinically significant improvements in progression-free survival (PFS), the primary endpoint, and OS, the secondary endpoint, were confirmed. These results clearly suggest that combination therapy rather than monotherapy should be adopted as a treatment strategy.However, it is difficult to see combination therapy in its current form as the definitive answer. While there is an OS benefit, it does not manifest identically in all patients. Depending on the mechanism of action, some patients may find the Rybrevant combination therapy more advantageous, while others may prefer 'Alimta (pemetrexed)'-based treatment. Additionally, the possibility remains open for new drugs with different mechanisms to be added as combination partners in the future.What is clear is that a distinct unmet need for monotherapy has existed. Specifically, the limitations of existing treatments were evident in high-risk patients, underscoring the need for a breakthrough therapeutic. Leclaza + Rybrevant presented a meaningful alternative for high-risk patient groups and further clearly demonstrated OS improvement. Based on this evidence, I assess that the treatment paradigm has now completely shifted.Q. How do you interpret the reason why the survival improvement effect of Leclaza + Rybrevant appeared consistently in Asian patients?If the global and Asian data are consistent across all subgroups and show no significant differences, this is the ideal clinical result.As a bispecific antibody, Rybrevant has the advantage of simultaneously inhibiting both the EGFR and MET pathways. Clinically, approximately 10–15% of patients exhibit MET-dependent resistance pathways. This patient group has a relatively poor prognosis, and Rybrevant has the potential to show long-term survival benefits in these patients.Q. Is it the ideal direction to proceed with combination therapy as the standard of care?While it is an ideal direction, I do not believe that combination therapy must be applied to every single patient.Recently, researchers have redefined high-risk patient groups for whom combination therapy should be applied, thereby refining the first-line treatment approach strategy, which I view as an essential step. In my opinion, the patient groups to whom I recommend combination therapy are those with a high tumor burden. For example, high-risk groups with confirmed bone, liver, or central nervous system (CNS) metastases.This also includes patients who show biologically aggressive characteristics, such as mutations being detected in circulating tumor DNA (ctDNA) tests. Additionally, because patients with the EGFR L858R mutation often do not achieve long-term response with monotherapy, combination therapy is considered.Looking at the distribution of EGFR mutations that constitute the high-risk patient group, exon 19 deletions and L858R substitution mutations are roughly split half-and-half. Specifically, I estimate exon 19 deletions at about 60% and L858R at about 40%. Among these, patient groups with high tumor burdens include those accompanied by bone or brain metastases. Among these patients, about 30–40% are high-risk EGFR-mutated NSCLC patients.Q. Overall, how do you evaluate the safety data regarding the adverse events of the Rybrevant combination therapy?Looking at the entire development process, from CHRYSALIS to MARIPOSA and MARIPOSA-2, the journey to find the proper indications was long. Because the drug's adverse events were clear, Johnson & Johnson conducted extensive research on them and simultaneously developed educational programs to help medical staff using the drug in clinical settings do so safely. The COCOON study was also conducted in this context.The COCOON study is significant in that it presented a standardized protocol for systematic management. Whereas management methods previously varied by medical staff or institution, a standardized management strategy that anyone can apply has now been introduced with the COCOON regimen.Rybrevant tends to have more adverse events than Alimta-based treatment. Since it is a drug targeting EGFR, there are areas where EGFR-related adverse events partially overlap when combined with Leclaza. In fact, combining Rybrevant and Leclaza for patients with exon 19 deletions or exon 21 (L858R) mutations may be more challenging than combining Rybrevant and chemotherapy for patients with exon 20 insertion mutations. This is particularly true for adverse events directly associated with EGFR mutation inhibition.A standardized adverse event management strategy was established through the COCOON study, which was designed to preventively manage skin-related adverse events associated with the Rybrevant combination therapy. For example, detailing the active use of chlorhexidine preparations or topical treatments when paronychia occurs is a novel part organized by the COCOON study.Overall, it was a very consistent and determined development strategy. The study did not avoid the drug's adverse events but addressed them directly, ultimately building a clinically applicable management system.Q. The subcutaneous (SC) formulation of Rybrevant was approved in the United States. What impact do you think it will have on clinical practice if introduced in Korea in the future?The characteristic of an SC formulation for anticancer drugs differs from that of methods such as insulin injections, which are administered in small volumes. Anticancer drugs require a specific dose to be administered, and a process is needed to wait for the drug to be absorbed by the body after administration. In cases like exon 20 insertion mutations, where it is combined with intravenous (IV) chemotherapy, a method of simultaneously administering IV and SC drugs during treatment may not be feasible.While there might be a possibility of administering the SC formulation alone with an oral targeted therapy, even in this case, the volume of drug to be injected is not small, and the local volume after administration is significant. Considering that one must wait for the drug to be absorbed into the local area after administration, we need to wait and see how applicable it will be in actual clinical practice. For these reasons, the application of the SC formulation in combination therapy is likely to be more limited compared to monotherapy.Q. With treatment options diversifying and long-term survival being expected, how do you view the issue of reimbursement for combination therapy?Currently, there are quite a few patients who maintain long-term treatment for over five years while taking an EGFR-TKI. In fact, there is a patient who has continued taking the same EGFR-TKI since the early days of our hospital's founding. Because these patients have a great fear of recurrence if they stop the drug, they continue treatment, having become accustomed to it.As the emergence of targeted therapies has led to an increase in stage 4 lung cancer patients surviving long-term at levels similar to patients who have undergone surgery, the 'Special Case Medical Expense Coverage System' is not being fully applied. There are cases where the exception ends after taking an EGFR-TKI for more than 5 years, and a realistic consideration is becoming necessary about how long the subsequent treatment can be recognized by insurance.Q.How do you expect the first-line treatment paradigm for EGFR-mutated lung cancer to change in the future?The MARIPOSA study shifted the paradigm for first-line treatment of EGFR-mutated NSCLC. In particular, it demonstrated the consistency of the therapeutic effect, as results identical to the global data were confirmed in the Asian patient group.Rybrevant’s mechanism of action clearly shows an overall survival benefit by comprehensively blocking not only the major signaling pathways centered on EGFR mutations but also MET-dependent bypass pathways, which occur in about 10–15% of cases. Considering these mechanistic characteristics, one can also expect a "long-tail effect," showing long-term survival after administration, in the latter half of the survival curve for the Rybrevant combination therapy.Of course, the possibility of accompanying adverse events is higher in combination therapy since two agents are used together. However, through studies like COCOON, an excellent protocol for adverse event management has been developed, establishing a foundation for easier application in the real world.
- InterView
- "Lilly's values in caring and discovery: social contrib."
- by Son, Hyung Min Jan 19, 2026 09:02am
- "Lilly's core values have always been 'Caring' and 'Discovery'. GDOS demonstrates that these values are not merely slogans, but a platform where employees can personally practice them within the community and reaffirm the company's reason for existence."On September 25 last year, Eli Lilly Korea held a volunteer activity attended by all employees to celebrate the 'Global Day of Service (GDOS).' GDOS is a global volunteer program launched by Lilly in 2008, which has accumulated over 1.2 million volunteer hours across 65 countries to date. Eli Lilly Korea has participated since 2010, contributing more than 20,000 hours of service.The Global Day of Service is a day when organizations and individuals worldwide volunteer for local communities and global issues. Lilly operates this day annually as its own GDOS, effectively establishing it as "the volunteering day for Lilly employees worldwide."The theme for the 2025 Eli Lilly Korea GDOS was "A Better World Made Together," centered on two pillars: environmental improvement and support for underprivileged children. Approximately 250 employees were divided into groups to conduct plogging in Jung-gu and Seodaemun, and to create donation kits for child support. The donations were delivered through Save the Children, in cooperation with the Community Chest of Korea. They provided necessary items to children at the Manan District Social Welfare Center in Anyang.2025 Eli Lilly Korea GDOS TF Team. From left: Hyung-min Kim (Regulatory Affairs), Minju Kang (Medical), Yu Seok Kang (ENC), Joohee Lee (Medical)Notably, they introduced 'GDOS Night; for the first time last year, featuring a charity performance by the in-house band and a donation program. A new attempt was made to expand the post-volunteer interaction into another act of sharing by accumulating the entire proceeds from food and beverages purchased by employees as donation funds.GDOS aligns with Lilly's global sustainability strategy, which includes: ▲ expanding access to medicines ▲ improving environments with limited medical infrastructure ▲ strengthening community resilience ▲ minimizing environmental footprints ▲ fostering innovation grounded in inclusion and diversity.We met with Minju Kang, Medical Director of Eli Lilly Korea, who managed last year's GDOS, and the TF team (Joohee Lee, Yu Seok Kang , and Hyung-min Kim) to discuss preparations, operations, achievements, and future directions.Q. Please describe your roles in last year's GDOS.Minju Kang (Medical): I am Minju Kang, head of the Medical Department and the sponsor for GDOS. Each year, a different department takes turns sponsoring GDOS, and since the Medical Department was in charge last year, I took on the role of sponsor.Hyung-min Kim (Regulatory Affairs): I am Hyung-min Kim from the Regulatory Affairs team. For this GDOS, I established the overall program plan, selected volunteer activities, and managed group assignments.Joohee Lee (Medical): I am Joohee Lee from the Medical Department. This program was led by three 'Champion Leaders' centered around Vice President Minju Kang. My role involved planning the program, encouraging employee participation, and setting the direction for which values we should focus on.Yu Seok Kang (ENC): I am Yu Seok Kang from the ENC team. Since GDOS is a long-standing event, my primary role was to ensure that the company's regulations and procedures were strictly followed throughout the support process.Q. What kind of event is GDOS?Minju Kang (Medical)Minju Kang: GDOS is a global corporate social responsibility program in which all Lilly branches worldwide, including the U.S. headquarters and Korea, participate. It has continued for nearly 20 years since its 2008 launch, and Eli Lilly Korea has participated consistently since 2010.It is highly significant because it goes beyond a simple annual event; it is a time for us, as members of a pharmaceutical company, to reconnect with our fundamental purpose asking 'Why do we do this work?'GDOS is operated as a volunteer-based activity involving all employees, and each year the TF Champions plan the program. The format changes annually. In some years, we conduct plogging to improve community environmental health, and other years we assemble donation kits for the underprivileged.Last year, we conducted plogging and kit production simultaneously. Furthermore, we introduced GDOS Night, aiming to extend the lasting impact of volunteer work into a time for sharing and interaction.Q. Compared to other CSR activities, what makes Lilly's program special?Hyung-min Kim (Regulatory Affairs)Hyung-min Kim (Regulatory Affairs): Generally, many volunteer programs rely on external vendors to handle the event's composition and operation. However, at Lilly, employees form their own TF to take the lead in the entire process—from planning and execution to reporting results.Members experience creating the program as protagonists rather than mere participants, gaining a sense of belonging and achievement. I believe this is the fundamental differentiator of Lilly's CSR activities.Joohee Lee: Having worked at other pharmaceutical companies, I found it particularly impressive that Lilly employees worldwide participate in volunteer work on the same day.It is rare to find CSR activities in which employees from every country, including the U.S. headquarters, work toward a common goal with a consistent identity as 'Team Lilly.'Additionally, not only the domestic TF but also TF Champions from each country meet in advance to discuss the planning direction and implementation methods for the GDOS program, and share the results after the event. This systematic approach, in which we learn from and spread the activities of different countries, sets GDOS apart as a sophisticated global volunteer program rather than a simple local activity.Q. We heard the 2025 theme was 'A Better World Made Together.' What were the central messages you focused on?Yu Seok Kang : The first pillar was 'Environmental Improvement.' To this end, we collaborated with the 1365 Volunteer Center to conduct plogging activities that could directly improve the local community's environment. The second pillar was 'Child Support.' In the past, we focused on visiting orphanages to improve their facilities. Last year, during discussions with the Community Chest of Korea, we explored ways to provide more practical help to children.As a result, we agreed that an approach of personally creating and delivering items that children need and would enjoy was more meaningful. By structuring the program around these two core pillars (environment and children), we prepared for the theme of "A Better World Made Together" to be portrayed through the activities.Q. Could you explain what "GDOS Night" was?Joohee Lee (Medical)Joohee Lee: In the past, after the volunteer work was completed, employees typically went their separate ways.Last year, we introduced GDOS Night to strengthen GDOS's communal values further.Many employees voluntarily attended the event even after the official activities ended, continuing the interaction and sharing. I believe this experience catalyzed GDOS to naturally expand its purpose.Minju Kang: GDOS Night was an event planned around the 'Lilly Band,' an in-house club.Employees who participate in the band as a hobby held a charity concert. We expanded the traditional 'one-day cafe' format by renting an external club space, selling food and drinks, and raising funds.It was meaningful because it provided a direct platform for employees to participate and spread a culture of donation beyond just a concert.Q. What is the meaning behind Lilly's core value, 'To unite caring with discovery to make life better for people'?Yu Seok Kang : We thought deeply about how to integrate caring and discovery into our activities harmoniously. In particular, introducing the unprecedented GDOS Night was meaningful because it expanded the event from a mere gathering of Lilly members into a festival where we could share and personally experience the values the company pursues.Furthermore, we interpreted the purpose of GDOS differently by introducing a structure that converted the program's proceeds into donations. I believe this series of processes was an attempt to meld the corporate purpose of caring and innovation into overall activities, and a case of seeking new ways through new methods.Minju Kang: Lilly holds 'Respect for People' as one of its core values, which is based on fundamental respect for human dignity and life. Caring, stemming from this value, has a practical meaning for a pharmaceutical company: providing patients with treatment opportunities and improving their quality of life. At the same time, for such caring to be realized in reality, innovative discovery and continuous R&D investment are essential.Therefore, Lilly simultaneously pursues a people-centered perspective and the discovery that realizes it. The discovery mentioned here refers to the development of new medicines, which directly links to our corporate purpose, as only innovative treatments can provide substantial benefits to patients. From this perspective, GDOS is not an event separate from the company's daily operations, but an extension of the process where caring and discovery for a better life are connected through activities.Q. Is there a message you would like to send to the TF preparing for this year's GDOS?Yu Seok Kang (ENC)Yu Seok Kang: Last year, we made various attempts based on collaborations with several volunteer organizations. However, since most volunteer groups operate with small numbers, there were constraints on schedules and operational methods during the coordination process for GDOS, which requires large-scale participation.In the future, we can enhance efficiency and meaning further if collaboration schedules are coordinated and prepared at an even earlier stage. Although there was some trial and error, the process itself, including discussing with TF members and brainstorming ways to maximize employee participation, was very valuable, and I am satisfied with the overall results.Joohee Lee: The feedback left by CEO after last year's GDOS was also memorable. The suggestion was to consider activities that can deliver hope to patients, caregivers, and the community, aligned with a pharmaceutical company's identity and role. From this perspective, I believe programs that create direct touchpoints with patients, such as the Lilly Band holding a concert for patients, are directions worth considering for the future.Kim Hyung-min: GDOS is not an activity directly linked to performance metrics, but it is an event we must participate in with a sense of mission, personally practicing the company's values as Lilly employees. Since it required preparation outside of working hours, personal motivation was necessary, and being able to provide momentum to each other while working as a team was a great help. I believe a TF involving members who can voluntarily offer opinions is a crucial element for the future success of GDOS.Minju Kang : The charity concert (GDOS Night), which was attempted for the first time last year, received a very positive response. If we consider how to further evolve this format for the charity concert next year, we can expand the meaning of GDOS even further.
- InterView
- Omjjara effective for high-risk myelofibrosis… access should be expanded
- by Son, Hyung Min Jan 13, 2026 06:59am
- “High-risk myelofibrosis patients with cytopenia have a clear unmet need due to the lack of appropriate treatment options. Omjjara is an option for these patients. This is why its reimbursement coverage is necessary.”Professor Sung-Eun Lee, Department of Hematology, Seoul St. Mary’s HospitalProfessor Sung-Eun Lee of the Department of Hematology at Seoul St. Mary’s Hospital emphasized the unmet needs in the myelofibrosis treatment landscape and the necessity of improving access to new therapies in a recent interview with DailyPharm.Myelofibrosis is a rare hematologic malignancy belonging to the chronic myeloproliferative neoplasm and is considered the most clinically severe condition within this group. Chronic myeloproliferative neoplasms are categorized based on the presence or absence of chromosomal abnormalities. Philadelphia chromosome–negative polycythemia vera and essential thrombocythemia generally show relatively slow progression.However, approximately 20% of patients with these conditions may progress to myelofibrosis and secondary acute leukemia over time, at which point the prognosis deteriorates rapidly.One of the most critical prognostic factors is hemoglobin level. Myelofibrosis is a disease where the bone marrow, the body's blood cell factory, is gradually replaced by fibrotic substances like collagen or reticulin, leading to a decline in normal hematopoietic function. As a result, blood cell production and maturation are disrupted, causing anemia.When anemia is present, the patient's quality of life significantly declines. Reports indicate that myelofibrosis patients with anemia have a survival rate approximately 3 to 4 times lower than those without anemia. For this reason, managing anemia is a major therapeutic goal in myelofibrosis treatment.In September last year, GSK’s Omjjara (momelotinib) received approval from the Ministry of Food and Drug Safety for the treatment of adult patients with intermediate- or high-risk myelofibrosis with anemia (including primary myelofibrosis, post–polycythemia vera myelofibrosis, and post–essential thrombocythemia myelofibrosis), thereby expanding treatment options.Omjjara differentiates itself through a unique mechanism of action. In addition to inhibiting JAK1 and JAK2 like conventional JAK inhibitors, it also inhibits ACVR1. This novel approach normalizes iron metabolism through suppression of hepcidin overexpression, a key cause of anemia.The introduction of Omjjara is expected to reshape myelofibrosis treatment strategies. However, as reimbursement coverage has not yet been granted, access to the drug remains limited for transfusion-dependent myelofibrosis patients in Korea.Professor Lee stressed the need to improve access to new treatments, emphasizing that myelofibrosis is a disease where patients can maintain social and daily life while undergoing treatment, and thus should not be restricted in all aspects of life.Q. Myelofibrosis is a relatively unknown rare blood cancer. Could you introduce the disease?Myelofibrosis is a disease closely associated with molecular genetic abnormalities, most commonly involving JAK2, CALR, and MPL mutations. JAK2 mutations are most frequent, followed by CALR and MPL mutations. Prognosis varies depending on the mutation type. Patients with CALR mutations generally have a relatively favorable prognosis, while so-called “triple-negative” patients are known to have the poorest prognosis. However, current clinical practice does not differentiate treatment strategies based on mutation subtypes.This disease presents with distinct symptoms and significantly impairs patients' quality of life. The course varies greatly depending on the patient's risk group and disease stage. As it is a functional disorder, there is currently no treatment that fundamentally halts disease progression. Therefore, selecting the appropriate treatment strategy based on patient status and risk assessment is critical.Median survival for intermediate- and high-risk patients is only 2 to 3 years, whereas early-stage patients may survive for more than 7 to 8 years. However, many patients are diagnosed only after symptoms develop, meaning many patients are already in the intermediate or high-risk group at diagnosis. In particular, patients with marked cytosis or the presence of blasts tend to show rapid disease progression.Q. How is myelofibrosis currently treated?The only curative treatment for myelofibrosis is hematopoietic stem cell transplantation. However, myelofibrosis predominantly affects elderly patients and is often accompanied by comorbidities, resulting in poorer post-transplant outcomes compared to other acute hematologic malignancies. Therefore, transplant candidates are selected very carefully based on age, general condition, comorbidities, performance status, and donor availability.For patients ineligible for transplantation, the primary treatment goals are to reduce splenomegaly and alleviate systemic symptoms. In this setting, JAK-STAT pathway inhibitors such as Omjjara, ruxolitinib, and fedratinib are used, each with distinct efficacy and characteristics. Because symptoms and clinical characteristics vary among patients, drug selection is personalized for each individual patient.Furthermore, securing a donor and improving the patient's overall condition prior to transplantation often requires a certain amount of time. During this period, drug therapy serves as a bridge to maintain the patient's condition in a stable state until transplantation.Q. What are the unmet needs in the current myelofibrosis treatment landscape?Historically, treatment options were limited for myelofibrosis. Ruxolitinib was effectively the only first-line therapy, followed by fedratinib. However, both drugs are difficult to use in patients with severe anemia or platelet counts below 50,000/µL.Anemia, in particular, has a profound impact on quality of life. Transfusion-dependent patients often require one or two units of blood per month and must spend 4–5 hours at the hospital for each transfusion. As the transfusion date approaches, systemic fatigue and weakness from anemia make mobility difficult. Patients often experience a pattern where daily functioning temporarily improves immediately after a transfusion, only to deteriorate again.When adverse reactions occur during treatment or the effect is insufficient, switching medications is necessary, but the limited alternatives have also been a major challenge in clinical practice. Consequently, patients who failed existing treatments or required additional therapy often had no options other than participating in clinical trials.Q. Omjjara was approved in Korea last September. Could you explain the clinical evidence supporting its approval?Unlike existing therapies, Omjjara has a mechanism of action that is favorable for anemia improvement and was therefore developed and approved specifically for myelofibrosis patients with cytopenia, an area with the greatest unmet need. Clinically, it can also be considered a treatment option for patients with severe thrombocytopenia, and improvements in anemia indicators are being observed in actual clinical practice.The SIMPLIFY-1 trial directly compared Omjjara with ruxolitinib and demonstrated non-inferiority in key endpoints such as spleen volume reduction. It also demonstrated clinically meaningful results in anemia-related parameters, providing the basis for the subsequent MOMENTUM study, which focused on anemia improvement and reduction in transfusion dependency.From a safety perspective, no new adverse effects of significant concern were identified compared to previous trials. While caution is required for infection management as with all JAK inhibitors, this falls within the familiar domain of healthcare providers treating hematologic malignancies and is deemed manageable in routine clinical practice.Q. Could you share any memorable patient cases or instances where notable changes occurred after using Omjjara?In Korea, experience with Omjjara has been accumulated through the Expanded Access Program (EAP), both as an initial treatment option or subsequent therapy after existing treatments in patients with myelofibrosis accompanied by anemia.Omjjara has also been used as a bridging therapy maintain stable conditions in patients awaiting hematopoietic stem cell transplantation. In one case, a patient with a baseline hemoglobin level below 8 g/dL and high transfusion requirements achieved recovery to approximately 11 g/dL after starting Omjjara. Furthermore, Omjjara is also being considered as an important treatment option for elderly patients for whom transplantation is difficult and who require long-term disease management.Q. What recommendations would you make to improve treatment access for myelofibrosis patients?Although myelofibrosis is a rare disease with a small patient population, unmet medical needs are substantial in clinical practice. In Korea, issues affecting large populations tend to receive more attention, while the voices of patients with rare, severe diseases are often overlooked. Consequently, the rarer and intractable the disease, the harder it is to secure reimbursement for new treatments.However, the unmet need is clear for high-risk myelofibrosis patients with cytopenia, as they lack appropriate treatment options. Furthermore, even intermediate-risk patients may experience changes in the clinical characteristics of their disease, making it crucial to secure multiple treatment options.Omjjara has accumulated robust clinical data in these patient populations. If reimbursement coverage is granted, patients who were previously constrained by repeated transfusions may be able to continue treatment more stably and return to daily life. Since myelofibrosis is a disease that allows patients to maintain social and daily activities while undergoing treatment, improving access to treatment will help ensure that patients are not forced to limit every aspect of their lives because of the disease itself.Currently, some patients continue Omjjara treatment through expanded access programs or at their own expense. Given the unmet needs in clinical practice, it is hoped that an environment will be established in which Omjjara can be introduced for more patients at the appropriate time.
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- [Reporter's View] CES 2026, Physical AI and utility task
- by Hwang, byoung woo Jan 08, 2026 07:31am
- One of the keywords from CES 2026, which opened in Las Vegas on January 6 (local time), was 'Physical AI.'As the world's largest tech exhibition, CES serves as a global stage for emerging trends in AI, digital healthcare, mobility, and smart homes.This year at CES, the primary themes in the medical sector were Medical AI with high clinical usability, automation-based diagnostic technologies, and innovations in women's health.While CES is intended to be a forum for the future of technology, this focus was even more pronounced this year.In this year's keynote, Nvidia put Physical AI at the forefront, emphasizing a flow of "Executable AI" spanning robotics, autonomous driving, and manufacturing. Even within the medical and healthcare sectors, Nvidia explained its utility cases in terms of products and partnerships rather than mere theoretical models.This shift is most visible in the digital health sector. CES designated digital health as a major pillar of its official programming.According to KOTRA analysis, the digital health sector saw a 7.4% year-over-year increase in participating companies, the highest growth rate among all industry groups at CES 2026.The term 'expansion' was also officially highlighted, with CES organizers noting that 2026 digital health programming would broaden to include women's health, AI, and wearables.However, the discussions surrounding Physical AI presents a new set of challenges for the domestic medical device industry, which has centered itself around digital health. The center of gravity is shifting from technological performance to the practical role played on-site.The industry is no longer satisfied with AI that proves its validity in research papers. It now demands evidence of how much it reduces bottlenecks in hospital workflows, cuts operating costs, and saves labor and time, ultimately translating these results into contracts.Digital health has long grown by "borrowing time from the market" under the guise of innovation. However, in the era of Physical AI, the market is asking whether that innovation can fundamentally alter cost structures and redesign daily clinical workflows.In this context, it is consistent with Lunit's position as an industry leader that has expanded its reach through global market share gains and strategic M&A that it continues to receive questions regarding its break-even point (BEP).This is not a task for any single company. The entire 'K-Medical Device' sector is expected to face a reality where medical software must integrate with hardware to prove tangible economic utility. For technology to become a reality, it must ultimately reach patients and medical professionals. The collaboration model recently demonstrated by Seers Technology and Daewoong Pharmaceutical is highly suggestive in this regard. A venture company with technical capacity, leveraging the robust sales network and trust of a traditional pharmaceutical firm to overcome the conservative barriers in the medical field, will be linked to future revenue demands.The question posed by CES 2026 is clear. Digital healthcare must now prove it is a tangible industry capable of generating revenue, overcoming its status as a mere 'future growth engine.'
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- [Reporter’s View] High-priced drugs: efficacy vs. efficiency
- by Son, Hyung Min Jan 07, 2026 08:46am
- The launch of high-priced anticancer drugs and treatments for rare diseases will continue. There is no question that the efficacy of new drugs is improving, with a growing number of therapies extending survival or even offering the possibility of a cure.The problem lies in how, for whom, and by what criteria these treatments should be used. This question is generating considerable debate in practice.According to the collective views of oncology specialists, the current reimbursement framework is producing paradoxical outcomes.This is because drugs for cancers with large patient populations are failing to secure reimbursement approval, even when they demonstrate strong clinical efficacy, simply because of concerns over financial sustainability. More problematic than the negative reimbursement decision itself is the lack of explanation regarding the rationale and criteria behind such decisions.In the past, in addition to the improvement in overall survival (OS), the contribution of domestic patients to clinical trials was a key evaluation factor during approval or reimbursement review for new drugs. If domestic patients contributed to the clinical results, this was also partially reflected in the approval and reimbursement decision-making process.Recently, however, a clear shift has emerged. Regardless of clinical benefit, drugs targeting cancers with large patient populations tend to be disadvantaged in reimbursement discussions, while coverage is increasingly concentrated on rare cancers with smaller patient populations. This perception is widely shared among clinicians.If reimbursement policies have been adjusted due to financial constraints, the criteria and reasoning behind those decisions must be explained more clearly. Otherwise, patients are left unable to understand why the treatment they need is denied reimbursement.Clinicians are well aware that healthcare resources are limited. However, there is growing concern over whether those limited resources are truly being allocated to where they are most needed.For example, during cancer follow-up care, CT, MRI, and PET scans are often repeated without clear clinical justification. Streamlining expenditures in these areas could free up a substantial amount of funding for essential treatments.The national health screening system also warrants reconsideration. Currently, cancer patients receive the same health screening notifications as the general population, leading to redundant examinations.Despite cancer patients undergoing regular follow-up and monitoring at hospitals, the central data system fails to administratively distinguish them. Simply excluding cancer patients from routine general health screenings could significantly reduce unnecessary expenditures.The government states that the ongoing drug price reforms and reimbursement system improvements are intended to safeguard the National Health Insurance finances and ultimately save patients with cancer and rare diseases. The direction itself, aimed at improving access to new drugs, cannot be denied. However, it is clear that before restricting treatment access, citing limited resources, we must first examine whether there are areas where public funds are being wasted inefficiently.In the era of high-priced innovative medicines, reimbursement decisions are not merely about reducing or expanding reimbursement. They are about choosing between efficacy and efficiency. If patient survival and access to treatment are truly the guiding principles, then the priority should be eliminating unnecessary spending and systemic inefficiencies.
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- [Reporter’s View| Global trials turn eyes toward Asia
- by Son, Hyung Min Dec 23, 2025 08:00am
- The key theme at the recent European Society for Medical Oncology Asia Congress ‘ESMO ASIA 2025’ held in Singapore went beyond simply global pharmaceutical companies presenting data on Asian subgroups.What became clear at the meeting was a deeper shift: the center of gravity for where clinical outcomes are generated, and where investment and development strategies follow those outcomes, is moving toward Asia.One of the most illustrative examples of this trend was AstraZeneca’s presentation at ESMO Asia. According to the company, 60–70% of patients enrolled in gastrointestinal cancer clinical trials are now being recruited in Asia. Trial designs are increasingly built around Asian data, with approximately 50 active clinical trial sites operating across the region.This signifies that, beyond mere participation rates, the core evidence determining the reliability of clinical outcomes is being generated in Asia. This is why projections indicate that incorporating Asia-centric data will become inevitable in future discussions regarding new drug approvals and reimbursement.The message global pharmaceutical companies are sending is now clear. Asia is no longer merely a site for secondary analysis in global clinical trials; it is becoming the point where clinical outcomes are first generated and where new drug development strategies originate. This signals that the day when the weight of primary endpoints shifts to Asia, rather than being relegated to subgroup analyses, is not far off.Within this wave of change, Korea's role becomes increasingly crucial. As long as Asia remains a diverse region rather than a monolithic entity, which country and which company will be chosen as a strategic partner ultimately depends on each company's level of preparedness. The question of what position Korea will occupy within this Asia-centered restructuring of clinical trials and investment has now become an unavoidable challenge.The shift in clinical outcomes naturally leads to changes in global pharmaceutical companies' investment strategies. From early development to late-stage trials, combination strategies, and follow-up studies, there is a clear move to place Asia at the center. In effect, clinical development and capital are relocating together. Essentially, a structure is forming where clinical trials and investment move simultaneously.A particularly noticeable change in this process amid China's growing presence. China is no longer merely a country with a large patient population. Building on national-level expansion of clinical infrastructure, regulatory innovation, and capital investment, it is rapidly accumulating the capability to independently drive development from early-stage clinical trials through to late-stage Phase III trials. Cases are also increasing where global pharmaceutical companies design clinical strategies by partnering with Chinese biotech firms for development.China's strengths extend beyond speed and scale. In specific mechanisms of action and indications, scenarios are emerging where China becomes the starting point for global development. This suggests that clinical trials and investments shifting to Asia could converge back to China.Korea has long established itself as a reliable clinical trial execution country in global trials. Rapid patient recruitment and excellent medical infrastructure are clear strengths. However, as China strengthens its design and leadership capabilities, maintaining a strategic presence through execution capabilities alone becomes difficult.What global pharmaceutical companies seek in Asia is not merely a large patient pool, but partners capable of jointly discussing development strategies. Engagement at the clinical design stage, interpretive capabilities for specific patient populations, and a research ecosystem capable of leading follow-up studies are becoming increasingly important.Korea possesses the conditions to move beyond being a mere participant and take on roles in clinical design, interpretation, and expansion. Its specialized clinical capabilities centered around large tertiary hospitals, accumulated experience with specific cancers and patient groups, and rapid data production speed are competitive even within Asia. The problem is that this capability remains confined to individual researchers or institutions and is not consolidated into a national-level strategy.The shift to Asia does not offer equal opportunities to all countries. While many nations conduct clinical trials, only a limited number accumulate results and sustain investment. As China rapidly moves to become a leading developer, what Korea needs is not a question of how much to participate, but a choice of how deeply to engage.
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- [Desk View] Thoughts on 18-year economic evaluation system
- by Eo, Yun-Ho Dec 22, 2025 08:54am
- Anticipation and concerns are remaining as the reform of the drug pricing system in 2026 is approaching.Amid the anticipated introduction of various regulatory frameworks and drug price-reduction measures, as a journalist covering multinational pharmaceutical companies, I am closely monitoring potential shifts in South Korea's economic evaluation system.It has been approximately 18 years since the economic evaluation system was first introduced as part of the 2007 Drug Expenditure Rationalization Plan. It has been more than enough time to identify systemic flaws and determine necessary improvements.Economic evaluation is a tool for assessing the cost-effectiveness of a drug. The majority of new medicines must undergo this process for insurance reimbursement listing.In principle, economic evaluation is straightforward. It measures clinical benefit against cost to determine how much more the payer is willing to spend. However, because these are new drugs, economic evaluations involve numerous assumptions, ranging from treatment duration and the extent of clinical efficacy recognized to endpoint selection, extrapolation, and weighting. Consequently, the structure is such that reimbursement is based entirely on which assumptions are accepted.The pace of new drug listing in South Korea is reportedly slower than in other major economies. Economic evaluation is cited as the primary cause. If it is such a simple tool, why does it take so long? It is because a consensus must be reached on those aforementioned assumptions. Every single underlying assumption requires total agreement.Because acceptance of these assumptions determines the Incremental Cost-Effectiveness Ratio (ICER) and, subsequently, the price, this process becomes a battle in which neither side can easily concede. We must reconsider whether spending this much time at this stage is truly the right direction.Depending on the assumptions made, a drug can be considered a cost-effective treatment or dismissed as unacceptably expensive. Currently, drug characteristics vary significantly not only across different therapeutic areas but even within the same disease category. However, the government’s actual evaluative criteria remain extremely limited. In contrast, looking at evaluation results from agencies such as the UK’s NICE, one can see instances in which manufacturer-proposed assumptions are accepted even when they deviate from traditional methods.Experts argue that, since assumptions in economic evaluations involve uncertainty, efforts must be made to minimize it. However, this is directly linked to the speed of new drug adoption. Minimizing uncertainty inevitably requires a significant amount of time. The real question is whether such a process actually reduces that uncertainty.For drugs with high clinical need, cost-effectiveness can be sufficiently demonstrated if different assumptions are applied. In other words, flexibility in evaluation can accelerate market entry. For more radical reform, we could consider a system that categorizes ICER ranges and determines drug prices accordingly.Similar to France's ASMR, South Korea could establish pricing criteria based on value, using scores for different ICER ranges as one component of the value judgment. It is finally time for a serious re-evaluation of the current system, where economic evaluation serves as the final stage of drug pricing.
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- ‘Adempas, effective alternative for patients with inadequate response to PDE5i’
- by Son, Hyung Min Dec 22, 2025 08:53am
- ‘The ultimate goal for PAH is to reach and maintain a low-risk status. If this goal is not achieved or the risk level is not sufficiently lowered, an early change in treatment strategy is essential.”Switching to Bayer’s sGC stimulator ‘Adempas (riociguat)’ is emerging as a practical treatment strategy for patients with PAH who do not show sufficient response to PDE5 inhibitors. With the recent establishment of reimbursement criteria in Korea, experts note that a turning point has been created in a treatment landscape that has long relied on sequential monotherapy.Vallerie McLaughlin, Professor of Cardiovascular Medicine at the University of Michigan Medical Center, and Wook-Jin Chung, Professor of Cardiology at Gachon University Gil Medical Center, recently emphasized the clinical potential of Adempas in PAH treatment during a recent interview with Daily Pharm.Vallerie McLaughlin, Professor of Cardiovascular Medicine at the University of Michigan Medical Center, Wookjin Jeong, Professor of Cardiology at Gachon University Gil Medical CenterPulmonary hypertension, characterized by abnormally elevated pulmonary arterial pressure, is classified into five groups based on etiology. Among these, Group 1 PAH and Group 4 chronic thromboembolic pulmonary hypertension (CTEPH) are considered rare diseases, accounting for only about 3% of all pulmonary hypertension patients each. Often beginning with nonspecific symptoms like shortness of breath, fatigue, and dizziness, patients frequently attribute these to aging or are misdiagnosed with other conditions at primary care facilities, leading to significant diagnostic delays.PAH is known to be a severe, life-threatening condition, with a mortality risk within 2-3 years if left untreated. In Korea, the number of diagnosed patients has increased due to greater physician awareness, efforts toward early detection, and wider use of right heart catheterization. However, concerns remain that the domestic treatment environment still diverges in part from global clinical guidelines.Unlike international guidelines that recommend initial combination therapy with endothelin receptor antagonists (ERA) and phosphodiesterase type 5 inhibitors (PDE5i) from the outset, sequential monotherapy-based treatment strategies remain commonly applied in Korea.PAH treatments are categorized by mechanism of action into: ▲ Endothelin receptor antagonists (ERAs) ▲ Nitric oxide pathway targeted therapies (PDE5 inhibitors, sGC stimulators) ▲ Prostacyclin analogues (PCAs) ▲ Prostaglandin receptor agonists (PRAs). Global guidelines recommend initiating treatment with a combination of ERA and PDE5 inhibitors from the outset, escalating treatment by adding agents with different mechanisms, such as PCA or PRA, when treatment response is inadequate. This strategy aims to maximize therapeutic efficacy by simultaneously targeting multiple pathophysiological pathways.In contrast, the domestic approach has primarily involved initiating treatment with ERA monotherapy, adding a PDE5 inhibitor if response is inadequate, and subsequently combining PCA or PRA if improvement is still lacking. However, studies report that despite the use of a combination of ERA and PDE5 inhibitors, some patients experience clinical deterioration due to inadequate response to PDE5 inhibitors or issues related to tolerability.In this context, Adempas (riociguat), an sGC stimulator, is gaining attention as a potential treatment option to replace PDE5 inhibitors.In the REPLACE study, which enrolled adult patients with symptomatic pulmonary arterial hypertension (PAH) who showed an inadequate clinical response to PDE5 inhibitor therapy, patients who switched from a PDE5 inhibitor to Adempas achieved a 2.78-fold higher rate of clinical improvement at 24 weeks compared with those who continued PDE5 inhibitor treatment. and a 90% reduction in the risk of clinical worsening.Based on this evidence, the 2022 ESC/ERS (European Society of Cardiology/European Respiratory Society) pulmonary hypertension treatment guidelines also recommend switching from PDE5 inhibitors to sGC stimulators in patients who fail to reach treatment goals despite ERA and PDE5 inhibitor combination therapy.Accordingly, reimbursement criteria for Adempas were newly established in Korea starting this June. Reimbursement is now granted for patients with PAH (WHO Group 1) in WHO functional class II-III who either ▲show insufficient response to ERA and/or PDE5 inhibitor therapy or ▲have contraindications to both ERA and PDE5 inhibitors. This is expected to serve as a new treatment strategy that can improve patient status before escalating to triple combination therapy.Amid these changes, the two professors emphasized, “Suspicion is the starting point in pulmonary hypertension. Alongside the importance of early diagnosis, treatment goals should be set to reach and maintain the low-risk status, with treatment strategies flexibly adjusted based on patient response.”Q. Please explain the main symptoms and causes of pulmonary arterial hypertension, along with its severity.Professor Vallerie McLaughlin: PAH is characterized by prominent shortness of breath during exercise or activity. While the timing varies among patients, they commonly experience dyspnea. In fact, many patients seek medical care primarily due to this shortness of breath. Other symptoms include fatigue, dizziness, chest pain, and leg swelling. These symptoms are non-specific and can commonly occur in other diseases, often leading to delayed diagnosis.PAH is a rare disease. While some cases are idiopathic with no identifiable cause, they can also result from genetic factors, specific medications, dietary habits, or conditions like connective tissue disorders, liver disease, or heart failure.Professor Wook-Jin Chung: The hallmark symptom of PAH is shortness of breath when climbing stairs or hills. Patients may not feel breathless on flat ground, but even mild exertion, such as ascending stairs, can provoke significant dyspnea. Diagnosis is often delayed because symptoms such as fatigue, dizziness, and chest pain are nonspecific.Although advances in drug development have made PAH a more manageable condition, prognosis remains poor. Therefore, prompt evaluation by specialized clinicians, accurate diagnosis, and appropriate treatment are critically important.Q. How do the treatment environments for pulmonary arterial hypertension differ between the United States and Korea?Vallerie McLaughlin, Professor of Cardiovascular Medicine at the University of Michigan Medical CenterProfessor Vallerie McLaughlin: In the United States, 13 PAH drugs have already received FDA approval and are available for use. In practice, many patients are using a diverse class of medications, including endothelin pathway–targeting agents (ERA), prostacyclin pathway–targeting agents (PCA, PRA), and nitric oxide pathway–targeting agents (PDE5 inhibitors and sGC stimulators).Patients classified as high risk require more intensive treatment. For these patients, triple combination therapy may be considered, including intravenous prostacyclin pathway agents in combination with endothelin or nitric oxide pathway therapies.Patients in the intermediate-risk or low-risk groups receive dual therapy based on oral medications, using agents such as ERAs or nitric oxide pathway therapies, including PDE5 inhibitors or sGC stimulators.Initial treatment, however, is only the first step. After 3-4 months of treatment initiation, the patient's risk level must be periodically reassessed using objective evaluation tools. If treatment goals have been achieved, the current regimen can be maintained. However, if the risk level remains insufficiently reduced, treatment intensity must be escalated. Strategies such as triple combination therapy or switching from the previously used PDE-5 inhibitor to an sGC stimulator can be considered. This re-evaluation process is repeated thereafter, with the ultimate treatment goal of achieving and sustaining a low-risk status for the patient.Professor Wook-Jin Chung: Globally, the development of PAH therapies gained momentum with the launch of epoprostenol in 1995. In Korea, effective treatment became possible in 2005, when Bayer’s Ventavis (iloprost) was approved for reimbursement.In the past, treatment escalation was typically initiated only after clinical deterioration. However, because PAH is difficult to cure, waiting for disease progression before intensifying treatment is highly risky and insufficient to reduce mortality. Consequently, recent treatment strategies follow the principle of guideline-directed medical therapy (GDMT), which emphasizes a goal-oriented approach—lowering risk early, achieving a low-risk state, and maintaining it over time.In addition to guiding patients toward a low-risk status, another important treatment goal is to normalize hemodynamic parameters as much as possible.The minimum goal is to sustain a low-risk state by maintaining a mean pulmonary artery pressure (mPAP) ≤40 mmHg and pulmonary vascular resistance (PVR) ≤4 Wood units. Some patients achieve near-normal levels, such as a mPAP ≤20 mmHg and PVR ≤2 Wood units.Injection therapy is required in only about 10% of all patients, and the majority of patients can achieve hemodynamic goals with oral therapies alone.Q. Please share any treatment challenges you have encountered in clinical practice.Wook-Jin Chung, Professor of Cardiology at Gachon University Gil Medical CenterProfessor Wook-Jin Chung: One of the main challenges in treatment is that several therapies used globally have not yet been introduced in Korea. While 13 types of medications are used globally, 4 of them have not been introduced in Korea. Even among the approved drugs, some are not covered by reimbursement, limiting patient access.According to the National Cancer Information Center, Korea's overall five-year cancer survival rate (2018-2022) is approximately 72.9%, whereas the five-year survival rate for PAH is 71.9%. Given that PAH has a poorer prognosis than cancer, government support is essential.Only a portion of PAH patients qualify for a special calculation exception, and even then, this is limited to idiopathic pulmonary arterial hypertension (IPAH). PAH is a high-cost disease where treatment is virtually impossible without insurance support. Therefore, beyond IPAH, it is necessary to expand special reimbursement programs to include PAH caused by other etiologies, based on accurate disease classification.In Korea, PAH treatment typically begins with ERA, with PDE-5 inhibitors added if the response is insufficient. However, there was no subsequent alternative for patients who did not respond adequately to PDE-5 inhibitors. Since intravenous formulations were not introduced domestically, subcutaneous injection and inhalation formulations of prostacyclin pathway-targeted therapies were used.With the recent inclusion of Adempas under reimbursement, a new option has emerged. As an sGC stimulator, Adempas produces a strong vasodilatory effect even at low doses, making it a powerful option for patients who do not respond adequately to PDE-5 inhibitors. It can be used immediately as a switch option in patients with poor PDE-5 inhibitor response and is also available in cases where PDE-5 inhibitors are contraindicated. Clinically, this represents a highly meaningful and practical addition to the treatment landscape.Q. Adempas has been used clinically in the US for a long time. How would you assess its clinical value?Professor Vallerie McLaughlin: Adempas has been used as a treatment for PAH in the US for over a decade, accumulating substantial clinical data over this extended period.Adempas is a drug that directly promotes cGMP production, independent of nitric oxide (NO) levels in the body. Unlike PDE-5 inhibitors, it induces vasodilation and achieves therapeutic effects for PAH in an NO-independent manner, even without the precursor substance NO.In the PATENT clinical trial, which confirmed the therapeutic efficacy and safety of Adempas compared to placebo, significant improvements were observed in exercise capacity (including the 6-minute walk test), pulmonary vascular resistance, and cardiovascular markers, including NT-proBNP, compared to placebo.In addition, the REPLACE study compared treatment outcomes by dividing patients previously receiving combined ERA and PDE-5 inhibitor therapy into two groups: one maintaining existing therapy and the other switching the PDE-5 inhibitor to Adempas. Results showed the clinical improvement rate upon switching to Adempas was 2.78 times significantly higher than in the group that continued PDE-5 inhibitor therapy.Given Korea’s limited access to PAH therapies, switching to Adempas represents an effective alternative for patients who show an inadequate response to PDE-5 inhibitors.Q. What is the value of using Adempas for the treatment of CTEPH?Professor Wook-Jin Chung: Adempas is the only medication available for patients with chronic thromboembolic pulmonary hypertension (CTEPH) and has been used worldwide for over a decade. However, in Korea, it is not covered by insurance, resulting in low patient access.The estimated number of CTEPH patients is nearly comparable to that of pulmonary arterial hypertension patients, but only about 300 to 400 patients actually receive treatment, such as surgery or interventions. Adempas is beneficial for CTEPH patients who still have residual pulmonary artery pressure after intervention or for those who are ineligible for surgery or procedures.Given its strong clinical evidence, it is essential to establish a KCD code for CTEPH and ensure access to the drug through reimbursement coverage. Academic societies plan to continue discussions with the National Assembly and HIRA on this issue.Professor Vallerie McLaughlin: Adempas is the only FDA-approved treatment for CTEPH and has been used in real-world clinical practice in the United States for over a decade.It is indicated for patients with CTEPH whose occluded vessels are too small for surgical or interventional procedures like pulmonary endarterectomy or balloon angioplasty, or for whom pulmonary artery pressure remains elevated after such procedures.Even among patients who undergo pulmonary endarterectomy, pulmonary artery pressure sometimes remains persistently high after surgery. Because persistently high pressure negatively impacts prognosis, pulmonary artery pressure is reassessed at 6 months post-surgery via right heart catheterization. According to UK research, patients whose mean pulmonary artery pressure does not decrease below 35 mmHg post-surgery are known to have a very poor prognosis. For these patients, measures to lower pulmonary artery pressure using Adempas are implemented.There is also evidence that pre-treatment with Adempas before balloon pulmonary angioplasty can reduce surgical risk. Based on these data, Adempas is actively used across various CTEPH patient populations, with successful clinical outcomes.Q. What treatment options are used for CTEPH patients who are not eligible for surgery or intervention?Professor Wook-Jin Chung: Some patients are currently using PDE-5 inhibitors without reimbursement, which reflects the treatment gap caused by the unavailability of Adempas. This highlights the urgent need to establish a KCD code and reimbursement coverage for CTEPH.Professor Vallerie McLaughlin: ERA-class drugs were studied in CTEPH patients in the past, but the results were not successful. Adempas is the only therapy that has demonstrated significant efficacy in CTEPH, and because there are no viable alternatives, the need for its access is even more pressing.Q. What recommendations would you make to improve the pulmonary hypertension treatment environment?Professor Vallerie McLaughlin: Raising awareness of the disease is paramount. Because pulmonary hypertension presents with highly nonspecific symptoms, diagnosis is often delayed. The media also plays a crucial role in this process. Providing the public with clear information about symptoms that should raise suspicion of pulmonary hypertension can greatly facilitate early diagnosis and treatment.Given that experts have already established a robust clinical network, specialized institutions should take on a central role as pulmonary hypertension referral centers. This would enable efficient referral systems, allowing suspected cases identified in primary care settings to be promptly transferred to specialized centers. Clinicians should perform appropriate risk assessments each time a patient visits the hospital and, when necessary, stepwise intensify treatment to reduce risk and lower long-term mortality.Professor Wook-Jin Chung: In Korea, expanding access to medications is the first priority. Introducing new drugs and using them appropriately according to guidelines is crucial for improving treatment outcomes.Furthermore, designating and operating specialized pulmonary hypertension centers is essential. The designation of these so-called ‘Centers of Excellence’ significantly impacts treatment outcomes. Therefore, government-level support is absolutely necessary. I would like to emphasize that a specialized pulmonary hypertension center covering all five types of pulmonary hypertension, not just PAH, needs to be established.We plan to continue the “Lung, Early” awareness campaign to improve understanding of pulmonary hypertension among the public, healthcare professionals, and policymakers, and to maintain discussions with pharmaceutical companies and the government to facilitate access to new therapies. We are also collecting data and conducting research to build a patient-centered treatment environment in Korea, with ongoing efforts to improve care across the entire spectrum of pulmonary hypertension, including PAH.Some patients use PDE-5 inhibitors without reimbursement, but this represents the treatment gap arising from the inability to use Adempas. This underscores the urgency of establishing a new KCD code for CTEPH and securing insurance coverage.
