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- 2026-03-10 00:57:08
- 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.
- InterView
- [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.'
- InterView
- [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.
- InterView
- [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.
- InterView
- [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.
- InterView
- ‘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.
- InterView
- Korea Is a key market… will turn neuroscience innovation into reality”
- by Son, Hyung Min Dec 19, 2025 09:09am
- Brad Edwards, Managing Director of Lundbeck KoreaLundbeck, a global pharmaceutical company specializing in central nervous system (CNS) drug development, has established a solid foothold in Korea's mental health treatment sector through antidepressants like ‘Brintellix (vortioxetine bromide)’ and ‘Lexapro (escitalopram oxalate)’.Recently, it has expanded its portfolio beyond mental illness to include rare neurological disorders, further clarifying its identity as a neuroscience-focused company.Amidst this transformation, Lundbeck Korea appointed its first foreign Managing Director in April, 23 years after the company's establishment in Korea. The new leader is Brad Edwards. He is an expert with nearly 30 years of experience at global pharmaceutical companies, including Pfizer, Shire, and Takeda, having led operations across Australia, New Zealand, and other emerging markets. Most recently, he was based in Singapore as Takeda's Head of Plasma-Derived Therapies, Growth and Emerging Markets.Edwards stated, “While working in Singapore, I never anticipated I would be working in Korea, but Korea is an attractive market with scientific prestige and clinical capabilities. I am greatly inspired by the Korean Lundbeck team's efforts to make a tangible difference in patients' lives through brain health.”“Introducing innovative products... strengthens patient-centric approach strategy”Edwards assessed Korea as a key hub for realizing neuroscience innovation within Lundbeck's global strategy. He noted that Korea is one of Lundbeck's 12 designated core markets, possessing clinical trial capabilities, scientific infrastructure, a digital health environment, and progressively improving regulatory and reimbursement policies.Edwards explained, “The Korean government has recently introduced various forms of regulatory flexibility to improve access to innovative medicines, including faster approval pathways, conditional approvals, and risk-sharing schemes. Policies aimed at reducing out-of-pocket costs for patients with rare and severe diseases, as well as improving diagnosis rates, are also positive developments.”He noted that these environmental changes are laying the groundwork for Korean patients’ faster access to new innovative therapies.Lundbeck Korea’s strategy goes beyond simply launching new drugs. The company envisions redesigning the entire patient access pathway, strengthening collaboration with patient advocacy groups and medical communities while addressing institutional factors such as regulatory approval, reimbursement, and pricing. Preparations for new product launches, pending domestic approval, are already underway.Edwards stated, “Our top priority is introducing innovative new products to the Korean market and improving the lives of Korean patients with brain disorders. To achieve this, we plan to continuously strive to make not only the products themselves but also our approach to patients more efficient and truly patient-centric.”From an organizational perspective, Lundbeck Korea is also built on a stable foundation. Within a culture that emphasizes employee engagement and diversity, the company has earned Great Place to Work certification for two consecutive years, while maintaining a balanced organizational structure across gender and generations.Edwards stated, “This kind of organizational culture is a critical foundation for delivering sustainable, long-term performance. I hope to continue the successful operating practices that Lundbeck Korea has built over the years.”Focus on Neuroscience and Rare Diseases… Accelerates Portfolio TransformationNeuroscience and rare diseases are the most critical pillars of Lundbeck’s mid- to long-term strategy. Globally, the company is reshaping its portfolio around rare neurological diseases, with Korea positioned as a key part of this effort.Edwards said, “Many patients with rare neurological diseases remain undiagnosed, resulting in significant unmet medical needs. By pursuing new scientific approaches and driving neuroscience innovation, we aim to improve patients’ quality of life. This aligns directly with Lundbeck’s goal of expanding an innovative and specialized portfolio in rare neurological diseases.”He added, “Lundbeck is shifting its focus within neuroscience toward rare neurological disorders. Based on our extensive experience working in the rare disease field across multiple countries, we expect Lundbeck Korea to contribute to this transformation process.”The antidepressant business continues to be an important pillar for Lundbeck Korea. Brintellix and Lexapro remain flagship products and have been widely used in Korea’s mental health treatment landscape for many years.Edwards said, “Mental health is both a core business area and a long-term responsibility. We will continue to ensure stable supply, education, and digital support for antidepressant therapies through close collaboration with healthcare professionals and partners.”From a global strategy perspective, Edwards emphasized that Korea plays a crucial role in connecting global R&D capabilities with local innovation.He stated, “Korea has excellent clinical execution capabilities and strong scientific expertise, making it an ideal partner for advancing neuroscience innovation. The government’s willingness to improve regulatory and reimbursement systems is particularly meaningful.”Edwards identified healthcare, digital, and policy (PA) as key areas for Lundbeck Korea to strengthen. He emphasized the importance of expanding real-world evidence (RWE) and data-driven insights, and leveraging new technologies like artificial intelligence (AI) to communicate more effectively with healthcare professionals. This aligns with Lundbeck's global ‘Focused Innovator’ strategy.The new CEO's mid-to-long-term vision is clear: steadfastly realizing Lundbeck's mission in Korea—‘improving the lives of patients with brain disorders and growing into an agile, high-performing organization through focused innovation in neuroscience.’Edwards stated, “Rather than pursuing abrupt internal changes, we will achieve natural evolution as our pipeline progresses toward commercialization. I hope Lundbeck Korea will grow into an agile, results-driven organization that contributes not only to Korea but also globally.”He concluded, “Lundbeck’s achievements to date have been made possible through collaboration and feedback from many partners and stakeholders. All progress in neuroscience begins with listening to their experiences and insights, and we will continue to engage in close dialogue going forward.”
- InterView
- "ADC use sufficient in neoadjuvant therapy of breast cancer"
- by Son, Hyung Min Dec 16, 2025 08:35am
- The potential of utilizing the Antibody-Drug Conjugate (ADC) Enhertu is being confirmed in the neoadjuvant setting.At the European Society for Medical Oncology Asia (ESMO Asia 2025) annual meeting in Singapore, clinical outcomes for Enhertu (trastuzumab deruxtecan) in patients with advanced HER2-positive breast cancer were recently disclosed.In an interview at the conference, Professor Chang Ik Yoon of the Division of Breast Surgery at Seoul St. Mary's Hospital evaluated Enhertu as an option with sufficient clinical value not only for metastatic breast cancer but also for advanced HER2-positive breast cancer requiring neoadjuvant treatment, demonstrating the potential to overcome the limitations of the existing standard of care.In the treatment of advanced breast cancer, pathological complete response (pCR) is a key indicator for lowering the risk of recurrence and improving long-term survival. However, a limitation of the existing standard of care TCHP, combining Herceptin (trastuzumab)·Perjeta (pertuzumab)·cytotoxic chemotherapy, is that approximately half of the patients fail to achieve pCR in real-world clinical practice.Professor Chang Ik Yoon of the Division of Breast Surgery at Seoul St. Mary's Hospital The Phase 3 DESTINY-Breast11 study, designed to address this unmet need, evaluated Enhertu's potential as a neoadjuvant therapy in 927 patients with advanced HER2-positive breast cancer. The result showed that the pCR rate in the Enhertu combination group was 67.3%, which was 11.2 percentage points higher than that of the existing standard treatment, the ddAC-THP group (56.3%). This double-digit gap in the early·advanced breast cancer setting is considered a clinically meaningful difference.Consistent improvement was also confirmed in subgroup analyses. In the Hormone Receptor (HR)- positive patient group, the pCR rate with the Enhertu combination therapy was 61.4%, 9.1 percentage points higher than in the control group (52.3%). Notably, the gap widened further in the HR-negative patient group, highlighting the therapeutic value of this approach in this high-risk population.Professor Yoon stated, "Even with earlier use of the treatment, there were no distinct disadvantages in terms of side effects. The safety profile showed a more stable pattern." Professor Yoon projected that "the scope of Enhertu's utilization in pre-operative treatment strategies will continue to expand."Q. We would like to ask about the background of today's session. Regarding the neoadjuvant data presented at ESMO, some medical oncologists expressed the view that the clinical significance was not substantial. Would you share your views?The answer to this question depends on one's perspective. There have already been several prior studies. The common and crucial endpoint examined in these studies was pCR.HER2-positive breast cancer accounts for approximately 20–25% of all breast cancers. The pCR rates following conventional chemotherapy range from about 50% for HR-positive, HER2-positive cases to up to 80% for HR-negative/HER2-positive cases. Combining these, approximately 65% achieve pCR, meaning roughly one-third, or 35%, do not.The pCR rate reported in the latest DESTINY-Breast11 study was around 67%. Placing this figure alone next to the existing data, the difference is not dramatically significant. However, the results were better than those of the control group, which received anthracycline (AC) chemotherapy followed by THP.However, some points don't align well with the South Korean setting. Doxorubicin (adriamycin) in the AC therapy is an agent with cardiac toxicity, and HER2-targeted therapies also carry a cardiac toxicity risk. Because using both together increases the risk of cardiac side effects, there is a clinical tendency in South Korea to avoid AC whenever possible. In contrast, it is still frequently used overseas. In that sense, the fact that Enhertu showed similar or even better results without using AC is a clear advantage.Q. Why is AC still used overseas despite its cardiac toxicity?AC is one of the major chemotherapy agents that has traditionally improved survival and recurrence rates in breast cancer treatment. It is a drug that, along with the taxane-type agents, has proven survival benefits. However, its use has somewhat declined since the introduction of HER2-targeted therapies due to concerns about cardiac toxicity.Despite this issue, there is still the understanding that significant problems do not arise when the cumulative dose is not high. Because the cardiac toxicity of HER2-targeted therapies is relatively manageable, they are still combined with AC in many overseas settings.Q. Why has the TCHP regimen been used more often in South Korea?The underlying perception in Korea was that there was no need to deliberately expose patients to the risk of cardiac toxicity if the difference in pCR rates was not significant. Therefore, the regimen most widely used has been the TCHP regimen administered over six cycles.Following the introduction of Enhertu, number of chemotherapy cycles can be reduced from six to four, and that earlier treatment does not incur a significant safety penalty; in fact, the safety profile appeared more favorable.Q. How clinically significant do you consider regarding pCR being approximately 10% difference in?In most studies, pCR is accepted as an indicator correlated with reduced recurrence rates and increased survival. Since actual survival data takes time to mature, pCR is utilized as an essential surrogate marker to predict long-term outcomes. Especially in HER2-positive and triple-negative breast cancer, pCR is almost synonymous with reduced recurrence risk and improved survival.However, the question of how meaningful a 10% increase is when pCR rates are already approaching 70-80% is fair. It is also possible to interpret that even if the pCR rate is slightly lower, the difference in long-term survival may not be significant because additional treatment is given during the subsequent adjuvant therapy.Q. Oncologists may have concerns about using such a powerful option too early in the neoadjuvant stage.Yes. Patients who do not achieve pCR are considered high-risk for recurrence, and choosing subsequent adjuvant therapy is critical. If a superior option has already been used in the neoadjuvant stage, the question of what to use next is unavoidable.While prospective data on subsequent treatment are currently insufficient, this does not mean that options are entirely nonexistent. As subsequent studies emerge, this aspect will be gradually resolved.Q. Do you believe the current results alone are sufficient to support the use of Enhertu in the neoadjuvant setting?In my opinion, it is sufficient for patients who achieve pCR. In these cases, the 5-year recurrence risk is less than 10%, indicating that the current standard treatment is adequate. The issue lies with patients who have residual disease.The KATHERINE study shows that HER2-positive breast cancer patients with residual disease after neoadjuvant therapy had an 8-year recurrence risk of about 20% even with the existing standard treatment. However, the DESTINY-Breast05 results suggest the possibility of reducing this recurrence risk to approximately 8% at 3 years. The hazard ratio indicates that the risk is nearly half.In other words, the reason not to be overly relying on pCR is that subsequent treatment options have improved, allowing for further intervention to lower the risk of recurrence.Q. Can Enhertu be used in the neoadjuvant setting and then used again later?It is possible. We already know that Enhertu is a better drug than Kadcyla, and a strategy of using it again, if necessary, can be considered. However, cost and reimbursement issues will be separate concerns.The core strategy is personalized treatment: de-escalating therapy for patients who achieve pCR and intensifying treatment for those with residual disease.For instance, with the old standard of care, a patient who achieved pCR would still need 14 cycles of Enhertu afterward. If pCR is achieved with only 4 cycles of Enhertu during neoadjuvant therapy, that patient would not require the additional 10 cycles. A patient group clearly benefits from this.Q. There was also criticism of the study design regarding why it only looked at the neoadjuvant stage and not the perioperative (pre- and post-operative) setting.The recent trend in research questions whether all patients need to complete the full course of chemotherapy. The approach is to see if treatment can be reduced for patients who show a good response.For example, studies like PHERGain adjust treatment based on an interim response evaluation. In this context, the design to identify early responders by using Enhertu upfront is a meaningful attempt. However, the failure to clearly define the subsequent strategy for patients with residual disease can be confusing, and may need further research.
- InterView
- ‘Imfinzi demonstrates rationale for perioperative immunotherapy in gastric cancer’
- by Son, Hyung Min Dec 15, 2025 11:02am
- The role of adjuvant systemic therapy is emerging as an important consideration even in patients with resectable gastric and gastroesophageal junction (GEJ) adenocarcinoma.Despite outstanding surgical outcomes in East Asia, a significant proportion of patients with stage II-III locally advanced disease still experience recurrence due to micrometastases. In this context, a perioperative treatment strategy, which involves systemic therapy before and after surgery, has emerged as a promising approach to enhance long-term outcomes.At the recent ESMO Asia Congress 2025 held in Singapore, results from an Asian subgroup analysis of the Phase III MATTERHORN study were presented, reinforcing the clinical value of Imfinzi (durvalumab) in combination with standard FLOT chemotherapy (5-FU, leucovorin, oxaliplatin, and docetaxel).In the study, Imfinzi plus FLOT demonstrated clinically meaningful improvements in event-free survival (EFS), 3-year overall survival (OS), and pathological complete response (pCR). These findings signal a clear shift toward earlier use of immunotherapy in resectable gastric cancer.Surgery remains the cornerstone of the cure in gastric cancer. However, there is growing global consensus, including in Asia, that surgery alone is insufficient for many patients. The MATTERHORN study demonstrated that administering a combination of chemotherapy and immunotherapy prior to surgery, followed by curative resection and additional therapy afterward, can significantly improve long-term outcomes.Based on these results, the U.S. Food and Drug Administration (FDA) approved Imfinzi for the treatment of adult patients with resectable, early-stage, and locally advanced (stages II, III, and IVA) gastric and gastroesophageal junction (GEJ) cancers last month. The approved regimen includes neoadjuvant Imfinzi in combination with chemotherapy before surgery, followed by adjuvant Imfinzi in combination with chemotherapy, then Imfinzi monotherapy.At ESMO Asia 2025, Daily Pharm spoke with Dr. Yelena Y. Janjigian, first author of the MATTERHORN study (Chief of the Gastrointestinal Oncology Service at Memorial Sloan Kettering Cancer Center), as well as Professor Sun Young Rha of Yonsei Cancer Center (President of the Korean Cancer Association) to discuss the clinical implications of this shift and the challenges associated with its implementation in Korea.Q. As the first author of MATTERHORN, what prompted the development of this perioperative immunotherapy strategy for patients with resectable gastric and GEJ adenocarcinoma, and what is the clinical significance of the results?Professor Yelena JanjigianDr. Yelena Janjigian: MATTERHORN holds significance as the first Phase III study in the resectable, early-stage setting to demonstrate improvements across pCR, EFS, and OS with chemo-immunotherapy.At ESMO Asia, we presented subgroup results from Japan, Korea, and Taiwan, which were consistent with the global findings. These data support the global relevance of the perioperative approach.With recent FDA approval for resectable gastric and GEJ adenocarcinoma, aligning treatment approaches across regions will help us move the field forward, especially given the global incidence of more than 1.2 million new cases each year.Q. The Asian subgroup showed efficacy and safety trends consistent with global results.Professor Yelena Janjigian: Three key points are important. First is the feasibility across regions. Perioperative Imfinzi was deliverable in Asian centers, and physicians were able to administer both FLOT and durvalumab safely.It also did not compromise surgical care. Rates of complete (R0) resection and the ability to proceed to surgery were maintained.Furthermore, the results showed improvement across all major efficacy endpoints—pCR, EFS, and OS. The ability to improve all three endpoints in one study is unprecedented in this setting and underscores the strength and value of the data.Q. What considerations should clinicians keep in mind when applying the regimen in earlier-stage patients?Professor Yelena Janjigian: The study enrolled a broad age range—from 18 to 84 years—and older patients benefited similarly to younger patients.That said, some patients, particularly in Asian populations, may present with nutritional compromise or lower baseline white blood cell counts. Asian investigators are familiar with these characteristics. Strategies such as early G-CSF administration and careful initial dose tailoring of FLOT can help maintain safety while preserving efficacy.Most importantly, close clinical monitoring, hydration, and supportive care are essential components of successful perioperative treatment.Q. Some Korean clinicians believe the regimen may be more suitable for stage III or IV than stage II. What is your view on this?Professor Yelena Janjigian: Clinical staging in gastric cancer is often imprecise. A patient staged clinically as T2 may be found at surgery to have T3N1 disease. Because many patients have difficulty tolerating intensive postoperative therapy after major gastrectomy, preoperative treatment becomes particularly valuable.We believe the greatest benefit of chemo-immunotherapy occurs when the tumor remains in place, allowing optimal priming and expansion of anti-tumor T-cells. This is supported by the stronger efficacy observed in the neoadjuvant component compared with adjuvant-only settings.In my clinical practice, even patients with clinically estimated T2 tumors—especially those with diffuse or signet-ring histology—are discussed in a multidisciplinary context for consideration of perioperative systemic therapy.Additionally, the shift in tumor epidemiology in Asia—with increasing proximal and GEJ tumors—further supports the need for downstaging approaches, as these tumors historically have poorer surgical outcomes.Q. How should we interpret the potential for non-operative management in early gastric cancer?Professor Yelena Janjigian: This area is highly exploratory and remains limited to very select patients within clinical trials or specialized centers.In certain patients who achieve a complete clinical and pathologic response and who can reliably adhere to intensive surveillance, non-operative strategies have been examined. However, this is not standard practice and remains controversial, particularly among surgeons.Nevertheless, these discussions reflect a broader evolution toward balancing cure with long-term quality of life. Gastrectomy, even when minimally invasive, alters eating patterns, sleep patterns, and body image. For selected patients, preserving the stomach—when supported by rigorous evidence and strict monitoring—could represent a meaningful advance, but this requires further research before it can be broadly implemented.Q. Gastric cancer has historically been viewed as a disease with favorable surgical outcomes, leading to a lesser emphasis on chemotherapy. Yet MATTERHORN highlights the importance of perioperative treatment. Why is this approach necessary?라선영 연세암병원 교수Professor Sun Young Rha: MATTERHORN enrolled patients with stage II and III gastric cancer eligible for curative surgery. While stage I “early gastric cancer” is often cured through endoscopic treatment or gastrectomy, stage II–III cancers are locally advanced and carry significantly higher recurrence risk.In East Asia, surgery followed by adjuvant chemotherapy has historically yielded strong outcomes, with five-year survival rates around 75–80%. Still, approximately 30–40% of stage III patients experience disease relapse, demonstrating a significant unmet need.Perioperative therapy aims to eradicate micrometastases after surgery and sustain systemic control afterward. In Western countries, perioperative FLOT has become the standard to achieve a better tumor resection, considering factors such as the increased obese population and different tumor extent at GEJ.Q. What are the key findings of MATTERHORN, and which patients are most likely to benefit from D-FLOT?Professor Sun Young Rha: The MATTERHORN study is significant in that it introduces a new treatment option for patients with locally advanced, resectable gastric cancer. Notably, a distinct survival benefit was observed in high-risk stage III patients with a high tumor burden. It is also noteworthy that the overall survival curve suggests the possibility that more than half of patients may remain alive at five years.However, it should be noted that MATTERHORN did not compare the experimental arm with the post-operative adjuvant chemotherapy that has been long used in Korea or Japan, but rather with FLOT, which is considered the global standard of care. While FLOT is highly effective, Asian patients may encounter challenges in completing treatment due to its relatively high intensity.Therefore, especially in Asia where surgical outcomes are generally more favorable, it may be more appropriate to selectively apply this regimen to patients with high-stage disease.Q. If the D-FLOT regimen becomes available in Korea, which patients would be the most appropriate candidates?Professor Sun Young Rha: If ongoing follow-up continues to support the favorable outcomes observed in stage III patients—particularly in terms of EFS and 3-year OS—this group will likely be prioritized in Korea.For stage II patients, surgery followed by adjuvant chemotherapy already yields a 5-year OS rate up to 80%, and the potential incremental benefit of perioperative D-FLOT must be balanced against its higher treatment burden and toxicity profile.In MATTERHORN, approximately 40–50% of patients completed the full perioperative regimen, reflecting the challenges associated with the adjuvant FLOT component. This underscores the importance of careful patient selection. Stage III patients, who have higher rates of micrometastatic disease and a 30–40% recurrence risk within two years, are expected to derive the greatest benefit.Clinical features such as extensive nodal involvement, T4 tumors, or biologically aggressive histology should guide decision-making. Biomarker-informed approaches will increasingly help tailor treatment intensity and minimize unnecessary toxicity.Q4. What distinguishes Asian patients from Western patients in MATTERHORN?Professor Sun Young Rha: A notable feature of Asian patients enrolled in the MATTERHORN study is that they generally had good performance status and a relatively lower tumor burden. In countries such as Korea and Japan, where early detection rates are high and surgical outcomes are excellent, patients enrolled in clinical trials can potentially have better overall performance status compared to their Western counterparts.While the proportion of stage IV patients in the study appears somewhat high, it should be interpreted as including patients with high-risk, advanced gastric cancer characterized by deep tumor invasion or extensive lymph node involvement, rather than those with unresectable or incurable M1 metastatic disease.In the MATTERHORN study, Asian patients represented approximately 20% of the total population, indicating that additional real-world experience will be needed to further strengthen the evidence base. It will be important to continue accumulating data from Asian patients to more clearly define the clinical significance in future practice.Q. What challenges must Korea overcome to adopt perioperative durvalumab?Professor Sun Young Rha: Above all, aligning treatment approaches between surgeons and medical oncologists is essential. Especially in Korea and Japan, where surgical outcomes are outstanding, and the paradigm remains surgery-centric. Consensus-driven discussions with surgeons are needed to establish the role of perioperative immunotherapy in high-risk advanced gastric cancer. To ensure patients do not lose access to optimal treatment options, multidisciplinary collaboration is vital for designing comprehensive treatment plans.From a medical oncology standpoint, individualized dose adjustment and proactive management of adverse events are key, particularly given the complexity and toxicity of the FLOT regimen. Active supportive care (such as antiemetics, G-CSF support, and dose modification) is necessary to help Asian patients complete therapy.Finally, reimbursement is a major hurdle. Without sustainable reimbursement pathways, early real-world adoption will be limited. Generating clinical experience and real-world data, especially in high-risk stage III patients who are most likely to benefit, is important to help establish the evidence base needed for broader access.
- InterView
- [Reporter's View] Generic price cuts…SME status
- by Choi Da Eun Dec 11, 2025 08:39am
- The price reduction of generic drugs in South Korea is once again putting severe pressure on small and medium-sized pharmaceutical companies. If the proposed price cuts are implemented, small and medium-sized pharmaceutical companies that heavily rely on generic sales will face greatest impact. For these companies, where over 70% of revenue comes from generics, a reduction of over 10% in drug prices could threaten their foundation.Drug pricing policy has been continually strengthened to ensue the sustainability of the National Health Insurance fund. Significant price reductions began in 1999 when the government reformed the drug reimbursement system, lowering prices for listed pharmaceuticals by an average of 30.7%. This was followed by the 'Drug Expenditure Rationalization Plan (DERP)' in 2006, which reduced the price of generics from 80% to 68% of the original product price.As high-cost drug usage and the launch of generics expanded in the late 2000s, concerns over the health insurance fund's financial deterioration intensified. The government undertook a major reform of the drug pricing system in 2012. This action eliminated the 'step-wise drug pricing system,' which gradually lowered prices based on the generic's market entry rank, and implemented a 'Comprehensive Price Reduction,' unifying the ceiling price for both off-patent original drugs and generics to 53.55% of the original price.Recently, the reform announced by the Ministry of Health and Welfare includes a plan to secure funding to improve access to innovative new drugs by further reducing generic prices. It is intended to lower the existing generic price standard from 53.55% to the 40% range. The government aims to implement this change in the latter half of next year.The limitation of the generic industry has long been predicted. The companies that are hit first and hardest by these price cuts are the domestic small and medium companies that lack robust R&D or global business foundations. Many firms introduced identical-component, identical-formulation products, fragmenting the market. Now, increased manufacturing costs in addition to further price cuts have severely dropped profitability.Excluding companies with high reliance on Contract Development and Manufacturing Organization (CDMO) or non-reimbursed products, the average operating profit margin for 100 domestic pharmaceutical companies over the last three years was a merely 4.8%, with a net profit margin of only 3%.The problem in these drug pricing reforms is simply focusing on reducing government health insurance expenditure. Conversely, the strategic support framework for the industry following these price cuts remains vague and ambiguous. While the government supports the pharmaceutical and bio industry as a future driver, it appears indifferent to the harsh realities facing the companies that are behind it. Current pricing regulations effectively become a price pressure mechanism that forces smaller companies out of the market first.The most significant expected adverse effects of the price reductions are cuts in R&D spending and corporate restructuring. Companies facing immediate operational cost depletion and severe financial distress will likely view R&D as a luxury, inevitably leading to a reduction in demand for specialized personnel. The market reality and policy objectives are clearly flawed and clash.Of course, pharmaceutical companies must also adjust their portfolios and profit strategies to cope with government regulatory pressure. For instance, they must maintain generic-based revenue while aggressively cutting down lower-profit items. If R&D burden is high, a cost-diversified research approach is necessary, such as through collaboration utilizing AI, platform technologies, and external R&D partnerships.It is difficult to deny that price reduction is a tool for stabilizing the health insurance fund and that more resources should be allocated to innovative new drugs. However, the approach of solving the problem solely through price cuts is overly simplistic. If financial efficiency directly undermines industry competitiveness, it will only worsen profit polarization among pharmaceutical companies, leaving the government's vision for the pharmaceutical industry a mere illusion.
