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- 2026-06-10 20:06:13
- Opinion
- [Reporter's View] Korean R&BD system and strategies
- by Whang, byung-woo Apr 10, 2025 05:57am
- “It is not easy for a new drug to succeed just with good technology. It is only a real success when the drug reaches the market.” This is what an executive of a global pharmaceutical trading company said to the reporter recently. The tides are changing in the pharmaceutical and biopharmaceutical industry. The focus is shifting from R&D (Research & Development) to R&BD (Research & Business Development). In other words, business-related strategies such as technology transfer, joint development, and investment attraction have become as important as R&D. The reality of the industrial ecosystem is behind this. South Korea is still choosing a growth strategy centered on technology exports rather than innovative new drugs. Although the number of pipelines is increasing, due to the nature of drug development that is accompanied by high cost and long-term battles, companies are focusing on technology transfer based on early-stage - such as Phase I and II - clinical results rather than the final approval, in order to achieve results. In fact, it takes a long time for a single candidate substance to reach global clinical trials, approval, and market entry. There is also a view that this is an unavoidable strategy in the domestic environment, where the pharmaceutical and bio-pharmaceutical ecosystem is relatively small, in terms of market size and investment conditions. At the global level, there is a growing recognition that it is difficult to survive without commercialization in mind from the early stages of R&D. R&BD, which adds business to R&D, has been spreading in line with this trend. Although Business Development (BD) was an existing job category, there is an overall shift beyond a simple change in terminology, and the occupation now entails roles in setting research direction, investment structure, and organizational operation methods. A typical example of such change is reflected in the recent activities of the Korea Drug Development Fund (KDDF). The KDDF has recently put its R&BD strategy at the forefront when selecting support projects. It comprehensively evaluates not only scientific feasibility but also the possibility of technology transfer, marketability, and global partnership plans. Whereas in the past the focus was on “good research,” now the emphasis is on “technologies with commercial potential.” To this end, KDDF is currently preparing a multi-layered support system for strengthening R&BD, including consulting, collaboration programs with global pharmaceutical companies, and VC networking. There are also cases where KDDF has arranged global partnering meetings for participating companies, which have led to positive trends in early-stage technology transfer. They are sharing 'how to speak in business language, not technology'. At the event, Yeong-Min Park, head of the KDDF, emphasized that “a sophisticated commercialization strategy is needed from the early stages for successful new drug development.” Ultimately, the key now lies in how to define the destination of research and development. Paper? Patent? Clinical trial? Now the destination is 'market'. The industry believes that no matter how excellent the technology is, it is useless if it is not connected to the market. Many pharmaceutical companies are reorganizing their structures by establishing new R&BD organizations or including BD personnel in their R&D departments. New drug development is both a science and a business. And it is the role of R&BD to create the intersection of the two. The capabilities of business developers are becoming as important as those of researchers. This is because, although there are many technologies, there are few success stories. Now is the time to ask what value the technology has in the market rather than the R&D results. Ultimately, the task of the pharmaceutical and biopharmaceutical industry is to nurture people who can bring the technology to the market.
- Opinion
- [Reporter's View] Why clinical outcomes require deliberation
- by Son, Hyung Min Apr 08, 2025 05:57am
- Even small differences in clinical outcomes can be highly meaningful. Especially for refractory diseases. Recent top‐line results from Compass Therapeutics’ Phase 2/3 COMPANION-002 trial for cholangiocarcinoma have sparked considerable debate within the industry. According to Compass Therapeutics, the treatment of bispecific antibody tovecimig in combination with the cytotoxic agent paclitaxel resulted in the objective response rate (ORR) of 17.1%, which is nearly three times higher than the 5.3% observed with paclitaxel monotherapy. However, some suggested that the 17.1% ORR is considerably lower than the 37.5% ORR recorded in a previous domestic Phase 2 study conducted by Handok. In a domestic Phase 2 trial, tovecimig+paclitaxel combination therapy had a 37.5% ORR. The latest clinical trial's ORR is less than half of the Phase 2 study. It is important to note that it is too early to make a definitive conclusion on the clinical trial outcomes. The patient populations differed significantly, 24 patients in the domestic Phase 2 trial versus 111 patients of varying races and ages in the global Phase 2/3 study. COMPANION-002 trial is still ongoing, and data on secondary endpoints such as progression-free survival (PFS), overall survival (OS), and duration of response (DOR) have yet to mature. Furthermore, the unique characteristics of cholangiocarcinoma must be taken into account. Although the overall patient numbers are relatively small, cholangiocarcinoma is notoriously difficult to diagnose early and is characterized by rapid metastasis and recurrence, leading to a 5-year relative survival rate of only 28.9% (2017–2021). In fact, seven out of ten cholangiocarcinoma patients eventually die. Another primary reason for the low survival rate is ineffective treatment options. In cases of locally advanced or metastatic cholangiocarcinoma where it is inoperable and first-line therapy has failed, there has been no approved targeted therapy available in Korea. Targeted drug studies have been challenging because of early diagnosis and a small population of patients. Although some targeted therapies for cholangiocarcinoma have shown less than a 1-month improvement in PFS compared to placebo, they have surpassed regulatory hurdles. FOLFOX-based regimens yield a median PFS of 4.0 months and an OS of 6.2 months. Some cancer types have many patients and easily detected in early phases. Clinical studies of those types rarely have data recording over ten digits of months of PFS and OS. However, in challenging fields such as cholangiocarcinoma and triple-negative breast cancer, where the development of targeted therapies is complex, even slight numerical differences can be highly significant. Therefore, it would be premature to draw definitive conclusions about Compass Therapeutics' cholangiocarcinoma treatment without waiting for the final data. As the pharmaceutical industry gradually overcomes long-standing challenges in developing effective therapies for cholangiocarcinoma, a range of new treatments is emerging. Recent developments include the addition of immunotherapies such as AstraZeneca's Imfinzi and MSD's Keytruda as first-line options, as well as the emergence of targeted therapies like Pemazyre and Tibsovo for second-line treatment, along with various agents including, tovecimig and rivoceranib. Experts emphasize that, given the scarcity of treatment alternatives for cholangiocarcinoma, even slight differences in efficacy relative to existing therapies can be significant. When interpreting clinical outcomes, it is essential to consider each cancer type's specific clinical context and biological characteristics. Ultimately, understanding the differences in clinical results between cancers with limited treatment options and those with a wide array of available therapies is critical for accurate data interpretation.
- Opinion
- [Reporter's View] Preparations for US pharmaceutical tariff
- by Kim, Jin-Gu Apr 03, 2025 05:55am
- US President Donald Trump designated April 2 (local time) as 'America's Day of Liberation.' The day the US will announce reciprocal tariff rates for various countries. US reports expect details on tariff-affected countries, rates, and applicable product categories to be disclosed as early as the evening of the 1st. The specific tariff rates will be known in South Korea by the morning of the 2nd or, at the latest, the morning of the 3rd in Korean time. The United States is Korea's largest market for pharmaceutical exports. Last year, Korean pharmaceutical exports to the US reached US$1.359 billion (approximately KRW 2 trillion), a 50% increase compared to 2023. When expanded to include the broader market, exports have surged from US$33 million a decade ago in 2014 to over 40 times that amount. The US accounted for 18% of Korea's total pharmaceutical export revenue last year, making it the largest single market among all countries. This fact underscores why US tariffs on pharmaceuticals could have a significant impact. Industry attention is now focused on whether Korean pharmaceuticals will also be subject to tariffs, and if so, which specific drugs and at what rates. A 25% tariff rate is the most likely scenario. During a press conference on March 18, President Trump even hinted that pharmaceutical tariffs 'could be 25% or more.' However, it remains unclear whether the forthcoming reciprocal tariff announcement scheduled for April 2 will include pharmaceuticals, or if tariffs on pharmaceuticals will be imposed separately. Some analysts predict that pharmaceuticals might be excluded from the tariff measures, arguing that imposing tariffs on them would likely lead to a surge in domestic healthcare costs. Indeed, recent projections indicate that the number of drugs experiencing supply shortages in the US could exceed 215 (up from 127), and US generic drug prices may increase by an average of 18%. One report even warned that US healthcare spending could skyrocket from a baseline of 5% to as high as 60%. Another opinion suggests that tariffs could be applied only selectively, targeting certain countries with large trade deficits, such as some European nations, or excluding critical categories like essential or supply-shortage drugs. Nonetheless, prevailing forecasts still lean toward the imposition of pharmaceutical tariffs. Should tariffs be levied on domestically produced drugs, it could deliver a severe hit to Korea's pharmaceutical and biotech industries. Companies are left with few options and must concentrate on creating exit strategies. The Trump administration has reiterated a 'tariff first, negotiate later' approach. Even if tariffs are eventually imposed on Korean pharmaceuticals, there remains potential to mitigate the impact through subsequent negotiations. Despite South Korea being identified as a 'notorious trade imbalanced country,' industry negotiators argue that it is important to emphasize that Korea has consistently run a trade deficit with the US in the pharmaceutical sector. In the medium to long term plan, South Korea must also accelerate reforms in its drug pricing and innovative pharmaceutical company certification systems. The United States Trade Representative (USTR) has previously released a report stating that Korea's drug pricing policies and innovative pharmaceutical company certification system disadvantage foreign companies. Both systems are currently undergoing revisions, and if given the opportunity to negotiate with the US, South Korea should actively highlight these improvements at the bargaining table.
- Opinion
- [Reporter's View]Unifying the price of pneumococcal vaccines
- by Eo, Yun-Ho Mar 28, 2025 06:38am
- The National Immunization Program (NIP) is the ideal “goal” for vaccine-holding pharmaceutical companies. This is because the government's policy of purchasing vaccines and providing them free of charge to the public, above all, ensures stable sales. Therefore, when the government announces that it has selected a preventive vaccine for a specific disease for NIP, companies with relevant vaccines begin to compete fiercely. Companies deploy various strategies to increase market share within the set market as well as to be selected for NIP. The National Immunization Program (NIP) of pneumococcal vaccines has recently been in the spotlight. The new addition of 'Prevnar 20' from Pfizer Korea, the original leader in the market, has stirred the battle between multinational pharmaceutical companies' premium vaccines. Pfizer’s pneumococcal vaccine pipeline, which ranges from Prevnar and Prevnar 13, has virtually dominated Korea’s market. In this market, a new 15-valent vaccine, MSD's Vaxneuvance, joined as a new player and driven change, so Pfizer is aiming for the throne again with its new 20-valent vaccine. At the core of the claim of the “superiority” of these new vaccines is serotype coverage. If 13 serotypes are covered, it is called a 13-valent vaccine; if it covers 15 it is a 15-valent, and 20 a 20-valent vaccine. The general perception is that a vaccine that covers more serotypes has a relatively higher level of preventive power. However, a higher serotype coverage does not necessarily mean better preventive power. However, the fact that a product “covers more serotypes” to prevent the same disease resonates with the consumers. In the NIP market, the vaccine with the highest price has always been the winner. However, there has been an interesting change in the stance of pharmaceutical companies entering the NIP with their pneumococcal vaccines. The companies have decided to supply their newly developed products at the same price as their existing vaccines. This is called price unification. The reason why the change is interesting is this: When Prevnar 13 was introduced to the NIP, Pfizer insisted on differentiating it from its strong competitor, GSK Korea's 10-valent vaccine ‘Synflorix,' and demanded a dual pricing system, which was implemented. The same goes for MSD. This company also requested a different price from GSK's 'Gardasil' and ‘Cervarix,' which are quadrivalent vaccines, when they entered the NIP for HPV vaccines. This is called the dualization of prices. The dualization of prices means the government's discrimination in the purchase price of NIP vaccines. If the prices of the 2 vaccines granted by the government are different even though they are free vaccines, the majority will choose to take the higher-priced option. The result was clear. However, for some reason, neither MSD, when it entered the market with its 15-valent vaccine, at a time when the 13-valent vaccine was the best, nor Pfizer, when it launched its 20-valent vaccine, demanded a price differentiation for their more serotype coverage. Both pharmaceutical companies chose to standardize their prices. Even though they have pride in their follow-up products. This is a reflection of the correct market logic. The situation in the pneumococcal vaccine market is different from the time when there was a price dualization. Rather than taking the time to raise the price of both the 15-valent and 20-valent vaccines, the companies decided to enter the market as quickly as possible. It is only natural for a company to pursue profit. The strategy may vary depending on the strategic decision to attract sales by competing with competing products. Before, the strategy was ‘dualization of price,' but now it is 'unification.' However, the message of 'for the health of the people' does not seem to be the reasoning behind the companies’ choice of unifying the price.
- Opinion
- [Reporter's View] Healthcare AI and diversity·expandability
- by Whang, byung-woo Mar 28, 2025 06:37am
- During the 'KIMES 2025,' held last week, artificial intelligence (AI) gained the most attention. The exhibition booths were filled with companies that showcased AI at the forefront, including hospital systems and diagnostic imaging to patient monitoring, chatbot consultations, and electronic medical records (EMR). Large booths prominently displayed slogans like 'AI-based' and 'AI solution,' drawing visitors’ attention to the cutting-edge AI technologies. With AI emerging as a spearhead of technological innovation, this emphasis was anticipated at the recent KIMES event. However, upon closer inspection, there were also elements of disappointment. Most of the AI technologies exhibited by these companies focused on aiding image interpretation and enhancing diagnostic accuracy. For instance, AI systems trained on existing medical imaging data from CT, MRI, and X-ray scans are being used to quickly and accurately identify specific diseases. Of course, in clinical settings, rapid and accurate diagnosis is critical, and a detailed review of each company’s AI innovations does reveal clear differentiators in their approaches. Although exhibition booths are limited in fully showcasing the company's strengths, one common question we encountered during our coverage was, "So, what's different?" This reaction suggests growing skepticism about whether the AI focus at this year's KIMES represents a departure from previous years. Recently, the government, industry, and the healthcare sector have all been touting the growth potential of digital healthcare and AI-driven medical innovation. Despite ongoing advancements, challenges remain with AI technology diversity. A conservative perspective still exists regarding their practical applications. As global medical device companies integrate AI into their products, domestic startups still face the challenge of overcoming capital gaps through technological advancement. Achieving diversity in AI technology is clearly a collective challenge that requires collaboration among companies, the government, and hospitals. A key hurdle is the limited accessibility of data. Due to privacy protections and legal constraints, medical data is confined mainly to major hospitals and select research institutions. To develop diverse AI solutions, diverse data from various medical institutions, patient populations, and lifestyle sources are essential. There is an urgent need to create an ecosystem that securely de-identifies such data for industrial use. It also includes accelerating the standardization of AI technologies. Differing computer systems, EMR formats, and data processing methods across hospitals impede the scalability of AI solutions. It is imperative to proactively establish related interfaces and data standards that facilitate seamless collaboration between AI companies and hospitals. Reflecting on KIMES 2025, it is clear that simply declaring 'we have adopted AI' is no longer enough to achieve differentiation. Instead, the focus must shift to 'diversity' so that AI is integrated throughout healthcare settings. Industry, policymakers, and the healthcare community must address this challenge together. We look forward to seeing even more diverse AI solutions at next year's KIMES event.
- Opinion
- [Reporter’s View] New Drug Trends and Choline Alfoscerate
- by Lee, Tak-Sun Mar 21, 2025 05:57am
- The Health Insurance Review and Assessment Service's Drug Reimbursement Evaluation Committee, which holds meetings every month, is the biggest hurdle to reimbursement in Korea and helps identify the latest new drug trends. For several years now, the keywords that have led the trend were “anticancer drugs,” “rare drugs,” “biological drugs,” and “high-priced drugs.” Contrary to how the trend leaned toward chronic disease treatments that owned a large patient population in the past, biologic targeted treatments for a small number of patients have become the trend these days. The characteristic of these drugs is that they are expensive. In line with the trend, the Health Insurance Review and Assessment Service and the National Health Insurance Service are in the process of establishing a system to manage such high-priced anticancer and rare drugs. Global pharmaceutical companies are now investing in anticancer and rare drugs with fewer patients and less competition, rather than developing synthetic drugs for chronic diseases, which become flooded with generics upon patent expiry. The companies’ investments have now been making results at the Drug Reimbursement Evaluation Committee level. However, the names of domestic pharmaceutical companies are rarely mentioned at DREC due to their lack of new drug development. Occasionally, the release of a new homegrown drug arises as a hot topic, but usually, the main players deliberated by DREC are multinational pharmaceutical companies. Domestic pharmaceutical companies are increasingly drifting apart from the new drug trend. Traditional pharmaceutical companies have missed the timing when biosimilar development began in the mid-to-late 2000s. When a bio-venture called Celltrion announced its challenge into the biosimilar field, the other companies ignored it, saying things like “there is no real profit” and “there is little chance of success.” When Samsung followed into the biosimilar market and achieved economies of scale, it was already too late. Currently, the top 2 pharmaceutical and bio companies in Korea are Celltrion and Samsung Biologics. I wonder what would have happened if domestic pharmaceutical companies had invested in biosimilars with the capital they had at the time, with a view to the future. Biosimilars are just one trend. The pharmaceutical market has been changing rapidly for the past decade. New diabetes drugs have been introduced one after another, and the cancer drug market has evolved from treatments targeting resistant mutations to immuno-oncology drugs such as Keytruda. The pace of pharmaceutical trends is too fast for Korean pharmaceutical companies to keep up with them, with the big pharma companies aggressively buying rare disease treatments through mergers and acquisitions. Nevertheless, Korean pharmaceutical companies are still stuck in the past. Many of the top-selling products of Korean pharmaceutical companies are the brain function enhancer, choline alfoscerate, which is already nearly 30 years old. In 2020, the government’s re-evaluation reduced the indications for the drug, and even though clinical trials are still underway due to doubts about their efficacy, sales of choline alfoscerate drugs have grown. It is a reliable cash cow for pharmaceutical companies. On the 13th, the Supreme Court ruled in favor of the government in a lawsuit filed by 26 pharmaceutical companies to cancel the notice of selective reimbursement of choline alfoscerate. With this ruling, it is likely that their selective reimbursement, which has been deferred with the lawsuit, will soon become a reality. If reimbursement for the drug is only applied to dementia diseases and excludes mild cognitive impairment, choline alfoscerate will no longer be able to serve as a cash cow for domestic pharmaceutical companies. While domestic pharmaceutical companies have managed to maintain their sales by defending the reimbursement reduction for their choline alfoscerate products for 5 years, this may have rather pushed companies to fall further behind in the market trends of anticancer drugs and orphan drugs. Of course, there are also pharmaceutical companies, such as Boryung and Handok that are increasing investment in the anticancer and orphan drug markets. However, there is concern whether most pharmaceutical companies will lose their investment timing as they struggle to maintain domestic sales, as was the case during the biosimilar boom. The global market's number one immuno-oncology drug, Keytruda’s follow-on is a biosimilar, not a generic drug. Celltrion and Samsung Biologics have just begun development efforts, but it is just a hope for domestic pharmaceutical companies.
- Opinion
- [Reporter’s View] Being a global clinical trial powerhouse
- by Kim, Jin-Gu Mar 18, 2025 05:56am
- Korea is a global clinical trial powerhouse. According to the Korea National Enterprise For Clinical Trials, Korea's share of global clinical trials was 4.04% as of 2023. It is in 4th place after the United States (22.02%), China (13.59%), and Spain (4.09%). By city, Seoul has been in first place since 2017. Korea’s outlook is also positive. Korea’s global clinical trial market share rank rose one step each year, from 6th in 2021 to 5th in 2022 and 4th in 2023. The share gap with Spain is only 0.05%, and if the current trend continues, Korea is expected to rise to become one of the Top 3 clinical trial countries in the world. However, there are some drawbacks. Although Korea has established itself as a clear clinical trial powerhouse in terms of quantity, there are indications that it is not so in terms of quality. The most straightforward example is the clinical trials related to antibody-drug conjugates (ADCs), which have recently emerged as a new trend in drug development. According to the Korea National Enterprise For Clinical Trials, as of May last year, more than 600 ADC-related clinical trials were registered on ClinicalTrials.gov worldwide, but there is not a single ADC clinical trial registered or being conducted in Korea. This cannot be simply attributed to Korea’s small population. This is because 27 ADC clinical trials are underway in Taiwan, which has a population of about half that of South Korea. It is time to improve the quality of Korea’s clinical trials. In particular, improving regulations related to clinical trials may be a way to improve the quality of clinical trials. From this perspective, it is encouraging that the first step has recently been taken in the development of “decentralized clinical trials (DCT)”. DCTs refer to de-hospitalized clinical trials that collect clinical data using wearable or mobile devices and deliver test drugs by mail, etc. Patients can participate in clinical trials without having to visit a medical institution in person. A representative success story of decentralized clinical trials is Moderna, which developed the COVID-19 vaccine. The industry has consistently called for the introduction of decentralized clinical trials. In response, the government has decided to launch a pilot project for decentralized clinical trials starting this year. The government plans to conduct 6 decentralized clinical trials during the pilot project period until 2027, and then institutionalize the system based on these trials. Korea’s high-quality infrastructure is considered the main reason why Korea has grown into a global clinical trial powerhouse. This includes excellent medical staff, hospital facilities, and the high level of education of clinical trial participants. This has enabled Korea to become a clinical trial powerhouse in terms of quantity. However, there is still much that needs to be done in terms of quality. Now is the time to improve the quality of clinical trials based on excellent infrastructure. Regulatory improvement is the surest way to improve the quality of clinical trials. This can foster an environment in which clinical trials of advanced drugs such as ADC can be conducted in Korea. Only when innovative new drugs can be developed in Korea will Korea be able to go beyond being a clinical trial powerhouse and become a pharmaceutical powerhouse.
- Opinion
- [Reporter’s View] Worth of ease in administration
- by Eo, Yun-Ho Mar 11, 2025 05:54am
- You can now orally take injectables, change the daily doses to monthly doses, and manage your disease with once a year injections. Such 'convenience of administration' has now become a competitive edge in the pharmaceutical market. Convenience, which had been mainly emphasized for chronic diseases, is now being highlighted in various other disease areas including anticancer drugs and autoimmune diseases. The emergence of one-shot drugs played a role, but other advanced new drugs have also been putting forth not only their efficacy but also their convenience. The convenience of administration literally means 'that taking the medication is convenient.' The question arises, 'If I'm taking medication because I'm sick, is comfort really that important? Shouldn't a drug’s efficacy be the most important aspect for the company?’ Nevertheless, pharmaceutical companies are quite obsessed with showing off their drug’s convenience. In many cases, convenience is the main slogan for marketing and sales of the relevant drugs. As such, “convenience” is emerging as a keyword in the healthcare industry. However, the value of such ‘convenience’ is not very recognized in the process of reimbursement listing. In particular, health authorities tend to be reluctant to accept the higher price of next-generation drugs that offer improved convenience compared to existing drugs for severe diseases such as cancer, which is a life-threatening disease. From the government’s standpoint, it’s a valid argument. If a drug receives a higher price simply because of its improved convenience, this may rather lower the opportunity cost for patients with other diseases that share the nation’s limited budget. However, there is a high possibility of new drugs that offer improved convenience while showing the same efficacy as existing drugs being introduced to the market. However, convenience is not unconditionally important. It depends on the situation. Common sense dictates that in the case of cancer, which is a life-threatening condition, there are not many cases where the prescription is changed for the sake of convenience. Therefore, the convenience of such anticancer drugs must be accompanied by a significant breakthrough in the method of administration or offer improved efficacy. A doctor would not give a patient a new drug if he or she is seeing efficacy with their currently prescribed drug, as this may cause unexpected side effects. In addition, the convenience of the drug may be reduced when it is used as part of a combination therapy regimen or when the patient has relevant diseases. However, it is also worth considering whether blindly rejecting the worth of convenience in administration is right. There are cases where such improvements in convenience can lead to improvements in treatment outcomes. There are also cases where long-acting drugs reduce the burden of monitoring and save health finances. In the current healthcare trend where cancer is also turning into a chronic disease, the 'quality of life' of the patients cannot be ignored. Convenience is a value that is difficult to either blindly support or ignore. However, if a drug has been developed to treat a major condition, that values convenience in administration, the value of this drug that met this need should be recognized, amid the increasing number of drugs that are facing difficulties in the listing process that offer improved convenience.
- Opinion
- [Reporter's View] K-Bio's global entry and transparency
- by Whang, byung-woo Mar 10, 2025 05:51am
- The active overseas expansion of domestic bio companies is considered to be one of the essential strategies for continuous growth. News is coming in on domestic companies participating in overseas academic societies and conferences. This has become an important strategy for strengthening competitiveness and building trust in the global market, beyond simply expanding the scope of a company's international activities. However, efforts are also being emphasized to ensure that their activities on the international stage do not simply end at 'participation' and lead to 'substantial results'. First, the transparent disclosure of the participation results is necessary. Many companies participate in academic conferences and conferences held abroad, but information about what they have achieved or what challenges they have faced is often not disclosed. This lack of information can affect not only internal development but also the long-term building of trust with investors and partners. From the company’s perspective, it may not share subsequent information due to a lack of performance or substantial results. However, transparently disclosing the results of participation, regardless of the achieved results, can send a positive signal to stakeholders and contribute to increasing the credibility of the company. Many domestic companies are entering the global arena, so no one expects much with a single participation. It is more reasonable to look at the lessons and experiences gained in this process as the drive to move on to the next step. That is why experts emphasize transparency and continuity as a way to ensure the success of companies. One-time participation may generate short-term interest, but continuous participation is believed to help companies build in-depth networking and long-term partnerships in the global market. In fact, many biotech company representatives say that when they participate in events like BIO USA, discussions develop further and progress in the following years with continuous participation rather than in the first year. Until now, the word “continuum” has been used much when mentioning government support for the development of the domestic pharmaceutical and bio industry. As a later entrant to the global market, continuous government support is an indispensable element for the development of the domestic pharmaceutical and bio industry. However, fundamentally, individual companies must lay the groundwork for the domestic pharmaceutical and bio-industry to become competitive on the global stage. Many companies have been seeking to enter overseas markets from the beginning of the year. Participation in overseas academic societies and conferences is an important opportunity for domestic bio companies to achieve sustainable growth in the global market. To this end, companies need to make efforts to strengthen the transparency of the companies' attempts to enter overseas markets and their continuous participation.
- Opinion
- [Reporter's View] Concerns about new drugs for rare diseases
- by Son, Hyung Min Mar 07, 2025 05:56am
- Duchenne muscular dystrophy is a rare muscular disorder caused by the lack of dystrophin, primarily affecting boys. The symptoms typically begin before the age of 3 and rapidly deteriorate, eventually leading to the loss of ability to walk before the age of 10 and above. To date, various genetic targeted therapies for Duchenne muscular dystrophy won FDA approval, including U.S.-based Sarepta Therapeutics' 'Amondys 45,' 'Exondys 51,' 'Vyondys 53,' and 'Elevidys,' the American subsidiary of Japan's Nippon Shinyaku NS Pharma's 'Viltepso,' and Italfarmaco's 'Duvyzat.' Since these new drugs have not been introduced to South Korea, so patients with Duchenne muscular dystrophy are relying on steroids. The Korea National Enterprise for Clinical Trials (KoNECT) ranked 'Amondys 45' as the No.1 new drug for domestic entry candidates through the '2021 Report priority ranking international new drugs not yet introduced in South Korea.' However, it has not been introduced to South Korea to date. New drugs have been introduced to various rare diseases, not only Duchenne muscular dystrophy but also metachromatic leukodystrophy and hemophilia, but patient access to these drugs is still limited. Rare diseases have limited treatment options and often patients have limited treatment opportunities. Fortunately, innovative new drugs for rare diseases have emerged in recent years. Introducing new drugs for rare diseases provides not just simply new drugs but opportunities for patients to improve their quality of life substantially. Notably, new drugs such as genetic therapy offer the possibility of fundamental treatment. Patients who have already received treatments give favorable reviews that their quality of life has significantly improved. However, the discussion on whether new drugs can be provided to Korean patients remains a crucial issue. Many pharmaceutical companies face challenges to commercialize new drugs for rare disease due to the lack of treatment infrastructure and limited treatment targets. Eventually, to promote domestic entry of new drugs for diseases with high unmet needs and limited treatments, supportive policies are essential, including government funding and insurance reimbursement. Until now, most evaluations indicate that the existing supports and policies toward patient access to new drugs for rare diseases have been inadequate. Clinical trial opportunities for Korean patients with rare disease must be discussed in depth. If the Korean market is not considered, there is no need for pharmaceutical companies to conduct new drug clinical trials subjecting Korean patients. If clinical trial opportunities involving potential new drug candidates are unavailable to patients without further treatment option, patients have no other resorts. It is of utmost importance to broaden patient access so that pharmaceutical companies would consider the Korean market. It is also essential for Korean pharmaceutical companies to pursue new drug introductions and make R&D investments. Importantly, pharmaceutical companies and the government must collaborate to establish ways to introduce effective new drugs. Fortunately, Korean pharmaceutical companies strive to introduce new drugs for rare diseases. Pharmaceutical companies like Dong-A ST and Boryung are pursuing new drugs in collaboration with KoNECT. Also, many Korean pharmaceutical companies like Handok have been distributing new drugs for rare diseases. No disease is unimportant, from rare diseases, cancer, brain tumors, severe diseases, and chronic diseases. However, patients should not have to travel abroad for domestically unavailable treatments.
