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- 2025-12-19 22:53:54
- Opinion
- [Reporter’s Note] Hasty move on anti-droplet masks
- by Jul 16, 2020 05:54am
- “Anti-droplet masks? They’ll be available from Monday. People with waiting number tickets given out from 9:30 in the morning can buy a box each.” This is the answer I got at a hypermarket in Seoul asking for an anti-droplet mask. The anti-droplet mask has been in the hypermarkets and convenient stores for a month now, but they are a rare find. I have visited a number of retail shops selling the masks in Seoul, but they were “not in stock.” These places said the masks are not stocked every day, and the limited supply lasts only a few hours after opening. The absence of anti-droplet masks is replaced by mass produced single-use masks. Most of them are made in China with a price tag ranging from 280 won to 1,600 won per mask. The prices and qualities of these masks are inconsistent and the consumers can hardly check if they are made in Korea or China. Unlike quasi-drugs, these mass produced masks are not certified by the Ministry of Food and Drug Safety (MFDS) for liquid blocking or anti-bacterial function. In other words, the quality is questionable. If not at a pharmacy, clerks at general retailers cannot tell which mask is which. Many of the retail shops I visited said the single-use masks are dental masks. Surprisingly, some of them were selling anti-droplet masks as general single-use masks. In such chaos, pharmacies and distributors who used to supply the public-distributed masks are now torn. They are seeking for ‘Korea Filter (KF)’ masks with assuring quality, but now that low-quality products are as highly sought after, they can barely find a manufacturer to produce high-quality masks. Even after finding a willing manufacturer, the high-quality masks would always be more expensive than the single-use masks. An insider from a distributor complained, “To be honest, many of those Chinese-made products (general mask) offer a price as low as 100 won. They are using cheap and questionable felt material that we definitely say no to,” although “we are looking for certified anti-droplet or dental masks, they are realistically very difficult to get our hands on. Even if we supply the rare masks, we get complaints on the high price as the people get confused with cheap and widely available mass produced products.” The chaotic mess in the market was caused, when the Korean government designated the anti-droplet mask as a quasi-drug without confirming the stable supply and hastily promoted it as a replacement for KF masks. The skewed demand on inaccessible anti-droplet and dental masks actually benefited single-use masks. The mask manufacturers apparently prefer mass producing uncertified single-use masks, instead of quasi-drug masks requiring a certification by the government. Even worse, the certified masks get restricted from overseas exportation. Some hints that importing extremely low-cost Chinese-made masks is more profitable. The government is now sluggishly saying they would enforce KC certification on general masks after observing the situation. As the summer is approaching, the high demand on thin-layered masks was predictable. And the government could have easily expected the industry growing resistant to switch production from KF mask to anti-droplet mask. The thin-layered masks are uniquely and highly demanded for the summer season only, but a manufacturer has to exhaustingly change up the parts of production line. The government’s hasty actions like designating and promoting anti-droplet mask as a quasi-drug or retrospectively reinforcing quality control on mass produced masks could have been prevented, if only they have surveyed the industrial environment better and faster. Hopefully, we can expect the government to take more proactive actions in the future than reacting to already-stirred up market.
- Opinion
- [Reporter’sView] A good administrative move by government
- by Eo, Yun-Ho Jul 06, 2020 10:44am
- Korea’s Ministry of Health and Welfare (MOHW) withdrew the plan to apply the stepped drug pricing reduction on drugs transferred by business restructuring in just four months to reflect the pharmaceutical industry’s opinion. It was first announced in February, and the revision was updated in June. The industry has been in a chaos for a while as the government mentioned of a possibility of applying stepped pricing on the transferred drugs. Initially, the stepped drug pricing, in effective from August, sets the upper limit pricing a drug, regardless of qualifying two criteria for top-level pricing, at 85 percent of either the lowest pricing or 38.69 percent of the first-in-class drug, if the number of listed same-substance drugs exceeds 20. However, the Pharmaceutical Decision and Adjustment Criteria amended in last February opened room for the stepped pricing to be used on a product that succeeds business status due to corporate restructuring—for example in case of M&A, corporate split or license sell-off. Accordingly, companies like Pfizer Pharmaceutical Korea (Upjohn) and MSD Korea (Organon) preparing for multiple transfer of original products to the respective split off companies, feared of the possible pricing reduction. Takeda Pharmaceuticals Korea that recently sold off an antidiabetic treatment pipeline to Celltrion could not be completely free from the same fear. Relevant industry organizations like Korean Research-based Pharmaceutical Industry Association (KRPIA) and Korea Pharmaceutical and Bio-pharma Manufacturers Association (KPBMA) submitted statements to urge MOHW to exempt transferred products from stepped pricing reduction, and requested for flexible interpretation of the regulation. Surprisingly, MOHW’s action was even more straightforward. The Pharmaceutical Decision and Adjustment Criteria were revisited again and announced the newest amendment last month. Fortunately, it subsided the industry’ complain fast. In this day and age of high-cost drugs, the Korean government puts forth ‘trade-off’ as the key regulatory approach to lessen the expenditure on off-patent drug for compensation on new drug coverage. As for the government, the initial plan was a great chance. Besides generics, lowering the pricing of the transferred original would put generics pricing higher than the original, and most likely the same substance drug pricing would fall, automatically. But the government accepted the industry’s logic behind its argument. A good communication can also lead to another opportunity. Hopefully, the government could result in good trade-off while listing new drugs or expanding coverage in the future.
- Opinion
- [Reporter’sView] Finding the key in platform technology
- by An, Kyung-Jin Jul 03, 2020 06:18am
- The world is eyeing on companies developing platform technology for novel drug. In last few years, Korean bio technology companies like ABL Bio, LegoChem Biosciences and Alteogen have signed deals worth over a trillion won with global pharmaceutical companies on rights over platform technology that facilitates drug delivery. Each in last November and June this year, Alteogen signed two licensing agreements with global pharmaceutical companies on human hyaluronidase (ALT-B4) technology that enables intravenously injected biologics to also work as subcutaneous injection. The company earned 35 billion won from the two companies licensed in the platform technology. The number already exceeds the Korean company’s sales in last year totaling at 29.2 billion won. From last year and on, LegoChem Biosciences have inked three deals on antibody-drug conjugate (ADC) technology. The technology mediates efficient delivery of drug to cancer cells by improving the unstable linkers between protein and antibody. In March last year, LegoChem licensed out three ADC-applied novel anticancer therapies to Millennium Pharmaceuticals, a subsidiary of Takeda Pharmaceutical. And in April and May this year, the Korean company signed deals for the U.K.-based Iksuda Therapeutics to hold exclusive rights to use the ADC technology and ADC-based cancer therapy. A platform technology is defined as source technology to apply on development of new drugs. Such technology can enhance medication convenience or benefit by changing injection to oral administration or switching intravenous route to self-injectable subcutaneous injection. The biggest appeal of the platform technology in novel drug development is the potential of expansion. Generally, the probability of candidate medicine to receive sales approval at Phase I clinical trial stage is 10.4 percent, at Phase II is 16.2 percent and at Phase III is 50.0 percent. Even a company with multiple new drug pipelines cannot be completely free from the probability of failure. On the contrary, platform technology holds less risk in failure as it could be used on different candidate drug when it fails in other. For instance, Hanmi Pharmaceutical took a blow when Sanofi returned the rights over efpeglenatide, but if the neutropenia treatment Rolontis (eflapegrastim) gets approval from the U.S. Food and Drug Administration (FDA) within this year, the Lapscovery technology’s potential could be reevaluated. Like the licensing agreements LegoChem or Alteogen signed, an exclusive rights or a novel therapy based on the technology could be transferred. But also the licensing profit could be maximized by signing a non-exclusive deal on the technology. Good news to the Korean industry is that the world’s perception on Korean pharmaceutical and bio companies have significantly improved in last few years. The Korean industry’s successful outcomes are yet to be tangible, but there are many companies with platform technologies on par with other global companies. Hopefully, the heightened interest on the platform technology would induce investment on these promising companies.
- Opinion
- [Reporter’s view] Listed drug reevaluation, now what?
- by Lee, Hye-Kyung Jul 01, 2020 05:56am
- The Korean health authority issuing a statement on reducing choline alfoscerate coverage is just a beginning. On June 11, Health Insurance Review and Assessment Service (HIRA) Drug Reimbursement Evaluation Committee (DREC) announced the decision to raise the patient copayment rate on choline alfoscerate, which has been covered by the National Health Insurance so far, from 30 percent to 80 percent for the effect and benefit of treating neurometabolic disease, emotional and behavioral change, and senile pseudo-depression. Increasing the copayment rate means the authority is to narrow the coverage on the drug. The selective reimbursement on the drug would impose 80 percent of copayment rate. The original full coverage would be limited the indication treating severe dementia case among the patients showing secondary symptoms of cerebrovascular insufficiency and degenerative brain-organic psychiatric syndrome. HIRA revisited the cost-effectiveness of choline alfoscerate products by studying the clinical efficacy, alternative options, and administration cost of the drug based on textbooks, clinical guideline, Health Technology Assessment (HTA) and clinical research literature (SCI, SCIE). Reviewing the clinical evidence and other considerations like social demand focusing on financial impact, medical importance and patient’s economic strain, the reimbursement on the drug except for the dementia indication was switched to selective reimbursement. Regarding the reevaluation result of the already-listed drug, a pharmaceutical company with a drug subject to reimbursement adjustment may appeal the decision for 30 days. Accordingly, the final decision on the coverage reduction would be enforced in coming August at earliest, after undergoing DREC re-deliberation and Ministry of Health and Welfare (MOHW) Health Insurance Policy Deliberation Committee (HIPDC) review. The government’s announcement has been expected since the National Health Insurance Master Plan was disclosed. MOHW has hinted of a listed drug reevaluation that generally takes account of clinical efficacy, financial impact and foreign country reimbursement status of the listed drug, as the ministry claimed reimbursement adequacy reevaluation mechanism reflecting the clinical efficacy was missing so far, unless the Ministry of Food and Drug Safety (MFDS) removes the coverage. For about a year, the public raised many issues regarding the first target of the reimbursement reevaluation. The reevaluation on choline alfoscerate was mentioned during the National Assembly Audit last fall, and eye drops were also mentioned as a target as well. However, it was narrowed down to the dementia treatment alone, as the eye drops have ongoing legal case. The pharmaceutical industry is reactively seeking for responsive plan after the announcement by HIRA DREC. Some say they would go as far as filing an administrative litigation to halt the execution, but it could be too late in the end. HIRA could have selected the second and the third listed drugs to reevaluate following choline alfoscerate. The next target would highly likely to be a drug prescribed as a supplement and not for unique effect or benefit. The pharmaceutical industry’s next action should be trying to predict the next target drug and concentrating on how to demonstrate clinical efficacy, cost-effectiveness and social demand better.
- Opinion
- [Reporter’s view] Unpredictable authorized generic policy
- by Lee, Tak-Sun Jul 01, 2020 05:54am
- The MFDS is planning to disclose the 'generic competitiveness strengthening plan' discussed through the public-private association in the near future. Some negotiated content has been reported. However, the main part of the generic policy seems to be known only when the final proposal is issued. The industry believes that the impact on the industry will also be enormous. These are tasks such as the introduction of INN (international nonproprietary names) in generic products. Currently, it is not possible to know whether the 'generic competitiveness strengthening plan' is a deregulation or a strengthening of regulations. The 'unification of authorized generics', which was first released to the media, was viewed as a deregulation in that the company does not need to undergo duplicate examinations. That is why the MFDS changed its policy after the co-biological equivalence limit policy failed at the Regulatory Reform Committee. However, the contents released afterwards are confused by strengthening regulations rather than deregulation. The challenge derived from the public-private councils, such as the development of quality indicators for livelihoods, disclosure of evaluation results, and enhancement of drug disclosure and information disclosure by pharmaceutical companies, is a policy of strengthening regulations to give generics a rank. As the policy direction of the MFDS is not shared, it is difficult for companies to pursue business. In particular, for companies seeking to expand the consignment manufacturing business, the method of limiting the co-biological equivalence test was frustrated, but the policy base was unpredictable, making it impossible to make an investment decision. This is explained by the fact that the contents of the discussion are shared only within the framework of the public-private council. The pharmaceutical industry points out that through the COVID-19 situation, the MFDS communicates in the course of policy making. In the case of the clinical reevaluation of recently announced Choline alfoscerate, it was difficult to know the exact contents until related companies were also announced. There seems to be no intention of policy communication. Press releases distributed to the media are everything The government's fate is to listen to as many opinions as possible and take criticism until the policy direction is decided. If policy promotion is inevitable, you can persuade them to fully understand. If this process is a reduced or deleted policy, the result will be bad. No matter how difficult it is to communicate with COVID-19, they have to disclose what they want to disclose and communicate with anyone who wants to communicate.
- Opinion
- [Editor’s View] Adjustment on choline alfoscerate coverage
- by Nho, Byung Chul Jun 11, 2020 06:23am
- Korea’s National Institute of Dementia (NID) found that 43 percent of senior citizens aged 60 to 69 fear dementia the most as far as disease goes. The number of patients with dementia in Korea has rapidly surged to date, and it is projected to exceed one million by 2024 and two million by 2039. And the cost of state-led dementia management is projected to see 15-fold jump from 8.7 trillion won in 2010 to 134.6 trillion in 2050. Due to the surge in dementia patient population and raised awareness of the disease, many of middle-aged people started demanding prescription by healthcare providers to prevent developing dementia. Accordingly, the prescription volume of cognitive function improving choline alfoscerate, used on patients with mild cognitive impairment (MCI), soared exponentially and claimed reimbursement of 325.5 billion won in 2019, making it a major prescription drug market. Noticing the sharp increase in choline alfoscerate reimbursement claim, however, Ministry of Health and Welfare (MOHW) has started reevaluating the adequacy of National Health Insurance (NHI) reimbursement on the medicine, while Health Insurance Review and Assessment Service (HIRA) is to convene Drug Reimbursement Evaluation Committee (DREC) to discuss the issue. The pharmaceutical industry sources report the government bodies are leaning towards designating the use of choline alfoscerate in a person with MCI as selective reimbursement with higher copayment rate. The groups opposing on providing reimbursement on choline alfoscerate claim the substance’ clinical efficacy is insufficient and excessive prescription is damaging the NHI finance. Nevertheless, the clinical experts say otherwise. Currently, a treatment for dementia does not exist; pharmaceutical giants like Pfizer, MSD and Lilly have invested on the pipeline immensely, but they gave up on it in the end. Although many hopeful studies spoke of seemingly ending the fight against dementia, not one dementia drug has received the U.S. Food and Drug Administration’s (FDA) novel drug approval since 2003. Considering the situation, the best means of treatment is to protect the cognitive function as much as possible from the MCI stage, right before developing dementia, and to constantly observe the progress of dementia. Finding the sign of dementia at early stage and treating it can significantly reduce the pain and burden of the patient’s family, in which the social cost can be lessened as well. And healthcare provider’s frustration surfaces from here. The prescribers are running out of options in cognitive function related drugs as donepezil’s indication to treat vascular dementia is removed, acetyl-L-carnitine’s indications were narrowed and now choline alfoscerate reimbursement would be adjusted. These changes are contradicting the Korean government’s emphasized goal to expand state-led dementia management. When an alternative option is unavailable for the mild cognitive impairment—a crucial stage to treat dementia early, increasing the patients’ copayment rate may immediately bring down the cost. But patients would miss the window to comprehensively control dementia development due to frequent visit to hospital, which eventually would result in treating even more dementia patients. And applying differentiated copayment rate by disease type would technically cut down reimbursement more and distort the clinical scene by inputting different disease code. Choline alfoscerate may have been criticized to have insufficient clinical evidence, but it has the highest number of evidences among cognitive function enhancing drugs. And in Russia, where the drug is designated as prescription use, had positive findings in 50 patients with amnestic mild cognitive impairment. Regarding the study, the researcher states choline alfoscerate has outstanding tolerability and confirmed safety, and recommends using the drug in people with high-risk of developing Alzheimer’s disease, such as people with mild cognitive impairment, as a preventive measure. Moreover, brain disease treatments struggle to find participants for large-scale clinical trial, so evaluating the drug’s benefit tends to be extremely difficult. A high number of reimbursement claims well depicts high social demand and the reality with no other option. The health authority should be advised that adjusting reimbursement on the drug and impeding prescription due to high cost could have the patients and their families to resort to untested folk remedy with growing anxiety and ultimately spend more money in the process.
- Opinion
- [Reporter’s View] Not penalty but incentive to generics
- by Lee, Tak-Sun Jun 09, 2020 06:26am
- Under the grandeur name of ‘strengthening market competitiveness,’ Korean-made generics are to face various changes in policy in the future. Concerned of excessive number of generics weakening the market competitiveness, the Korean government has already attempted to restrict joint bioequivalence test and reduce pricing of generics with cosigned bioequivalence test result. But it was scrapped by the Regulatory Reform Committee. However, Ministry of Food and Drug Safety (MFDS) is unceasingly reviewing a number of plans to better manage generics with public-private consultative body. So far, they have mentioned of specially labeling package of drugs that conducted individual bioequivalence test, setting bioequivalence quality assessment indicators, and building a database on generic products by each substance. Regardless, only the time will tell, if all these actions would effectively improve generic products’ competitiveness. Unfortunately, most of the competitive enhancement plans are focused on ‘penalty.’ The policy to bring down the pricing of generics without individual bioequivalence test result is a good example. The public-private consultative body was on the same page about being more selective with generics. Naturally, the products at the end of the line would feel penalized. Nothing is more inspiring than a compliment. And we would have to see if the Korean-made generics, tattered by restricting regulation, would survive in the market with so-called ‘strengthened competitiveness.’ Rather, these restraints may even advertise generic as a lower-quality version of the original. Therefore, a means to grant incentives to well-developed good-quality generics should be discussed as well. For instance, the government should consider policies to provide benefit on patent-evading generic with preferential sales approval, generic with an overseas export deal and products contributing to save NHI finance through lowered price. Only when the government promotes the good side of generics, the market would be confident enough to trust those generics. Hopefully, the government would seek for policies granting incentives, besides the penalties, to improve generics’ market competitiveness.
- Opinion
- [Reporter’s view] Pharmacists are tired of impurities
- by Kim JiEun Jun 04, 2020 06:13am
- Another task has recently been added to the pharmacy. From Valsartan, Ranitidine to Metformin. This is because pharmacies have to clean up with repeated impurities situation. The Metformin situation was stopped by the sale of some items, so there was not much confusion than the previous Valsartan or Ranitidine events. However, due to the past two cases, alternative medicines showed a shortage shortly after the announcement of discontinuation. In particular, some of the drugs that have been discontinued this time have fewer drugs to replace, and alternative drugs were sold out of major online drug stores less than an hour after the announcement of discontinuation. It had a hard time for pharmacists who couldn't order medicine quickly to get out of stock. Again, pharmacists who have come into contact with the media are in chaos. They had to clear the stock of drugs that had been discontinued since the morning, and they were busy going into and out of an online mall to order a replacement drug and inquiring about the drug at a wholesale dealer. The pharmacy dispensary was filled with new medicines that were ordered immediately after the announcement of the suspension. If the demand is unpredictable and they don't know when it will be out of stock, it is best to stock up on the new drugs. Even in the case of Valsartan and Ranitidine, pharmacies with many prescription preparations had to give up pharmacy space for drugs that had been ordered in advance for several months. This is not all. The pharmacy had to respond to patient inquiries that followed immediately after the announcement. Like any pharmacist who said, "We are not wrong, we must explain and persuade the pharmacy to be wrong every time." Even in this situation, pharmacists had to explain why the drug they were taking was discontinued and how to deal with it. It is not only a problem that will end with Valsartan, Ranitidine, and Metformin in an era when impurities have emerged as a new era of drug safety management. Although the original responsibility and management of impurity medicines ultimately belongs to the pharmaceutical industry and regulatory authorities, there is an unfair aspect in the current situation where pharmacies must stand at the center of the situation whenever it happens. Of course, it is a top priority to prevent the current situation from being repeated with clear standards and strict regulations. However, as impurities have emerged as a new paradigm for drug safety management, it seems that the government will not be able to ignore the introduction of the international common name and furthermore, the introduction of ingredient names. The fact that this year is the 20th anniversary of separation of prescribing and dispensing drugs will also be a part of empowering discussions on these systems.
- Opinion
- [Reporter’s view] We must prepare for the post impurity era
- by Kim, Jin-Gu May 27, 2020 06:04am
- The situation wasn't the worst. Unlike Ranitidine, not all Metformin sales have been stopped. It is expected that the confusion of patients and the potential damage of pharmaceutical companies will not be greater than in the case of the Valsartan·Ranitidine crisis. The MFDS decided to stop selling 31 of the 288 finished Metformin products in Korea on the 25th. The reason was the same as in the previous situation. N-nitrosodimethylamine (NDMA) was detected above the provisional management criteria. As a result, the case of impurities, starting from Valsartan and Ranitidine to Metformin, has been closed. At present, there are no other ingredients that raise concerns about impurities. However, it is not the end, it’s the beginning. Impurities have become a new disease and standard for drug safety management. The time has come for us to manage unexpected impurities in advance. It must be a contradictory situation. It is contradictory to anticipate and manage unexpected impurities in advance. However, this contradictory situation is the reality faced by the pharmaceutical bio industry. The paradigm of drug safety management has changed completely. The MFDS has been obliged to submit proof of safety for carcinogenic impurities, metal impurities, etc. when pharmaceutical companies apply for drug approval from September. It is only possible to preemptively check for harmful substances that can occur on its own and to prove safety. There are many problems that have not been solved. First, it is a standard for impurity management. Currently, NDMA·NDEA are all impurities that have been established as management standards. However, NDMA·NDEA are only some of the many impurities. The possibility of Nitrosamine-based impurities such as NMBA, DIPNA, and EIPNA cannot be excluded. If the range is extended to impurities other than Nitrosamines, theoretically, impurity problems close to infinity occur. These impurities should be cataloged, and separate control standards should be prepared for each impurity. The MFDS has announced that this work will be done in cooperation with regulatory authorities in each country. However, as COVID-19 suddenly became world-wide, it was postponed. The issue of responsibility derived from this is another problem. The regulatory authorities and the pharmaceutical industry may not be able to predict in advance, but the damage caused by the pharmaceutical industry should be more concerned. Discussions with the regulatory authorities and the pharmaceutical industry should begin to rationalize responsibility. At present, the Metformin situation is thought to be over. However, the new era of drug safety management has just begun. You can't have the same confusion every time. We look forward to setting new standards for the new era as soon as possible.
- Opinion
- [Reporter’s View]Raising the voice of rare disease patients
- by Eo, Yun-Ho May 20, 2020 06:11am
- Seems like rare diseases put patients in utmost pain, because of its ‘rarity.’ Rare disease treatments can hardly prove cost-effectiveness and predictability with limited patient size for the reimbursement listing approval. Regardless, the Korean government is aware of the struggle. The revised drug pricing system the Ministry of Health and Welfare (MOHW) unveiled is in process of legislative preannouncement until June 11. The key revision in the new drug pricing system is to expand eligibility in pharmacoeconomic evaluation (PE) exemption and RSA. The revision would stipulate the Korean government to sign risk sharing agreement (RSA) not only with first-in-class drugs, but also with follow-on drugs. And also the revision would expand subjects for PE exemption and coverage with phase III clinical evidence in development. Even the PE exemption, previously restricted to anticancer and rare disease treatments, would be applicable on National Essential Drugs, such as tuberculosis treatment, antibiotics and emergency antidotes. However, many of the public claim rare disease treatments would still face various obstacles before receiving RSA and PE exemption benefits. In Korea, rare disease is defined as a disease with patient size less than 20,000 with difficulties in diagnosis to survey accurate number of patients. The affected patients are desperate to get access to life-saving treatment as the diseases are difficult to diagnose and treat, but many of the diseases cannot even start a clinical study with barely sufficient number of patients. Pharmaceutical companies are hesitant to develop a novel drug for rare disease, as the limited number of patients means limited marketability, while the chances of successfully proving the cost-effectiveness of the drug through PE would be thin. Many countries around the world are introducing regulatory exceptions in legislation, special approval or reimbursement listing for better access to rare disease treatments. Korea has also provided regulatory exceptions like medically essential drug management, PE exemption system and RSA in reimbursement listing or drug pricing. Regardless, the limitations to treatment access still exist. In fact, data comparing listing rate by each drug type before and after implementing RSA and PE exemption systems for bettering the access to high-cost treatments showed a significant improvement in general drug (79.6 percent to 98.6 percent) and anticancer treatment (77.1 percent to 91.7 percent), but the improvement in rare disease treatment (71.1 percent to 71.4 percent) was rather unnoticeable. As a solution to the much needed access to rare disease treatment, some argue the PE exemption eligibility should be expanded for the rare disease treatment. They say the reimbursement review system should be flexible to exempt PE or adjust the number of PE sample patients similar to the special case reimbursement standard, when a drug has no other alternative option or has been approved with placebo-controlled study results. For instance, the U.S. Food and Drug Administration’s (FDA) Breakthrough Therapy Designation (BTD) or European Medicines Agency’s (EMA) Priority Medicine (PRIME) designation could be referred as a review standard. The PE exemption system aims to provide patients the access to rare disease and anticancer treatments unable to produce PE data, while protecting the initial purpose of positive listing system. So to serve its purpose, the Korean health authority should contemplate on the flexibility of the healthcare reimbursement listing procedure. Same goes for RSA. Compared to numerous anticancer treatments that received coverage through RSA, many of rare disease treatments have failed in passing the barrier to RSA due to lack of PE data. As far as quality-adjusted life-year (QALY) goes, the majority of rare diseases results in severe physical damage in patient’s body, lowers quality of life, and shortens life expectancy. And because of extremely small number of users, rare disease treatments are inevitably high-priced and show poor cost-effectiveness. The rare disease treatments can hardly prove cost-effectiveness, when applied with incremental cost-effectiveness ratio (ICER) thresholds on par with other general drugs. The regulation stipulates the health authorities to flexibly set ICER thresholds on rare disease treatments, but the drugs are challenged with PE barriers higher than that of anticancer treatments due to their practices so far. This is why many urge more adaptable ICER thresholds should be given to rare disease treatments for their PE. When the Rare Disease Management Act came in effect in 2015, the Korean government designated May 23 as the Rare Disease Day to raise awareness and understanding of patients fighting against rare diseases. Rare diseases have a handful of treatments for a handful of patients, which is why the voice to demand their healthcare benefit is so easily lost. Hopefully in the near future, the government and pharmaceutical companies would pay a closer attention to those patients, as much as they do for cancer patients.
