- LOGIN
- MemberShip
- 2026-04-14 03:36:11
- Policy
- Patients with SCLS shouldn't get Janssen vaccine
- by Lee, Tak-Sun Jul 16, 2021 05:54am
- The MFDS said Janssen COVID-19 vaccine should not be vaccinated for people with a history of Systemic Capillary Leak Syndrome (SCLS). This is in accordance with the European EMA recommendation and is similar to the action that was taken on the AstraZeneca vaccine on the 14th of last month. The MFDS released the Dear Healthcare Professional Letter on the 12th. EMA's PRAC announced that Systemic Capillary Leak Syndrome should be added as a new side effect of the Janssen vaccine. It reviewed three cases of Systemic Capillary Leak Syndrome in Janssen COVID-19 vaccine recipients, one with a history of Systemic Capillary Leak Syndrome and two with fatalities. Systemic Capillary Leak Syndrome is a rare and serious condition that causes fluid leakage in small blood vessels (moscevascular), mainly resulting in edema in arms and legs, hypoglycemia, blood concentration, and hypoglycemia (critical blood protein). The MFDS said it will add related precautions to the manual by compiling the current status and safety information at home and abroad, stressing that people with a history of Systemic Capillary Leak Syndrome should not be vaccinated against Janssen vaccine.
- Policy
- Generic for Galvusmet was first approved
- by Lee, Tak-Sun Jul 16, 2021 05:54am
- The timing of the sale varies depending on the results of the Supreme Court's lawsuit against the validity of the patent. Hanmi has been approved for the first time in Korea as a generic for Galvusmet, a diabetes complex affiliated with Novartis' DPP-4 inhibitor. Hanmi developed Vildagliptin HCl and licensed both single and multi-drugs including Vilagliptin. Attention is focusing on whether it will preoccupy markets as it has made efforts to commercialize them early. The MFDS approved Hanmi's Metformin HCl-Vildagliptin HCl. Vildaglmet is the first generic for Galvusmet to be licensed in March 2008. It contains the same Vildagliptin HCl as the single Vildagle approved by Hanmi in February this year. The original Galvusmet is a salt-free Vildagliptin. Vildaglmet is the first licensed Galvusmet in Korea, so it is likely to be released first in the market. Material patent of Vildagle can be released earlier than other companies. The material patent expires on 4 March 2022. An-Gook and Hanmi have reduced the term of 187 days on the Intel Property Trial and Appeal Board and 55 days on the Patent Court through a lawsuit against the extension of the patent. It was shortened in the second trial. If the Patent Court cites the Intellectual Property Trial and Appeal Board's verdict, both companies' products can be released within the year and can be released next year according to the Patent Court. However, the Supreme Court may not recognize the claim for invalidation of the extension. If Supreme Court accepts the an extension of patent term, Ahn-Gook and Hanmi, which filed a patent suit, can be the first to put the product on the market before other companies. Hanmi can release both products at the same time because both single and comination drugs are licensed. This is because all patents related to compound drugs have been avoided. Hanmi has experience in co-selling Galvus with Novartis from 2014 to 2016. After the expiration of the copromotion, Hanmi worked on generic development and set up a strategy to preoccupy the market. It sought early release only with permission for efficacy and effects that did not violate patents, but lost a patent lawsuit. Since then, the product has been withdrawn. Outpatient prescription for Galvus and Galvusmet amounts to ₩8.1 billion and ₩36.4 billion, respectively. Hanmi does not have DPP-4 inhibitors. Therefore, early release of Vildagle and Vildaglmet is expected to expand in the diabetes market.
- Company
- Novartis most challenged for its patents, followed by Pfizer
- by Kim, Jin-Gu Jul 16, 2021 05:54am
- Among pharmaceutical companies, Novartis was found to have received the most amount of patent challenges since 2016. Over the past five and a half years, 33 generic companies had filed a total of 121 patent suits against Novartis' 15 patents on 8 products. In addition to Novartis, generic companies also targeted Pfizer, Boehringer Ingeheim, Astellas, and AstraZeneca. The same went for domestic companies. United Pharma, Alvogen Korea, Boryung Pharmaceutical, and Chong Kun Dang were also challenged on their patents by generic companies. ◆Generic companies challenge patents of 8 Novartis products On the 13th, Dailypharm’s tally of patent challenges in the biopharmaceutical sector since 2016 showed that Novartis has received the most challenges from generic companies. Generic companies had challenged 15 patents of 8 drugs owned by Novartis. A total of 121 requests for appeal and trial were filed by the companies against Novartis. In the same period, the total number of appeals and trials claimed (scope of right + invalidation suits) amounted to 1,042. In other words, 1.2 cases in every 10 patent suits filed had targeted Novartis. The 8 products that received patent challenges were;▲heart failure treatment ‘Entresto’; ▲DPP-4 inhibitor antidiabetic ‘Galvus’; ▲Breast cancer treatment ’Afinitor’; ▲ glaucoma treatment ‘Simbrinza eye drops’; ▲ conjunctivitis treatment ‘Pazeo eye drops’; ▲ COPD treatment ‘Xoterna’; ▲ chronic iron overload treatment ‘Exjade FCT’; and ▲ immunosuppressant ‘Certican’ The interesting point is that only 2 of the drugs among those in legal dispute are blockbuster drugs that bring over ₩10 billion in annual sales. According to the pharmaceutical market research institution IQVIA, only Entresto (₩21.7 billion) and Afinitor (₩14.9 billion) recorded over ₩10 billion in sales among the 8 Novartis products. The other products, Exjade (₩8.5 billion), Galvus (₩7.3 billion), Certican (₩6.9 billion), Xoterna (₩6.2 billion), Pazeo (₩4.1 billion), and Simbrinza (₩2 billion) all recorded less than ₩10 billion in annual sales. The analysis is that generic companies are now actively seeking patents not only for the well-sold large items but also for small and medium-sized sales items. Five or fewer generic companies have requested a judgment for each of the products that sold less than ₩10 billion. Also, Novartis targeted the most is because the company has that many patents. As of the 12th, Novartis has 151 patents (on 69 drugs) listed on Korea’s patent list. This is the most amount of patents registered among domestic and multinational pharmaceutical companies combined. In other words, despite the aggressive patent challenges filed by generic companies over the past 5 and a half years, Novartis has kept over 80% of its patents safe and sound. ◆Followed by Pfizer with 106 patent cases > Boehringer Ingelheim with 95 cases > Astellas with 89 cases The runner-up was Pfizer. Pfizer received the second-most amount of patent challenges after Novartis since 2016. A total of 106 patent trials were filed on 8 patents of 6 drugs. Pfizer's sedative ‘Precedex,’ the pneumococcal vaccine ‘Prevenar 13,’ smoking-cessation treatment ‘Champix,’ antidepressant ‘Prestiq,’ JAK inhibitor immunology drug ‘Xeljanz,’ and neuropathic pain treatment ‘Lyrica’ were the targets. The 4 products other than Precedex and Pristiq were blockbusters that raised over ₩20 billion in sales last year. Also, Boehringer Ingelheim (95 cases), Astellas (89 cases), and AstraZeneca (80 cases) received patent challenges from generic companies. Boehringer Ingelheim received patent challenge for its SGLT-2 inhibitor diabetes treatment ‘Jardiance,’ NOAC ‘Pradaxa,’ and Parkinson’s disease treatment ‘Mirapex.’ Astellas was challenged for its patents on its OAB treatment ‘Betmiga' and immunosuppressant ‘Advagraf.’ AstraZenca’s SGLT-2 inhibitor ‘Forxiga’ also was a target of focus for patent challenges. Among domestic companies, United Pharma, Alvogen Korea, Boryung Pharmaceuticals, Dong-A ST, and Chong Kun Dang were also actively targeted for their patents. In the case of United Pharma, 4 patents on its gastrointestinal symptom treatment ‘Gastiin CR,’ antiplatelet drug ‘Cilostan CR,’ and expectorant ‘Levotics CR,’ were targeted by generic companies. A total of 28 pharmaceutical companies had filed 80 patent trials. Alvogen Korea was challenged by 50 pharmaceutical companies for one single drug - its antithrombotic drug ‘Sarpodipil SR.’ Also, 44 generic companies challenged Boryung Pharmaceuticals 'patent on its antihypertensive combination drug ‘Dukarb.’ Dong-A ST also was challenged by 31 generic companies on its gastritis drug ‘Stillen 2X.’ Chong Kun Dang was challenged for the patent of two of its drugs – its GERD treatment ‘Eso Duo’ and its antihypertensive combination ‘Telminuvo.’ The patents held by domestic companies that the by generic companies targeted are all Incrementally Modified Drugs (IMDs). In other words, the drugs do not separately hold a substance patent and are therefore relatively easy to target. The analysis is that the patent challenges focused on IMDs as most were well-sold products that brought over ₩10 billion a year.
- Policy
- Kymriah's benefit is urgently needed
- by Lee, Jeong-Hwan Jul 15, 2021 07:07pm
- Leukemia patients have urged rapid health insurance registration of the first end-stage leukemia-Lymphoma CAR-T treatment, Kymriah. Korea Leukmia patients organization asked the government to register Kymriah's benefit and pharmaceutical companies to come up with reasonable financial burdens to strengthen access to medicines for leukemia patients. In a statement, the Korea Leukemia patients organization repeatedly called on the government to push for rapid health insurance benefits, including the introduction of Kymriah, at the fifth Cancer Drugs Benefit Appraisal Committee to be held on the 14th. The organization called for a rapid health insurance registration system for new drugs directly linked to life, such as Kymriah. It also added that Novartis Korea should come up with reasonable financial sharing measures to prevent the recurrence of some immuno-cancer drugs, which have delayed health insurance benefits due to controversy over high-dose financial sharing. "Even if Kymriah is passed at the Cancer Drugs Benefit Appraisal Committee on the 14th, the health insurance benefit will not be completed until November this year at the earliest because the NHIS' negotiations and health insurance policy committee procedures are required," said the Korea Leukemia patients organization. It said, "Most patients with recurrent or end-stage acute lymphocytic leukemia or lymphoma die within three to six months. If Kymriah is covered by health insurance in November, many of the 200 patients expected to be treated annually." It said, "It is a pity that patients who cannot afford to pay about 460 million won (non-reimbursed medical expenses disclosed on Samsung Medical Center's website) have to die while waiting for health insurance to be applied." "The government should complete health care benefits within six to seven months, just like Leclaza, the late-stage lung cancer treatment," the organization said. The organization said, "In the case of Kymriah, the scope of 'new drugs directly related to life' should be determined through social discussions, and health insurance should be applied first by the HIRA." The organization also said that "the HIRA's Cancer Drugs Benefit Advisory Committee, the NHIS' Pharmacological Benefit Advice Committee, and the Health Insurance Policy Committee will take the lead." "Glibec, the world's first targeted treatment for chronic myeloid leukemia, was approved by the MFDS on June 20, 40 days after the FDA approved it in May 2001. "In November, five months after the approval, the MOHW announced health insurance benefits. Twenty years later, the Kymriah process should not be delayed than Glivec. "Since August 30, 2017, when Kymria was approved by the U.S. FDA, the issue of high-priced drug prices has been raised."
- Company
- Xospata is approved as a targeted anti-cancer drug
- by Eo, Yun-Ho Jul 15, 2021 07:06pm
- The emergence of FLT3 targeted anticancer drugs is also causing new changes in the area of acute myeloid leukemia, which lacked treatment options. Chronic Myeloid Leukemia (CML), which was considered incurable, has benefited many patients from the commercialization of the targeted anti-cancer drug Glivec(Imatinib), but Acute Myeloid Leukemia (AML) has relied on next-generation chemotherapy, despite being a life-threatening serious condition. Astellas' FLT3 target anti-cancer drug "Xospata ( Gilteritinib fumarate)" has been approved by the MFDS as the first FLT3mut+ recurrence or nonresponsive AML target treatment. AMLs are identified by individual chromosomes or genetic mutations. Guidelines published by the U.S. NCCN and the European ELN classify patients with FLT3-ITD mutations or TP53 mutations as the highest risk group. Xospata is a drug that targets both FLT3-ITD, FLT3-TKD, and FLT3-TKD mutations, which can be treated at home without frequent visits to hospitals. It also showed higher effectiveness and safety compared to conventional chemotherapy. It obtained permission from the U.S. FDA, Ministry of Health, Labor and Welfare of Japan in 2018 and the European EMA in 2019, and was also classified as Category 1, the highest recommended class for treating relapsing or nonresponsive AML patients with FLT3 mutations. It has recently been approved by Asian countries such as China and Singapore. Since its approval in March last year, it has passed the DC of major hospitals in Korea, including Seoul National University Hospital, Seoul St. Mary's Hospital, and Samsung Medical Center, and is under review at other general hospitals. It passed the HIRA's Cancer Drugs Benefit Appraisal Committee in February. Medical staff are also looking forward to it. Kim Hee-je, a professor of hematology at Seoul St. Mary's Hospital, said, "With Xospata's permission in Korea, patients can relieve their anxiety." "Of course, there is still a cost problem, but we expect it to become a standardized treatment as soon as possible after the salary is registered." "In FLT3mut+ R/RAML patients, the prognosis is poor and the disease can worsen rapidly, so it is important to provide proper treatment quickly. "That's why Xospata is being prescribed even though it's non-reimbursement." In May, the Korea Alliance of Patients organization delivered its opinion on the rapid benefits of new drugs, including Xospata, at a meeting with the MOHW' insurance and medicine department. "It is very unfortunate that AMLs are rapidly deteriorating and cannot be applied with already licensed treatments," said an official at the Korea Alliance of Patients organization. "We are also making efforts to ensure that the treatment can be used quickly for patients who need the only new medicine."
- Opinion
- The patient's life comes first
- by Lee, Jeong-Hwan Jul 15, 2021 07:06pm
- On March 5, the MFDS approved Kymriah(Tisagenlecleucel), the world's first treatment for CAR-T, as the first advanced bio-medicine under the Advanced Regenerative Bio Act. Targets are patients with B-cell acute lymphocytic leukemia and submicrobial giant B-cell lymphoma under the age of 25 who are recurrent and nonresponsive. Kymriah was already licensed in August 2017 by the U.S. FDA and in March 2019 by the Ministry of Health, Labor and Welfare of Japan, where there was no further treatment due to recurrence or non-compliance, resulting in treatment for late-stage blood cancer patients. Emily Whitehead, who was diagnosed with acute lymphocytic leukemia at the age of five and failed to treat it, has been living in good health since joining the first clinical trial of the CAR-T drug Kymriah in 2012. However, in Korea, there was news of Eun-chan who recently died while preparing for Kymriah treatment. Emily Whitehead's experience is touching around the world, but we are facing sad news that we are not being treated in time. Eun-chan's mother left a wish on her blog, saying, "It will be a lifelong regret that Eun-chan was not able to receive CAR-T treatment, but I hope that other patients in similar cases will not lose their lives like Eun-chan." Kymriah has been given only once in a lifetime, or "one shot," to allow for near-cure treatment and long-term survival. Therefore, if the existing treatment does not work, or if patients with multiple recurrences of blood cancer can rely on it for the last time.In acute lymphocytic leukemia, 82% and lymphoma 39.1% show Complete Response Rate. The effectiveness of treatment above a certain level can be verified. When Kymriah was licensed in Korea, not only were it already licensed in more than 30 countries, but there are also many countries that have health insurance registered, including Japan. What if Kymriah's domestic approval had progressed a little faster? In Korea, it would have been a medical environment where CAR-T treatment could be done as quickly as in Japan, and health insurance coverage would have been carried out as quickly as in Japan. If that were the case, Eun-chan might have been able to receive Kymriah treatment by now. A sad situation like Eun-chan should not be allowed to happen again. Korea also needs a system that can quickly register new drugs directly related to life without alternatives and has more than a certain level of therapeutic effect like Kymriah. The scope of the "new drug directly related to life" should be set through social discussions, and the MFDS should set a "temporary drug price" at the same time as the approval and apply health insurance to save the patient's life first The HIRA's Cancer Drugs Benefit Advisory Committee and Pharmacological Benefit Advice Committee, the NHIS negotiations, will be carried out quickly to ensure that the "final drug price" is finalized. Afterwards, the government shall actively engage in the introduction of a new drug health insurance rapid registration system directly related to life by post-settlement. The reason why pharmaceutical companies developed new drugs is to save people's lives, and the reason why the government creates and operates a health insurance system is to prevent patients from losing their lives because they have no treatment costs. Pharmaceutical companies and governments should put priority on patients' lives over profits and health insurance finances. Novartis Korea should actively cooperate with the government to quickly provide health insurance benefits by preparing a socially acceptable rational CAR-T treatment Kymriah financial sharing plan.
- Company
- Keytruda overcomes its 4-year hurdle, with Tecentriq
- by Eo, Yun-Ho Jul 15, 2021 06:30am
- The immunotherapy ‘Keytruda’ finally overcame one giant hurdle for its reimbursement, with ‘Tecentriq.’ According to industry sources, the PD-1 inhibitors – MSD Korea’s ‘Ketyruda (pembrolizumab) and Roche Korea’s ‘Tecentriq (atezolizumab)’ – passed deliberations by the Health Insurance Review & Assessment Service’s Cancer Drug Review Committee for its first-line indication for non-small cell lung cancer (NSCLC) yesterday (14th). However, differences exist between the criteria under which the two drugs gained the nod. Keytruda’s is for monotherapy and in combination with chemotherapy for patients with a PD-L1 expression rate of 50% or higher, and Tecentriq’s is as monotherapy for those with a PD-L1 expression rate and PD-L1 stained tumor-infiltrating immune cells (IC) covering 10% or more of the tumor area. Regardless of the specifics, the introduction of a cancer immunotherapy option in first-line NSCLC is reassuring news. The last move made by MSD Korea, which waited nearly 4 years for reimbursement of Keytruda in NSCLC, seems to have finally paid off. However, Keytruda’s nod comes with a condition attached. The Cancer Drug Review Committee again mentioned the equity of Keytruda and Tecentriq and requested additional modifications to be made to Keytruda's cost-sharing plan. This indicates that the drug pricing negotiations with the Pharmaceutical Benefits Appraisal Committee or the National Health Insurance Service will act as a variable in discussing Keytruda's benefit expansion to the lung cancer indication. Keytruda’s benefit expansion has been discussed for almost 4 years since September 2017. Among the many barriers, the biggest unresolved issue was the condition presented by the government to all pharmaceutical companies with cancer immunotherapies – requesting the company to ‘cover the initial 3 cycles’ worth of administration cost’ Roche, which owned the then-latecomer ‘Tecentriq,’ was the only company to accept the government’s proposal then, and 2 types of PD-1 inhibitors – Keytruda and ‘Opdivo(nivolumab)’ were unable to accept the offer. Since then, MSD had repeatedly proposed compromises and revisions to the government. The last discussion was held in August last year, during which the decision for the drug was put on hold as the committee believed that MSD Korea’s proposal lacked compromise on the company’s part. In September of the same year, HIRA handed the proposal back to MSD Korea and requested a re-revision. A month later, MSD submitted a re-revised proposal, which the reimbursement standard sub-committee meeting discussed but to no avail. The agenda was push back and not deliberated at the Cancer Drug Review Committee meeting. This time, with MSD Korea’s new managing director Kevin Peters personally attending to persuade the government, finally, the long-awaited “YES” came from the Cancer Drug Review Committee. Meanwhile, Keytruda was first listed for insurance benefits in August 2017 through the mixed refund and expenditure cap type of the Risk Sharing Agreement (RSA) with a PD-L1 expression rate standard.
- Company
- Will Rivoceranib+Iressa really be a game changer ?
- by Jul 14, 2021 06:26pm
- Can Rivoceranib+Iressa therapy really be a game changer? This is the title of the press release distributed by HLB on the 9th. "We have confirmed high synergy with the first-generation blockbuster EGFR TKI drug in phase 3 clinical trials," HLB said. "We expect a next-generation treatment due to a complete improvement in one person and PFS." Rivoceranib (Aptinib) is a targeted anticancer drug targeting endothelial cell growth factor receptor 2 (VEGFR-2), which plays an important role in the tumor development process. It is a plan that combines this with Iressa (Gefitinib), a first-generation EGFR target anti-cancer drug, to create synergy in treatment. The combination of the two drugs is positive. In fact, other VEGFR2 inhibitors "Cyramza" and first-generation EGFR TKI "Tarceva" combined therapy were approved as the first treatment for EGFR mutation non-small cell lung cancer in the U.S. last year. Let's take a look at the phase III results recently published by developer Jiangsu Hengrui Medicine in the Journal of Thoracic Oncology, a journal of the World Lung Cancer Society. This study compares Rivoceranib and Iressa combinations with Iressa sole therapy (Iressa+placebo) in patients with EGFR-positive non-small cell lung cancer who have not received existing chemotherapy. EGFR Exxon 19 defect or Exxon 21 L848R variant were deployed one-on-one in both counties. The primary validity indicators are PFSs evaluated by the IRRC, which consists of radiologists, and secondary validity indicators include clinical evaluation PFSs, overall survival periods (OS), and quality of life (QoL). In the study registered by 313 people, the primary variable, the median progressive survival period (mPFS), was approximately 3.5 months longer than 13.7 months for the combined group and 10.2 months for the single group (HR 0.71, p=0.0189). The 12-month PFS was 53.4% to 35.6%. OS data is immature at the time of analysis. CR was observed in one person. Grade 3 or higher adverse reactions were higher in the Rivoceranib group, with high blood pressure (46.5%), proteinuria (17.8%), and higher serum ALT in the control group (10.3%), and higher AST (3.2%), but there were no statistical changes in the quality of life between the two groups. It is true that the combination of Rivoceranib and Iressa therapy in phase 3 demonstrates the significance of effectiveness over Iressa alone therapy. However, considering the results of Tagrisso (Osimertinib), a third-generation targeted drug, which is currently a global standard for treating EGFR-positive non-small cell lung cancer, or Cyramza+Tarceva, the same mechanism, the results are somewhat insufficient to become a "game changer." Although clinical designs and patients are different, let's refer to the results of FLAURA study, which served as the basis for Tagrisso's acquisition of primary indications. Tagrisso compared Iressa and Tarceva, the first-generation drugs at the time, to patients with EGFR-positive non-small cell lung cancer who have no experience in chemotherapy. mPFS had 18.9 months for Tagrisso and 10.2 months for control (HR 0.46 and p
- Company
- Bavencio can be prescribed in general hospitals
- by Eo, Yun-Ho Jul 14, 2021 06:03am
- Merck and Pfizer’s immunotherapy ‘Bavencio’ can now be prescribed at general hospitals. According to industry sources, the PD-L1 inhibitor Bavencio (avelumab) passed the Drug Committees (DCs) of the Big-5s general hospitals in Korea - Seoul National University Hospital (SNUH), Asan Medical Center (AMC), Seoul St. Mary’s Hospital, Samsung Medical Center (SMC), and Severance Hospital – as well as other major medical institutions in the nation including Inje University Seoul Paik Hospital, Ajou University Hospital, Ewha Womens University Hospital, Wonkwang University Hospital, Wonju Severance Christian Hospital, Chosun University Hospital, and Inje University Haeundae Paik Hospital. Bavencio, the 6th cancer immunotherapy and the 3rd PD-L1 inhibitor to be approved after Tecentriq (atezolizumab) and Imfinzi (durvalumab), was first approved in Korea in March 2019 to treat the rare condition, Merkel cell carcinoma (MCC), then listed for insurance benefit under the RSA (Risk Sharing Agreement) scheme for the same indication in September last year. In Part A of the EMR100070-003 study that was conducted on patients with MCC whose disease had progressed on or after chemotherapy, the objective response rate (ORR) of those treated with Bavencio inj. as monotherapy was 33.0%. 11% of patients experienced a complete response and 22% of patients experienced a partial response. At the time of analysis, tumor responses were durable, with 93% of responses lasting at least 6 months and 71% of responses lasting at least 12 months. Also, interim analysis results in Part B of the study showed that the ORR in this patient group was 39.7%, with 13.8% of patients experiencing a complete response and 25.9% experiencing a partial response However, the company has been facing difficulties in expanding Bavencio's indication. Since December last year, the benefit approval is being delayed due to negative evaluations made on the efficacy of Bavencio for renal cell carcinoma by the Central Pharmaceutical Affairs Council. The council pointed to the overall survival (OS) data of the drug as the key cause. Also, recently, Bavencio’s indication as a first-line maintenance therapy for urothelial cancer that has been approved by the U.S. FDA was rejected in the U.K by NICE. NICE recognized the drug’s value in addressing the unmet need that exists in the population up for review but raised the question on its cost-effectiveness.
- Company
- Will a new reimbursement model be introduced for Zolgensma?
- by Jul 14, 2021 06:03am
- The move to list Novartis’s new drug ‘Zolgensma (onasemnogene abeparvovec-xioi)’ for reimbursement benefits is now underway. The industry’s eyes are on which types of the RSA scheme will be combined for this ultra-high-priced ‘dream drug.’ Whether Zolgensma will be listed for insurance benefits is the focus of attention in the industry. The industry-wide interest was approved in May in Korea as a single shot of Zolgensma costs over 2 billion won. Of course, when compared to other existing drugs that require spending 300 to 500 million won every year for the rest of one's life, this ‘one-shot’ treatment cannot strictly be called an ‘ultra extensive' drug. Nevertheless, it is true that the government burden would be immense, as it would have to bear billions of won as its cost each time. The reasoning for Zolgensma’s high price is in that it can treat rare diseases that cause death with just a single injection. Zolgensma treats spinal muscular atrophy (SMA), a rare condition in which the SMN1 gene is innately deficient or mutated to result in progressive muscle atrophy. In the case of SMA Type 1, the most common and severe form of SMA, over 95% of the motor neurons are damaged within 6 months, and 90% die before the age of 2 if left untreated. Zolgensma is a gene therapy that has a functional replacement for the missing or non-working SMN1 gene. When the drug is administered to a patient, the replacement delivered with Zolgensma produces SMN proteins and radically cures the disease. This completely different mechanism of action, unlike those of existing drugs that increase the production of SMN protein using the ‘backup’ SMN2 gene, is why Novartis calls Zolgensma an ‘innovation'. In the Phase III SPR1NT study, all pediatric SMA patients with two SMN Type 2 gene copies (Cohort 1) that were treated prior to the onset of symptoms survived without requiring ventilatory or nutritional assistance and achieved sitting independently for 30 seconds or more. Most (11/14) patients achieved age-appropriate motor milestones within the World Health Organization (WHO) window of normal development. Also, in the real world, Zolgensma improved or maintained motor development scores in patients 6 months of age and older, regardless of their prior use of existing therapies. The efficacy and safety of Zolgensam were confirmed for over 6 years, with treatment experience accumulated for patients aged 6 months to 2 years. Novartis applied for Zolgensma's reimbursement benefit in June and is currently discussing ways to allow its reimbursement. The key agenda lies in how to incorporate Zolgensma in the current reimbursement system. As of now, the RSA scheme is being considered the most realistic alternative, and among the various types of RSA, it is likely that a mix of several models would be used to cover the drug. The company has proposed a performance-based RSA system. Miri Shin, Director of Zolgensma's Business Unit at Novartis Korea said, “We have reviewed various measures that can effectively reduce the burden on NHI finances and proposed the performance-based payment system to the government.” The performance-based payment system is an RSA conditional upon evidence development. Under this RSA type, reimbursement is decided according to the results of a clinical trial that is conducted after listing. The only drug listed with the RSA conditional upon evidence development was ‘Evoltra,’ a treatment for acute lymphoblastic leukemia. If this performance-based RSA is applied for Zolgensma, setting the criteria for reevaluation will be important. Also, new types such as installment payments can be incorporated into the system, under which the price of the single shot would be paid in installments. The U.S. has also adopted this method to minimize its financial burden. In addition, finance-based RSA types such as the expenditure cap type may also be applied to Zolgensma. “We are flexibly discussing various measures for Zolgensma’s reimbursement with the government," said Director Shin. “We will continue discussions to work out the details.”
