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- 2026-05-21 00:43:42
- Opinion
- [Column] Legal disputes over rebate penalty reducing price
- by Lee, Hye-Kyung Dec 05, 2019 06:12am
- This year would be a year to remember as various issues regarding drug pricing broke out, such as ‘lump-sum price reduction on single-use eye drops’, ‘revised generic pricing system’, and ‘litigation against pricing reduction penalty for providing rebate’. Currently the drug pricing system is geared towards paradigm shift, starting with abolished ‘same substance same pricing’ policy. Pharmaceutical companies would be helpless but to seek for other survival tactics in the coming year while the drug pricing ecosystem changes. There are many issues to be talked about regarding drug pricing, but today it would be about a few updates on meaningful court decisions made on pricing reduction as an illegal rebate penalty. However, it would mainly be summarized points of the issues as the lower court made the decisions and the issues are still open for long-running disputes. As I introduced in a column titled ‘Rebate and Kick-back’ published December last year, the Korean Ministry of Health and Welfare (MOHW) imposed maximum reimbursement price reduction on 340 items from 11 pharmaceutical companies accused of providing rebate. Currently, the most of affected pharmaceutical companies have filed administrative litigation against the matter. The pricing reduction penalty has not been imposed for years and related legal dispute has not been talked, either. So the recent legal disputes were raised since various issues occurred with MOHW imposing penalty of the massive scale. Going through each dispute issue, the first issue is about whether to consider nature or property of drug pricing reduction penalty as a sanction or not, according to the Item 12 of Paragraph 4 of Article 13 of Regulation for Criteria for Providing Reimbursed Services in the National Health Insurance, stating “a drug that has been confirmed as having disturbed trade orders by offering money or good for sales promotion, etc”, or also known as former rebate regulation. The distinction of the sanction is crucial, because the ministry’s jurisdiction could change depending on the recognition of the discretionary sanction. In other words, when the court recognizes the penalty as discretionary sanction, the judiciary would then decide the penalty was legitimate respecting the administrative agency’s judgment, if without a significant flaw, but if not then the court could revisit the issue. On the issue, the lower court decided drug pricing reduction penalty imposed based on the former rebate regulation ‘could not be seen as sanction, but rather the maximum reimbursement price adjustment should be judged as discretion of reasonable penalty.’ Therefore, the court meant that it would be considered as a legitimate penalty within the discretionary jurisdiction only when the jurisdiction is considered reasonable. With the said premise, the court ruling made decision on jurisdiction of discretionary authority for each specific disputed issue. First, the court ruled that the Minister of Health and Welfare was not obligated to lay down detailed basis of maximum reimbursement price calculation to affected companies, when imposing the price reduction penalty. The court did not see the legitimate reason as for the minister to consider the company as direct subject, because the regulation defines subjects for notice on reimbursed drug are mutually applied among healthcare institute, National Health Insurance Service (NHIS), policyholder, and dependent. Among drugs provided from Pharmaceutical Company B to Hospital A, should the price be reduced only for drugs prescribed by the rebate-received medical profession? Or should maximum reimbursement prices of all drugs supplied by Company B and prescribed by Hospital A be reduced? The court stated all drugs from Company B could be subject for the maximum price reduction. Rebate provision itself is highly likely to have been provided to promote sales of a specific company’s product, and there was no objective evidence to prove the rebate was provided for a specific product instead. So the court decided the Ministry of Health and Welfare’s penalty was within its jurisdiction of discretionary authority. Then what about a case of Hospital A providing both reimbursed and non-reimbursed drugs. How should the maximum reimbursement price reduction rate be calculated? Should the rebate on non-reimbursed drug be disregarded from the calculation of price reduction rate? The court decided proportionally dividing rebate amount on reimbursed drug, while completely disregarding non-reimbursed drug, was a faulty calculation of maximum reimbursement price reduction rate. The calculation formula for the price reduction rate was wrongful as rebate could have been provided for the non-reimbursed drug, and removing the amount provided to non-reimbursed drug from the calculation would have resulted in excessive reduction rate. Lastly, if the rebate provided to a pharmacist was for the cost of the provider’s prescription drug, would it be possible to reduce the maximum price including the rebate cost? Besides from violating Pharmaceutical Affairs Act, the court saw that the company’s act of providing rebate is difficult to relate back to prescription and sales of the prescription drug. The principle and the norm of dispensing and sales of prescription drug is decided by doctor’s prescription, so the court judged it is unlikely to see the correlation between rebate provided to pharmacist and ‘promotion of dispensing and sales of prescription drug’, except for a special occasion. Therefore, the court stated reduction rate should be calculated without the rebate cost provided to the pharmacist. As for the last decision, the court reviewed standard and process of imposing maximum reimbursement price adjustment penalty more specifically than other previous rebate decisions, which sets judging standard to see if the maximum reimbursement price reduction penalty was reasonable based on the ministry’s discretionary authority. The decisions were made during respective first trials and they are waiting for the appeal. Attention on the issues is heightened to see if the preceding decisions would be sustained in the appeal. In fact, there is a possibility of the change in decision during the appeal, and whichever decision is made at the Supreme Court later, the cases would definitely be the precedents setting a standard of the rebate-induced drug pricing reduction penalty. The heated legal disputes seem inevitable for the healthcare sector, as it is Korea’s new economic growth engine with visible rapid expansion in quality and quantity. Besides, the highly political and technical drug pricing is right in the center of the dispute. Previously mentioned drug pricing paradigm shift seems like it would bring more interesting topics on the table than just the rebate case. Surely the drug pricing policy would attract even more attention in the coming year 2020.
- Company
- Ildong exclusively sells 9 kinds of GSK generic drugs
- by Chon, Seung-Hyun Dec 04, 2019 06:41am
- Ildong Pharmaceuticals announced on the 2nd that it has signed 9 co-promotional contracts with GlaxoSmithKline Consumer Healthcare Korea, including general medicines. With this agreement, Ildong Pharmaceuticals is a comprehensive cold medicine ‘ theraflu’, an ophthalmic drug ‘Otrivin’, a non-smoking supplement ‘Nicotinell’, a hyperhidrosis cure, ‘Driclor soln’, an external anti-inflammatory analgesic ‘ Voltaren’, a denture attachment ‘Polident’, toothpaste ‘Sensodyne’, ‘Paradontax’, and band medical expander,‘Breathlite’. Last year, domestic sales of nine products stood at about ₩46 billion. It is equivalent to 9% of last year's consolidated revenue. Ildong Pharmaceuticals will be in charge of distribution, sales and marketing of co-promotional products to the pharmacy market from next year. GSK Consumer Healthcare Korea will support brand marketing and customer service related tasks. Dong-wha Pharmaceticals was in charge of distribution and sales of products that Ildong Pharmaceutical decided to sell. Dong-wha Pharmaceutials entered into a copyright agreement with GSK until 2020, but due to the merger of GSK and Pfizer Healthcare, the company was terminated and decided to terminate the contract by the end of this year. Ildong Pharmaceutical anticipated that, starting next year, the company will be able to achieve annual sales of more than ₩200 billion only through the OTC pharmaceutical business. Ildong Pharmaceuticals is known to have differentiated competitiveness in the field of OTC and consumer healthcare by possessing a number of well-known brands such as Aronamin, the No.1 best selling drug in Korea, and specialized sales and marketing organizations and human resources. The company explained that the company's online drug store 'Ildong Shop' was able to increase profitability as well as sales through efficient distribution and inventory management. A representative of Ildong Pharmaceutical said, “We have expanded our cooperation with GSK Consumer Healthcare Korea and OTC / Consumer Healthcare after co-promotion of flu medicine, Relenza with GSK Korea. We will continue to look for partnerships that will allow us to work together and grow together in the medium to long term”.
- Company
- Narcolepsy treatment, Nuvigil settled down in Big 5 hospital
- by Eo, Yun-Ho Dec 04, 2019 06:41am
- Narcolepsy treatment, Nuvigil are facing the entry in Big 5 hospitals. According to the industry, Handokteva's Nuvigil (Amordafinil) has passed the SNUH, Severance Hospital, SMC, and ASAN Medical Center's drug commitee (DC). In addition, prescription code was generated at St. Vincent's Hospital of Catholic University. The system of St. Mary's Hospitals is centralized, and must be approved by the Seoul St. Mary's Hospital before the code is applied to the entire St. Mary's Hospital. It has entered into an unusual case. Nuvigil was released last September in the health insurance reimbursement list in June last year for the treatment of hypersomnia related to adult narcolepsy. Narcolepsy is a symptom controlled through drug treatment. Currently, there are few treatments, so the choice is very limited. Nuvigil is an R-isomer of Amordafinil, which is currently used as a treatment for narcolepsy, and is characterized by an improved drug convenience by improving the duration of drug efficacy. Nuvigil, the active isomer of sleep seizure treatment Provigil (Modafinil), is a blockbuster drug with sales of about $80 million (about ₩94.8 billion). Teva acquired Nuvigil in 2011 by taking over Cephalon inc, the original developer. In Korea, it was approved as an indication for 'excessive drowsiness related to narcolepsy'. Nuvigil is prescribed for sleep attacks, obstructive sleep apnea, and shift work sleep disorder. In particular, a study comparing Nuvigil and placebo in 245 patients with shift work sleep disorder was noted. In this study, the proportion of patients whose sleep latency was more than 5 minutes, measured by multiple sleep latency tests, was 38% in the Nuvigil’s group, 17% above placebo. An official of the Sleep Society said, "Nuvigil is a drug that improves waking conditions without interfering with the sleeping state. Although there is a late entry into Korea, it is expected to be used in various ways".
- Company
- 8 multinational companies name new CEOs in Korea
- by Eo, Yun-Ho Dec 03, 2019 03:21pm
- From top left; Vice-chair Lee Youngshin, CEO Moon Hee-seok, CEO Shin Jung-beom, President Alberto Riva, CEO Kang So Young, CEO Lee Hye-young, CEO Kim Jinyoung, and CEO Kang Sangwook Sources report eight global pharmaceutical companies have appointed new CEOs of Korean affiliates this year only. Daily Pharm surveyed a roster of major 30 multinational companies’ Korean affiliate CEOs as of Dec. 4, and found about 28 percent of the companies have either replaced or newly appointed the top executive personnel. While several companies had a regular personnel reshuffle, others had a new personnel this year for specific reasons like merger, corporate split, and disciplinary dismissal. Roster of Korean affiliate CEOs of multinational pharmaceutical companies (as of Dec. 2019) First, Takeda Pharmaceutical and Bristol-Myers Squibb completed their acquisitions of Shire and Celgene, respectively, and welcomed new CEOs to their merged organizations. As for Takeda Pharmaceutical Korea, when the former CEO Mahender Nayak left the position it was vacant until the former CEO of merged company Shire Korea, Moon Hee-seok took over. With such development, the pharmaceutical industry had their eyes on the recently merged Bristol-Myers Squibb (BMS) and Celgene Korea’s CEO. BMS ultimately chose a promotion from within. BMS also had a vacant CEO position for a while as the former CEO Park Hye-sun left suddenly before her term ended in May. And the former CEO of Celgene, Ham Tae-jin was re-elected for another term. Although the BMS headquarters interviewed number of major candidates from Korean pharmaceutical industry, including CEO Ham Tae-jin, it appointed then acting CEO and former head of Legal and Compliance division of BMS Korea as an official CEO of the merged company. Some top executives, who served their company for a long term, left the position. A former general manager of Abbvie Korea, Yoo Hong-Ki had his retirement ceremony in last March after leading the company for over a decade, even before Abbvie was split from Abbott. As he left the company, he appointed former vice-president Kang So Young as a new general manager of the Korean branch. Menarini Korea also saw the former president Albert Kim leave last year, who served the company since before Invida was acquired by Menarini. The position was replaced by Park Hyeyoung. Pfizer Upjohn Korea, established with the recent Pfizer split, appointed Lee Hye-young as the first head of the company. Before she was appointed as a CEO, Lee used to lead Pfizer Korea’s off-patent product division. In addition, Lilly Korea appointed Alberto Riva, LEO Pharma Korea appointed former Roche personnel Shin Jung-beom, and GSK Consumer Healthcare invited former L'Oréal personnel Kang Sangwook as the new heads of their Korean affiliates. The Korea Research-based Pharmaceutical Industry Association (KRPIA) also welcomed a new CEO, Youngshin Lee. Meanwhile, this year’s ratio of Korean personnel, as a CEO of Korean affiliate of multinational pharmaceutical company, soared to 66 percent from 50 percent last year. And overall female CEO ratio in the multinational companies reached 30 percent.
- Policy
- Opdivo label changes but reimbursed scope unchanged
- by Lee, Hye-Kyung Dec 03, 2019 05:55am
- Ono Pharmaceutical’s immunotherapy Opdivo’s (nivolumab) 240 mg dose secured an approval from the regulator and expanded scope of administration on the label, but apparently the treatment’s reimbursed level of dose would remain unchanged. Health Insurance Review and Assessment Service (HIRA) recently collected public comments on the revised ‘notice on medicine prescribed and used for cancer patients.’ The agency then clarified the insurance reimbursement would only be granted for Opdivo used to treat patients with non-small cell lung cancer or melanoma with dosing schedule of 3 mg/ kg every two weeks. It would be in effect from Dec. 9. After Ministry of Food and Drug Safety (MFDS) approved of Opdivo 240 mg in April, the immunotherapy received expanded approval on dosing schedule of ‘240 mg for every two weeks or 480 mg for every four weeks’ and added it to the label of Opdivo 20 mg, 100 mg and 240 mg, on Nov. 7. However, HIRA decided not to approve of reimbursement on the new dosing schedule as Opdivo’s clinical result did not demonstrate meaningful differences between several different doses. The agency explains because Opdivo 240 mg product is not listed for reimbursement, the cost-effectiveness of additional higher dose and administration is uncertain. “HIRA has decided to grant insurance reimbursement only for using the immunotherapy under the initial dosing schedule of ‘3 mg/ kg every two weeks’, as stated on the MFDS-approved label”, HIRA official said.
- Policy
- Hyperalgesia possibility if Fentanyl mucosa not control pain
- by Lee, Tak-Sun Dec 03, 2019 05:54am
- 대표적 펜타닐 점막투여제 Fentanyl mucosal drug, a typical narcotic analgesic used for the treatment of cancer patients, may not be controlled by hyperalgesia and resistance, will be reflected in the permit. This was done by the Agency by reviewing safety information from the European Medicines Agency (EMA). The permission change adds that fentanyl mucosal medications may cause hyperalgesia if pain is not controlled, and that dose reduction and discontinuation may be considered. In particular, new content is added to the usage and dosage located in the upper line of the permit. The additional phrase is, "If pain is not properly controlled, there is a possibility of hyperalgesia, tolerance and underlying disease progression, which should be taken into account." Hyperalgesia is an abnormally sensitive condition for a painful stimulus. In addition to the general precautions, "Optimal drug-induced hyperalgesia should be considered when there is a lack of pain control compared to increasing fentanyl doses, as with other opioids. Fentanyl dose reduction or discontinuation may be considered." Adverse events such as drug abuse and Synching Withdrawal Syndrome from postmarketing experience are also added. The MFDS asked for a review by December 12. Fentanyl mucosal drugs approved in Korea are 26 items in six companies, and the items are Narco Sublingual Tab. 200μg of BC World Pharmaceuticals, Actiq oral transmucosal tab of Hyundai Pharmaceuticals, Abstral sublingual tab of Menarini Korea, Daewoong Pharmaceutical's 'Instanyl Nasal Spray', Pharmbo’s Fentakhan sublingual tablet, Teve-handok’s Fentora Buccal tab. Of these, Fentora's sales amounted to ₩6.8 billion in 2018 and Abstral recorded ₩5.9 billion based on IQVIA.
- Company
- Counterattack of Valsartan’s Pharmaceuticals
- by Chon, Seung-Hyun Dec 03, 2019 05:54am
- Pharmaceuticals filed an alleged class action lawsuit against health authorities. A lawsuit was filed preemptively that the government can not accept the Valsartan claim. Cost liability for follow-up of impurity drugs has been determined in court. According to the industry, 36 pharmaceutical companies recently filed a lawsuit to confirm the absence of debt against the National Health Insurance Service in the Seoul Central District Court. It is a lawsuit stating that it is not responsible for the valsartan damages claimed by the National Health Insurance Service. The NHIS asked to pay a donation of ₩2.03 billion to 69 pharmaceutical companies in last October. It is a follow-up to the Ministry of Health and Welfare's decision to return the amount of money invested in pharmacies after exchanging impurity valsartan for the remainder of their prescriptions last year. It is unprecedented that pharmaceutical companies filed a lawsuit against the government in a group. In 2012, the government made a move to file a lawsuit against the collective price cuts of health authorities, but did not lead to a legal workshop. In 2013, the NHIS and pharmaceutical companies filed a lawsuit for damages related to drug substance preferential treatment. However, at this time, the NHIS filed a lawsuit. Originally, pharmaceutical companies considered the joint response to the NHIS 'lawsuit. However, they agreed to take a hard-line response by preemptively bringing up class action. The number of companies participating in litigation is also on the rise. Pharmaceutical companies were the first to discuss whether they would co-operate for the first time immediately after receiving a bill from the NHIS in early October, at that time, 20 companies showed a positive position. When it decided to file a lawsuit last month, it increased to 35 companies, and it was reported that one large pharmaceutical company was willing to join an additional company immediately after submitting the complaint. Most companies with large damages have refused to pay. According to the data submitted to the Democratic Party's member, Nam In-sun, 26 pharmaceutical companies paid ₩440 million in compensation. The payment rate was only 21.5%. The pharmaceuticals refused to pay about 80% of the recourse amount. Pharmaceutical companies are claiming no responsibility for the government-claimed Valsartan damages. The NHIS has proposed a product liability law on the grounds of valsartan compensation. It is judged that there is a defect in the manufacturer's product and safety, and according to the product liability law, it is possible to claim for damages due to the product defect. It is based on Article 3 of the Product Liability Act, which states that 'manufacturers shall reimburse those who suffer damages to life, body or property due to defects of the product'. Pharmaceutical companies stress that there are no manufacturing and design flaws with impurity valsartan. Carcinogen N-nitrosodimethylamine (NDMA), detected in Valsartan’s issue, is a hazardous substance in the valsartan raw material that has no standard. Neither governments nor pharmaceutical companies were aware of the risk of NDMA detection in Valsartan. According to the Product Liability Act, it is clear that if the manufacturer proves that a defect was not found at the level of science and technology at the time the manufacturer supplied the product, it would be liable for damages. After the valsartan’s issue, the MFDS derived a test method for detecting NDMA from Valsartan raw materials and set new standards. The MFDS set the NDMA standard for Valsartan to 0.3 ppm or less by reviewing the guidelines recommended by the International Pharmaceutical Regulatory Coordination Committee (ICH M7), domestic and international data, and expert advice. Moreover, the hazard of impurity Valsartan was not revealed. The MFDS said, “ in last December, based on individual doses and duration of patients actually taking NHA-detected drugs using Valsartan by Huahai , the possibility of additional cancer was negligibly low“ Pharmaceutical companies argue that the National Health Insurance Service does not include claims for damages. The NHIS has charged Valsartan medical fees and dispensing fees, but pharmaceutical companies are not liable for compensation under the Product Liability Act. An official of a pharmaceutical company said, “We took huge losses from the recovery and disposal of products that are not exposed to human hazards, and it is unfair to pay for represription fee and redispensing costs. Following Valsartan, sales of Ranitidine and Nizatidine were stopped, and we decided we need to weigh the injustices of the government action”.
- Company
- ECCK “Revised Pricing Negotiation Guideline Violates FTA"
- by Kim, Jin-Gu Dec 03, 2019 05:53am
- Chair Julien Samson of ECCK Healthcare Committee (VP& General Manager of GSK Korea, fourth from the right) and other ECCK members presented White Paper 2019 and presented recommendations to Korean government. Europe-based global pharmaceutical companies with offshoots in Korea have filed official complaints on ‘additional requirements under side agreement’ added to drug pricing negotiation procedure in Korea. They claim the new changes could violate the principles of Korea-EU Free Trade Agreement (FTA). European Chamber of Commerce in Korea (ECCK) convened a press conference on Nov. 29 at Four Seasons Hotel Seoul, and addressed issues regarding each industry sector and 180 recommendations to the Korean government. ECCK’s Healthcare Committee (Chair Julien Samson, VP & General Manager of GSK Korea) also contributed 34 recommendations. The highlight of the recommendations was the ‘Revising the New Requirements Imposed through Drug Price Agreement.’ National Health Insurance Service (NHIS) imposed several ‘side agreement’, or ‘additional requirements’ to the pharmaceutical companies during the price negotiation since last year and regulated them in June, 2019. ECCK official warned the additional requirements were “regulated without any public comment period. Some of them are overly strict with little relevance to pricing negotiation. This could violate the principles of Korea-EU FTA”. The committee argues Korea-EU FTA stipulates when revising regulation or guideline regarding pharmaceutical pricing or insurance reimbursement, both government bodies should communicate with industry. “It is recommended to consult with the industry stakeholders on any new requirements or revisions in line with the principles of the Korea-EU FTA”, ECCK officially advised. In fact, the side agreement requirements have become the most talked about issue of recent drug pricing negotiation cases. Lipiodol incident was the critical point triggered the Korean government. The change is interpreted as the government’s effort to prevent any other already-listed item refusing to supply products to Korean market. The new requirements impose penalty on pharmaceutical company not fulfilling obligation to supply drug products, and stipulate the companies to provide compensation for patient when suspending supply. Also the side agreement requires both negotiating parties to keep negotiation details non-disclosed. Biogen’s Spinraza, Janssen’s Darzalex, Amgen’s Prolia and other items that completed pricing negotiation this year had to meet the additional requirements. Although it had the negotiation last year, CJ Healthcare’s K-Cab had to fill out the side agreement as well. And three new drug items exempted from negotiation were denied from the last reimbursement review procedure in April, because they were “missing side agreement”. It was unexpected as reimbursement used to be granted usually without an issue for an item passed by Health Insurance Policy Deliberation Committee (HIPDC). At the end, Whanin Pharm’s Agotin, AstraZeneca’s Faslodex and Takeda’s Alunbrig were successfully listed after compiling side agreement. But the happening reaffirmed the gravity of side agreement in the negotiation procedure. In June, the side agreement submission was regulated as a law. NHIS’ revised drug pricing negotiation guideline now requires pharmaceutical companies to submit an agreement about supply obligation, patient protection measures, and confidentiality when negotiating drug pricing. The Article 9 of Drug Pricing Negotiation Guideline was revised and now imposes new requirements to pharmaceutical companies to protect Korean patient, to provide compensation for patient damage, and keep negotiation confidential. The pharmaceutical industry is mainly complaining about the government omitting the industry comment period. For an instance, a global pharmaceutical company organization, or Korean Research-based Pharma Industry Association (KRPIA), submitted an official statement claiming “Imposing of additional requirement or obligation for drug pricing negotiation should come with an administrative notice according to the Administrative Procedure Act”. The organization also reprehended the new requirements are considered as ‘side agreement’, but it actually puts NHIS on an authoritative position for it to unilaterally impose obligations to pharmaceutical companies. The ECCK’s complaint is basically an extension of the criticism. Established in 2012, ECCK has headquarters in Europe and it consists of 360 active member companies. Every year the Chamber represents the voice of European businesses by collecting recommendations and relaying them to the Korean government. Last year, the Chamber has presented ECCK White Paper 2018 with 123 recommendations, and almost 40 percent of the recommendations received positive feedbacks from the Korean Ministry of Trade, Industry and Energy (MOTIE).
- Policy
- HIRA rejects reimbursed use of Spinraza on 14-year-old
- by Lee, Hye-Kyung Dec 03, 2019 05:53am
- Approval on reimbursed use of Spinraza (nusinersen) on a 14-year-old male patient with 5q spinal muscular atrophy (SMA) was denied. The application submitted by the hospital was rejected as it did not clarify if the patient had developed clinical symptoms of SMA before 36 months. On Nov. 29, Health Insurance Review and Assessment (HIRA, President Kim Seung-taek) posted seven cases of Treatment Review and Evaluation Committee deliberation in October. Although Spinraza was listed on the Drug Reimbursement List from last April 8, a healthcare institute planning to use the treatment has to submit a preliminary drug use approval application due to the ultra-expensive maximum reimbursement price reaching 92,359,131 won per 5 ml vial. In last month, 15 preliminary drug use approval applications on Spinraza, including 13 first loading doses and two maintenance doses (application submitted once every four months), were submitted. But the committee disapproved only one case, which involved a 14-year-old boy. Other 11 first loading doses and two maintenance doses were approved, and one first loading dose was approved with conditions. The conditional approval was granted to a five-month-old female infant trying to get detached from ventilator. The committee approved of reimbursed use of Spinraza as long as the patient submits medical profession’s statement on use of ventilator prior to Spinraza administrations and medical record of respiratory function. Before the Spinraza review, the committee reviewed 34 preliminary applications on use of Soliris (eculizumab), including 23 cases of paroxysmal nocturnal hemoglobinuria (PNH) and 11 cases of atypical hemolytic uremic syndrome (aHUS). For the first loading dose, one PNH case was accepted and other one was rejected. Whereas three cases of aHUS patients were accepted and other three were rejected. Use of Soliris on a PNH case denied as the patient’s medical record was missing repetitive abdominal pain-induced hospital administration, but showed a recent increase in use of narcotic analgesic. The patient’s record did not meet scope of reimbursed use of the treatment. An aHUS case was rejected because the patient’s status was not clear cut as stated in the criteria. The patient’s symptom was considered as a secondary thrombotic microangiopahty induced from rheumarthritis and its treatment, and from multiple myeloma and its treatment. And also the patient showed rise of CEA level, delayed prothrombin time and activated partial thromboplastin time (PT/aPTT), and fall of fibrinogen level. The committee also decided the patient cannot expect positive effect from the treatment as the patient needs dialysis for end-stage renal disease. A preliminary approval review on reimbursed ventricular assist device (VAD) treatment was conducted as well. A male patient aged 64 years was registered on the heart implant waiting list with dilated cardiomyopathy (DCM), who was diagnosed with end-stage heart failure by an echocardiography with dobutamine substance and motor function test. The patient did not recover from medication treatment and is dependent on intravenous cardiotonic agent. HIRA approved reimbursement on the patient’s VAD treatment, because the case met the reimbursement standard of ‘indication for VAD implant on end-stage heart failure patient, who has been registered on heart implant waiting list as a makeshift treatment’. Also the patient not showing any contraindication helped the decision. Other details of the Treatment Review and Evaluation Committee’s October meeting can be found on HIRA website (www.hira.or.kr).
- Company
- Drug Committees pass oral Fabry treatment Galafold
- by Eo, Yun-Ho Dec 02, 2019 05:57am
- Galfold (migalastat) The world’s first orally taken Fabry disease treatment ‘Galafold’ is landing its code on tertiary hospital drug list. According to pharmaceutical industry, Handok’s Galafold (migalastat) has been passed by drug committees (DC) in specialized healthcare institutes for rare disease care, like Seoul Asan Medical Center, Ajou University Hospital, and Pusan National University Yangsan Hospital. Other major general hospitals and Big 5 hospitals are also processing the drug code. Galafold is prescribed to patients aged 16 years or older with a confirmed diagnosis of Fabry disease and who have an amenable mutation. The treatment should be taken one capsule every other day. Galafold’s Phase 3 ATTRACT study switched therapy for 57 Fabry disease patients, who had enzyme replacement therapy (ERT) for at least 12 months, with Galafold. The investigational treatment demonstrated statistically equivalent annual change of estimated glomerular filtration rate (eGFR) with 18-month of ERT. While the group who remained on ERT had a decline of left ventricular mass index (LVMi) by -2.0 g/m2 over 18 months, the group who switched from ERT to migalistat had a significant decline in LVMI by -6.6 g/m2 over the same period. Developed by an U.S.-based Amicus Therapeutics, Galafold was approved by the U.S. Food and Drug Administration (FDA) in October, 2018, and it is now available in the U.S., EU, Australian, Canadian, Swiss, Israeli, and Japanese markets. The treatment has been designated as an investigational orphan drug to meet urgent medical needs, and received approval from Korean Ministry of Food and Drug Safety (MFDS) in 2017. Its insurance reimbursement was also granted from last March. Currently, Galafold’s competitive medicine are Sanofi Genzyme’s Fabrazyme (agalsidase beta) and Shire’s Replagal (agalsidase alfa). The two competitors are ERT sharing a similar mechanism in injection formulation. Replagal is injected once every other week into intravenous line with dose of 0.2 mg per kg body weight, whereas Fabrazyme is injected once every other week into intravenous line with dose of 1.0 mg per kg body weight. Fabrazyme is comparatively a high-dose treatment. Another notable difference is that Replagal is produced in a human cell line, and Fabrazyme is produced in Chinese hamster ovary cell line. An official from Korean Society of Medical Genetics and Genomics commented, “Rare disease like Fabry disease has small patient size to begin with, which makes conducting a credible clinical trial quite difficult. Therefore, medical professions are more likely to be loyal to older treatments. However, having more treatment option for the disease is good news”.
