Orphan drug: Development trends and strategies.

This study aimed to identify orphan drug accessibility and impact on pharmaceutical budgets in South Korea by analyzing the status of orphan drug designation, approval, reimbursement, and pharmaceutical expenditure. We analyzed the dataset on orphan drugs designated, approved, and reimbursed from 2007 to 2019 based on long-term real-world data. The designated and approved orphan drugs were 165.

South Korea, products that have already obtained orphan designation in elsewhere can benefit from a partial exemption from dossier submission for orphan designa-tion and market authorization. ConClusions: OD regulation and incentives are similar by nature but differ in magnitude. It is unclear how the profitability criteria are applied in US as multiple orphan products are blockbuster. USA.

Pharmaceutical IP and competition law in South Korea.

South Korea’s National Assembly voted on amendments to the Act on the Management of Narcotic Drugs that would allow non-hallucinogenic dosages of medical marijuana. Patients will be required to apply to the Korea Orphan Drug Center, an organization that allows access to rare medicines.Japanese Ministry of Health, Labour and Welfare grants orphan drug designation for diseases that affect fewer than 50,000 patients in Japan, and for which significant unmet medical need exists MET exon 14 ( MET ex14) skipping alterations and MET amplifications are present in 3-5% of non-small cell lung cancer patients and correlate with poor prognosis.Minoryx Therapeutics granted FDA Orphan Drug Designation in Friedreich’s Ataxia for new drug leriglitazone Minoryx Therapeutics is a company that specializes in the development of innovative treatments for rare diseases of the central nervous system (CNS), the part of the nervous system that contains the brain and spinal cord.


The main regulatory authorities are the MFDS and the Ministry of Health and Welfare (MOHW). In principle, the MFDS has the authority to oversee drug distribution from a safety perspective, while the MOHW has the authority to regulate the drug distribution market from a public interest perspective (for example, prevention of kickbacks, and an increase of transparency between pharmaceutical.An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy in many countries and has yielded medical breakthroughs.

Hunterase, the world's second commercialized treatment of Hunter syndrome, was released in South Korea in 2012 and designated as an orphan drug by the FDA a year later, and is undergoing phase II clinical trials in the U.S. Recently, it has pushed into the markets of the Middle East, Africa and South America. There are only about 70 patients suffering from Hunter syndrome in South Korea.

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Orphan Drug Designation. An Orphan Drug Designation is a status assigned to medicines developed for rare condition. ODD are grounded on proven medical plausibility of the orphan condition, and potentially significant benefits of the proposed treatment. ODD in the European Union. A medicinal product can be designated as orphan if it fulfils the.

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Further, South Korea has fully introduced a drug approval-patent linkage system (similar to that of the Hatch-Waxman Act in the US) from 15 March 2015. Under this system, a patentee can file an application to list its patents within 30 days from the drug approval date or 30 days from the patent registration date (if the patent is granted after drug approval). With respect to the listed patents.

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CMIC's South Korea office was established in 1996 in Seoul, South Korea as the first CMIC’s CRO business incorporated outside of Japan to provide full functional services in clinical trials under the Pharmaceutical Value Creator (PVC) model. With more than 25 years’ experience in South Korea for projects of global and local scales, we understand.

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South Korea has become the first east Asian country to legalise medical cannabis in a surprising move to expand the treatment options for patients with epilepsy and other rare diseases in the.

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The medical cannabis system of South Korea is controlled by the Ministry of Food and Drug Safety (MFDS) and the Korean Orphan Drug Center (KODC). The KODC is a government body that facilitates access to rare medicines, and together with the MFDS, they decide if a patient is eligible to import and use a specific CBD medicine.

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Orphan Medicines are intended to treat rare diseases. Both, the EU and US drug legislation have financial and regulatory incentive systems in place to encourage developers engaging in these neglected therapeutic areas. Nevertheless, often development of a new orphan medicine follows alternative development and regulatory pathways which require specific scientific and regulatory expertise. Our.

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South Korea moved toward the legalization of medical cannabis and became the first East Asian country to legalize medical marijuana. Korea’s move to legalize medical marijuana is “a big step,” but much detail requires to be sorted out concerning the regulations and little has been published beyond what is declared in the revision bill— Rev. Sung-seok Kang, with the Organization of.

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SALT LAKE CITY, USA, June 19 2017 — LSK BioPharma (LSKB, Company) announced today that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to apatinib for the treatment of gastric cancer. Apatinib, which has been approved and is marketed in China for patients with gastric cancer, is currently being investigated in a global phase 3 clinical trial sponsored by LSKB.

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This study aimed to identify orphan drug accessibility and impact on pharmaceutical budgets in South Korea by analyzing the status of orphan drug designation, approval, reimbursement, and.

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