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‘World Rare Disease Day’ on the 29th… Global orphan drug market expected to be worth KRW 647 trillion by 2032

Recruiting patients is difficult… Market exclusivity benefits for several years when designated as an orphan drug

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(Seoul = Larose.VIP) Reporter Hyunsoo Kim = The coming 29th is ‘World Rare Disease Day’ and ‘Day to Overcome Rare Diseases.’

Treatments for rare diseases with few patients have been the exclusive domain of global pharmaceutical companies, but domestic pharmaceutical and bio companies are also accelerating their development thanks to market exclusivity and speedy screening benefits.

According to the pharmaceutical and bio industry on the 25th, World Rare Disease Day was established in 2008 by the European Rare Disease Organization, taking into account the ‘rarity’ of the last day of February ending on the 29th once every four years. Various campaigns are held in over 100 countries to improve awareness of rare diseases and support patients.

Rare Disease Overcoming Day is a legal holiday in Korea that has been observed every year on May 23 since 2015, and will be held on the last day of February starting this year to raise awareness.

The definition of a rare disease varies slightly from country to country.

In the United States, rare diseases are defined as diseases with fewer than 200,000 patients, and in Europe, rare diseases are defined based on a prevalence of less than 5 per 10,000 people. In Korea, the number of patients is less than 20,000, and in Japan, the number of patients is less than 50,000.

Currently, there are approximately 500 to 8000 rare diseases in the world, and the number of patients is estimated to be 350 to 400 million.

Because the number of patients is small, it is not easy to conduct clinical trials, which are an essential prerequisite for the development of rare disease treatments.

For this reason, governments around the world are providing benefits such as rapid review and exclusivity through ‘orphan drug designation’.

The United States grants exclusive rights to drugs for rare diseases for 7 years from the date of marketing approval, and provides tax benefits of up to 50% on research and development (R&D) costs and a priority review system.

Exclusivity means guaranteeing marketability by prohibiting the approval of identical or similar drugs for a specific disease. In Europe, this period is longer, at 10 years.

In Korea, after approval for a shorter period of time, market exclusivity is granted for 4 years, and conditional approval can be obtained only through phase 2 clinical trials.

Seoul National University Hospital Pediatric Cancer and Rare Disease Project Group Symposium

Seoul National University Hospital Pediatric Cancer and Rare Disease Project Group Symposium

Gemvax & KAEL, a new drug development company, received designation as an orphan drug in the development stage for ‘GV1001’, a treatment for progressive supranuclear paralysis (PSP), from the Ministry of Food and Drug Safety on the 22nd.

Vigencell, an immune cell therapy development company[308080]Last year, ‘VT-EBV-N’, a treatment for NK/T cell lymphoma, a rare incurable blood cancer, was designated as an orphan drug by the European Medicines Agency (EMA), and in 2019, it was also designated as an orphan drug in the domestic development stage. The company completed phase 2 clinical trials in September last year and is currently observing the progress.

Hanmi Pharmaceutical[128940]It is known that the number of domestic and foreign orphan drug designations for new drugs under development is 20, which is the largest among domestic companies.

Daewoong Pharmaceutical[069620]GC Green Cross, NeoImmuneTech[950220]Toolgen[199800] Other treatments under development have also been designated as overseas orphan drugs.

Analysis suggests that the fact that it is a ‘blue ocean’ compared to existing medicines is one of the factors contributing to such active development of treatments for rare diseases.

According to market research firm Presidence Research, the orphan drug market is expected to grow at an average annual rate of about 12.2% from about $172 billion (about 229 trillion won) last year to about $484.7 billion (about 647 trillion won) in 2032. .

Global pharmaceutical companies enter the market through phase 2 clinical trials through the orphan drug designation system and then implement a strategy of expanding the scope of treatment to non-orphan diseases.

According to a report by the Korea Innovative Pharmaceutical Consortium (KIMCo Foundation), multinational pharmaceutical company Roche’s anticancer drug ‘Avastin’ received orphan drug designation for direct metastatic bowel cancer, and expanded its treatment range to include non-small cell lung cancer, ovarian cancer, and breast cancer, resulting in sales. It has almost doubled.

An official from Roche Korea explained, “The trend is to start research on rare diseases with high unmet medical needs, and then expand the indications (therapeutic range) to other diseases once the drug’s efficacy is verified.”

An official in the bio industry said, “Rare diseases have various variables in clinical patient recruitment, research and development, etc., but there are advantages such as being granted exclusive sales rights from relevant organizations or receiving tax benefits if they are designated as orphan drugs at home and abroad.” “Companies with a sense of mission are turning their attention to the development of orphan drugs to share the pain and difficulties of patients with diseases and their families,” he said.

hyunsu@yna.co.kr

Report to KakaoTalk okjebo 2024/02/25 08:00 Sent

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