Korea Accelerating Efforts To Develop, Launch Orphan Drugs
This article was originally published in SRA
South Korea's Ministry of Food and Drug Safety has issued revised rules to ease the designation and approval of orphan drugs. The changes are expected to create an environment that would support aggressive development of orphan drugs by the domestic pharmaceutical industry and result in more treatment options for patients.
The changes will allow high-priced medicines to be designated as orphan drugs. Earlier, to be designated as an orphan, the medicine's production or import value had to be equivalent to $1.5m or less. This requirement has now been removed.
Other designation criteria remain in place. Which means that in order to be designated as an orphan: the drug should be used for the treatment of a disease affecting fewer than 20,000 people; there should be lack of appropriate treatment methods or drugs for the concerned disease; and/or the candidate drug's safety and efficacy outcomes should represent a substantial improvement over existing therapies.
The ministry has also extended the validity of marketing approvals granted to orphan products. Specifically, once an orphan drug gains marketing authorization, then a renewal of the marketing approval will not be required for 10 years; earlier, renewals were needed after every five years. This has been done in recognition of the fact that rare diseases have a very limited patient population and it is difficult to collect data to evaluate the safety and efficacy of a drug to support the renewal of the marketing approval.
The changes brought about the ministry will also ease the conditions to evaluate drug manufacturing and quality control.
The changes have been brought about on account of the rare disease management act that was passed by the National Assembly passed late last year. Under this, beginning in 2017, the MFDS can prioritize the approval of orphan drugs over other medicines. The government will also give tax incentives to orphan drug producers or sellers as well as ease the financial burdens on patients by supporting treatment costs for rare diseases.
In addition, every five years, the health ministry has to formulate comprehensive plans to manage rare diseases. The health minister can also officially designate medical institutions that meet certain conditions as rare disease-specialized centers.
The rare disease management act was drawn up because the early diagnosis of such disorders is difficult and also they lack appropriate treatment methods or therapies. Due to low profitability, the pharma industry's research and investment in these conditions has also been relatively weak compared to other areas.
So far, South Korea has approved 353 orphan drugs from 93 companies and the number of approved orphans has been rising each year since 2010. In 2014, the domestic production of orphan drugs rose by 45% to KRW31.4bn ($26.2m), while imports of orphan drugs increased 6% to $131.8m.
A version of this article has been published in Pharm Asia News. Scrip Regulatory Affairs brings selected complementary coverage from our sister publications to our subscribers.