Big Pharma are making all efforts to preoccupy orphan drugs

KIMCo Published 'Global Rare Disease Market Research Report

In the U.S. and Europe, they dominate the global market with strong incentives such as tax incentives and priority screening

Korea is relatively behind in development and the usage environment such as benefit policy is insufficient

New drugs are emerging one after another for rare diseases such as multiple Castleman's disease, beta thalassemia, Fabri's disease, and spinal muscular dystrophy, which were not well known and had no medicine.

 

Global pharmaceutical companies such as Johnson & Johnson, AstraZeneca, and Roche are leading new drugs for rare diseases.

 

It was suggested that Korea should also provide institutional support and active development in the rare disease market dominated by global companies.

 

◆ Global company focused on rare drugs.

 

26% of total sales in 2026 According to the "Global Rare Disease Market Research Report" published by KIMCo, the global orphan drugs market is expected to grow by 12% annually, and sales of orphan drugs are expected to account for 20% of the total prescription drug market by 2026.

 

Global Big Pharma is aggressively dominating the market through active technology transfer and mergers and acquisitions.

 

In fact, the M&A case of a global pharmaceutical bio company in the first half of this year also reveals the high interest of Big Pharma in rare diseases.

 

Pfizer acquired Biohaven, a developer specializing in treatments for rare nervous system diseases, for $11.6 billion.

 

It is the largest deal in the first half of this year.

 

GSK and UCB also spent $1.9 billion each to buy biotechs that developed treatments for rare cancers and rare epilepsy.

 

Last year, AstraZeneca spent $39 billion to acquire Biotech Alexion, which specializes in treating rare diseases.

 

It is predicted that 79% of the global new drug pipeline currently being developed is rare drugs, and the trend of global new drug development in the future will focus on more rare drugs.

 

It is predicted that sales of rare drugs will reach an average of 26% of Big Pharma's total sales in 2026.

 

◆"Korea Has Less Development and Poor Support.

 

Development strategies for orphan drugs" Behind the global big Pharma's rush to turn to rare diseases is the policies of major countries that give strong benefits to the development of orphan drugs.

 

The U.S.

 

Food and Drug Administration (FDA) provides up to 50% of tax benefits for R&D costs for new drugs designated as orphan drugs.

 

It also advises on the design of subsidies and protocols for clinical development and exempts for orphan drugs for application for examination.

 

If a new drug is commercialized, the exclusive right period will also be given seven years, two years longer than five years of general new drugs.

 

This year, it also established a priority review for orphan drugs.

 

Europe also has tax benefits for orphan drugs, exemption from examination application fees, and priority review systems.

 

Monopoly rights can be granted up to 10 years from the date of marketing authorization, and the monopoly period is set through re-evaluation five years after marketing.

 

Tax benefits are given from the development stage and various incentives such as priority review, post-marketing monopoly rights, and extension of item permits can help pharmaceutical companies secure growth engines in the long run.

 

Global Big Pharma is taking a strategy to maximize drug sales by continuously expanding the indication of non-rare diseases after entering the market after phase 2 with an orphan drug designation and rapid screening program.

 

Korea has implemented the Rare Disease Control Act since 2017, relatively late, and has prepared a legal basis for incentives for orphan drugs.

 

Under this law, orphan drugs are given a 10-year validity period.

 

In addition, the priority review system can be applied to orphan drugs, and fees will be reduced.

 

The exclusive right shall be granted for four years from the date of marketing authorization.

 

Some point out that the domestic benefit policy for orphan drugs, which are relatively expensive, is still insufficient.

 

At the "policy debate to improve the environment of pediatric rare diseases" held at the National Assembly on the 13th, Professor Lee Bum-hee of AMC argued that orphan drugs designated by the Ministry of Food and Drug Safety and rare disease treatments are separated.

 

Professor Lee Bum-hee pointed out that even if a new drug is developed successfully, the market will not be able to be activated unless an environment that can be used properly is supported.