Updated on 15 December 2014
What is the current status of orphan drugs in Asia?
Governments in many Asian countries like Japan, Taiwan and Korea are becoming increasingly aware of orphan diseases. Individual countries are stepping up efforts to solve this crisis. Japan has an Orphan Drug Act that regulates drug approval and new research for orphan drugs. Taiwan and Korea too have some regulatory policy and some action plan to address these diseases.
How is the Japanese government encouraging orphan drug research?
The government has declared two major actions to promote the development of orphan drugs:
- Until last year, the government had identified 56 diseases under the rare diseases category to support. This year the government has identified 300 diseases.
- In Japan, the conventional drug review process is a yearlong. The government is aiming at fast-tracking this process to six months by recruiting more reviewers.
Are there any financial incentives available from the government for orphan drug developers?
The government of Japan is investing a lot of capital in R&D. The government has identified 10-15 universities as Centers of Excellence. For these centers the government provides financial assistance to support new research.
What are the biggest challenges faced by drug developers in orphan drug R&D?
Most of these rare diseases are very difficult to diagnose. The rarity of these conditions means that most doctors have very limited experience with them, so that many rare disease patients wait years before receiving an accurate diagnosis. Even then, treatment options may be limited or nonexistent: Fewer than 5 percent of all rare diseases have approved therapies available. Due to the paucity of approved therapies, medicines are sometimes used off-label to treat rare diseases.
There is usually little understanding or information about the natural progression of the disease to inform trial design, and investigators often have difficulty identifying and enrolling a large number of patients. Basic research tools, such as validated animal models, may not exist. These challenges increase the uncertainty that a research program will lead to a new therapy. Given small patient numbers, there has historically been less investment in rare diseases than in more common diseases.