Pharma Focus Asia

Orphan Drugs - Asia

The New Frontier

Mary Dunkle, Vice President of Communications, National Organization for Rare Disorders,Connecticut, USA

As the home to more than half the world's population, Asia is poised to become an area of tremendous interest to pharmaceutical and biotechnology companies engaged in research on the thousands of diseases considered rare.

Asia has emerged in recent years as the newest frontier in the development of treatments for diseases classified as rare. Several Asian nations either have enacted, or are considering, orphan drug legislation.

In some cases, Asian countries are patterning their orphan drug policies on the United States’ Orphan Drug Act of 1983, which provides seven years of marketing exclusivity and other financial incentives for companies that develop products for rare diseases. In other cases, Asian policy-makers are charting their own course, but with an eye to what has been done successfully elsewhere to date.

Either way, as the home to more than half the world’s population, Asia is poised to become an area of tremendous interest to pharmaceutical and biotechnology companies engaged in research on the thousands of diseases considered rare. The definition of “rare” varies from country to country, but in the U.S. the word is applied to any disease believed to affect fewer than 200,000 Americans.

Asia on the Move

In several Asian countries, patient communities have been actively involved in advocacy for orphan drug development. For instance, in December 2005, the Taiwan Foundation for Rare Disorders (TFRD) hosted an international conference in Taipei on “Genetic Medicine and Promoting Healthcare for Rare Disorders.” Speakers from the U.S., Europe and Australia participated. Patient organisations represented on the program included the National Organization for Rare Disorders (NORD) of the U.S. and EURORDIS, the European Organization for Rare Diseases.

In September 2006, the Korean National Institute of Health (KNIH) and its Genetic and Rare Diseases Center hosted a two-day symposium in Seoul aimed at bringing together Asian and U.S. speakers to present information on orphan product research and development. KNIH has also recently established a web site to provide information in Korean for rare-disease patients and families, and it has signed a Memorandum of Understanding with NORD to provide content for that site. This is a major step forward, because little information on rare diseases has been available to Korean patients and their families in the past.

The two conferences and the new web site underscore the emphasis on international collaboration in Asian countries interested in expanding their involvement in rare diseases. A key figure in orphan product development in the United States, Marlene E. Haffner, MD, agrees that international cooperation is important.

“Twenty-five years ago, a program targeted at patients with rare diseases was only a hope. Today, the concept has been adopted around the world from the U.S. to Japan, to Taiwan and South Korea, to Australia and Europe,” said Dr. Haffner, who is the Director of the Office of Orphan Products Development at the U.S. Food and Drug Administration. “Millions of patients now have some treatment for their disease. And much new cutting-edge research has been undertaken. Rare diseases know no borders. It is only by working together that we will achieve treatments for patients in need.”

The American Experience

Since the U.S. Orphan Drug Act was passed by Congress and signed into law by President Ronald Reagan in 1983, more than 1,400 products have been designated as “orphans,” and 290 have been approved for marketing by FDA. Before the law was passed, very few companies were developing products for rare diseases because it was almost impossible to recover the initial investment.

Today, many companies, both large and small, are finding a profitable niche in orphan product development. And, for the patient community, this translates to treatments for diseases that previously had none. This year alone, FDA has approved several new treatments including, for example, enzyme replacement therapies for Pompe disease and Hunter syndrome, two life-threatening metabolic diseases that previously had no specific therapy. It is estimated that more than 300 products for more than 600 rare diseases are currently in clinical trials or awaiting FDA approval.

Asia to Date

Japan was the first Asian nation to enact orphan drug legislation. Its Orphan Drug Development Program was launched in 1993, 10 years after the U.S. law went into effect. The program has been successful and has attracted involvement from non-Japanese drug companies. Japan’s policy is similar, but not identical to, America’s Orphan Drug Act.

In the 1980s, in the U.S., it was the patient community, working through a coalition that later was formalized into NORD, that made the critical difference in drawing attention to the need for orphan drugs. The same is happening today in several Asian countries.

For instance, in Taiwan, before rare diseases legislation was implemented, patients with rare diseases and their families faced several challenges: Drugs for rare diseases were expensive and were not covered by the National Health Insurance, there was a shortage of genetic specialists, and little information was available on rare diseases.

The Taiwanese Department of Health recommended legislation patterned on the U.S. model to address orphan drug development, but ultimately the legislation adopted more closely reflected that proposed by the Taiwan Foundation for Rare Disorders, an advocacy organisation founded by two parents of children with rare diseases, Li-Yin Chen and Min-Chieh Tseng. The Rare Disease and Orphan Drug Act enacted in 2000 in Taiwan covers a broad spectrum of needs, including a subsidy for expenses not covered by the National Health Insurance, increased public education awareness and education, and licensing a genetic consultation network.

According to Dr. Tseng, one of the founders of TFRD, over a period of three years after the implementation of the Rare Disease and Orphan Drug Act, the number of orphan drugs covered by the National Health Insurance increased from 27 to 52. Individual patient cases covered by the insurance increased from 435 to 874, with an increase of total payment cost from US$3.3 million to $7.9 million.

The Taiwanese government has also expanded its newborn screening programs for genetic diseases. The expanded screening program uses tandem mass spectrometry to test for nearly 30 inherited metabolic disorders, and the TFRD sponsors newborns of aboriginal heritage so that their screening is free. From 2002 to 2005, Dr. Tseng said, 105 cases of metabolic disorders were detected early as a result of the expanded screening.

South Korea is also seeking to modernize and advance its rare-disease research and orphan product development. The Korean National Institute of Health includes within its structure an Orphan Drug Center, a Genetic and Rare Disease Center, and a Department of Biomedical Sciences that is committed to increasing research on rare diseases. As in Taiwan, there is an active patient community in Korea, organised by the Korean Organization for Rare Diseases (KORD).

At the recent conference, speakers addressed issues such as the feasibility of expanded orphan drug development in Korea and the possibility of international partnerships to spark increased research on orphan products. The interest is strong, and Korea appears poised on the brink of an explosion of activity in these areas. It is significant that three members of the Korean National Assembly spoke at the recent conference, emphasising the importance of the event.

Other Asian entities, such as Hong Kong and Singapore, represent small but well-to-do populations with relatively sophisticated health delivery systems that would support additional activity related to orphan products. And of course China represents a huge, but to date largely undeveloped market for these products.

Abbey S. Meyers, the President of NORD, who is widely considered the leading consumer advocate for the U.S. Orphan Drug Act, sees the awakening Asian interest as a welcome and healthy sign. “We in the U.S. hear of increasing interest in orphan drugs in Asia, and we would be willing to help in any way that we can,” she said. “The Orphan Drug Act has brought hope to many patients and families who had none in the U.S. We are encouraged and delighted to hear that other countries are also addressing the plight of these once-forgotten people.”

Mary Dunkle is Vice President for Communications of the National Organization for Rare Disorders (NORD). The NORD web site is at

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