The status of orphan drugs in China

Chia Wen Lee and Betty Su

CMAx and RXB Consulting LLC

As we near the end of our “emerging markets” focus month, Chia Wen Lee and Betty Su share their thoughts on the status of orphan drugs in China, one of the largest emerging markets in the world. With over 10 million Chinese people suffering from rare diseases, Chia Wen and Betty look at the legislation and funding around rare diseases status and what can be done to improve this.

International context

Governments determine rare disease status by prevalence of the disease and this varies by country. In the United States, an orphan drug is defined as intended for a disease with a prevalence of fewer than 200,000 (0.075 per cent of the total population). In the European Union (EU) it is approximately 0.05 per cent. In the last 20 years, orphan drug legislation (ODL) that has been adopted in several countries around the world (USA, Japan, Australia, and the EU) has successfully promoted R&amp,D investments to develop new pharmaceutical products for the treatment of rare diseases.

Examples of a rare condition are disorders resulting from inborn errors of metabolism (IEM). These disorders affect a very small number of individuals. The entire population, however, of patients suffering the results of inherited metabolic disorders is large, and has been of increasing concern to patient groups and health care professionals in the United States as well as other countries throughout the world. The 1983 US Orphan Drug Act (ODA) serves to facilitate the development of drugs to treat rare diseases by providing several economic incentives. Orphan drug legislation in the US has benefited victims of IEM by encouraging development of drugs for metabolic deficiencies affecting populations that otherwise would be ignored1.

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“Today, international support for rare disease research is providing stimulus and motivation to overcome the financial barriers…”

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America’s solution to the orphan drug problem has had worldwide impact. The success of this legislation was a factor leading to the 1993 orphan drug law in Japan, the 1997 implementation of a process whereby most FDA-approved orphan drugs and biological products will be similarly approved in Australia, and, in 1999, regulation on orphan medicinal products in the European Union (EU). Today, international support for rare disease research is providing stimulus and motivation to overcome the financial barriers and encourage development of treatment for very rare diseases throughout the world.

Situation in China

Based on WHO’s estimate, over 10 million Chinese suffer from rare diseases. Though today in China the relevant laws and legislations have no official definition on “rare disease”, there seems to be a consensus from the clinical experts to define Orphan disease (OD) as prevalence less than 1 / 50 million, or neonatal morbidity less than 1 / 10,000, and the diseases are classified by ICD-10 categories. Among commonly recognized rare diseases, haemophilia has received most attention hence some funding. Nevertheless there is no earmarked funding for rare diseases.

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“Based on WHO’s estimate, over 10 million Chinese suffer from rare diseases.”

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Despite the fact that rare disease has been an area of overlook where medical resources are firstly spent in ensuring the primary care delivery, lately there have been some positive developments indicating the increased awareness and attention from the policy makers. On the Rare Disease Day of 2011 2, 3 , the Shanghai Medical Association held the inaugural ceremony for the Society of Rare Diseases, China’s first organization in China focused on rare diseases. Again in Shanghai, the government has published policy to protect patients with 12 rare diseases with some reimbursement in three specialty hospitals within Shanghai municipality. At the State level, Ministry of Human Resources and Social Security announced that the State considers providing reimbursement of orphan disease interventions including the possibility of incorporating them into Essential Drug List EDL and / or Category B of National Reimbursement Drug List (NRDL).

References

1. Haffner ME. Developing treatments for inborn errors: incentives available to the clinician. Mol Genet Metab. 2004 Apr, 81 Suppl 1:S63-6.

2. http://www.hanjianbing.org/

3. http://www.chinararedisease.cn/index.php

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About the authors:

Dr Chia Wen Lee

Dr Chia Wen Lee, PhD, is Co-Founder and Managing Partner of CMAx.  She is a versatile executive with significant scientific and business development experience, across various delivery systems in Europe, Asia and the US. Dr Lee co-founded CMAx (www.ChinaMarketAccess.com) in 2010 and is the Managing Partner. She currently heads up Market Access for the Emerging Markets for a major biotechnology company head quartered in the US.

Dr Chia Wen Lee was born and raised in Taipei, and received PhD in epidemiology and biostatistics from London School of Hygiene and Tropical Medicine.

Betty Su

Betty Su is Partner &amp, Managing Director of RXB Consulting LLC.  She has extensive hands-on experience in the pharmaceutical industry in China. She started her career as the Executive Assistant to the CEO of AstraZeneca China. She was then promoted onto Corporate Affairs role responsible for external stakeholder management and internal communication, then Brand Manager with major responsibility of Oncology portfolio. Ms. Su holds MBA degree from BI-Fudan University in Shanghai and co-founded RXB Consulting in 2009 (www.rxbllc.com) providing market entry advices and solutions for pharma and device players developing their business in Chinese market.

How can we better support patients with rare diseases in China?