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Orphan Drug And Rare Diseases: Global News

March 4, 2013

Recent orphan drug and rare disease global developments are in the news. Here is a list and description :

I – China

The State Food and Drug Administration (SFDA) in China is responsible for policies and programs on the administration of drugs, health food, medical devices, and cosmetics. The SFDA releases new proposed guidelines for its drug review process. Drugs that address an unmet need will be given greater priority. Greater emphasis on drugs for children is also proposed. The SFDA aims to speed up approval for the products that address the most critical needs.

Pfizer receives China’s approval for U.S. orphan drug Xalkori (Crizotinib) for patients with Non-Small Cell Lung Cancer (NSCLC) that has the Anaplastic Lymphoma Kinase (ALK) mutation. The ALK-positive mutation has to be shown by a biomarker test. Per Pfizer, China’s approval comes 11 months after submitting a new drug application to the SFDA.

A recent March 4, 2013 article “Government urged to ease financial urden on patients with rare diseases”,  discusses the request by doctors and leaders of social organizations to improve efficiency in screening for rare diseases and for the government to lower the financial burden facing rare disease patients. According to Ding Jie, chairman of the rare disease branch of the Beijing Medical Association, it is estimated that there are more than 10 million with rare diseases in China who go undiagnosed due to the lack of doctors’ knowledge about rare diseases. Currently in China, there is no legislation regarding treatment and health insurance for rare disease patients. According to Ding, access to medicine is another problem for rare disease patients – medicine is effective in treating only about 1% of rare diseases.

In February 2013, Dyax announces a partnership with CVie Therapeutics to develop and commercialize orphan drug Kalbitor for Hereditary Angioedema (HAE), in China, Hong Kong, and Macau.


According to a February 28, 2013 article, Dr. Fatima Bastaki, consultant pediatrician in clinical genetics and metabolis, says that the Centre for Arab Genomic Studies has catalogued 350 rare genetic disorders in Arab populations. Because of the lack of a rare disease central registry of any type, the incidence of rare diseases in the UAE cannot be accurately estimated. Eight of 10 cases of rare diseases have genetic origins and according to Dr. Fatima, “Consanguinity increases this risk, especially as intermarriages between relatives is common in countries like the UAE …. Patients with rare diseases and their families feel isolated and vulnerable. We need to change the societal attitude towards people with rare diseases and look at inclusion at all levels ….”.

III – India

Recently, Roche has had drug patents under attack in India. Roche is planning to manufacture some drugs in India, “indicating it will drop the prices by as much as 50%, even as it faces a new threat from India’s compulsory license law”. Roche will drop prices of several drugs in India including the following orphan drugs :

  1. Herceptin  (Trastuzumab) from its current 110,700 rupees ($2,000 US) to 75,000 rupees ($1,366), a drop of 31%
  2. MabThera (Rituximab) from its current $1,456/month to $682/month, a drop of 53%.

IV – Europe

The European Commission (EC) announces February 28, 2013, that €144 million of new funding will be provided for 26 research projects, with the objective to deliver 200 new therapies for rare diseases by 2020. It is estimated that this will help to improve the lives of 30 million Europeans. Over 300 participants from 29 countries and beyond will be involved in the projects.

Please Note: “Globe” by Augiasstallputzer at Wikimedia Commons [Public domain] | Wikimedia Commons.

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