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Orphan Drugs And Rare Diseases: Global News (04.15.13)

April 15, 2013

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

I – Europe

The European Commission (EC) approves drugs for all twenty-seven members of the European Union (EU).  Each country is responsible for deciding whether to pay and what it will pay for a drug. With the global economy in cost-cutting mode for the past several years, it is no surprise that drug prices, especially orphan drugs in particular, are under close scrutiny because of their high prices. Several countries have taken action against the high price of orphan drugs per a Bloomberg article :

•  UK rejects a recommendation to expand the use of Alexion Pharmaceuticals’ orphan drug Soliris (Eculizumab) for two blood disorders, despite an advisory panel recommendation. The government refers this issue to the National Institute for Health and Care Excellence (NICE)
•  Ireland recommends against the government paying for Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF), resulting in the company significantly reducing the price of the drug
•  The Netherlands demand a cut in prices for Enzyme Replacement Therapies (ERTs), including Sanofi’s orphan drug Myozyme.

Dutch Health Minister Edith Schippers says in January 2013, “Sanofi and Shire must cut prices for drugs against 2 rare genetic disorders, Pompe and Fabry diseases, to “acceptable levels” to remain in the nation’s basic insurance package covered by all health insurers.”

As noted in a previous Blog Post, under the UK’s new Health and Social Care Act of 2012, NICE’s newly established Health Technology Assessment (HTA) group starts April 1, 2013 to review and evaluate the high cost of orphan drugs. The criteria that NICE will use to evaluate the pricing of orphan drugs has not been released.

II – India

As noted in a previous Blog Post, India’s Supreme Court in April 2013, decides to block patent protection for Novartis Pharmaceuticals’ blockbuster cancer orphan drug Gleevec (Glivic in India). BBC News last week interviews Ranjit Shahani, head of Novartis India, who expresses disappointment that India’s Supreme Court refuses to grant a patent for an updated version of Glivec. Mr. Shahani goes on to say that Novartis would not invest in drug research and development in India in the future, and suggested that other pharmaceutical firms would follow suit. The decision by India’s Supreme Court will result in generic drugmakers continuing to sell copies of the drug at a lower price in India. The interview can be viewed.

III – El Salvador

In El Salvador, under the General Medicine Law approved of in February 2013, prices “for at least 6,200 medicines will be reduced by a minimum of 30%, and up to 60% for those most often prescribed”. El Salvador is considered one of the countries with the most expensive medication worldwide. The new law creates an entity known as the General Directorate of Medications, which will be authorized to supervise activities in the supply chain, such as imports, prescriptions, and distribution. It will also oversee the classification of drugs that require both prescriptions and that are available over-the-counter (OTC). It will be interesting to see what impact if any this has on orphan drugs.

IV – Canada

On April 12, 2013, the creation of the International Consortium of Gene Therapy is announced. Professor Jacques Tremblay, Professor of the Molecular Medicine Department of Laval University, gets 50 researchers to write a letter of intent in the latest issue of Molecular Therapy, for the creation of this organization. The mission of the organization is to speed up the development of treatments, like gene therapy, to help patients suffering from rare diseases.

V – Australia

In Australia, the administrative authority involved in orphan drug regulations, is the Therapeutic Goods Administration (TGA). TGA is part of the Australian government’s Department of Health & Ageing. The alphabetical list of designated orphan drugs in Australia from the TGA is updated as of April 2, 2013.

VI – Iran

In March 2013, the Rare Diseases Foundation of Iran unveiled a full-color comprehensive atlas of rare diseases.

VII – United States

Two new FDA Breakthrough Therapy Designations are announced :

•  Pfizer’s non-orphan drug Palbociclib for breast cancer
•  Janssen Pharmaceuticals and Pharmacyclics’ oncology orphan drug Ibrutinib receives its 3rd FDA Breakthrough Therapy Designation for Chronic Lymphocytic Leukemia (CLL).

The annual World Orphan Drug Congress (WODC) USA is held for 3 days, April 9-11, in Washington DC. The next meeting is scheduled for April 23-25, 2014, in Washington DC.

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

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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