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Orphan Drugs And Rare Diseases: Global News (04.05.13)

April 5, 2013

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

I – UK

Under the UK’s new Health and Social Care Act of 2012, the National Institute for Health and Care Excellence’s (NICE) newly established Health Technology Assessment (HTA) group starts April 1, 2013 to to review and evaluate the high cost of orphan drugs for people who suffer with rare conditions.

According to an April 3 article, there seems to be confusion over the pricing process in the UK for Alexion Pharmaceuticals’ orphan drug Soliris (Eculizumab) for the treatment of atypical Haemolytic Uraemic Syndrome (aHUS). The Advisory Group for National Specialized Services (AGNSS), a group set up to advise government ministers, gives Soliris a positive recommendation for reimbursement for aHUS in January 2013. The Department of Health rejects the recommendation. With the dissolution of AGNSS, responsibility is now with NICE, which plans to start the Soliris assessment this month. It will be interesting to observe what actions NICE will take, as Soliris is the first product to be assessed by the HTA. According to a statement by the NHS, a decision on Soliris’ reimbursement in the UK will be delayed until the spring of 2014. This contradicts recent assertions by Alexion that Soliris is soon to be made available in the UK through an “unprecedented access program.”  Per the article, “it now appears that management expects more immediate access to the drug to be facilitated by local/regional NHS trusts ….”.

II – Japan

Daiichi Sankyo Company announces April 1, 2013 the opening of the Orphan Disease Treatment Institute, with the focus on developing therapy for the treatment of Duchenne Muscular Dystrophy (DMD).

III – India

India’s Supreme Court on April 1, 2013 decides to block patent protection for Novartis Pharmaceuticals’ blockbuster cancer orphan drug Gleevec (Glivic in India). According to the article, this case “highlights a process common in the US knows as “evergreening”, by which pharmaceutical companies introduce new drugs that are just slightly modified formulations of older drugs in order to effectively maintain patent protection.” The Indian court rejects Novartis Pharmaceuticals’ patent for Gleevec that is currently under patent protection in the US, on the basis that “the formulation was not significantly different or better than previous drugs on the market.” Fascinating story that can be read at The Wall Street Journal (may need subscription).

IV – United States

On April 1, 2013, the US House of Representatives, by a vote of 375-27, approves a bipartisan bill, the National Pediatric Research Network Act, NPRNA (House Resolution (H.R.) 225). The bill authorizes the National Institutes of Health to “facilitate the creation of up to 20 pediatric research consortia focused on developing treatments and cures for pediatric diseases, including rare conditions like Spinal Muscular Atrophy (SMA).” The bill was introduced after Rep. Lois Capps, D-Santa Barbara, California, met the Strong family whose daughter is diagnosed with SMA as an infant. The purpose of NPRNA is to improve and increase research on children’s illnesses, especially rare and complex diseases. NPRNA has not been voted on yet in the Senate.

PatientCrossroads, recently announces to rare disease organizations, an opportunity to become Registry Partners in a new shared, open-access patient registry where data can be collected on any disease, from the most rare to more common disorders.”  Rare disease organizations are defined as patient advocacy groups, pharmaceutical companies, and medical researchers. This program is supported by a grant from Novartis Pharmaceuticals. PatientCrossroads is to announce this month, the selected organizations and the launch of CONNECT, the openly available worldwide patient registry. There is scheduled first, a short beta test period with selected disease communities, before officially launching. The PatientCrossroads’ registry platform is currently powering the NIH Global Rare Diseases Registry program. PatientCrossroads operate the registry and is the custodian of the data.

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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