Blog entry by Meguid El Nahas

Anyone in the world


In a mini review soon to be published in Nephron,  Professor Neveen Soliman highlights the issues related to Orphan and Rare Kidney Diseases (ORKD). This is a timely reminder of the challenges facing patients who suffer from orphan diseases as well as their healthcarers.  Orphan drug development has been at the forefront of renal and nephrology research in the last decade, the cost of such treatment is increasingly prohibitive. Western governments have supported orphan drug development, granting incentives of up to 10 years of exclusivity for manufacturers of such drugs receiving regulatory approval. In recent years, up to 1500 drug applications have been filed in the EU alone with more than 70 drugs have been approved in Europe.  Also regulatory bodies have facilitated rapid marketing by granting conditional approvals based on limited data and conditional to ongoing monitoring and risk of withdrawal of approval if treatment impact was negative.  However, work on orphan drugs could be under threat due to the economic downturn and the exhorbitant cost; a single patient with an orphan disease can cost between £100,00 to £400,000 a year!  Consequently, the foreseen EU expenditure on orphan drugs will reach 4.6% of total EU pharmaceutical expenditure by 2016. It is therefore not surprising that due to the economic downturn, Western economies are finding such cost increasingly unaffordable. On the other hand,  emerging economies, such as Egypt, have never been able to afford the price of orphan therapies. 
Access to Orphan therapies has encouraged a new brand of activism bringing patients groups, researchers and specialists together to facilitate access to healthcare for orphan disease. Professor Soliman herself has set up EGORD (Egyptian Group for Orphan Renal Disease) with for a mission raising significant funds and increasing awareness for research and treatment of Orphan disease such as cystinosis. This group, along with other organisations and networks highlighted in this mini review by Professor Soliman, may be shifting the pharmaceutical industry’s attitude to developing and marketing Orphan drugs. Whilst some industrialists continue to justify the cost based on years of research and the small market, others such as Moncef Slaoui, head of research at GSK, recently stated that the industry need to take a more responsible approach to pricing. It is imperative that pressure is put on the industry to provide affordable care for Orphan and Rare Disease. It is unacceptable that economic downturns impact on the healthcare of those who are sick and most vulnerable in our societies. It is one of the responsibilities of our nephrological community to engage manufacturers of orphan drugs in partnerships to deliver affordable healthcare to those with orphan disease.  Nowhere are such initiatives more urgent than in developing countries. 
The ORKD Campaign on OLA hopes to position itself as a key player in the fight for access for Affordable Therapies for Orphan and Rare Kidney Disease.

Abbott A. Rare-disease project has global ambitions. Nature. 2011 Apr 7;472(7341):17.

 Simoens S, Cassiman D, Dooms M, Picavet E. Orphan Drugs for Rare Diseases: Is it Time to Revisit Their Special Market Access Status?

Drugs. 2012 Jul 30;72(11):1437-43. 



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