Blog entry by Meguid El Nahas
A letter to the editor in this month NEJM:
highlights the difference between the outcome of Nephropathic Cystinosis in developed and developing countries.
A survey undertaken by the authors in 17 developing and 13 developed countries showed massive differences in renal survival (ESRD) between the two groups; in fact the outcomes of developing countries nephropathic cystinosis in the 21st century is similar to that observed in developed countries 30 years ago before the advent of cysteamine treatment!
This once more highlights, many issues and problems with healthcare for Orphan and Rare Kidney Diseases (ORKD) in developing countries:
1. Poor awareness
2. Late diagnosis
3. Restricted access to medication
This in spite of the well known fact and observations that initiation of Cysteamine therapy before the age of 5 years impacts on the progression of cystinosis, renal failure, diabetes, hypothyroidism as well as patients survival.
It is high time the GAP between rich and poor countries is bridged when it comes to healthcare, but for this to be accomplished in ORKD concerted efforts have to be made between Governments, NGO as well as the Pharmaceutical Industry.