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World J Methodol. Sep 26, 2014; 4(3): 163-188
Published online Sep 26, 2014. doi: 10.5662/wjm.v4.i3.163
Published online Sep 26, 2014. doi: 10.5662/wjm.v4.i3.163
World health dilemmas: Orphan and rare diseases, orphan drugs and orphan patients
Christina N Kontoghiorghe, Nicholas Andreou, Katerina Constantinou, George J Kontoghiorghes, Postgraduate Research Institute of Science, Technology, Environment and Medicine, Limassol 3021, Cyprus
Author contributions: Kontoghiorghe CN reviewed the ethical issues on orphan and other drug treatments; Andreou N reviewed the orphan drug topics; Constantinou K reviewed the disease profile in developed and developing countries; Kontoghiorghes GJ reviewed the issues related to iron chelating drugs, orphan drug development and the role of pharmaceutical companies and he also designed, wrote and edited the manuscript, which is part of a postgraduate project.
Correspondence to: George J Kontoghiorghes, PhD, Professor, Postgraduate Research Institute of Science, Technology, Environment and Medicine, 3 Ammochostou Street, Limassol 3021, Cyprus. kontoghiorghes.g.j@pri.ac.cy
Telephone: +357-26-272076 Fax: +357-26-272076
Received: January 29, 2014
Revised: April 5, 2014
Accepted: June 27, 2014
Published online: September 26, 2014
Revised: April 5, 2014
Accepted: June 27, 2014
Published online: September 26, 2014
Core Tip
Core tip: The major world health problems are related to poverty and other monetary health issues, including the supply of orphan drugs for the treatment of rare and orphan diseases. Differences in disease profile, disease burden and monetary health policies influence the mortality and morbidity rates of patients in developed and developing countries. The inexpensive developmental procedure of the iron chelating drug, deferiprone, used in thalassaemia is proposed as a paradigm for orphan and rare drug development. Improvements in worldwide health policies including procedures for inexpensive drug development and alleviation of poverty could reduce the mortality and morbidity rates of patients worldwide.