Negotiating prices of drugs for rare diseases

Séverine Henrard & Francis Arickx, Bulletin of the World Health Organization 2016;94:779-781. doi:10.2471/BLT.15.163519

Due to the increasing number of treatments for rare diseases that have been approved and are under development, and hence the rising costs for countries’ health systems, there is an ongoing debate about health policies for rare diseases and particularly about reimbursement of rare disease drugs

Number of applications for orphan designation for medicinal products to the European Medicines Agency over the years 2000–2015

A Cost of Illness Study Evaluating The Healthcare And Societal Burden of Friedreich’s Ataxia In The United Kingdom

K Hanman, A Griffiths, A Bobrowska, J Vallortigara, J Greenfield, RS Thompson, Value in Health, Volume 19, Issue 7, November 2016, Pages A584-A585, ISSN 1098-3015, doi:10.1016/j.jval.2016.09.1372

Overall, the total COI of FRDA patients to the NHS was £8,038,645 per year, with a mean annual cost per patient of £3,556. The development and delivery of new treatment options, particularly to address the loss of mobility and cardiac abnormalities experienced by FRDA patients, will substantially reduce the economic burden of this rare and devastating disease.