Access To Orphan Drugs in France: The Case Of Siklos For The Treatment of Sickle Cell Syndrome Before The Council of State
2014 Value in health
Murteira, S. | El Hammi, E. | Kornfeld, A. | Toumi, M. |
France, orphan drug, Value,
Hydroxycarbamid is an antineoplastic molecule commercialized since 1968 in France under the name Hydrea®. In the absence of an alternative licensed drug for the treatment of Sickle Cell Syndrome (SCS), Hydrea® has been routinely used off-label for over 15 years in this indication. In 2007, Siklos® became the first licensed orphan drug indicated in the symptomatic treatment of SCS. However, during the pricing and reimbursement procedures, the French Transparency Committee decided to compare Siklos® to Hydrea® and as such Siklos® was evaluated as bringing a weak additional benefit (ASMR IV) which had a major impact on the price given by the French Economic Committee on Health Care Products (CEPS).