Orphan medicines: special treatment required?: case study 3: Glivec and chronic myeloid leukaemia

Document type
Case study
Corporate author(s)
2020health (Think tank)
Date of publication
1 November 2012
Health Services
Social welfare
Material type

Download (124KB )

This case study on the drug Glivec and treatment of chronic myeloid leukaemia accompanies a report that examines the evidence to see if there are ways of improving decision making on treatments for people with rare diseases in the NHS. Glivec (imatibib) is an anticancer medicine, manufactured by Novartis. Glivec has been shown in clinical trials to improve clinical outcomes for patients with chronic myeloid leukaemia (CML) in terms of surrogate outcome measures for survival, including: overall haematological and cytogenetic response rates, and progression-free survival. Glivec was the first in a new class of cancer drug, resulting in reductions in the uncontrolled proliferation of white blood cells which occurs in CML.

Glivec was awarded orphan drug designation for its use in CML and approved for use by the European Medicines Agency in 2001. Glivec was therefore one of the earliest products to be able to benefit from incentives under the Orphan Drug Legislation.

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