A record number of fatalities in many categories of patients treated with deferasirox: loopholes in regulatory and marketing procedures undermine patient safety and misguide public funds?

Abstract

A record 4113 fatalities were reported in 2012 in a postmarketing surveillance of patients treated with deferasirox, despite warnings of life-threatening toxic side effects, and the need for regular monitoring and prophylactic measures. In an EMA report, the mortality rate was estimated at 11.7% and a warning was issued for increasing the dose from 30 to 40 mg/kg/day. In an earlier FDA report of 2474 individual fatality cases, it was revealed that deferasirox was used in many categories of patients. Among the fatal cases reported were many young individuals and about 500 patients with normal iron stores such as cancer, leukaemia, cardiovascular and neurological diseases. The iron-loaded patient categories included myelodysplasia, sickle cell disease, haemochromatosis and thalassaemia. The rate of fatalities and the number of patient categories involved suggest that there has been an indiscriminate and uncontrollable use of deferasirox. These findings raise major concerns on patient safety and question the role, practices and procedures adopted by pharmaceutical companies, regulatory authorities, physicians, etc. in the development of new drugs and their safety. The generic drugs deferiprone, deferoxamine and their combination offer a safer, less expensive and complete treatment of iron overload in thalassaemia and other iron loading conditions.

Keywords::

• chelation therapy
• deferasirox
• deferiprone
• deferoxamine
• drug safety
• ethics
• non-iron-loaded diseases
• thalassaemia
• toxicity and fatalities
• transfusional iron overload