Comment on: 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?

The editorial by GJ Kontoghiorghes [1] contains significant factual inaccuracies, selectively omits important information about deferasirox and lacks the robust epidemiological methodology typical for analysis of pharmacovigilance data for safety signals. The aim of this letter is to correct these inaccuracies, providing correct information for doctors and patients. Several key references in the editorial do not meet the criteria for scientific data integrity. For example, data are cited from ehealthme.com, a website that includes social media among its data sources, and fda-reports.com, the website of a US personal injury law firm. In addition, data cited from the Institute for Safe Medication Practices (ISMP) report are interpreted out of context, cover a 4-year time period and are highly flawed as a result of a reporting problem acknowledged by ISMP itself [2]. Several other references cited are the author’s own opinion/review articles and many reflect treatment practices in place over 5 years ago, when less was known about available iron chelators. Furthermore, the author, a chemist, is the inventor of the drug deferiprone (a fact which is not disclosed). We believe that peer-reviewed, published clinical trial data and health authority reports provide far more reliable information. The article lacks robust epidemiological methodology, as shown in Figure 1 [1]. Four mortality data sets are drawn upon to allegedly demonstrate a progressive increase in the number of fatalities among patients treated with deferasirox. The author compares 1-year data with cumulative data obtained since deferasirox FDA approval, from different and highly unreliable sources. Several of the cited references do not report the data referenced. The author also wrongly cites an EMA ‘warning’ ‘issued for increasing the dose from 30 to 40 mg/kg per day’. The actual EMA Report approves the 40 mg/kg/day dose and states that ‘increased risk of renal adverse events with Exjade doses above 30 mg/kg cannot be excluded’. Deferasirox has been approved in 117 countries. Since first approval in 2005, over 5900 patients have been enrolled in trials with up to 5 years of follow-up, and there have been over 150,000 patient-years of exposure. The author’s claim that reporting of ‘mortality and morbidity incidence [...] is scarce’ and suggestion that Novartis would ‘promote only positive aspects of the drug but downplay