Improving antidote development: A business perspective

As clinical toxicologists we must accept that we have a dismal track record in bringing effective antidotes to market. In the past 40 years only a handful of new drugs have been approved in the US (DigiBind, CroFab, DigiFab, Antizol, NAC), with others available only on a compassionate use on named p...

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Bibliographic Details
Published in:Clinical toxicology (Philadelphia, Pa.) Pa.), 2005-05, Vol.43 (5), p.434-434
Main Author: Heath, A J
Format: Article
Language:English
Online Access:Get full text
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Summary:As clinical toxicologists we must accept that we have a dismal track record in bringing effective antidotes to market. In the past 40 years only a handful of new drugs have been approved in the US (DigiBind, CroFab, DigiFab, Antizol, NAC), with others available only on a compassionate use on named patient basis (coral snake antivenin, CPG-2, anti-scorpion antibodies). In Europe, the picture is similar. Although proven, safe and effective technologies exist (specific polyclonal antibodies; recombinant enzymes) few drugs are being developed. The process is long, and often fails although the drug itself maybe clearly effective. Why is this so? This presentation attempts to identify where we have gone wrong, and what we can do better. Key to any improvement is an appreciation of the role and motives of the three stakeholders - the investigator (the clinical toxicologist), the sponsor (usually a pharmaceutical company) and the regulator (national authority/EMEA). For the clinical toxicologist, regulatory trials present multiple hurdles. A scarcity of patients can make obtaining an adequate sample size highly unpredictable, and lack of a placebo control makes the burden of proof much harder. For the sponsor, a rare condition may make the cost of pursuing the project just too high when compared to other opportunities. For the evidence-based regulator, the compromises necessary for burden of proof as regards both efficacy (usually involving surrogate endpoints) and safety (size of material studied) maybe just be too uncomfortable when compared with the standards by which regulatory approval in controlled trials are justified. A better understanding of the ethical, political, and financial conflicts arising from the often opposing interests of these three stakeholders is essential if we are to improve our track record and collectively get effective antidotes more quickly to our patients.
ISSN:1556-3650