"The Misperception That Clinical Trial Data Reflect Long-term Drug Safety" is the title of a commentary in this week's Archives of Dermatology.
The authors focused on one particular drug - Raptiva (efalizumab) for psoriasis - and the fact that the European Medicines Agency (EMEA) has recommended the suspension of the marketing authorization for it after concluding that its benefits no longer outweigh its risks. But their conclusion carries a much broader message than just about this one drug:
There is the misperception by the public and many physicians that drugs are "safe and effective" as is often concluded in (approval) randomized clinical trials (RCTs). This phrase suggests, without equivocation or nuance, that drugs work and do not have (serious) adverse effects. The real meaning of this phrase is that a particular drug--for the approved indication, in the approved patient population, and at the approved dosing schedule and administration--has an efficacy profile that warrants being marketed despite its known toxic effects. Or more concisely, at time of approval, the known benefits outweigh the known safety risks. To fully appreciate the benefits of a drug, it should be compared with existing standard therapies and not merely show superior benefit compared with placebo. How a new drug will act and how patients in more complex real-life settings than in those eligible for RCTs (eg, healthy patients) will react is not known and needs to be robustly monitored in postmarketing safety studies that include a large heterogeneous patient population for at least 5 years and are performed swiftly after drug approval so that they may have an impact on clinical care.
Yet, as we've seen, the track record for swift postmarketing safety studies is not good.