DÄ internationalArchive24/2012Analysis Not Appropriate
LNSLNS

The authors of this publication mentioned some 50% placebo controlled pivotal studies for 39 medicinal products that gained approval in 2009–2010, and implied that the pharmaceutical companies shied away from real comparisons between treatments. What I cannot understand in this context is that this criticism was also applied to preparations for which no other option existed but to test them against placebo (in patients who are beyond treatment; no therapeutic standard to date). Pointing this out, the authors excluded medications for rare diseases (orphan drugs) from their study from the outset. However, these reasons apply to more common diseases too, as was shown in a 2009 publication of European drug approvals (1). My own analyses of pivotal studies for 2010 and 2011 confirm this. An appropriate analysis would have therefore differentiated between studies that were placebo controlled out of necessity and those that were not. This would have shown that new medications are almost always tested against standard treatments—if this makes sense medically. Placebo controls are used almost exclusively in a setting where no standard treatment exists—for example, when a drug is tried in patients who are beyond treatment.

DOI: 10.3238/arztebl.2012.0436a

Dr. rer. nat. Siegfried Thorm

Berlin

E.Theimer@vfa.de

1.
Götte D: Arzneimittelmarktneuordnungsgesetz: Bedeutung für die Identifizierung von Zielparametern zum Nachweis klinischer Wirksamkeit innovativer Arzneimittel. Dtsch Med Wochenschrift 2012; 137: 274–80. CrossRef MEDLINE
2.
Ujeyl M, Schlegel C, Walter S, Grundert-Remy U: New drugs: evidence relating to their therapeutic value after introduction to the market. Dtsch Arztebl Int 2012; 109(7): 117–23. VOLLTEXT
1.Götte D: Arzneimittelmarktneuordnungsgesetz: Bedeutung für die Identifizierung von Zielparametern zum Nachweis klinischer Wirksamkeit innovativer Arzneimittel. Dtsch Med Wochenschrift 2012; 137: 274–80. CrossRef MEDLINE
2.Ujeyl M, Schlegel C, Walter S, Grundert-Remy U: New drugs: evidence relating to their therapeutic value after introduction to the market. Dtsch Arztebl Int 2012; 109(7): 117–23. VOLLTEXT