The investigators note that almost all of these medications were approved by the European Medicines Agency for use throughout Europe.
“Thus our results also reflect the outcome of European drug development processes and policies,” they write.
They suggest that the situation has come about because regulators still allow placebo-controlled studies even though health technology assessment bodies have long recommended active controlled trials, which provide more useful information.
Of the 125 drugs lacking data on added benefit in the analysis, 64 had no studies vs an active comparator.
For another 42 drugs, studies compared the drug with an active comparator — but the comparator was inappropriate for such reasons as off-label drug use or inappropriate dosing regimens. The remaining 19 new drugs were tested against an appropriate comparator (standard care) but did not show an advantage.
The investigators acknowledge the argument that drugs can be approved without showing “added benefit” data to allow speedy access to new products, with the promise of future comparative studies. However, they note, such promises are often never fulfilled.
“A critical and well known problem with post-marketing studies is they often do not happen .... Globally, regulators do little to sanction non-compliant companies,” the researchers write.
“High levels of uncertainty about treatment benefit jeopardize quality care and impede decision making, particularly on highly priced drugs in economically strained situations,” they add.