Consistently continue on the right path for children's medicines

22.12.2014 17:12

In the first procedure for early benefit assessment for an approved children's medicine (Pediatric Use Marketing Authorization - PUMA), the G-BA should consistently follow the right path taken by IQWIG when making its decision. In its assessment, the IQWIG awarded the drug Hemangiol a significant additional benefit, but this only in one of three patient groups. In two others it was questioned whether the appropriate comparator therapy had been implemented correctly. "The IQWiG comes to the conclusion that the appropriate comparative therapy was interpreted correctly for one of the patient groups, but it could have been done differently for the other patient groups. And this is used as a reason not to attribute any additional benefit to them. The transfer of the results to both The company has given good reasons for further subpopulations. This was also discussed with the EMA when it was approved and received their approval. Therefore, the G-BA should recognize the additional benefit for the entire patient group," says Henning Fahrenkamp, General Manager of the BPI.

In the opinion of the BPI, this is particularly true because a PUMA drug generally represents an additional benefit in itself. For the first time, children can be treated with a medicine that, based on study results, is specifically approved for children. The G-BA's decision on this drug also has a signaling effect for other PUMAs. In the present case there was an outstandingly good level of evidence. This is also due to the fact that the PUMA indication is not identical to the approved adult indications. Independent studies were therefore carried out. However, even with this outstandingly good evidence base, the IQWIG did not recognize the best available evidence in two subpopulations. It should be clear that the specific nature of the evidence situation for PUMA must be taken into account. “It is the task of the G-BA to ensure an adequate assessment of children's medicines beyond this special case. The special nature of research with children must be taken into account. It must be taken into account that clinical trials involving minors are limited to a minimum for ethical reasons alone. That's why PUMA also uses evidence outside of RCTs in a very special way for approval. This must also be taken into account in the social law assessment process,” says Fahrenkamp.