Do registers for orphan drugs become mandatory in Germany soon?

Thu, 2017 / 10 / 12

58 orphan drugs (drugs treating rare diseases) have been evaluated in the German early benefit assessment to date since AMNOG was introduced in 2011. To those drugs an additional benefit is automatically attributed by law irrespective of the present evidence, as long as their annual turnover rate does not exceed a threshold of 50 million euros at the expense of the statutory health insurance. For orphans, the IQWiG only assesses patient numbers and annual therapy costs, but not does not perform the methodical evaluation of the additional benefit. If sales exceed the annual limit of EUR 50 million, the active substances are re-examined in a full evaluation procedure.

This approach, is sometimes seen as a "free ticket”, is being increasingly questioned by the chairman of the Joint Federal Committee (G-BA), Prof. Josef Hecken. While in the first two years, only three or five active substances had harnessed the benefits of this legal regulation for drugs treating rare diseases, there were 19 in the past year alone. In more than 50% of the previous evaluation procedures involving orphan drugs, the G-BA has opted for a non-quantifiable additional benefit due to the uncertain evidence. In two cases, the G-BA has even determined no additional benefit in the re-evaluation after exceeding the sales limit. The quality of the clinical data for orphan drugs is getting worse, according to Josef Hecken. In an article of Ärzte Zeitung online, Prof. Hecken states: "We have more and more orphans for which the evidence at the time of the evaluation by the Joint Federal Committee is close to zero. Over the past years, we reached the point where we only have evidence in the range of 10 to 15 percent. "

In addition, annual therapy costs for orphan drugs often exceed, by far, costs of normal drugs and increase constantly. According to the medical newspaper, these now range between 200,000 and 800,000 euros. Therefore, the G-BA calls for measures that allow to deal with this often missing evidence. Already now, more and more decisions regarding the benefit assessment are made for a limited period of time and are linked to conditions such as downstream studies or mandatory registries. However, implementation by pharmaceutical companies is not always easy, since those conditions are not legally binding, as the specialized consulting agency SKC Beratungsgesellschaft has experienced.

The establishment of registries to generate further evidence is not only difficult from SKC’s point of view for several reasons: On the one hand, the non-interventional character of a registry prevents a systematic comparable survey of patient-relevant endpoints. On the other hand, medical decision makers are not always willing to include patients in already established registries. Reasons for this are diverse, but the focus is certainly on the temporal burden, as the time-consuming complete data collection is impossible to implement in clinical practice.

Here, Josef Hecken calls for an obligation to include patients in registries. He does not see an alternative to "forcing the doctors to report the patients to the registry so we can get an idea, which tendencies the treatment has". In his opinion, "this is a demand after election of the new German parliament (Bundestag)".
For most pharmaceutical companies, the creation of a registry represents a totally new territory and therefore, a challenge. The specialized consulting agency SKC Beratungsgesellschaft has already been involved in numerous benefit assessments for the establishment of registries and is thus familiar with the arising challenges and strategies as well as with the views of the decision-makers.