The benefit assessment of orphan drugs according to AMNOG basically follows the same process as the evaluation of classic patented drugs. Following the European approval, orphan drugs can be placed on the German market without restriction. A benefit assessment based on the AMNOG dossier submitted takes place within 6 months, followed by price negotiations with the GKV Spitzenverband, which in turn take 6 months. After 12 months, the new, negotiated reimbursement amount applies. The principle of free pricing at market entry equally applies to orphan drugs.
Additionally, orphan drugs have an exceptional position in the German benefit assessment. This is derived from the approval, as orphan drug status is also associated with a (fictional) additional benefit for patients. As orphan drugs have already an additional benefit with the approval, only the extent of the additional benefit is assessed in the early benefit assessment. The Joint Federal Committee (G-BA) assesses the extent of the additional benefit directly, IQWiG assesses only the number of patients and the associated costs according to Module 3 of the AMNOG dossier.
Unlike classical drugs, orphan drugs do not require an appropriate comparative therapy in order to determine the degree of added value. The study data relevant for the approval are used for the benefit assessment. In the case of rare diseases there is often no appropriate comparative therapy in the approved indication.
However, there are exceptions to the exception: if the sales volumes of such a drug exceeds 50 million euros within a year in the outpatient sector, a classical benefit assessment is initiated. This assessment is based on the criteria for innovative drugs, especially in comparison to an appropriate comparative therapy. With a sales volume of more than 50 million euros, a new benefit assessment is therefore carried out, which also leads to new price negotiations.
The degree of added value for orphan drugs is assessed by the categories significant, significant, low and non-quantifiable. The majority of orphan drugs were classified as „non-quantifiable“ in the past. This is ultimately an expression of insufficient data for describing the extent of added value in many cases according to evidence-based medicine criteria.