German early benefit assessments of orphan drugs under full dossier conditions

In Germany, market access provides several advantages for orphan drugs in the course of the early benefit assessment, since the additional benefit is already granted by law. However, this simplification only applies up to an annual turnover of 50 million euros. Medicines that exceed this threshold have to demonstrate their additional benefit again but in comparison to an appropriate comparative therapy, taking a higher level of evidence into account.

SKC has analyzed the already completed procedures that have exceeded the threshold of 50 million euros in annual sales in terms of available evidence (1), appropriate comparative therapy (2), G-BA (Federal Joint Committee) methodology (3) and negotiated prices (4). Most of these procedures referred to oncological drugs (Jakavi®, Imnovid®, Imbruvica®, Darzalex®, Kyprolis®), only one of the six (Opsumit®) referred to a cardiological drug. Besides the high annual therapy costs of these drugs, the overweight of oncology drugs is also triggered by label extensions.

All but one oncology drug were able to confirm the previously granted additional benefit, the cardiological one failed to prove an additional benefit. Kyprolis® and Jakavi® were even able to prove a higher benefit category within the second procedure. In most of the procedures the considerable additional benefit was based on prolonging overall survival. Based on the benefit assessment, a second price negotiation takes place for each product. The analyzes show that all but one drug negotiate an even higher rebate in the second procedure. This subsequently negotiated rebate demonstrates the major challenges of a reassessment under non-orphan dossier conditions. But it is necessary to await further reassessments of rare and high-prices drugs outside oncology to be able to draw further conclusions, especially in term of consequences for the market potential and sales expectations of these drugs.

SKC consulting has many years of experience in the field of market access for innovative active substances. As specialists for orphan drugs, we can give you strategic advice on your pricing rationale, launch sequence or other process-related challenges.

BY Prof. Matthias P. Schönermark, M.D., Ph.D., managing director and Thora Mrosowsky, M. Sc. Health Economics