Repurposed Drugs Launched Under the AMNOG in Germany

Drug repurposing, the use of established drugs in new, previously unrelated indications, offers many potential advantages, such as lower development costs and higher success probabilities in clinical trials, which is thought to be attractive especially for orphan indications. This analysis was conducted to assess the success of repurposed drugs (RD) in German HTA procedures and subsequent price negotiations based on AMNOG.

A drug was defined as repurposed if it was launched under the AMNOG in more than one therapeutic area as defined by the German Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA). Utilizing a database, containing information on dossier content, benefit assessment, G-BA resolution and negotiated rebate of all AMNOG procedures ever performed, we compared procedure outcomes of RDs to those approved in more than one indication in a single therapeutic area.

Eighteen of 494 active substances (~5 %) which passed through AMNOG were identified to be approved in more than one therapeutic area. The most common therapeutic areas were diseases of the musculoskeletal system followed by dermatology, neurology, respiratory tract diseases and gastroenterology. The most frequent combination of therapeutic areas was diseases of the musculoskeletal system together with dermatologic indications for six active substances. Although on average RDs didn't receive a higher added benefit in the G-BA resolution compared to those approved in only one therapeutic area, the current negotiated gross box rebate was lower (20% vs. 33%) for RDs.

The lower negotiated rebate for RDs cannot solely be explained by the extent of the granted added benefit. Hence, in addition to the launch sequence for RDs, the same applies as for other drugs: Besides the granted added benefit, the result of the price negotiation also depends on a convincing value story, (sub-)population sizes, costs of the appropriate comparative therapy and a harmonized strategy for pricing throughout the complete AMNOG process.