ATMPs in Germany - Hemgenix®

👉 The pricing policy of Hemgenix presents a possible option to overcome high upfront costs for one-time therapies.

Hemgenix (active substance: etranacogene dezaparvovec) was authorized in February 2023 for the treatment of haemophilia B. The gene therapy Hemgenix is intended to enable patients to produce the previously insufficient FIX. The pivotal study is the single-arm trial HOPE-B. Hemgenix is available in Germany.

🏆German HTA rating:

No direct comparative data for Hemgenix exists. Accordingly, the German HTA rating in October 2023 resulted in a non-quantifiable additional benefit, only due to the orphan drug status.

Intraindividual comparisons were submitted for the HTA, comparing Hemgenix to FIX therapy with Idelvion. As in the HTA for Roctavian, these comparisons had methodological limitations and were not considered sufficiently valid to be used for HTA.

💵 Neither the launch price nor the reimbursement price were listed so far.

Instead of listing a price for the one-time treatment Hemgenix, the market authorization holder proposes “Tag-EINS” contracts (“day ONE” contracts) to health insurance companies. These contracts are adaptive, annual, P4P-models to avoid high upfront costs for the health insurance companies. The annual payments amount to €390,005.88. This price represents the costs of existing long-term therapies for haemophilia B plus a premium for the additional benefit. In the event of therapy failure or in case a maximum payment period is reached, no further payments are made. Within this model the reimbursement price might also be adjusted to price cuts for long-term therapy, eliminating the risk of uneconomical reimbursement.

📘Special features of reimbursement:

Hemgenix is subject to the ATMP Quality Assurance Guideline and the routine practice data collection. Therefore, it will only be reimbursed if the requirements of both regulations are met.

💡The pricing policy of Hemgenix represents a very innovative approach. The impact of such an approach in the German healthcare system remains to be evaluated.