Client Challenge
Innovative gene therapies can offer patients with serious illnesses an alternative treatment option. Unfortunately, they also pose risks for patients, as the risks, and especially the long-term risks, are unknown, as are the side effects and their causes. However, gene therapies can mean a cure or significant improvement for the patient after just one administration, which is also an enormous relief for doctors and the healthcare system. The pharmaceutical company bears the risk during the development of innovative gene therapies.
Single-dose gene therapies could reduce the costs for the National Association of Statutory Health Insurance Funds (GKV-SV) over a long period (possibly a lifetime). However, for the pharmaceutical company, the costs of research and development must also be recouped, and an appropriate return on investment must be generated. Alternative reimbursement models, such as pay for performance, can achieve a good result in this regard. A successful outcome through the Federal Joint Committee (G-BA) is essential for securing a high reimbursement amount in negotiations with the GKV-SV.
The challenges for pharmaceutical companies entering the German market include the high quality standards for gene therapies mandated by the Federal Joint Committee (G-BA), as well as potential requirements for data collection during application or a registry. These must be met by the pharmaceutical company to ensure that the drug actually reaches the patient.
To ensure successful market access in Germany, the client commissioned SKC Consulting to clarify crucial questions in an early phase through G-BA consulting, in order to subsequently have the opportunity to address these issues up to market access.
- Is the study duration long enough for the present disease?
- Are the endpoints patient-relevant and sufficiently validated for the present disease?
- Is the planned population size in the study (depending on the low prevalence) sufficient to achieve an additional benefit in a direct comparison of the treated patients to the control patients?
- Which is a suitable comparator (relevant if the 50 million euro limit is exceeded)?
Kintiga Approach
To identify and assess all challenges the client faces regarding the AMNOG process, a comprehensive risk assessment is the solution. Following this, a consultation with the G-BA (Federal Joint Committee) clarifies any remaining questions , allowing specific requirements to be considered in study planning and AMNOG process preparation. Crucially, the questions and wording must be chosen strategically to prevent the G-BA from using the consultation as an opportunity to impose further requirements.
Another advantage of an early consultation with the G-BA (Federal Joint Committee) for this client was that the disease, the product, and the pharmaceutical company could be presented to establish a relationship of trust. The pharmaceutical company was able to position itself to the G-BA as an open-minded and reliable company with an effective and innovative product for a disease with a high unmet need for therapy.
Solutions
Kintiga Germany conducted a systematic risk analysis using a comprehensive grid, examining all key aspects of the AMNOG process. We successfully completed the project thanks to its extensive experience and numerous engagements within the AMNOG process.
By taking an external perspective on the product and the company, our team identified and assessed risks and opportunities, such as how the stringent quality requirements for gene therapies might affect the product’s success in the German market, or how to address the uncertainty surrounding the potential establishment of a registry. Study-specific questions were clarified in a consultation with the Federal Joint Committee (G-BA). Thanks in part to specific inquiries from our team, strategically important comments were obtained from the G-BA and documented in writing, thus laying the foundation for successful market entry.
Glossary of terms:
- AI – Artificial Intelligence
- AMNOG – Arzneimittelmarktneuordnungsgesetz (German Pharmaceutical Market Restructuring Act)
- HTA – Health Technology Assessment
- G-BA – Gemeinsamer Bundesausschuss (Federal Joint Committee)
