Relatives and doctors were delighted when the Danish Medicines Council recently recommended the drug Hemgenix for patients with severe and moderately severe haemophilia B. Thanks to this new drug, patients can look forward to replacing lifelong treatment with a new gene therapy they only have to do once.
The recommendation from the Danish Medicines Council was based on the negotiated agreement between the pharmaceutical company CSL Behring and Amgros, which states that the Regions only have to pay if the gene therapy works in the long term.
To follow up on the effect of the gene therapy, we have built a new setup for digital data collection in close collaboration with the Regions, hospital pharmacies and clinics.
Establishing the new setup was hard work. We had to develop a completely new digital platform for the clinics to report on the effectiveness of Hemgenix, and we had to involve actors from the Regions, the Danish Clinical Quality Program – National Clinical Registries (RKKP), CSL Behring, and Amgros.
- We believe that the digital model we've developed will make it easier for clinicians to report the effectiveness of Hemgenix on patients. This knowledge is necessary for us to make sure that we don’t pay for a treatment that doesn’t work, says Flemming Sonne, Amgros CEO.
This is the second time Amgros has entered into an outcome-based agreement. The first agreement was signed in 2020 for the drug Luxturna for patients with a rare hereditary eye disease.
Developing a new setup to follow-up on an outcome-based agreement has been resource-intensive. It has required a lot reflection on how best to do it and how to avoid overburdening clinics with administrative requirements.
- We hope that experience with the new setup will make it easier for us to enter into more outcome-based agreements in the future. However, it’s still important to stress that it must make sense to share the risks associated with the efficacy of a medicine. But we have an impression that our suppliers are very understanding of this, says Flemming Sonne.
RKKP is behind the new digital platform, KIP. However, Amgros has worked closely with many actors in the Regions to develop the best solution so that we can follow-up on the outcome-based agreement.
- I’d like to thank our close partners in the Regions, at RKKP, at hospitals and at hospital pharmacies for helping to develop the new setup. Without their commitment and eagerness to contribute to follow-up of the outcome-based agreement with CSL Behring, we would never have reached our goal, says Flemming Sonne.
Patients are already queuing up for Hemgenix treatment.
The Danish Medicines Council recommended Hemgenix for standard use in public hospitals on 19 June 2024.
Read more: Danish Medicines Council recommends gene therapy treatment Hemgenix after new impact-based price agreement (medicinraadet.dk) (in Danish)
The Danish Clinical Quality Program– National Clinical Registries (RKKP) provides knowledge to improve the quality of the health services.
Read more: We develop knowledge to improve health services - RKKP (in Danish)
Contact
Karen Torgny
Senior Specialist- Communication
I’m responsible for overall communication at Amgros, including PR. And I help to ensure that we communicate with the surrounding world in an open and engaging manner.