Following aHUS India’s response came two replies from The Netherlands and UK in Europe. Their expectations reflect a very different position on the timeline of access to eculizumab i.e. if or when it is no longer needed how safe is it to withdraw treatment.
The European Medicines Agency approved a licence for eculizumab for both PNH and aHUS on the same basis that treatment was life. The two diseases are different PNH is a benign “cancer” of the Bone Marrow and aHUS is a Complement Disorder.
Three years European aHUS researchers approached an EU funding agency to support a pan European study on whether, and when, eculizumab could be withdrawn safely. Funding was declined on the rather politically incorrect basis that it would have only benefited a few people ( the kind of thinking that Rare Disease patients face time and time again)
Nevertheless researchers from individual nations progressed alone. The Netherlands and UK had to because such studies were a condition for universal access set by the health policy decision making authorities in both countries.
These are their expectations for 2018.
The Netherlands expectations from 2018- aHUS Netherlands
The UK’s expectations for 2018- Answers for aHUS.
As the National Specialised Service for aHUS ,overseen by an expert group in The National Renal Complement Treatment Centre in Newcastle upon Tyne, reaches the end of its second year of operation, there is an expectation that fewer aHUS patients are being misdiagnosed and more will be accessing eculizumab sooner to avoid anyone going into chronic kidney failure. Also that those still awaiting a transplant supported by eculizumab will soon benefit.
At the other end of the spectrum the UK’s Research Study of the Stopping Eculizumab Treatment Safely (SETS) is expected to begin in the Spring and continue until 2020. Focused on a safe care pathway, and properly monitored the Study will establish a risk management approach to the withdrawal decision. Participants in the Study will have the opportunity for their views on the experience to be recorded in a parallel qualitative study of quality of health issues.