Last weekend we announced on our website the changes that Roche were making to their compassion access program in light of Covid-19 challenges. While this was a welcome move we also published a statement questioning the decision not to go further with access. Following that statement we held a call with Roche and their UK Managing Director: We wanted to provide you a summary of that conversation.

The conversation was positive and one that everybody felt was extremely useful. Richard Erwin, MD of Roche UK, disagreed with our assertion that this was a financially/business driven decision to only expand to those who are having difficulty continuing with Spinraza treatment, but also agreed that we as a Patient Advocacy Group are here to hold Roche to account when it comes to decision-making. He has acknowledged that while Roche could defend themselves from this assertion it is actions that speak louder than words. Roche remains completely committed to getting risdiplam out to the community as soon as possible and confirmed that they continue to have a team working on a submission to the MHRA for an Early Access to Medicine Scheme (EAMS) as the highest priority for this project.

The other key topic of discussion was around the breadth of access that will be provided as part of the EAMS. Roche did confirm that at this time they will not submit a request for access to type 3 patients due to Type 3 patients not being part of the global pre-approval access / compassionate use programme.

With that said, Roche confirmed that they will be submitting data to the European Medicines Agency (EMA) in the summer and aim to bring risdiplam to all patient groups, including Type 3 patients, as quickly as possible.. TreatSMA pushed extremely hard on this point and made it very clear of the importance of treating all SMA groups, and we genuinely believe that Roche are doing everything they can to deliver on their promise.

In conclusion, everything discussed was positive and all points raised were addressed to our satisfaction. We have a much clearer picture now in terms of the process Roche is following and we believe that they remain committed to delivering as soon as regulatorily possible.

We are continuing the dialogue with Roche at all levels, and while we are confident in their intentions, as Richard himself said, actions speak louder than words.