A quiet success story: How the Robert and Agnes Hunt Orthopaedic Hospital rolled out givinostat to patients across Shropshire and beyond
We visited Oswestry to meet the dedicated team at the Robert and Agnes Hunt Orthopaedic Hospital (RJAH), which has been delivering givinostat to patients since the end of May. The team has now dosed eight boys and continues to rollout out the treatment.
We spoke to the team at RJAH, which is headed by Professor Tracey Willis, about their approach and how they were able to get the Early Access Programme (EAP) up and running so quickly. It was one of the first hospitals to do so.
We also had the pleasure of meeting 11-year-old Ruben, who lives with Duchenne muscular dystrophy (DMD) and was one of the first patients to receive the medicine at RJAH, along with his lovely family.
Interview with the team at RJAH, Professor Tracey Willis (Consultant Paediatric Neurologist with a Specialty Interest in Neuromuscular Disorders), Dr Richa Kulshrestha (Consultant in Paediatric Neurodisability and Neuromuscular Disorders), Natalie Grigg (Neuromuscular Clinical Nurse Specialist and Research Nurse) and Connor Wainwright (Senior Pharmacist for Homecare and High Cost Medicine).
Q: Professor Willis, you’ve been delivering givinostat for eligible patients through the EAP since the end of May. How many patients have received it so far?
A: We now have eight patients being treated with givinostat through the EAP and we are continuing to roll it out to eligible patients.
Q: That’s fantastic, RJAH was one of the first hospitals to make givinostat available through the EAP how did your team make this happen?
A: Following the successful clinical trial of givinostat, which we are proud to be a part of, we were aware of the EAP before it opened, so as soon as it became operational in the UK, we were ready to get going.
Givinostat was quickly approved by our Drugs and Therapeutics Committee with no issues and having been one of the clinical trial sites, we were familiar with the medicine, so we were confident in making it available to our patients and understood what was required in terms of monitoring.
We had pre-briefed eligible patients during their routine clinic appointments so that they were aware givinostat and the process for administering it, when it became available.
We sent out the necessary consent forms and had a Zoom meeting with families to ensure they had all the information they needed. We consented families in groups of four at a time to make things as quick and efficient as possible.
Within just a few weeks, we were able to dose seven patients, whilst most tertiary hospitals were still in the process of organising a service delivery plan for patients. The ‘can do’ attitude of the team, supported by management and an excellent pharmacy team has been key to enabling us to deliver this.
Q: Are there specific things you put in place to make this possible and who has been involved in the process?
A: Our specialist nurse, Natalie Grigg, led on the administration side of things for the EAP keeping in touch with families and organising the necessary follow up appointments and monitoring required to ensure patient safety, including bloods and ECGs. There is a lot to keep track of, for example, the timing of follow up appointments might need to be altered, if there any dose adjustments required. Natalie has been fantastic at making sure we are on top of all of this, with everything tracked in a spreadsheet that can be easily updated to ensure patients are being seen at the right time and monitored appropriately.
Our pharmacy team under Connor Wainwright’s leadership have also worked hard to set up the service and were very supportive from the outset.
Having a multi-disciplinary team all working together has been crucial in delivering givinostat through the EAP. Everyone has played their part and wanted to make it happen to ensure we are delivering the latest and very best care possible to our patients at RJAH.
Q: Connor, as the senior pharmacist who led on this, can you tell us about your experience?
A: Setting up a givinostat service has been such an exciting prospect for pharmacy. In the end it was a relatively straightforward process, and it has been great to be part of the multi-disciplinary team and helping provide access to treatment.
Q: Natalie, RJAH cares for patients across a large geographical area. How have you managed the logistical challenges posed by that in terms of monitoring these patients?
A: We are working closely with patients’ local NHS trusts and appreciate their support in patient monitoring to minimise disruption to the families and enable their continued treatment.
In some cases, patients have still had to travel to us here in Oswestry for bloods if there isn’t the necessary capacity locally, but where possible we have tried to facilitate this for them as close to home as possible. As we care for patients from many different areas, we had already built strong relationships with local Trusts and teams across the region. Those relationships have been a real asset to us in making givinostat available to our patients.
Q: Is there anything else you have had to consider in terms of prescribing givinostat for your patients?
A: In some cases, we’ve had to prescribe earlier than when the next prescription is due to be filled to ensure patients have the supply they need and prevent any issues arising. This has particularly been the case for those who live some distance away or if families have a holiday planned, but everything has gone smoothly so far. We have had no issues with supply or in dealing with the pharmaceutical company.
Q: Professor Willis do you have any final thoughts to add?
A: There are a number of things to consider in making this treatment available, but the monitoring involved, including ECGs and full blood counts are relatively routine and not too resource intensive, so with the right process in place it is deliverable.
The main challenges in making givinostat available to our cohort of patients at RJAH, were logistical ones and we were able to overcome them by working closely with colleagues in other areas and taking a multidisciplinary approach.
For us, this process has been about working to make everything as manageable for families as possible so we can ensure equity of access and that no one is left behind.
Q: Dr Kulshrestha, any final thoughts on this or anything you would like to add?
A: As a clinician this is a positive phase of Duchenne care, that brings motivation to excel the service. We are very happy to be a part of delivering this important new medicine and working to ensure that our service is providing the best care possible.
Q: Poppy, it’s lovely to meet you. You are Ruben’s mum, what does it mean to you and your family for Ruben to be able to access this medicine?
A: As a parent I feel extremely grateful that Ruben has been given the opportunity to start givinostat. It has filled me with hope for the future knowing that it will slow the progression of DMD down.
Ruben will be able to continue doing the things he loves for longer such as playing football with his friends and being creative.
It also gives more time for research which fills me with hope that the future for our DMD community will be bright.