Delivering Hope: How Leicester’s Early Access Programme Became a Lifeline for Duchenne Patients
Emily and Alex took to the road to meet the team at Leicester Royal Infirmary to find out how they were able to be the first site to set up the Early Access Programme (EAP) for givinostat.
They spoke to Dr Dhinesh Baskaran about the creative approach he took to getting the EAP up and running.
And they met the delightful Felix and his mum Charlotte who fought to get the EAP started
An interview with Dr Dhinesh Baskaran, Consultant in Paediatric Neurology at Leicester Royal Infirmary
Q: Dr Baskaran, when did you start running the clinic at Leicester Royal Infirmary?
A: I began in October 2022 as a Consultant Paediatric Neurologist. Since then, we’ve been committed to ensuring our patients with Duchenne muscular dystrophy (DMD) get access to the most promising treatments as early as possible.
Q: Leicester became the first hospital to deliver an EAP for a promising new Duchenne drug. How did that come about?
A: To be honest, we didn’t set out to be the first. We learned about the drug through the NorthStar network and followed the research closely. Once the Medicines and Healthcare products Regulatory Agency (MHRA) approved it in December 2023, and the company offered it for free under the EAP, we knew we had a responsibility to act. The evidence was promising, and we wanted to offer this hope to our patients. As such, we have made a local agreement to pilot its use for a small number of patients who are already under our service.
Q: Was it a straightforward decision to go ahead?
A: Not entirely. We needed to consider whether we had the resources to deliver it safely. Our Chief Executive, Richard Mitchell, was very supportive, which made a huge difference. But we still had to take it through our local pharmacy committee to ensure it was safe and feasible within our existing policies and capacity. Thankfully, they approved it for a small, well-monitored group of existing UHL patients.
Q: What challenges did you face with logistics and resources?
A: We had to think creatively. Everything—from prescriptions to blood tests—requires coordination. We limited the initial rollout to between five and ten patients, which helped us manage resources safely, and unfortunately, we do not have the resources to safely extend the pilot scheme to any additional patients at present. The drug monitoring involves weekly blood tests initially, and we’ve had to keep a close eye on parameters like platelet counts and triglycerides, which can fluctuate significantly.
Q: What made your approach to delivering this treatment unique?
A: We put patients at the centre of every decision. Rather than treat this as a routine rollout, we treated it like a pilot, focusing on safety, logistics, and clear communication. For instance, instead of seeing each family individually for consent, we hosted a group session, where I gave a full presentation and answered questions. Families left informed and empowered.
Q: How have the families responded?
A: The response has been overwhelmingly positive. Many families have expressed deep gratitude. Charlotte, one of the parents, was especially thankful. It’s humbling. This has been a joint effort—from clinicians to pharmacists, nurses to our Chief Executive — and we’re all just trying to do our best for these children.
Q: How many patients have you dosed so far?
A: We’ve dosed five patients and have four more lined up. The monitoring continues, and we’re cautiously optimistic.
Q: What happens if the National Institute for Health and Care Excellence (NICE) doesn’t approve the treatment long-term?
A: The pharmaceutical company has committed to continuing supply free of charge as long as it’s helping and NICE hasn’t rejected it. However, if NHS funding is ever required and not granted, we wouldn’t be able to continue under current trust policy. All parents have been made aware of this, and it’s included in the consent form.
Q: Any final thoughts?
A: This is about doing what’s right for our patients. We’ve taken every step carefully, ensuring safety and transparency. I know other centres are working toward the same goal, and I hope this model can help inspire further access for Duchenne patients across the UK.