The givinostat EAP – a promising new medication with hurdles to implementation
In this guest blog, an NHS doctor explains the challenges and barriers they've met while trying to take part in the Early Access Programme (EAP) for givinostat.
As part of our Time is Muscle campaign, Duchenne UK has been hearing from doctors about the hurdles they encounter while trying to take part in the EAP – a compassionate use scheme which provides a treatment for Duchenne muscular dystrophy (DMD), called givinostat, for free to the NHS.
At our webinar on 19 December, Dr Adnan Manzur, clinical leader of the expert NorthStar network, gave an update on the EAP – and as part of his presentation discussed some of these hurdles. You can watch that webinar back here.
A lot has changed since December, and during conversations with the NorthStar Network we agreed that it would be helpful for the community to learn more about the barriers and challenges of implementing the EAP. As a result, Dr Sandya Tirupathi (Consultant in Paediatric Neurology at the Royal Belfast Hospital for Children), has kindly written this blog which was reviewed by other NorthStar clinicians before publication.
We hope this will provide some insights into what clinicians are experiencing, and Duchenne UK will be using the points raised by Dr Tirupathi and the NorthStar Network when we discuss with decision-makers how to urgently overcome barriers to the EAP. Time is Muscle, and we want access to givinostat for everyone eligible in the UK with DMD as soon as possible, so they all have the chance to keep their muscles moving for longer.
The givinostat EAP – a promising new medication with hurdles to implementation
By Dr Sandya Tirupathi, Consultant in Paediatric Neurology at the Royal Belfast Hospital for Children
The availability of a promising new medication for Duchenne muscular dystrophy (DMD) is as exciting to physicians as it is to families. Givinostat has recently been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of DMD in boys aged 6 and older.
Givinostat is currently under consideration by the National Institute for Health and Care Excellence (NICE), and NICE guidance is awaited. In the meanwhile, givinostat is being made available via an Early Access Programme (EAP) by its manufacturer.
Compassionate use schemes like the EAP work well for therapies and conditions in which no close monitoring of patients is required, and when no significant side effects are anticipated.
Additional resources
However, when monitoring is a necessity, as in the case of givinostat, clinical centres have a need for additional resources (to be able to carry out the necessary blood tests and hold additional clinics, for example) to undertake this monitoring in a safe and sustainable manner.
Resources for additional clinical monitoring are not usually provided by NHS Trusts, Regions or Health Boards (as applicable to different UK countries) unless a therapy is NICE-approved. This is not currently the case for givinostat.
Blood tests to monitor
Givinostat treatment, under the EAP, requires close monitoring. This includes blood tests every 2 weeks for the first 8 weeks of treatment, followed by additional blood tests at 12 weeks and every 3 months thereafter. These tests are used to look for the presence of significant side effects, such as low platelet count (which can lead to bleeding tendencies), high triglyceride levels in the blood, and gastrointestinal side effects like diarrhoea.
If these tests show any abnormality, the test must be repeated within a week and the givinostat dose may be adjusted.
Additional nurse support
Each neuromuscular centre taking part in the EAP will have a substantial number of patients eligible for givinostat, meaning that it is important that there are sufficient resources in place to ensure smooth supply and good monitoring.
This means that each participating centre will have to secure additional nurse specialist time and clinician time – initially to ensure that inclusion and exclusion criteria for the EAP are discussed with families, that families provide informed consent, that givinostat is prescribed as necessary, and that exit strategies are discussed; and subsequently to monitor blood test results every two weeks, supply necessary prescriptions and make changes to givinostat treatment regimes in the event of adverse effects.
Pharmacy resource
In addition, pharmacy resources are required to ensure the safe and appropriate monitoring of treatment, as well as the procurement, storage and dispensing of treatment continuing after commencement.
Phlebotomy services
Clinical centres will also need good phlebotomy services with sufficient capacity, a suitable physiotherapy service to objectively measure the functional effects of givinostat treatment, and psychological services if there is needle phobia.
At present, many neuromuscular teams have little spare capacity to undertake this work, meaning that solutions will need to be found if the programme is to be delivered fully.
Ongoing discussions about resource
Clinicians UK-wide are in discussions with their local Trusts and Health Boards, to understand whether and how additional resources can be put in place, to facilitate implementation of the EAP.
In addition, as there is no national process in place for programmes like the EAP, neuromuscular centres must apply individually to their local Trust/Region/Health Board’s drug and therapeutics committee (DTC), or local equivalent, for permission to take part in the EAP. These applications require significant clinician time.
These conversations, around permissions and resources, are in progress, but together they are delaying individual neuromuscular centres’ ability to begin givinostat treatment.
Solutions to these issues are actively being sought by clinicians and NHS services, so that givinostat treatment can be introduced safely, and as quickly and efficiently as possible.