Translarna/ataluren is used to treat Duchenne muscular dystrophy that is caused by nonsense mutations.
Translarna is currently accessed in England on the NHS under what is known as a Managed Access Agreement (MAA). These agreements between NHS England, NICE, and a drug manufacturer allows patients to receive treatments with promising potential while further data is gathered to strengthen the clinical evidence.
On 13 May 2021 NICE announced that the Translarna MAA, which began in 2016, is to be extended from its original end date of July 2021 to January 2023.
As patient representatives on the Managed Access Oversight Committee, MDUK and Action Duchenne are working with NICE to relay questions from the Duchenne community to NICE and to publish the answers that they provide in response.
This Q&A will be regularly updated. If you would like to add a question to the Q&A please email info@musculardystrophyuk.org. MDUK will share all questions with Action Duchenne and we will jointly address them with NICE. If you have any other questions, you can email Neil on neil@actionduchenne.org.
Last updated 19 May 2021
Q: What is Translarna/ataluren and who could benefit from it?
In August 2014, ataluren (Translarna) became the first drug to be given a conditional approval, by the European Medicines Agency (EMA), to treat an underlying genetic cause of Duchenne muscular dystrophy.
Ataluren has been developed to target a particular genetic mutation, called a ’nonsense mutation’ that causes 10-15 percent of cases of the condition. Ataluren is the first drug that targets an underlying genetic cause of Duchenne muscular dystrophy to be recommended for use by the NHS in England.
To start taking ataluren, children must be:
- amenable by genetic mutation
- aged two years
- and over able to walk 10 steps unaided
Q: Why does NICE make recommendations for managed access?
NICE committees have a difficult job to ensure that decisions about new NHS-funded treatments are based on evidence that shows that patients experience the benefits claimed.
These decisions take account of a wider range of factors and sometimes the evidence available is limited, or it only relates to some patients with a condition and not others, or important information about clinical benefits is missing. NICE committees can usually only make a positive or a negative recommendation.
However, for treatments with promising potential but with limited evidence, NICE committees can make a recommendation for managed access, if they think the clinical uncertainties can be addressed through collection of further data to strengthen the clinical evidence.
This means that NHS England and the drug manufacturer are asked to agree how patients could have access to treatment through the NHS and how treatment is funded, while NICE works with the company, clinicians and patients to make sure the data needed is able to be collected.
If all of this can be agreed, patients are able to access treatment for a time limited period while further evidence is collected. While most topics recommended for managed access go on to be recommended for routine use on the NHS, there is no guarantee that it will be recommended when it is reviewed by NICE.
Q: What is a Managed Access Agreement?
NICE makes recommendations on new medicines by reviewing clinical and cost effectiveness evidence. When a medicine shows promising potential but there are gaps in the clinical evidence, it may be recommended for time limited NHS access in England as part of a MAA.
This is a way that doctors and the NHS can assess the long-term benefits of a new medicine by collecting agreed test results over a given period of time in patients who have certain symptoms of a condition.
At the end of the MAA period, NICE will review the new evidence to make a final recommendation as to whether the medicine will be available to access via the NHS in the long term.
Q: What does it mean for a treatment to be available through a Managed Access Agreement?
When a treatment is available through a managed access agreement:
- NICE and clinical experts and the drug manufacturer will agree the terms for patients to access treatment for a time-limited period. This is usually based on the evidence presented to NICE about which patients receive a benefit, and those who do not. Commercial terms are agreed between NHS England and the manufacturer, based on the cost-effectiveness evidence presented to NICE.
- Data about the benefits for each patient (and often the benefits and impacts for their family too) will be collected to inform a review by NICE of the benefits of the treatment.
Q: If NICE agreed ataluren showed functional benefit, why wasn’t it just approved for use on the NHS?
- NICE’s committees are required to assess the clinical benefits for patients and balance this against the cost of the treatment. Ataluren has been assessed as showing some promise of functional benefit to patients in a small study. The NICE committee has asked to see evidence to confirm how long patients experience benefits from treatment and how big this benefit is relative to patients not on treatment.
- With new information on these issues the NICE committee feels it will be able to make a final decision about whether ataluren should be available on the NHS, based on whether the cost of the benefits is deemed a good use of NHS resources.
Q: Why is the managed access agreement being extended? Why is ataluren not being re-evaluated now for routine use?
The COVID-19 pandemic meant that the clinical visits required to monitor patients had to switch to home care and remote monitoring. During this period patients continued to receive treatment but some of the data collection was suspended.
Additionally, NICE and NHS England wanted to ensure PTC Therapeutics (the drug manufacturer) has access to more complete data on patients receive ataluren in preparation for their resubmission to NICE before the end of the MAA.
NICE, NHS England and NHS Improvement and PTC Therapeutics (the Company) have reached an agreement that will extend the Managed Access Agreement (MAA) for Translarna (ataluren) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene.
This agreement extends the MAA, that was originally scheduled to end in July 2021, to January 2023. This will enable PTC Therapeutics to access more complete data in preparation for their resubmission to NICE and the re-evaluation of ataluren before the end of the MAA, the outcome of which will determine how ataluren will be commissioned in the long term.
Q: Can newly diagnosed patients start treatment during the period of the extension to the managed access agreement?
For the duration of the managed access agreement extension (up to January 2023) newly diagnosed patients will be able to start treatment with ataluren.
Q: How do I know if my child is eligible for the treatment and how can I access the MAA?
Your child’s clinician will consider whether or not your child will benefit from treatment with ataluren. Please see the published MAA for further details on the clinical eligibility criteria for treatment access.
Q: What can patients do to support the re-evaluation?
Patients receiving treatment with ataluren will be asked to attend face to face appointments at their treatment centre. During these visits further data on the functional benefits of treatment will be collected and you and your family will be asked to answer questions about the impact of treatment on your daily life.
By attending these appointments and providing this data you will be enabling the collection of the most complete data possible, which will be used in the final decision about whether ataluren should be routinely available on the NHS.
Please speak to your treating clinician and their team for information about measures in place to protect you and your family from COVID-19 when attending face to face appointments. We appreciate that every family will have to weigh up how comfortable they feel about attending face to face appointments, but we hope you might feel able to attend so your data can be included in this evaluation.
Q: Will the community be kept informed of how things are developing between now and January 2023?
Action Duchenne and Muscular Dystrophy UK will work with NICE to provide progress updates to the community throughout the extended data collection period and the NICE evaluation.
We will also add further FAQs in due course to this list to provide a central reference for all your questions and to show how both organisations are representing the interests of patients and working with NICE throughout this period.
Q: Why are the evaluation processes in England and Scotland separate?
Scotland and England have parallel decision-making bodies who review whether new treatments should be available on the NHS – the Scottish Medicines Consortium and NICE. This is because the NHS in Scotland is independent of the NHS in England.
While both the SMC and NICE aim to time our decision-making processes to coincide, sometimes one country is ready to make a decision sooner and will proceed with an announcement to provide patients with certainty about what is likely to happen.
As far as possible, both organisations aim to ensure patients in all parts of the UK have broadly similar access to the same treatments.
Q: Can patients in Wales and Northern Ireland access ataluren?
While Wales and Northern Ireland typically allow access for patients who would be eligible under the terms of the managed access agreement, NICE is following up with colleagues in both countries to confirm that they will be able to extend the terms for access to ataluren. We aim to provide an update to this response by 4 June.
Q: When will we know if ataluren has been approved for NHS use beyond January 2023; and if it is will it change the way people access it?
NICE will schedule the evaluation of ataluren so that a final decision is published before January 2023.
Information about the progress of the evaluation will be published on the NICE website at regular intervals, alongside engagement with the patient community.
NICE will work with clinicians and patient groups to consider the impact of NICE’s decision on patients and their carers and provide early notice of any decisions and how any impacts of these decisions might be managed.
Further reading
Update on Translarna NHS treatment for Duchenne muscular dytrophy
An amazing 10 in 10 for Action Duchenne
