NICE modulator appraisal

There are different appraisal bodies across the UK.

• England – National Institute for Health & Care Excellence (NICE)
• Wales – All Wales Medicines Strategy Group (AWMSG)
• Northern Ireland – NICE/Department of Health and Social Care (DHSC)
• Scotland – Scottish Medicines Consortium (SMC)

Once a new treatment has a licence, drug appraisal bodies weigh up the price a company has asked for against the clinical trial and other relevant data. They then recommend whether the drug will be clinically- and cost-effective for the NHS, in the context of available budget.

The NHS is legally obliged to fund drugs recommended by these bodies. However, it also works the other way. If a drug is not recommended, then the NHS is not obliged to fund it.

The UK CF Registry has a significant role in the process, monitoring the real-world effectiveness of CFTR modulators over the last four years. Data that is already routinely collected in the UK CF Registry – thanks to people with CF agreeing to donate their data, and to their clinical teams for entering it –has been analysed by the UK CF Registry team at the Trust to create a report that will be crucial for the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) to evaluate Orkambi, Symkevi and Kaftrio. This data will paint a true picture of the medium- and long-term impacts that the treatments can have for people with cystic fibrosis.

Alongside providing data to NICE as part of the NICE appraisal process, we are also a stakeholder. This means we have been able to submit additional evidence, call for a wide range of outcomes to be considered as part of the assessment and continue to campaign for all people with CF to have access to innovative treatments.

Part of the NHS deals to grant access to modulator therapies included the agreement that data would be collected on their effectiveness. The treatments would then be looked at by NICE, the arm’s-length body that assesses the clinical and cost effectiveness of treatments.

At the end of 2022, Cystic Fibrosis Trust provided feedback to NICE on how they proposed to approach the appraisal. We highlighted:

• background information on CF and the different types of medicines people with CF need to take
• which existing medicines to compare the modulator therapies against to understand how effective they are
• whether the appropriate outcome measures have been included that will capture all the benefits of modulator therapies
• the incredible impact of these medicines for most people with CF who are eligible to take them.

Earlier this year, many of you shared your experiences of Kaftrio, Orkambi and Symkevi to inform Cystic Fibrosis Trust’s evidence submission. Thanks to over 1,100 of the CF community, we were able to demonstrate the significant impact living with CF can have on your quality of life, and that “there is no day off from CF”. The CF community identified a wide range of benefits to these medicines, including:

• improved physical health and wellbeing
• improved lung function and reduced coughing
• fewer medical interventions
• less time in hospital
• reduced treatment burden because of increased health stability.

66% of people who took our survey felt that access to Orkambi, Symkevi and Kaftrio has significantly improved their quality of life, and a further 25% reported an overall improved quality of life. 80% of people felt significantly more positive about the future of living with CF because of access to Orkambi, Symkevi and Kaftrio, and 15% reported feeling slightly more positive.

In October, NICE’s independent committee met and presented on Orkambi, Symkevi and Kaftrio. Clinical experts, and patient experts participated. Representatives from Vertex Pharmaceuticals were also present and responded to questions from the committee. The second half of the meeting was held in private, and the committee agreed their recommendations.

The committee met again in December 2023. On 31 January 2024, NICE gave an update to say that the appraisal process is now paused, and Vertex, who make the drugs, can start commercial negotiations (where a price and terms and conditions can be discussed between the NHS and the company). On 4 March 2024, an update was issued to say that commercial discussions are ongoing.

Following a second committee meeting in December 2023, NICE provided an update on 31 January 2024 to say that the appraisal process is now paused. On 4 March 2024, an update was issued to say that commercial discussions are ongoing.

NICE’s committee concluded that Orkambi, Symkevi and Kaftrio are clinically effective treatments, with important benefits for people with CF but that they cannot be considered to offer sufficient value for money at the current price for them to be recommended for routine use on the NHS. You can find out more about the methods and processes used to assess NHS medicines on NICE’s website.

The committee recognised the range of challenging symptoms caused by CF, the large treatment burden it requires, the significant effect on the quality of life for those with CF and their families, as well as the large and robust evidence base after the four years of data collection through the interim access agreement.

After the update on 31 January 2024 where the NICE appraisal was paused, on 4 March 2024, the NHS confirmed that commercial discussions are ongoing.
 

NICE and the NHS have confirmed that no one who is already prescribed a modulator drug will lose access. Kaftrio is now licenced for everyone aged over 2 years old who is eligible.

Because the appraisal is paused, there are no set timescales at the moment. We don’t know how long commercial discussions will take as we are not part of the discussions. We expect these discussions to take some time.

Nothing changes to access arrangements while discussions are ongoing, so under existing arrangements, anyone eligible for Kaftrio, Symkevi or Orkambi can be prescribed the drug from two years old. Because the appraisal is paused for commercial discussions, there are no set timescales at the moment.

The NHS across the UK has previously told us they will all follow the recommendations made by NICE, so this pause in the process affects the whole of the UK.

Throughout the appraisal process, we’ve been ensuring the voices of the CF community have been heard and telling NICE how important these drugs are for people with CF. It’s now up to Vertex and the NHS to continue their commercial discussions. As these are commercially sensitive, we can’t be involved in this stage of the process, but we’ll continue to take any opportunity to highlight that everyone who is eligible now and in the future should have access to these medications.

The CF community has done a brilliant job of highlighting exactly why access to these drugs is so important. We are now waiting for Vertex and the NHS to reach a new agreement to supply Kaftrio, Orkambi and Symkevi. We’re not asking you to take any action at the moment, but we continue to monitor what’s happening, and if there is a time for action, we will let you know.

We know NICE’s update about its appraisal on 3 November is concerning for many of you and we are here to support you. Please contact our Helpline who will get back to you as soon as they can.

You can contact us on 0300 373 1000, by emailing [email protected] or by sending us a message on WhatsApp on 07361 582053.

It’s important to emphasise that this is not a final decision from NICE and doesn’t affect anyone currently taking modulators.

We are hopeful that there will be a swift resolution to this uncertainty and are urging Vertex, NICE and the NHS to work together to find a solution.