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 can 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.

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.

Following the consultation period, a second committee meeting will take place on Thursday 14 December to discuss the consultation responses. Cystic Fibrosis Trust has responded to the consultation.

You can register to observe the public part of the committee meeting through the link on this page. Registration will close at 4pm on Thursday 7 December 2023.

While the NICE appraisal is ongoing, access to treatment will continue. This will be the case for people starting the treatments as well as those already taking the treatments.

No. As part of the interim access agreement, there is a flexible commercial mechanism to ensure continued access for patients already receiving any of the licensed treatments following the conclusion of a full NICE evaluation to determine the value of the medicines for the NHS. 

As a registered stakeholder, Cystic Fibrosis Trust will be informed of the outcome in confidence shortly after the meeting. The outcome will be publicly released once the guidance is published on the NICE website several weeks after the meeting.

NHS England have confirmed to us that  if someone is on Orkambi at the end of the NICE appraisal process, they will still be able to move to Kaftrio, whatever the outcome of the appraisal.  This applies to anyone who needs (based on clinical advice) to move between the drugs covered by the appraisal: Kaftrio, Orkambi and Symkevi at any point in the future. We hope this will be the same in Wales, Scotland and Northern Ireland but we are trying to get confirmation in writing.

On 15 November, the MHRA announced approval for Kaftrio for eligible 2–5-year-olds and NHS England also confirmed that all children eligible today can be confident about their long-term access to these treatments whatever the final outcome of the NICE process. You can read more about this here.

While the NICE appraisal is ongoing, access to treatment will continue. This will be the case for people starting the treatments at any stage during the appraisal as well as those already taking the treatments. This is not a typical process for NICE to undertake and we do not know exactly what will happen at each stage. The initial recommendation announced by NICE on 3 November 2023 is not a final decision, it’s a step in the wider appraisal process and we’ll be continuing to campaign for access for everyone who could benefit, now and in the future.

As shown on the NICE consultation page, the key dates for this evaluation are:

• Closing date for consultation comments: Friday 24 November 2023 (5pm)
• Second evaluation committee meeting: Thursday 14 December 2023
• The outcome will be publicly released once the guidance is published on the NICE website several weeks after the meeting.
• If a decision is not reached at the second committee meeting, a further period of consultation may take place after which a further committee meeting will be scheduled.

Timescales for NICE appraisals are set regardless of what the medication or technology they are assessing. What a future timescale is depends on the outcome of the next stage and may be impacted what Vertex or NHS England decide to do.

We expect any final decision by NICE will be aligned across all four nations in the UK. In Wales, guidelines produced by NICE apply (subject to approval by the Welsh authorities) and in Northern Ireland the majority of NICE guidelines apply (subject to approval by the relevant Northern Ireland authorities). In Scotland, the Scottish Medicines Consortium have confirmed that they are inputting into the NICE process and whilst they will produce separate guidance and advice documents, the recommendations will be aligned.

The consultation period has now closed. 

Following the consultation period, a second committee meeting will take place on Thursday 14 December to discuss the consultation responses. Cystic Fibrosis Trust has responded to the consultation.

You can register to observe the public part of the committee meeting through the link on this page. Registration will close at 4pm on Thursday 7 December 2023.

The consultation questions covers some technical questions where NICE would like further information. The Trust will be responding to these in our consultation response. You do not have to respond to these questions but can focus on things like:

• how the day-to-day severity of CF has changed since access to modulators
• what you can do now that you couldn't before
• how you perceive life with CF
• what the actual impact of increased lung function is for you
• how you would describe the treatment burden
• what the non-health related implications are on your quality of life.

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 helpline@cysticfibrosis.org.uk 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.