Latest update on the NICE appraisal of Orkambi, Symkevi and Kaftrio
UPDATE: As part of the NICE appraisal, NICE have asked for people to submit their feedback to the NICE consultation by 5pm on 24 November, sharing the views and experiences of people with CF and their families. Thank you to everyone who has contributed, but for those who don't get the chance, don't worry, the Trust has also submitted on your behalf.
For more information, please watch our video from Dr Keith Brownlee below.
Four years ago, when the deal was reached to give people with CF access to Orkambi and Symkevi (Kaftrio was added in 2020), it was agreed that data would be collected on the effectiveness of modulator therapies ahead of a formal appraisal by the National Institute for Health and Care Excellence (NICE) which advises NHS England on the cost effectiveness of treatments.
The NICE appraisal process started at the end of 2022 and earlier this year, over 1100 members of the cystic fibrosis community shared their experiences of Kaftrio, Orkambi and Symkevi to inform the Trust’s evidence submission.
NICE have today (3 November) published an update on their evaluation. They have said that while Orkambi, Symkevi and Kaftrio are clinically effective treatments, with important benefits for people with CF, they are too expensive at the list price for them to be able to recommend them for use on the NHS.
This is not a final decision, and there will now be a 4-week consultation period followed by a second committee meeting in December to discuss the consultation responses. As part of this, Cystic Fibrosis Trust will be able to respond to the consultation, sharing the views and experiences of people with CF and their families.
NICE, the NHS and Vertex have also already confirmed that the NICE process does not apply to anyone with CF who is currently taking a modulator therapy.
NICE’s initial recommendation today that the modulator drugs Kaftrio, Orkambi and Symkevi are highly effective for people with cystic fibrosis, but just too expensive to be available on the NHS is disappointing news. It is important to emphasise that those already taking any of the modulator drugs are not affected by the NICE process because of the agreements already in place but this update creates uncertainty for those not yet on treatment. Vertex, NICE, and the NHS must now urgently work together to find a solution to make these treatments available for all those who could potentially benefit. We must never return to a situation where people with CF die far too young, knowing there’s a treatment that could change that.David Ramsden, CEO of Cystic Fibrosis Trust
Since 2019, thousands of people with cystic fibrosis have been able to benefit from licensed treatments – firstly Orkambi® and Symkevi® and then Kaftrio®, following its marketing authorisation in 2020. Access to these treatments is provided under the terms of a commercial agreement reached between Vertex and NHS England, with the full support of NICE.John Stewart, National Director for Specialised Commissioning at NHS England
Within the agreement between NHS England and Vertex 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.
Today’s draft publication is an initial step in the review of these medicines and does not affect patients’ continued access to these drugs on the NHS in any way. Eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these drugs, as clinically appropriate.
NHS England remains committed to ensuring that these life-changing cystic fibrosis medicines are available to everyone who may benefit, now and in the future, and will continue to work with Vertex and NICE through the next stages of the appraisal process to make this possible in a way that is fair to patients, and fair to taxpayers.
November 2023 update
What has happened to date with the NICE appraisal?
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.
What are the draft 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.
What happens now?
There will be a 4-week consultation period followed by a second committee meeting in December to discuss the consultation responses. Cystic Fibrosis Trust will respond to the consultation.
The Trust has written to the Secretary of State for health, urging Vertex, NICE, and the NHS to swiftly work together to find a solution to make modulator treatments available to everyone who could benefit.
What happens to people about to start treatment?
Whilst 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.
If the committee’s final recommendation is that the medicines are not cost effective, will people lose access?
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.
When will we know the outcome of the second committee meeting?
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.