Petition Fund continued access to modulators drugs for Cystic Fibrosis patients

Please help cystic fibrosis patients have continual access to life-changing modulator medication, after the news on the 3rd November from NICE that it believes these clinically proven medications are not cost effective for the NHS to use.

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CF is a life limiting, genetic condition that affects the lungs and organs. Patients take numerous types of medication daily to help the symptoms of this disease. These modulator drugs are the first to target the faulty CFTR gene.

The modulator drugs have improved the lives of many cystic fibrosis patients already.

We want the Government to ensure access to these life changing drugs.

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Government responded

This response was given on 8 December 2023

Existing patients will be eligible for continued treatment during and following the NICE evaluation. NICE is working with all parties to address the issues raised in its draft guidance.

Read the response in full

The Government recognises that cystic fibrosis can have a huge impact on the physical and mental health of those affected. We recognise that cystic fibrosis presents significant challenges to the quality of life of patients and their families, and are committed to supporting timely access to effective new treatments in a way that is fair to all parties.

It is important we ensure that NHS funds are spent in a way which provides the most health benefit for society, as every pound that is spent by the NHS on medicines is money that is not available for other health spending. The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing evidence-based recommendations on whether medicines should be routinely funded by the NHS based on an assessment of their costs and benefits.

This process means that the NHS can be confident that spend on NICE recommended medicines will not displace other more effective treatments. These are very difficult decisions to make and it is right that they are made independently of Government on the basis of the available evidence. NICE recommends the vast majority of medicines for use on the NHS, often as a result of commercial arrangements struck between the NHS and companies. The NHS is legally required to fund medicines recommended by NICE and many thousands of patients have benefitted from access to new medicines as a result of its important work.

Since 2019, NHS patients have been able to access the modulator therapies Orkambi, Symkevi and Kaftrio through an interim access agreement between NHS England and the manufacturer. The agreement has made the medicines available for a limited time at a reduced price, whilst enabling the collection of data to inform a NICE appraisal to determine whether the medicines should be routinely funded by the NHS. On 15 November 2023, the Medicines and Healthcare products Regulatory Agency approved a new licence extension and children as young as two years old with cystic fibrosis will now be eligible to receive Kaftrio through the interim access agreement.

NICE’s appraisal of Orkambi, Symkevi, and Kaftrio is now underway and NICE and has unfortunately been unable to recommend the medicines for routine funding at the price proposed by the company in its draft guidance published on 3 November. While the Department understands this has been extremely disappointing to those affected, it is important to note that recommendations on medicines can, and often do, change during the NICE process. The draft guidance was subject to a public consultation which closed on 24 November and NICE is continuing to work with the manufacturer, NHS England and other stakeholders to address the issues it raised.

NICE recognises that its guidance has real and important consequences for patients and only publishes final recommendations on the use of a medicine after a very careful consideration of the available evidence. NICE’s committee will take all comments received during the consultation into full account when developing its final guidance which is currently expected to be published on 20 March 2024.

Under the terms of the interim access agreement, eligible children and adults with cystic fibrosis can continue to receive and be initiated onto treatment with Orkambi, Symkevi and Kaftrio while NICE’s appraisal is ongoing. It is also important to note that there is a flexible commercial mechanism in place between NHS England and the manufacturer to ensure that patients already receiving these treatments will have continued access following the publication of NICE’s final guidance, regardless of the outcome.

Department of Health and Social Care

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