How we’re campaigning to improve treatment options for people with CF who can’t benefit from modulators

CFTR modulator treatments like Kaftrio, Kalydeco and Alyftrek have transformed the lives of many people with CF. But they don’t work for everyone. Some people with CF have gene variants that modulators can’t help, and others cannot tolerate them due to side effects. 

The Trust recently responded to a call for evidence from the MHRA on the Rare Disease Framework. The framework is looking to change the way treatments for rare diseases, like CF, are approved for use in the UK, particularly where there is limited medical evidence from trials due to the small numbers of people taking part. 

The framework has some useful solutions for improving access to new treatments, but we believe it can still go further to truly make a difference for people with CF. These are some of the key asks we are pushing for:

• We want to ensure that people with CF who have rare mutations and are not able to benefit from CFTR modulators meet the proposed threshold for the new rare disease treatments pathway, even if CF as a whole does not. Although a rare disease, CF is the most common rare disease and the framework currently recognises rare as 1 in 50,000, meaning that CF as a condition wouldn’t automatically qualify. However, people who have CF and who can’t benefit from modulators are an even rarer sub-group. It is vitally important that groups of people with CF with rare genotypes are included under the criteria. 
• People with CF should be at the heart of decision making about which treatments are approved. It is important to understand what success looks like to people with CF and their families when developing a new treatment. For example, is the treatment burden acceptable? Do the benefits outweigh the risks? What are the health and daily life outcomes that matter to people with CF?
• We want to address challenges with other parts of the process so that treatments can reach clinical trials quicker, and be made available to people who can benefit from them as soon as possible. 
• We want resources like our UK CF Registry to be used to support decisions about new treatments. The Registry already collects important information from routine CF care. This information can help assess treatments when there are too few people to run large clinical trials.

Everyone with CF deserves the chance to benefit from effective treatments. That’s why finding new options remains one of our top research priorities. 

We are calling on the Government, the MHRA and other agencies to create research and regulatory systems that help new treatments to be developed and approved for people with CF who are not able to benefit from current modulators. We won’t stop until everyone with CF can live longer, healthier lives.