Every CF team in the UK enters data to the UK CF Registry and over 99% of people with CF consent to their data being collected. Thanks to the dedication and support of clinical teams and people with CF and their families, we have a powerful dataset ready and waiting to support the development and rollout of new therapies for people with cystic fibrosis.
Leading the way for drug safety studies
In 2012 we began our Drug Safety Study program, which demonstrated that the Registry could be used to monitor drugs long term. Because the Registry has detailed and accurate data on almost the whole CF population in the UK, we are able to monitor outcomes in the group of people being prescribed a specific drug and compare these with outcomes in those not taking the drug. This helps make it clear whether any effects are likely to be connected to the drug, or not.
Conducting drug safety studies within the Registry means they are independent from the pharmaceutical company, which is an important consideration for the EMA when reviewing the results. Through the Registry, the Trust is leading the way in improving the quality of long-term safety studies of new medicines and developing best practice for working with industry to do this.
Back in 2016, we wrote an open letter to NICE suggesting that ‘managed access’ (also called ‘interim access’) should be the solution for speeding up access to the pipeline of Vertex therapies for cystic fibrosis. In managed access agreements, a drug is made available for a period of two to five years during which data is collected to monitor the effectiveness of the treatment before it is appraised. This solution was adopted for Orkambi, Symkevi and Kaftrio.
Since managed access agreements were put in place for Orkambi and Symkevi across the UK at the end of last year, we have proven that we can monitor the effectiveness of these therapies on behalf of the medicines watchdogs responsible for appraising their cost effectiveness. To do this, we moved to an ‘encounter-based’ approach, which means we’re providing funding to CF centres to collect data more frequently, which has the added benefit of boosting the power of the Registry for all of the other research we do.
What about Kaftrio?
Kaftrio will be appraised by NICE in four years. We’ll be monitoring Kaftrio over this time to present NICE with a true picture of the medium- and long-term impacts that the treatment can have for people with cystic fibrosis. This will arm the decision makers with the best evidence to negotiate well and make the right decisions so that people with CF can continue to benefit from the drug.
Through Registry data, the CF community has a fundamental seat at the table for securing ongoing access to these life-saving drugs.
Looking to the future
The Registry continues to support access to the best therapies for everyone with cystic fibrosis. We’re working with Registry data to describe the characteristics of the people with CF in the UK who will not benefit from the CTFR modulators due to their age, genotype or transplant status. This will mean support and therapeutic development can be focused on them and the model of care can be adjusted to support them properly. The information will also help the Trust to make sure our services are appropriately targeted.
We’re also using data from the past to predict the future health needs of people with CF who will be accessing the new therapies.
There have been some momentous changes in the treatment of CF but there is still so much more that we need to do to ensure everyone gets access to life-changing treatment and support. If you are able, please consider making a donation today to ensure we can continue our work for everyone with cystic fibrosis.