The story so far
On 11 November 2019 you heard from me about the role of the UK CF Registry in evaluating the effect of Orkambi and Symkevi following interim access agreements in Scotland and England. Two days later, access had also been confirmed in Wales and Northern Ireland.
Since then we have been working hard make sure that the best possible evidence will be available, at the right time, to support access to life-saving drugs for people with CF in England, Scotland, Wales and Northern Ireland.
Vertex’s Data Collection Agreement with NHS England and NHS Improvement, required the pharmaceutical company to have a research plan, and a contract with the Trust’s UK CF Registry to carry out that research, by 11 February 2020. The plan and contract are now in place. NICE has provided reporting deadlines for 2020 and 2021, and the final report will be made available to regulatory authorities in Scotland, Wales and Northern Ireland. Scotland’s Pricing Agreement for Orkambi and Symkevi allows for reports to be given over a five-year period if necessary.
Our first task was to decide which information should be included in the reports on Orkambi and Symkevi. Analysis will be carried out by the UK CF Registry statisticians and delivered to regulators as a report with summary data. This means that nothing about individual people with CF will be included in the report. The Trust, two independent clinical experts and Vertex have agreed which clinical measures, drawn from the UK CF Registry, will be reported to NICE to help them appraise Orkambi and Symkevi in 2021.
These decisions were based on what NICE and the Scottish Medicines Consortium (SMC) have said were areas of uncertainty in the original submissions of Orkambi and, in the case of the SMC, Symkevi. The study, which will analyse data from people with CF across the whole of the UK, will focus on lung health changes over time, nutritional outcomes, changes in medications and rate of exacerbations requiring IV antibiotics. The results for people taking Orkambi and Symkevi will be compared to people with CF who aren’t taking them, to help us to understand the effect of the treatments.
What about quality of life?
Clinical data alone doesn’t tell the whole story. Over the past few months many people have asked me how changes in the quality of life of people with CF taking these medicines will be measured. For that reason, the Trust has created a new section of the UK CF Registry to allow CF centres to enter quality of life data, calculated using a validated questionnaire called ‘CFQ-R’. This survey is used commonly in CF studies all over the world, which will make it easier to interpret the results for people with CF in the UK. Everyone involved agrees that measuring changes to quality of life, as reported by people with CF themselves, is incredibly important. Exactly how to measure and report this for Orkambi and Symkevi is a topic for discussion at today’s committee meeting (more on that later).
What’s at stake?
The agreements giving access to Orkambi and Symkevi in England and Scotland require monitoring of the impact, through the UK CF Registry, of the schemes. NHS England and NHS Improvement and Vertex have agreed a flexible commercial mechanism which will enable continued patient access for those who start treatment during this interim access period. Equivalent arrangements have been made in Northern Ireland and Wales, and the Scottish Pricing Agreement covers a five-year period.
Vertex’s Data Collection Agreement with NICE and NHS England and NHS Improvement stipulated that Vertex should cover the costs of the additional monitoring of its treatments that are needed to evaluate them as part of a Health Technology Appraisal. This month, the Trust agreed a contract with Vertex to commission us to write the reports for NICE. This agreement is similar to our ‘Pharmacovigilance’, or Drug Safety Studies, where the Registry provides reports to the European Medicines Agency (EMA). As well as covering the internal costs of performing the analysis, the contract enables the Trust to support CF centres to enter more frequent data onto the Registry, by making more than £1 million available to get them the resources they need.
This information will be a legacy of the study, supporting research and quality improvement in CF across the whole community for years to come. For the UK CF Registry, containing this more frequent ‘baseline’ data will be incredibly helpful when it comes to evaluating and quantifying the effectiveness of the Vertex’s triple combination therapy (known as Trikafta in the US), which is being evaluated by the EMA now, with an outcome due by the end of 2020. Last week NICE announced their timelines for assessing the triple combination therapy, which will be in line with the EMA’s.
Because all the analysis is being done ‘in house’ by the Registry team, no information about individual patients, or information that could personally identify someone with CF, will be released as part of this study.
First NICE Interim Access meeting
Today, Tuesday 25 February, is the first meeting of the NICE Interim Access Oversight Committee (IAOC). The Committee, established by NICE, will keep an eye on the operation of all aspects of the interim access agreement for Orkambi and Symkevi in England. This includes monitoring data collection and analysis and the number of people accessing the medicines, and considering any proposed amendments to the Data Collection Agreement. Discussions in these meetings will mostly be confidential, but important milestones will be communicated publicly. The committee is made up of representatives from the Cystic Fibrosis Trust, clinicians, NICE, NHS England, CF Voices and Vertex. A clinical subgroup has also been set up, with representation from the Specialised Respiratory Clinical Reference Group and the UK CF Medical Association.
At the first meeting we will discuss the roll-out of the interim access agreement, progress with clinical data collection from the UK CF Registry, and collection of Patient Reported Outcome Measures.
The first ‘interim report’ to NICE is due in May 2020. This is our first opportunity to tell the IAOC how data collection is going, but it will still be too early to start proper analysis of the impact of the medicines themselves. Between now and then we will be communicating closely with CF centres about how to access the funding they need to enter enhanced data onto the Registry. Without the hard work of people in those centres, and the support of people with CF and their families who consent to have their data submitted to the Registry, we wouldn’t be able to write these reports, which are so crucial to getting access to life-saving drugs, now and in the future. Quantifying the impact of new medicines as they are made available is a real team effort – a team I’m very grateful and excited to be a part of.
Get the latest on access to the triple combination therapy here.