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Advocating for Access to New Treatment

This is an exciting time as new treatments and new treatment approaches for bleeding disorders are constantly being developed. However, not all of these new advances are getting approved for use in the UK.

In this article we have used our work in seeking to get access to BPL’s Coagadex as an example of how this process functions and how people with bleeding disorders are not always seeing the benefit of advances in treatment. Coagadex is a treatment for Factor X (ten) deficiency. It is a replacement factor product produced from human plasma by pharma company Bio Products Laboratory who are based in the UK.

The standard treatment for Factor X deficiency has for many years been Prothrombin Complex Concentrate (PCC) which is a mix of different clotting factors but includes some Factor X. It can be effective at preventing and stopping bleeds in people with Factor X deficiency but is not an ideal treatment as it also includes other clotting factors that the people being treated with it are not lacking. There are less than 400 people with Factor X deficiency in the UK and less than 10% need replacement factor treatment each year.

BPL begun work on a pure Factor X product over ten years ago and in September 2007 their proposed product Coagadex was designated an “orphan medicine” by the European medicines agency. This is an acknowledgement that they are working on a treatment for a rare condition and provides certain incentives to help them through the development process.

Following clinical trials, which included people in the UK, Coagadex was licensed for use in the EU by the European Medicines Agency in March 2016. Currently, EMA licenses cover the UK and this allows the companies that own those products to begin negotiating with UK Government for their use in the NHS.

There are a series of different routes for new treatments for rare diseases to be approved for use and in this case BPL decided that they would submit the treatment to be considered through a prioritisation process run by NHS England. Treatment on the NHS in England is overseen by the Specialised Blood Disorders Clinical Reference Group (CRG) a group of commissioners including doctors, other health professionals and patients.

NICE (a UK government body) began a process of reviewing the clinical evidence on Coagadex and on behalf of NHS England convened a panel of clinicians and a patient representative from The Haemophilia Society to produce a proposed policy for its use in England. The proposed policy alongside an analysis of the impact on the NHS budget goes before a committee of NHS England called the Clinical Priorities Advisory Group (CPAG).

The proposed policy for Coagadex was first reviewed by the CPAG in June 2018. The CPAG rates each policy submitted to them on a scale of 1-3 for effectiveness and a scale of 1-3 for cost. This allows them to rank all the policies they have considered into 5 levels of priority. In this way they are comparing treatments for bleeding disorders against other new advances in conditions such as pulmonary hypertension and corneal blindness.

In this first review Coagadex was placed as a level 4 priority and was therefore not funded for use in England. We were disappointed by this and made representations to NHS England that they were not sufficiently considering the impact on people with Factor X deficiency of this improved treatment. It was clear that Coagadex’s high price was a major consideration but a level 4 rating implied also that it had only be considered of moderate clinical effectiveness.

Following, our submissions Coagadex’s was reviewed again at the next CPAG meeting in November 2018. We found out in December that once again NHS England had decided not to allow routine use of Coagadex for people with Factor X deficiency. However, the minutes of the meeting showed that since the summer they had raised its priority from level 4 to level 3 following our intervention.

We were invited in February 2019 to provide a patient impact report to help CPAG consider Coagadex for a third time in May 2019 and we worked with one of our members to detail the major impact that being treated with PCC rather than the improved treatment with Coagadex has on their life. However, we have since been informed that BPL have withdrawn the product from consideration as they do not expect it to be approved. Allowing it to be reviewed this year would be their third and final time that it can be considered. This is a major disappointment to us and to the 40-50 people who are being denied a treatment that would improve their quality of life.

If NHS England can’t fund this step from PCC to a pure factor product how can people expect that in future further new treatments such as recombinant products and other treatments will be made available to this small patient group. We have similar concerns that in the current system new recombinant products for von Willebrand’s and Factor 13 deficiency and new treatments in haemophilia including gene therapies, sub-cutaneous products and longer half-life products will also be rationed or not provided at all.

In light of these challenges in access to new treatments The Haemophilia Society through its work with the All Party Group on Haemophilia and Contaminated Blood has launched an Inquiry into the current systems of licensing, procurement, commissioning and prescription of treatments for genetic bleeding disorders in the UK. 

It will document the current processes with a view to how these processes impact on the treatments that patients are offered and can be offered by the NHS. They wish to show where the processes are working well and when they are not and make recommendations for how the processes could be changed to improve access to treatments and outcomes for people with bleeding disorders. 

The APPG wish to seek extensive input from all stakeholders: Government, the NHS, industry, clinicians and people with bleeding disorders and would welcome submissions including documents and data and any other information.

Formal submissions and any other evidence can be sent to [email protected] 

Jess Milton

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