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Is NICE fit to review orphan drugs? UK MPs aren’t convinced

By Max Rex, Senior Analyst

Email: mrex@partners4access.com

Jacob Rees-Mogg, Member of Parliament (MP) for North East Somerset on one of his last days as a backbench MP, was granted an Urgent Question in Parliament on July 22nd to discuss the failure of the National Institute for Health and Care Excellence (NICE) to recommend BioMarin’s orphan drug Brineura, and the subsequent failure of NHS England and BioMarin to come to an agreement on reimbursement.

One of Rees-Mogg’s constituents is a young boy who has been diagnosed with Batten disease. Batten disease is a severe degenerative disease of the central nervous system that is fatal in all patients, with most patients dying in childhood. The patient could not get access to Brineura, a potentially life-changing enzyme replacement therapy that halts the progression of the disease. Rees-Mogg and other MPs found it hard to believe that NICE would not recommend Brineura for reimbursement on the NHS when the disease itself is so severe and the treatment has been shown to be effective at stopping disease progression. MPs quoted the NICE assessment, where NICE concluded that there was a good chance that Brineura could be associated with over 30 QALYs. The Shadow Minister for Public Health, Sharon Hodgson, was equally unimpressed by NICE, claiming that NICE assessments are unfit for purpose when it comes to assessing rare disease drugs. The vast majority of MPs who spoke in the session made similar statements, with some MPs also asking the Government’s representative whether there was any opportunity for Parliament to circumvent the NICE decision in this instance to secure NHS reimbursement. MPs pointed out that many other countries reimburse Brineura, including Germany, France and even Wales.

The UK Parliamentary Under Secretary of State for Public Health, Seema Kennedy, the representative sent to respond to the question, did not offer many new information or give much hope to the MPs. Although she had sympathy for the patients with this horrific disease, she stressed how important it is that NICE is an independent body comprised of internationally renowned experts. Her view remains that these decisions should be left to the experts. NICE is currently undertaking its regular review of its processes and procedures, and Mrs Kennedy claimed that NICE is always looking to improve by implementing feedback from all stakeholders. Overall, she stuck to the party line that NHS England and the Ministry of Health are ready to negotiate with BioMarin, and that BioMarin should enter the negotiations seeking a “fair and reasonable price”. However, there might be some disagreement about what constitutes “fair and reasonable”.

This Urgent Question is unlikely to be the singular event that causes NICE to reconsider its processes – it was just a Parliamentary discussion, after all. However, this topic is very emotive and perhaps there will now be increased public pressure on the different parties to come to an agreement for the good of patients. NICE is currently re-assessing its final decision on Brineura following an appeal against the decision, and perhaps this negative press could spur more advocates to push the committee to change its mind. It will also potentially galvanize certain stakeholders who have long considered the NICE process unfit, including certain patient groups and pharma companies.

However, what is also interesting to note is that a Labour Shadow Minister called NICE unfit for purpose in assessing rare disease drugs. With the residing uncertainty of Brexit looming over all aspects of UK politics, there is still a non-zero chance that a general election could be called before the end of the year. Politics appears to be more volatile than ever, so a change of government is not out of the question. If the Shadow Minister speaks for Labour, then there is a chance that wider reform of NICE could be on the cards.

 This debate also seems to have a very different feel to the ongoing NHS England / Vertex debate. Vertex and NHS England have failed to come to an agreement to provide its cystic fibrosis treatment Orkambi on the NHS, despite NICE initially rejecting Orkambi in July 2016. This disagreement has since turned hostile, with both sides blaming the other for their inflexibility and not negotiating in good faith. Generally, parliamentarians have seemed to blame Vertex as they have recorded very high profits in recent years. However, in the Brineura discussion, there was very little (if any) blame put on BioMarin by MPs for their role in this, with all of the anger directed at NICE. The upcoming review of NICE processes will no doubt further stoke this debate, but NICE has historically been very resistant to large scale changes. Though MPs and other stakeholders will advocate for reform, widescale changes are still unlikely.

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