On 18th July 2019, NHS England published its decisioni, that it would not fund Sapropteriniii for people with Phenylketonuriaiii iv
This was a devastating blow for hundreds of people in England with PKU who would be responsive to Kuvan and have their lives transformed by it, especially those unable to successfully manage their condition through alternative means and therefore who suffer permanent cognitive impairment due to brain injury as a result.
Sapropterin is available in Austria, Belgium, Bulgaria, the Czech Republic, Denmark, Estonia, France, Germany, Hungary, Italy, Latvia, Lithuania, Luxembourg, the Netherlands, Norway, Portugal, Romania, Russia, Slovakia, Slovenia, Spain, Sweden, Switzerland, Turkey and Ukraine, as well as the United States of America. However, except for a small number of women during pregnancy, it is not available in England on the NHS.
In a significant development however, NHS England have now agreed to set aside its decision, following a legal challenge to the decision brought by an 11 year old child with PKU.
The legal challenge was started on the 6th August 2019 when a letter was sent to NHS England notifying them that legal proceedings were to be brought against them in respect of their decision not to fund Sapropterin on the grounds that (1) the decision making process was unfair (2) the decision-making procedure was irregular (3) the decision makers had failed to address the right questions (4) the decision failed to comply with the government’s international obligations to children (5) the decision breached statutory public sector equality duties under s.149 Equality Act 2010 & The National Health Service Act 2006 as amended by the Health & Social Care Act 2012.
The 11 year old was granted legal aid for the legal challenge by the Legal Aid Agency.
NHS England has now responded to the threat of legal proceedings and conceded there were problems with its decision and agreed to re-consider. As a result the decision will be re-taken afresh.
Peter Todd, solicitor and partner in the firm of Hodge Jones & Allen who represented the Claimant in this legal challenge said :-
“Sapropterin has been licensed for use in England since 2008 and after 11 years of waiting it is bitterly disappointing that patients in England are still unable to access it. It is widely available elsewhere. We call upon NHS England to do the right thing for people with PKU, and like many other countries, to fund Sapropterin for those who would benefit by it.”
An ultimatum for NHS England to agree to fund Sapropterin by the end of October 2019 has been sent, or further proceedings will be taken to challenge the failure to fund it.
In a related case, legal proceedings have also been commenced against the National Institute for Healthcare & Clinical Excellence (“NICE”) in relation to its appraisal of Sapropterin. The Claimant challenges the decision by NICE to appraise Sapropterin on its standard technology appraisal track rather than the highly specialised track. Peter Todd explains :-
“Proceedings have been issued and served against NICE seeking to challenge the way it proposes to appraise Sapropterin. The claim is that NICE and NHS England are manipulating the NICE appraisal processes in order to obtain a pre-determined outcome. It is unusual for NICE to commence an assessment for a drug which is not new and has already been licenced for over 11 years. We consider that Sapropterin meets all the criteria to be assessed as a highly specialised technology and as such it would be found by NICE to be a cost effective treatment, and NHS England would then have a duty to fund it. We are therefore disturbed by manipulation of the criteria to appraise it as a standard technology, so it would be deemed not cost effective.”
i The decision is published at https://www.england.nhs.uk/commissioning/publication/sapropterin-for-phenylketonuria-all-ages/
ii (a drug manufactured by Biomarin Pharmaceuticals with the brand name ‘Kuvan’)
iii Phenylketonuria (“PKU”) is a congenital genetic disorder, causing inability to metabolise phenylalanine, an amino acid present in most protein-containing foods. About 1 in 10,000 babies are born with PKU, so about 6,000 people in England have the condition. In PKU, the accumulation of phenylalanine in the blood causes damage to the brain resulting in very low IQ unless kept within limits. The only alternative method of controlling the levels of phenylalanine within such limits is life-long dietary management involving a draconian reduction of intake of protein in the diet. In addition, life-long blood level monitoring, the taking artificial protein substitute and of amino acid to help metabolise phenylalanine are required. Children are especially vulnerable to cognitive damage caused by uncontrolled PKU. The difficulty of adhering to dietary management means that most adults are unable to continue with dietary management and suffer a range of adverse effects as a result.
ivFor further information about PKU and Sapropterin see www.nspku.org and http://www.nspku.org/sites/default/files/publications/NSPKU_A4_Kuvan_Longform_1.pdf