Christine Lavery, MBE, Group Chief Executive of the MPS Society in the United Kingdom, has sounded the alarm about the National Institute for Health and Care Excellence’s recently-announced plan to change the arrangements for evaluating and funding drugs and other healthcare technologies assessed through NICE’s Highly Specialized Technologies (“HST”) appraisal. From April 1, 2017, NICE plans on the introduction of a £100,000 quality-adjusted life year (“QALY”) threshold for medicines evaluated via NICE’s HST program, which assesses treatments for ultra-rare diseases.
The MPS Society in the UK has responded to this plan by pointing out that this threshold will effectively stop the flow of new medicines reaching patients with ultra-rare and complex diseases. Many treatments for ultra-rare conditions that are currently funded by NHS England have costs per QALY of more than £500,000, including the three medicines that have been approved by NICE’s HST process to date. The MPS Society states that QALY thresholds are not appropriate for evaluating medicines for ultra-rare diseases, due to the small patient populations, and often limited data.
Christine Lavery said, “Being born with an ultra-rare disease, a disease affecting less than 110 people in England, is not a lifestyle choice; it is no one’s fault; it happens albeit very rarely; it happened to my son. At that time, there was no treatment and Simon died aged 7 years. I can only imagine now how it might be, to be faced with a child with an ultra-rare disease who could be treated with a highly-specialized medicine, but is denied treatment on cost grounds. The pain for the family of seeing their child condemned to death by Andrew Dillon, Chief Executive of NICE; Simon Stevens, Chief Executive of NHS England; and the UK Government, is unimaginable. Let us also be clear to Members of Parliament, many of the babies and children who will be affected by this catastrophic decision are ‘yet to be born or diagnosed’ members of your constituencies.”
MPS Society Chairman Paul Moody said, “A decision by NICE and NHS England to implement this new policy . . . will affect the most vulnerable in UK society, and confirms that children and young adults with ultra-rare diseases going forward are economic pawns in a failing NHS, and cheaper dead than alive. The UK government now needs to act at lightning speed and reverse this initial NICE & NHS England policy, not just in the context of patients with ultra-rare diseases, but also that of the life sciences industry, who will see no incentive to investing in the UK market if their innovative medicines and technologies have no prospect of reaching the patient.”