Slow decision making about funding new treatments for rare diseases creates fears that the NHS is abandoning people with extraordinary care needs while generating no real financial saving. It also risks discouraging future private industrial investment in the British bio-pharmaceutical research base, finds a new UCL School of Pharmacy Practice and Policy Briefing, published today1. Ethics, Compassion and the Nation's Interests highlights the recent example of Morquio syndrome treatment, in which some individuals and families affected by an ultra-rare disease which restricts normal growth and causes painful and other symptoms have been made to feel that they are having to beg for NHS funded access to a new treatment, as a form of cruelty which is neither morally nor financially justifiable in any modern society.
The UCL authors accept that overall spending on medicines must be kept within affordable limits, and note that pharmaceutical companies will this year pay back in the order of £1 billion in revenues that exceed the fixed NHS spending cap on innovative drugs and allied products. They argue this is all the more reason for making timely decisions with regard to supplying new therapies which offer hope as well as relieving suffering, and in some cases extending life. Co-author Professor David Taylor (who has had 30 years' experience in NHS Board and allied roles) commented:
'The NHS is a great national asset, which promises universal care. But in areas like rare disease treatment it needs strong political leadership to counter bureaucracy and insensitive managerial approaches that do not fully reflect voters' priorities. No-one should be left feeling abandoned, especially when this could cost the nation much more than it saves.' 'It is good that the company that funded the trial of the Morquio syndrome treatment which led to its licensing by the European Medicines Agency is continuing to supply it without charge to the patients who were included in this research. But other people have been left in need of better treatment. If the NHS were required to supply all licensed rare disease therapies up until NICE has published robust evidence that they are not cost-effective it would help stop injustices and speed evaluations.
The MPS Society represents people Morquio syndrome and allied conditions. The UCL Pharmacy Practice and Policy team responsible for Ethics, Compassion and the Nation's Interests have agreed with the MPS Society that they will research this issue again at the end of this year with a view to offering a joint report on treatment access improvement early in 2016.