![Professor Bradshaw]()
Professor of Health Policy Peter Bradshaw on Sam Brown, the young boy from Otley, who has the rare Morquio’s Syndrome and the rationing of the drug Vimizin that could he could benefit from.
The rationing of expensive drugs for ultra-rare diseases – a moral dilemma
Sam Brown is a six year old from Otley who has the inherited Morquio’s Syndrome. This is a complex metabolic disturbance due to mucopolysaccaride dysfunction. It affects just 78 sufferers in England. Its consequences prevent growth above a maximum of 3.5 ft. Adult sized organs become compressed into the space available resulting in severe physical disability, hearing, sight, heart and lung problems and a life expectancy on 25 years. There was no known remediation until the recent creation of a new drug, Vimizin. Sam was a clinical trialist for this and apparently derived great benefit.
Rationing of Drugs for Rare Diseases
Vimizin is designated as having ‘Orphan Drug Status’. These preparations are inevitably expensive because they target ultra-rare orphan diseases of which little are fully understood. Clearly these drugs are in conflict with resource allocation and with economic evaluation. They cost up to £200k per patient per year and struggle to meet conventional cost: benefit analyses criteria used for funding other expensive drugs. Yet restricting orphan drugs means patients with rare conditions risk being untreated.
Orphan drug legislation
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product but trials are bedeviled by the low number of participants available. So the market by definition is small and thus largely unprofitable for pharmaceutical companies.
Whither Equity?
The debate about whether to fund such treatment goes to the heart of the equity debate and what is fair and socially just? The NHS historically treated us on the basis of clinical need and not on our perceived financial cost or our ability to pay. Hence a dilemma. The utilitarian argument reasonably proffers that the NHS exists to bring the ‘greatest benefit for the greatest numbers’. On this assumption orphan drugs fare badly. Supporting this, Sir Andrew Dillon, the chief executive of the National Institute for Health and Care Excellence (NICE) has said that it is irrational to fund approved drugs which NICE has already rejected.
But critically, should cost effectiveness be the sole determinant of their availability? For Sam’s parents and supporters of the Vimizin cause, the ‘rules of rescue’ , through a sustained commitment to ‘non-abandonment’ means that Vimizim should be available to Sam irrespective of its cost .
It could be argued thereby, that orphan drugs should be differentiated from other very costly Drugs. The Government created a ring-fenced fund for these. But now this is becoming restricted with 42 drugs being reassessed.
Advocates for orphan drugs argue that these are distinct and ought not to be construed as Cancer Drugs which they are certainly not. They urge government intervention to motivate manufacturers through tax incentives, patent protection and marketing rights and the financial subsidisation of research and development. If we live in a nation with a world-renowned system priding itself on the principle of universal health care, how can there be any other solution?
Read the full story on the BBC News website.
