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How much is Harry Kane worth? The growing cost of rare diseases

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Date: 26/5/2016

A dispatch from the 2016 pH Summit

Professor Mehta, Consultant Haematologist at the Royal Free Hospital, likes his football analogies. Back in 2002 when Manchester United parted with an eye-watering £30 million to secure Rio Ferdinand, the NHS were spending the same figure on enzyme replacement therapies (ERT) for rare diseases, such as Fabry and Gaucher disease. Fast forward to 2013, the very same scenario: £85 million spent on Gareth Bale by Real Madrid, matched by ERT spend in the NHS. Now, in 2016, it’s possible that the cost of treating patients with these progressive, incurable conditions will actually outpace that of the astonishing world of transfer fees. Where does it end?

‘Individually rare, but collectively common’

You would be forgiven for thinking that rare diseases are just that, rare. But, ironically, with so many of them already identified, the chances of YOU being one of the global 350 million people with a rare disease is probably greater than you think. And those are just the conditions we know about…

Add to that, the number and scale of hurdles that the pharma industry must clear in order to deliver life-saving rare disease medicines to patients are immense, sometimes insurmountable. Just considering the following:

·        Little clinician exposure = poor disease awareness

·        Early clinical manifestations can be silent = delayed diagnosis

·        Disease heterogeneity = misdiagnosis

·        Pockets of patients, dispersed widely

·        Geographically spread of specialists = fragmented research

The power of n

Having somehow managed to overcome these challenges, the next herculean task for the pharma industry is to design and a run robust clinical trial, in order to establish safety and efficacy. As Sebastian Stachowiak, of Shire summed it up: ‘Statistical significance? Forget it!’

It’s a wonder that any rare, or ‘orphan’ disease medicines have made it over the NHS finish-line. Where indeed is the commercial incentive for R&D, if the size of your market dictates you’ll never recoup capital investment? Enter, the Orphan Drugs Act, stage right. And it was right: it pathed the way for galvanising the pharma industry into action. Incentives, such as lowering the bar for demonstrating safety and efficacy, and longer periods of patent protection, have proven powerful stimulators of research and development. Orphan drug designation levels the playing field, meaning that individuals with rare diseases can no longer be side-lined. But, these high technology, life-saving therapies cost!


The bottom line


So, how does the NHS answer the financial question posed by orphan drug development? We cannot stop the march of orphan drugs, that would be ethically wrong. Instead, could real world evidence hold the key? Some orphan drug designations already come with a proviso to collect longitudinal patient data, free from the rigours and contrivances of the clinical trial. The use of disease and drug registries is increasing, allowing clinicians and companies to evaluate long-term treatment safety and patient outcomes, in the real, unconstrained, world. Looking at the outcomes of sub-populations of patients will also help. The more evidence clinicians have, the greater their ability to target these high technology, high cost therapies for maximum benefit, both for patients, and for the ever-constricted NHS purse-strings. 

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