Ramblings of a CEO: a call to arms

Ramblings of a CEO: a call to arms

As CEO of the MPS Society, and a Dad of these two “Hunter” boys, I live with a rare disease every day. These lads and all like them did not choose to have a rare disease but they do not let it define them. They are an example of how to live life despite it being stacked against them.

It appears however that not all value their lives as much as they should and they argue that new treatments and therapies should not be made available because of the cost. An example of this was NHS/NICE’s decision to refuse Cerliponase Alfa (Brinuera) as an innovative new therapy option for neuronal ceroid lipofuscinosis type 2, CLN2.

Why was it turned down? It cannot be its effectiveness because it has been evaluated at having potentially at least 30 QALYs (Quality Adjusted Life Years). Yes, that’s right, 30 years’ benefit. So why? Sadly value for money is the sad fact and the health economics model used to determine the value of a person’s life.

Society has chosen to save people’s lives no matter the cost; for example when someone is lost at sea huge resources are deployed at great expense to save one life. Why? Because as human beings we choose to do the right thing, be compassionate to our fellow human beings despite the financial costs.

These new treatments are expensive but that is because of the patient populations. Does that mean we should not be compassionate just because of the cost? Companies invest millions in developing these new therapies and treatments and, unless they can show a profit, no one will invest in them. So NHS/NICE need to be realistic about reimbursement of these drugs and realise that there needs to be a sustainable model that works. If not both adults and young children will continue to die or suffer just because of cost and where is the morality and compassion in that?

Rare diseases constitute approximately 2% of NHS spend if you exclude cancer treatment costs. The Pharmaceutical Price Regulation Scheme (PPRS) is the mechanism used by the UK Department of Health to ensure that the NHS has access to good quality branded medicines at reasonable prices. It involves a non-contractual agreement between the UK Department of Health and The Association of the British Pharmaceutical Industry (ABPI). The scheme applies to all branded, licensed medicines available on the NHS. The purpose of the scheme is to achieve a balance between reasonable prices for the NHS and a fair return for the pharmaceutical industry.

If the money levied against the relevant pharma companies, via the PPRS, was ploughed back in to the rare disease drug reimbursement then this would become a sustainable model. However, as usual with the NHS it disappears into a black unaccountable hole.

Our government can find the extra billions to create yet more mess over Brexit but they cannot find a few million to save our most vulnerable in society. They have put politics before patients and, unless we the general public demand a change, they will continue to let people suffer and die.

It is time for change, time for compassion and time to do the right thing. The MPS Society is standing up for rare diseases, giving the unheard a voice and, together with other partners such as the LSD Collaborative, we are fighting for our and your rare community.

People like my two sons may be RARE BUT THEY ARE NOT ALONE . #rarebutnotalone#ramblingsofaceo

We are the only registered charity providing professional support to individuals and families affected by MPS, Fabry or a related disease in the UK.

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Repton Place, White Lion Road,

Amersham, Buckinghamshire,

HP7 9LP, United Kingdom

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