The average cost of a rare disease patient

A major argument in favour of developing treatments for rare diseases is that this will ultimately lead to a reduction in cost to national health services and society. Indeed, our experience of AKU is that a patient, particularly one who is severely affected, can end up costing a small fortune to the health service, mainly because of poor medical management, doctors who don’t know about the disease, errors in treatments and all kinds of other issues.

I’m guessing this is pretty much the same case across the rare diseases. Nevertheless, is it possible to quantify exactly how much a rare disease patient costs on average when there’s no treatment? That’s the question we tried to answer for AKU.

Michael Craig, a chartered accountant who has worked at a large international consultancy firm for the past six years, carried out a study for us on the average cost of an AKU patient to the NHS. He interviewed a range of patients, spoke to doctors, NHS finance experts, and did lots of desk research.

He wrote an extensive report, but here are his key findings:

• Individual AKU patient costs vary greatly depending on the stage of the disease they are in and the resulting number of surgeries and patient care required. Direct costs from one patient for one year can be in excess of £90,000 without taking into account drug therapy and physical aids required to live as normal a life as possible.  These additional costs bring the single possible direct patient total to over £100,000, and possibly even larger given the limit to which direct costs were captured for each patient.
• A conservative approximation of the total costs of AKU in the UK including indirect costs (i.e. lost wage and production) is approximately £1.4 million to £2.0 million per year with the upper limit possibly being as high as £7.0 million, for 80 known patients.

Much of this cost, as mentioned above,  is due to poorly coordinated care, lack of access to treatment, doctors who don’t know the condition, and errors with diagnosis. For us, this makes the case for why specialised care in a specialist centre is so much more effective. If all AKU patients were treated in national specialist centres by medical experts who knew about the condition, the costs to the health service and society would be much smaller.

In the end, however, it’s about the benefit to the patient. Rare Disease UK published research earlier this year that showed how rare disease patients suffer from isolation, lack of understanding and poor care. Specialised centres address many of these challenges.

So here’s something for you: if you’re a rare disease patient group, have you carried out a study to figure out the average cost of your patients?