Does Scotland have the answer to dealing with rare diseases?

The NHS is under crippling pressure and one of the great political questions of our time is to ensure it’s able to cope.

Addressing the demands of resources covers the full spectrum of NHS services – from GPs to A&E, and complex treatments for conditions such as cancer. But the pie is only so big, and this means some patients run the risk of missing out. At particular risk are the small number of patients with ‘rare’ conditions or diseases, who require particularly specialised – and therefore expensive – care and medication

There are between 5,000 and 8,000 diseases considered ‘rare’, which means they affect less than five people in every 10,000. Each such disease might affect just 0.1 percent of the UK’s population.

You would think this would mean it’s a small problem. Hardly. If 0.1 percent of the population is affected by each of these diseases, then a total of three million people require specialist treatment. That’s far from small beer for the NHS.

Because the number of people requiring drugs for each rare disease is so comparatively small, funding in England is decided at a national level through NHS England’s specialised commissioning budget. And these decisions on what should and should not be funded is informed by guidance produced by the National Institute for Health and Care Excellence (NICE). The problem is that drugs for rare diseases are far more costly than the spend on more common conditions. This makes it difficult for NHS England and NICE to assess value for money. More importantly, it means patients often struggle to get access to the medications they need.

The Government wants to improve access to innovative medicines and technologies, including those used to treat and manage rare diseases. Part of its approach involves the launch of the Accelerated Access Review, which is considering the barriers to the development, adoption and diffusion of innovation – including how NHS England can commission drugs more effectively. But the fundamental issue this review must address is funding, and the nature of these rare diseases means that drugs are likely to remain very – sometimes prohibitively – expensive.

But could the Scottish example provide a solution? In 2013/14, the Cancer Drugs Fund in Scotland was merged with the Rare Condition Medicines Fund. The newly named ‘New Medicines Fund’ ensured that overall funding was quadrupled from £20 million to £80 million. This included funding from the rebate made to the Scottish Government by the pharmaceutical industry as part of the Pharmaceutical Prime Regulation Scheme (PPRS).

Scotland has also taken steps to improve patient and clinical evaluation, known in the sector as PACE. What this means is that patient groups or drugs companies can request a PACE assessment – involving clinicians, patients and patient groups – to ensure issues such as quality of life and money saving across the wider care pathway are considered. In other words, the cost of an individual drug is not the be all and end all for its availability to patients.

If England were to adopt a similar system, it wouldn’t mean more funding magically appeared overnight. Nor would it avoid hard decisions that have to made on which drugs should be available to patients, at a time when the NHS is trying to make (or at least figure out how to make) £22 billion in efficiency savings. But it would mean patients’ voices are heard as part of the decision making process for rare diseases. In a system often condemned as opaque and glacially slow-moving, this could help alleviate frustration while ensuring both value for money and the best treatment for patients.

If the launch of the Accelerated Access Review is meant to be a leap forward, then considering the lessons of the Scottish model would be progress indeed.