PharmExec Blog

UK Plan for Rare Diseases: Little Focus on Access to Medicines

By Leela Barham.

The European Commission mandated that all member states have a plan for rare diseases back in June 2009. With the EC deadline just weeks away, the UK has now finalized its own.

But for those who were hoping it would have a clear focus on access to medicines, there’s little to be optimistic about.

The plan’s ambitions cover much of what you might expect about how best to deliver treatment and care to those with a rare disease, namely:

  • empower those affected by rare diseases;
  • identify and prevent rare diseases;
  • diagnose and intervene early;
  • coordinate care; and
  • improve research.

In relation to medicines there’s a nod to some of the issues; the plan highlights that there needs to be “appropriate procedures for evaluating the benefits and costs of treatments when they become available” and that “procedures should be transparent and robust enough to be able to take account of the particular challenges that occur when evaluating treatments for rare diseases.”

But left unsaid is who will determine whether the procedures succeed in achieving the above; patients and industry may well disagree with Government on such points. And since the National Institute for Health and Care Excellence (NICE) is responsible for assessing Highly Specialised Technologies (ultra-orphan drugs to you and me), this will no doubt generate a lot of interest.

The plan also references Innovation, Health and Wealth, which is an English effort to speed up diffusion of innovation. It also says that the UK will explore how to change systems that collect information on uptake of medicines.

With regard to drug development, it suggests that much can be done to build on the European partnerships that already exist. The UK Clinical Trials Registry may also provide information for patients and their families on experimental medicine trials for rare diseases.

As many have pointed out, it’s not only what it written that matters but the action that is taken. For that we need to wait and see, but the plan has to be implemented by 2020. Seven years is not long (in healthcare-system terms) to get it all done.

Leela Barham is an independent health economist. You can find out more about her at and contact her on

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