Cancer52’s briefing paper on Orphan Medicines

Cancer52, the alliance of rare and less common cancer charities of which AMMF is an active member, has published a briefing paper on Access to Orphan Medicines in the UK, today 5th June 2014.

More deliberation and less maths – Making reasonable decisions about access to orphan medicines in the UK

In summary:

Orphan medicines treat diseases that affect no more than 5 in 10,000 people in Europe. Ultra-orphan treat those diseases that are even more rare. Rarer cancers make up close to 40% of orphan medicines.

It’s a difficult and long journey for an orphan medicine to reach the patient, and difficult decisions need to be made about the benefit and risks of orphan medicines when we can’t always have the robust evidence that regulators and the NHS wants.

Orphan drugs can be assessed not just for their clinical benefits and risks, but also their value for money. Three main agencies do this across the UK using cost per Quality Adjusted Life Years (QALYs) calculations. But this approach doesn’t capture everything that it should for orphan medicines. Partly in recognition of this, but also due to political pressure, changes are being made. These focus on considering more criteria (like absence of other treatment options) and more engagement with patients and carers: more deliberation and less maths should inform decisions to recommend or reject orphan medicines.

Cancer52 thinks changing the way that access is determined is a step in the right direction: that such decisions need to account for the special circumstances faced in treating rare diseases. And more decisions about smaller patient groups will be needed in the era of precision medicines.

But we might not need every agency to do that job. Cancer52 is asking for:

  • Full and open approach to evaluation of changes.

    This should include how far proposals/changes in appraisal affect research priorities, including research for those with rare and less common cancers. Cancer52 is willing to work with all stakeholders to build on the opportunities of reform and to improve access to medicines in the UK

  • A debate on the merits of a single UK wide approach to appraising ultra orphan medicines

  • Faster decisions from agencies.  Patients can’t always wait the time it takes for recommendations to be made


To read Cancer52’s briefing paper on Access to Orphan Medicines in the UK in full, click here

June 2014