Question to the Department of Health and Social Care:

To ask the Secretary of State for Health, what products for rare and ultra-rare disease treatment have received European Medicines Agency marketing authorisation and orphan designation since 2013; and which of those products is routinely available to patients in England.

Information on products that have received marketing authorisation and orphan designation since 2013 is contained in the attached table.

Since April 2013 there have been 48 drugs designated as orphan against 62 indications. Of these indications, 28 are routinely commissioned, of which:

- eight are recommended in National Institute for Health and Care Excellence (NICE) technology appraisal guidance;

- three are recommended in NICE Highly Specialised Technologies guidance;

- 11 are commissioned under an NHS England Policy; and

- six are available through the Cancer Drugs Fund.

26 indications are not currently routinely commissioned. Of these:

- 15 are being assessed through NICE’s technology appraisal or highly specialised technologies work programmes;

- four are on NHS England’s programme;

- three are not launched in the United Kingdom; and

- four are launched in the UK, but not currently being considered for routine funding.

Clinical commissioning groups are responsible for one of the remaining eight products and information is not held centrally on their availability. Information is not available on the remainder of products.