January 2025
News Release:
The inequality, injustice and failure to treat rare cancer disease patients of Langerhans
Cell Histiocytosis and Erdheim Chester disease with the drugs Dabrafenib and Trametinib
under the system NHS Specialised Commissioning.

Histio UK has written to the Secretary of State for Health and Social Care, it’s Member of
Parliament and NHS England specifically with regard to the inequality, injustice and failure to
treat rare cancer disease patients of Langerhans Cell Histiocytosis and Erdheim Chester
disease with the drugs Dabrafenib and Trametinib under the system NHS Specialised
Commissioning.

This communication is on behalf of patients of these two types of rare cancer for which we
urgently need to draw the attention of Parliament they are Langerhans Cell Histiocytosis and
Erdheim Chester Disease which both fall under the category of Histiocytic Neoplasms.

For the last 4 years the Histio Policy Proposal Group for Dabrafenib and Trametinib for
Histiocytic Neoplasms have been engaged with NHS Specialised Commissioning to gain
approval for targeted cancer drugs for a group of rare blood cancers (histiocytic neoplasms –
including Langerhans cell histiocytosis and Erdheim Chester Disease). After 4 years of
prevarication, it was announced in December that there was ’no headroom’ to pay for these
treatments.

The door is now firmly closed for patients with rare cancers seeking access to life-saving
treatment. Many of these drugs are tried and tested in more common cancers and work
extremely well in the rare cancer setting because they share the same genetic mutation. As an
example, the drugs, dabrafenib and trametinib, are used at volume to treat melanoma.
We asked for funding to treat about 20 patients per year, including 5 infants under 1 with life
threatening disease. Surely there must be some route for such a limited number of patients?

The UK, and England in particular are complete outliers in lack of access to these drugs.

Collectively, rare diseases are ‘common’ affecting up to 1% of the population. The lack of a
functional specialised commissioning process is a complete let down for our patients, and an
abject failure of Government to deliver priority 4 of the Rare Disease Framework.