The Rarer Cancers Forum (RCF) has recently published a report – Off limits – revealing wide variations in the way in which NHS organisations process applications for the use of drugs outside their licensed indications, to treat those with rarer cancers. The report can be accessed here.

Because there are usually very few, if any, licensed treatments for such cancers, these patients tend to be prescribed drugs which are actually licensed for the treatment of more common cancers whose underlying disease process is similar to the rarer disease. Such so-called “off-label” use presents difficulties for patients because NICE only appraises drugs for use within their licensed indications. The use of drugs off-label, or as the RCF calls them “near-label”, cannot therefore be the subject of mandatory technology appraisal guidance and patients must accordingly look to their local PCTs for funding in their particular case.

The report makes the following observations:

  • The attitudes of NHS hospitals to near-label prescribing vary enormously, with some trusts actively discouraging its use, while others recognise its necessity.
  • Over the last three years, over 1,000 patients out of the 3,000 who applied to their PCTs for near-label funding, have been rejected.
  • Near-label prescribing is significantly more common in Europe.  

The report makes 25 recommendations to improve matters for such patients, including:

  • The Secretary of State should order PCTs who operate a blanket ban on near-label prescribing to lift that ban.
  • The Department of Health should reiterate to PCTs the specific circumstances in which treatment requests are to be treated as exceptional cases. Funding requests for near-label treatments for use in patients with rare cancers should not be treated as exceptional.
  • The use of near-label drugs for rarer cancers should be funded at national level.
  • Mandatory guidance should be issued to the NHS on the most frequently used near-label treatments.
  • The pharmaceutical industry should contribute to the cost of running and auditing the new system.

Whilst access to treatment has increased for many patients, in the wake of the various initiatives following publication of the Richards report, patients with rarer cancers seem rather to have been left out in the cold.