There are huge variations in the way local NHS organisations in the UK are processing applications for using treatments for patients with rare cancer outside their licensed indication, according to a new report.

Such off-label treatments are an issue for people with rare cancers because there are usually very few licensed products for rare cancers, if any, and instead patients are prescribed treatments by their doctors which are licensed in more common cancers, but where the cancer which they have has a similar underlying disease process, according to the Rarer Cancers Forum, which conducted the study.

The Forum notes that it has coined the phrase “near-label treatments” to describe therapies which fall into this category, and emphasizes that this issue is challenging for patients because, since the treatments they need are not licensed for the use for which they are needed, no mandatory national guidance can be issued.
This means that patients must instead apply for the treatments they need to their local NHS organisations.

The Forum’s new report, the first analysis of how this system impacts on patients with rare cancers, finds that:
· the attitudes of NHS hospitals to near-label prescribing vary widely. Some Trusts appear to actively discourage the practice whilst others acknowledge its necessity in high-quality patient care;
· over the last three years, more than 3,000 patients have had to apply for funding for off-label treatments through their doctor to their local Primary Care Trust (PCT), and more than 1,000 have had their requests rejected; and
· patients with rarer cancers in the UK are less likely to receive off-label treatments if they need them than patients in France and Germany.

The report makes 25 recommendations to improve the system, including that:
· near-label cancer treatments used rarely should be funded at the national level;
· mandatory guidance should be issued to the NHS on the near-label treatments used most frequently;
· the pharmaceutical industry should contribute to the costs of running the new system, provided that patients with rarer cancers benefit from improved access to medicines; and
- the Department of Health (DoH), the Medicines and Healthcare products Regulatory Agency (MHRA) and the Association of the British Pharmaceutical Industry (ABPI) should work together to produce guidance clarifying the circumstances in which pharmaceutical companies can provide information on the off-label usage of their products to clinicians, charities and policymakers. This should reaffirm that the promotion of medicines off-label is not acceptable, but make clear that there is a distinction between marketing an off-label treatment, and responding to legitimate requests for information about its use.

Commenting on the report's findings, the Forum’s executive director Stella Pendleton said: “no patient should be denied a treatment recommended by a doctor simply because the cancer it treats is too rare for the medicine to be licensed. If a doctor thinks that a patient with a rare cancer should be given a drug, then the NHS should fund it. The NHS is forcing desperate patients into the cruel situation where the chances of their being given the treatment they need depend on where they live.”

“Drugs companies, politicians and the NHS have a responsibility to patients to fix this system,” she added.