Around 200 patients each year could benefit from combining the standard use of two marketed drugs to treat rare blood disorders in NHS patients.
Novartis' Tafinlar (dabrafenib) and Takeda's Adcetris (brentuximab vedotin), in combination with bendamustine, will offer patients an additional treatment option when other therapies have been ineffective.
While these drugs are not entirely new to the UK's National Health Service, the new approvals allow them to be used routinely to treat rare blood disorders.
In the UK, Tafinlar was first approved for the treatment of a certain type of melanoma in 2013, and Adcetris was approved a year earlier for the treatment of certain types of lymphoma. These drugs have been available and regularly funded by the UK National Health Service for these indications for many years.
Tafinlar is now available off-label as a standard treatment for histiocytic neoplasms with the BRAFV600E mutation when standard treatment has failed. Histiocytic neoplasms are rare and potentially fatal blood disorders. Without effective treatment, approximately one in ten children with the high-risk disease dies within a year of diagnosis, and seven in ten adults within five years.
Novartis's oral targeted cancer therapy works by blocking cancer cell growth signals. Unlike chemotherapy, its outpatient nature allows patients to receive treatment at home, rather than in a hospital, where close monitoring and hospitalization are required.
Meanwhile, a combination of Adcetris (co-developed by Takeda and Pfizer) and bendamustine will benefit children aged eight years and older with Hodgkin lymphoma that has relapsed or failed to respond to initial treatment.
NHS National Clinical Director for Cancer, Professor Peter Johnson, said: "This is a landmark moment for people with histiocytic neoplasms and Hodgkin lymphoma, giving them access to new treatment options that may otherwise be out of reach.".
«For people living with the uncertainty associated with these rare cancers, these innovative treatments can offer something hard to find – new hope – and allow many patients to be treated at home rather than in the hospital, so they can spend more time enjoying life.».
Access to two treatments for patients with rare blood cancers is in line with the UK government's National Cancer Plan for England. Published in February 2026, the strategy aims to ensure that three-quarters of patients diagnosed with cancer are free of cancer or living a full life within five years. Over the next decade, the government will implement a number of strategies, including improving access to new treatments and strengthening patient support during treatment.
Medicines are introduced through the National Health Service England Clinical Priorities Advisory Group (CPAG), which annually evaluates dozens of specialized medicines, medical devices, and treatments based on their patient benefit, clinical effectiveness, and value for money. Unlike NICE, this committee prioritizes specialized procurement policies, often for rare diseases and unapproved uses of existing medicines.
According to NHS England, CPAG experts noted that the discovery of genetic factors that cause cancer in patients means that targeted therapy has become an additional treatment option. This provides more personalized care with fewer side effects, NHS England added.
Leslie Coombs, a 69-year-old woman from the UK, received Tafinlar four years ago through Novartis's preferential access program. Although chemotherapy cured her lymphoma, the histiocytosis persisted. She then began taking Tafinlar, which led to remission.
Coombs stated, "I started taking [the drug] 10 days before my scheduled radiation treatment, but within three days, the tumor began to shrink rapidly. My family and I were shocked—the radiation treatment had to be suspended, and I am still in remission. Thanks to the drug, I can continue to lead a very active lifestyle.".
The article "Novartis and Takeda drugs win approval for routine use on the UK National Health Service for rare blood cancers" was originally created and published by Pharmaceutical Technology, a brand owned by GlobalData.