Infants, young children and adults with rare blood cancers are among around 200 patients a year expected to benefit from two new treatments approved for use by NHS England.
The health service said the medicines would offer new options for patients whose cancer has returned, has not responded to standard treatment or cannot be managed with conventional therapies. The treatments include dabrafenib, a tablet for histiocytic neoplasms, and a combination of brentuximab vedotin and bendamustine for some children and adults with Hodgkin lymphoma.
NHS England said the rollout begins from today following assessment by its Clinical Priorities Advisory Group, which reviews specialist medicines, medical devices and treatments according to patient benefit, clinical effectiveness and value for money.
Dabrafenib is designed to treat histiocytic neoplasms, a group of rare and potentially fatal blood cancers. The medicine works by blocking proteins that help cancer cells grow, which can slow or stop the disease. NHS England said its approval would allow many eligible patients to take the drug at home rather than spending more time in hospital for more invasive treatment.
The condition can be particularly serious in patients with high-risk disease. NHS England said that, without effective treatment, around one in 10 children with high-risk disease die within a year of diagnosis, while seven in 10 adults die within five years.
The second treatment, a combination of brentuximab vedotin and bendamustine, will be available for children as young as eight with Hodgkin lymphoma that has returned or has not responded to initial treatment. Hodgkin lymphoma is a rare cancer affecting around 1,800 people in England each year.
For between 10% and 30% of patients, equivalent to about 180 to 540 people a year, the disease comes back or does not respond to standard treatment. NHS England said the new combination targets cancer cells more precisely by binding to a protein on the surface of the cancer cell and delivering a cancer-killing drug directly into it.
Patients receiving the combination treatment will have it through a drip into a vein. NHS England said it has been shown to achieve high remission rates, is generally well tolerated, and can help patients become well enough to receive a potentially life-saving stem cell transplant.
Professor Peter Johnson, NHS national clinical director for cancer, said: “This is a landmark moment for people with histiocytic neoplasms and Hodgkin lymphoma, giving them access to new treatment options they might not otherwise have had.
“For people living with the uncertainty of these rare cancers, these innovative therapies could offer something that can be hard to find – renewed hope – while allowing many patients to take their treatment at home instead of in hospital, so they can spend more time living their lives.
“Improving the treatment for rare cancers is a key ambition of the new 10 Year Cancer Plan, and the NHS is committed to ensuring more patients have access to advance, safe and effective treatments, which can save or transform their life.”
NHS England said the approvals reflect changes in the treatment of rare blood cancers as researchers identify specific genetic drivers of disease. Its advisory group noted that many patients can now benefit from targeted therapies, including BRAF and MEK inhibitors, shifting treatment towards more personalised approaches for those who do not respond to, or cannot tolerate, conventional options.
Professor James Palmer, national medical director for specialised services at NHS England, said the treatments could be life-changing for children and adults affected by rare blood cancers. He said: “I want to thank the many clinical, commercial and patient experts that have helped NHS England arrive at this point because their expertise and support has been invaluable.
“The rollout of these new treatments are excellent examples of the NHS’s commitment to offering access to innovative medicines for those living with rare conditions.”
One patient who has already received dabrafenib is 69-year-old Lesley Coombs, from Cambridgeshire. She was given the medicine four years ago through a compassionate access programme offered by the manufacturer. NHS England said she had been undergoing treatment for follicular lymphoma alongside histiocytosis with the BRAFV600E mutation, which she described as a “1 in 60 million” combination.
Without the medicine, Lesley may have required a stem cell transplant as well as radiotherapy, which would have involved a long recovery even if successful. She said: “I started the dabrafenib 10 days before my planned radiotherapy, but within three days of starting the drug the tumour had rapidly started to shrink. My family and I were amazed – the radiotherapy was put on hold and to this day I continue to be in remission.”
She said the treatment had allowed her to continue an active lifestyle, including cycling, walking and spending time with her grandchildren. “Access to the drug and the care of incredible staff at both Hinchingbrooke and Norfolk and Norwich University Hospitals allowed me to pick up my life again after cancer and I’m delighted many more people will now be able to benefit from this medicine too,” she said.
Blood Cancer UK welcomed the announcement. Dr Rubina Ahmed, the charity’s director of research, policy and services, said: “Today’s announcement is welcome news for people affected by some of the rarest blood cancers. Blood cancer is the UK’s third biggest cancer killer, and some of the rarest forms can be particularly hard to treat, with few options available if the disease comes back or stops responding to treatment. Every new treatment offers renewed hope for patients and their families.”
Lynn Jackson, executive director at Histio UK, said NHS access to dabrafenib for eligible patients with BRAFV600E mutation positive histiocytic neoplasms would “change lives and outcomes now and in the future”.
NHS England said the main symptoms of Hodgkin lymphoma include a painless lump or swelling, usually in the neck, armpit or groin, pain in glands after drinking alcohol, a high temperature, night sweats, shortness of breath, itchy skin and sudden weight loss. Symptoms of malignant histiocytosis can include one or more rapidly growing lumps, localised pain, numbness, tingling and persistent fever.
The Clinical Priorities Advisory Group considers drugs and treatments that are not being assessed by the National Institute for Health and Care Excellence, including where a medicine is not licensed for the clinical indication in question.
Public Health Minister Sharon Hodgson said the treatments were an important step for people living with rare blood cancers. She said: “By backing innovation and working closely with the NHS to bring promising medicines into routine care, we are helping ensure more patients can benefit from the latest breakthroughs, while giving families greater confidence that world-class treatment is available closer to home.”
