NICE recommends breakthrough life-extending combination treatment for advanced bladder cancer.
The new treatment offers hope to thousands living with advanced urothelial cancer, with clinical trials showing overall survival rates were almost twice as long compared to standard treatment.
NICE has recommended a new first-line treatment for adults with advanced bladder cancer, marking a significant breakthrough for patients facing this devastating disease.
Enfortumab vedotin (also known as Padcev and made by Astellas) with pembrolizumab (also known as Keytruda and made by MSD) is recommended for treating unresectable or metastatic urothelial cancer for people who are eligible for platinum‑containing chemotherapy.
The combination treatment has been hailed by clinical experts as a ‘step change’ in cancer care, with clinical trial evidence showing it can significantly extend both progression-free survival and overall survival compared to current standard chemotherapy treatments.
The treatment is available in the NHS from today (Thursday, 21 August).
This is a highly promising and effective new drug, with clinical trial results highlighting the tremendous difference it could make to the length and quality of people’s lives.
Advanced bladder cancer is a devastating condition which can have a substantial impact on people’s daily lives, often leading to them struggling to work, travel or maintain physical activity.
Today’s recommendation highlights that NICE is committed to helping get the best care to people fast, while ensuring value for the taxpayer.
More than 10,000 people are diagnosed with bladder cancer each year in the UK and only around 10% of people with stage 4 bladder cancer will survive five years or more after they are diagnosed.
It is estimated over 1,200 people per year could benefit from this targeted treatment, which is given as an infusion in a clinic or hospital. It is made up of a monoclonal antibody which recognises and kills certain cancer cells.
The standard treatment for unresectable or metastatic urothelial cancer is platinum-based chemotherapy (cisplatin or carboplatin) and a range of other medications.
The recommendation is based on evidence from a clinical trial involving 886 adults with untreated unresectable locally advanced or metastatic urothelial cancer. Progression-free survival almost doubled for people taking enfortumab vedotin and pembrolizumab to 12.5 months compared with 6.3 months for those on chemotherapy and overall survival increased to a median of 33.8 months for those on the combination treatment compared with 15.9 months for those on chemotherapy.
Around 30% of people on the trial achieved a complete remission following treatment with enfortumab vedotin and pembrolizumab, meaning all detectable signs of the disease had disappeared.
Urothelial cancer occurs in the cells that form the inner lining of the bladder and urinary tract, with most cases originating in the bladder. Urothelial cancer accounts for around 90% of all bladder cancers. It is considered metastatic when the cancer has spread outside the pelvis. Unresectable cancer cannot be completely removed by surgery.
In advanced bladder cancer, symptoms may include pelvic or bone pain, unintentional weight loss, swelling in the legs, difficulty urinating, lower back pain, and fatigue.
Patient experts highlighted the limited first-line treatment options available for unresectable or metastatic urothelial cancer. They described living with the condition as "intensely challenging and emotionally exhausting," with the debilitating disease affecting all aspects of a person’s life and having a significant impact on their families and friends.
Currently, only about 12% of people on platinum-based chemotherapy see an improvement in their condition, highlighting the urgent need for more effective treatments.
ABC UK, bladder cancer patients and their families welcome this much-needed, step forward in treatments available for this hard-to-treat cancer. This new drug has the potential to increase how long people have before their cancer gets worse and how long they live compared with the current, limited, treatment choices available. It’s also of importance that this treatment can mean these patients can experience a better quality of life with less hard to tolerate side effects.
For people with cancer, every moment matters.
This breakthrough treatment will give people precious extra time with their families. This is exactly what modern healthcare looks like - saving lives by providing access to the best support available.
Through our Plan for Change, we're turning the tide after years of cancer services being run into the ground and delivering the world class treatments people to deserve, that will create an NHS fit for the future.
The independent appraisal committee considered this evidence alongside testimony from patient and clinical experts and were able to apply a severity modifier of 1.2. This allows medicines that are used to treat conditions classed as high or medium severity to have a higher threshold at which they can be considered cost effective.
Commercial arrangements have been put in place through patient access schemes which make both drugs available to the NHS with a confidential discount.