MSD’s Keytruda (pembrolizumab) has secured a significant recommendation from the UK’s National Institute for Health and Care Excellence (NICE), signaling a groundbreaking moment in cancer immunotherapy evaluation. In a historic move, NICE has endorsed Keytruda based on results from a ‘basket trial,’ a novel approach that enrolls patients with varying tumor locations but sharing a common genetic mutation, a concept pioneered by MSD.
This milestone decision brings Keytruda into the spotlight as the first cancer immunotherapy to gain NICE approval through a basket trial. Basket trials, by evaluating treatments across multiple cancer types simultaneously, optimize participant numbers and shorten trial durations. These efficiencies accelerate the path to treatment access by bypassing the need for separate studies.
Keytruda’s recommendation by NICE extends its usage within the National Health Service (NHS) to encompass a range of cancers characterized by microsatellite instability (MSI) or mismatch repair deficient (dMMR) mutations. Previously treated patients dealing with endometrial, biliary, colorectal, gastric, and small intestine cancers with MSI-H or dMMR positivity can now consider Keytruda as a second-line alternative to chemotherapy.
Amongst the approved alternatives for second-line treatment in colorectal cancer stands GSK’s Jemperli (dostarlimab), a PD-1 inhibitor also designated for MSI-H and dMMR tumors. NICE granted its support for Jemperli last year via the Cancer Drugs Fund (CDF).
Backing the recommendation are the outcomes of two phase 2 open-label studies: KEYNOTE-164 focusing on colorectal cancer and KEYNOTE-158 encompassing endometrial, gastric, biliary, and small intestine cancers. These studies unveiled objective response rates ranging from 33% to 35%, accompanied by a notable two to four months enhancement in progression-free survival (PFS) for patients.
“MSI-H/dMMR tumours are associated with a poorer prognosis in advanced cancers which means there is a high unmet for patients with these gene features. The results of the basket trial for colorectal, endometrial, gastric, small intestine and biliary cancer will help to address a high unmet clinical need going forward. The Medicines and Healthcare products Regulatory Agency (MHRA) approved Keytruda for the five MSI-H/dMMR tumour types last year. This treatment option offers a small but important step forward in the treatment of some people with cholangiocarcinoma for whom, to this point, there has been so very little.”
– David Lond, head of oncology at MSD UK.
This development has been met with enthusiasm by patient advocacy groups, including AMMF, a representative of cholangiocarcinoma patients. With this recognition, which comes at a time when cholangiocarcinoma diagnosis often occurs in advanced stages, these patient communities have newfound hope for more effective treatment options.
As Keytruda forges a pioneering path through the realm of cancer immunotherapy evaluation, it redefines how therapies can be evaluated and approved across diverse cancer types. This innovative approach aligns with the urgency to provide better solutions for patients facing complex and often late-stage cancers, underscoring the transformative potential of precision medicine.
