NICE has recommended the use of MSD's KEYTRUDA (pembrolizumab) as a perioperative immunotherapy for patients with resectable locally advanced head and neck squamous cell carcinoma (LA HNSCC) whose tumours express PD-L1 with a Combined Positive Score (CPS) ≥1.
The decision marks the first regulatory advance in this treatment setting in more than 20 years.
The approval covers pembrolizumab administered as neoadjuvant therapy prior to surgery, continued adjuvantly in combination with standard-of-care radiotherapy, with or without concomitant cisplatin and then as monotherapy.
It represents the first perioperative immunotherapy to be recommended for LA HNSCC in England.
The decision is supported by data from the Phase III KEYNOTE-689 trial, which demonstrated a statistically significant improvement in event-free survival (EFS)—the study's primary endpoint—versus adjuvant radiotherapy alone.
In the CPS ≥1 population, the pembrolizumab-based regimen reduced the risk of disease recurrence, progression or death by 30%. Median EFS was doubled: 59.7 months in the pembrolizumab arm versus 29.6 months in the comparator arm.
Why this matters
Head and neck squamous cell carcinoma accounts for approximately 90% of all head and neck cancers, which represent the ninth most common cancer type in the UK.
With around 12,800 new cases and 4700 deaths annually (and mortality rates projected to rise by 12% by 2038-40), the unmet need in this indication is considerable.
Standard treatment had remained largely unchanged since the early 2000s, limited to surgery followed by post-operative radiotherapy with or without chemotherapy, with persistently high recurrence rates and poor long-term outcomes.
Participant-reported outcomes from KEYNOTE-689 indicated stable health-related quality of life during neoadjuvant pembrolizumab therapy, with no overall detrimental impact on disease-related symptom scores versus the control arm.
The safety profile was broadly comparable between groups for most adverse events, though immune-mediated adverse events of any grade were more frequent in the pembrolizumab group (43.2% vs 10.2%).
Pembrolizumab is already approved in the UK for metastatic or unresectable recurrent HNSCC. The NICE recommendation extends its use earlier in the treatment pathway, offering a new option for patients with surgically resectable disease and expanding the role of PD-1 inhibition across the HNSCC continuum of care.
John McNeill, Oncology Business Unit Director at MSD in the UK, said: "We welcome today's decision from NICE, which represents an important step forward in expanding treatment options for patients with resectable, locally advanced head and neck squamous cell carcinoma."
Head and neck cancer remains an area of considerable unmet need, with many patients facing complex treatment pathways and a high risk of recurrence.
"This decision is a meaningful milestone, particularly for patients who may benefit from new treatment options."