The National Institute for Health and Care Excellence (NICE) has issued draft guidance not recommending elacestrant, known as Korserdu, for treating advanced estrogen receptor-positive, HER2-negative breast cancer with an ESR1 mutation in England. This decision impacts approximately 1,000 women who have developed resistance to hormone therapies. The rejection stems from concerns over the robustness of clinical trial data regarding the drug's effectiveness compared to existing treatments.
Clinical Significance of Elacestrant
Elacestrant represents a novel approach as an estrogen receptor degrader, targeting and degrading estrogen receptors to impede the growth of estrogen-dependent cancer cells. This mechanism is particularly relevant for cancers that have developed ESR1 mutations, which render them less responsive to traditional hormone therapies. Up to half of advanced or metastatic breast cancers treated with hormone therapy develop these mutations, leading to disease progression and poorer survival rates.
Claire Rowney, chief executive at Breast Cancer Now, emphasized the potential benefits of elacestrant, stating it "could bring precious additional time with loved ones" for patients with this type of secondary breast cancer. She expressed deep concern that patients might be denied this chance due to the current rejection.
NICE's Concerns and Data Uncertainty
NICE acknowledges the distress and stress associated with incurable secondary breast cancer for patients and their families. However, Helen Knight, director of health technology evaluation at NICE, highlighted the "considerable uncertainty" surrounding estimates of how long elacestrant can prevent disease progression compared to current clinical practice. This uncertainty also prevented the committee from confirming whether a severity modifier could be applied.
The decision was based on indirect comparisons with current drugs, and NICE is requesting more clinical trial data from Menarini Stemline to better assess the drug's long-term efficacy. The draft guidance is open for public consultation until October 22, offering an opportunity for further data and perspectives to be considered.
Call for Collaboration
Breast Cancer Now is urging NICE and Menarini Stemline to collaborate closely to address the data concerns and potentially reverse the provisional decision. Rowney stated, "Unless this happens, patients could be denied the precious chance of being offered this targeted treatment option that gives them more time before their cancer progresses."
NICE has indicated its willingness to work with the company to resolve the identified issues. Historically, a significant percentage of topics initially not recommended by NICE at consultation have turned positive before final guidance publication, offering hope for a potential reversal in this case.
