The overall level of unmet need in hepatocellular carcinoma (HCC) is high due to its severity and low survival. However, the needs will soon be relatively well met, as many new therapies entering the market have different mechanisms of action and are aiming to meet needs in different treatment settings, says GlobalData, a leading data and analytics company.
The variety of pipeline products in the adjuvant setting (Barcelona clinic liver cancer [BCLC] Stage A), intermediate (BCLC Stage B), and advanced (BCLC Stages B and C) stages bring hope for improvements to the cure rate for early-stage disease and survival in all stages over the currently marketed therapies.
The five key unmet needs and opportunities in the HCC market are as follows: better HCC surveillance and prophylactic treatments, curative therapies in early-stage HCC, improved survival for intermediate-stage HCC, predictive biomarkers, and treatments for patients with impaired liver function.
Key opinion leaders (KOLs) interviewed by GlobalData noted that better HCC surveillance and prophylactic treatments are highly desirable since a delay in diagnosis puts the patient at risk for diagnosis of a higher BCLC stage, where they may be deemed ineligible for curative treatment options.
Mandana Emamzadeh, PhD, Pharma Analyst at GlobalData, comments: “While other oncology indications experienced improved cure rates from adjuvant therapy, there are currently no adjuvant therapies available for HCC patients. Current studies are investigating immuno-oncology (IO) agents alone or in combination with Avastin after ablation or resection as an adjuvant therapy, which is intended to reduce the recurrence rate of HCC.”
Furthermore, the pipeline products in the intermediate setting are aiming to combine IO agents, tyrosine kinase inhibitors, and angiogenesis inhibitors with transarterial chemoembolization (TACE). KOLs revealed that combining systemic therapies with TACE might improve TACE’s efficacy in comparison with TACE alone, but the exacerbated adverse effects should not be overlooked. In addition, more studies are required to evaluate the safety and efficacy of systemic therapy versus TACE and to investigate the replacement of TACE with the advanced HCC treatments for most, if not all, intermediate patients.
Emamzadeh continues: “A large number of pipeline agents with innovative approaches are under investigation for adjuvant, intermediate, and advanced therapy. However, KOLs asserted that the assignment of patients to a suitable therapy is not feasible with current methods. Therefore, new biomarkers are urgently needed to select the most appropriate treatment for each individual HCC patient. GlobalData anticipates that the opportunities will remain for further biomarkers to predict high responders.”
Emamzadeh adds: “GlobalData’s primary research revealed that the most underserved populations are Child-Pugh class B and C patients with decompensated liver cirrhosis. The Child-Pugh score describes the functional status of the liver and is one of the criteria used in BCLC staging, ultimately deciding the most suitable treatment. According to KOLs, little is known about anticancer treatment in these patient populations since they are usually excluded from clinical studies due to tumor-related complications. Moreover, the current palliative treatment is not beneficial in treating the underlying liver disease or increasing the survival rate in these stages. As a result, a significant unmet need remains.
“It is expected that the situation will continue to improve over the next decade as new therapies are launched on the market. However, further research is in high demand to provide HCC patients with a better quality of life.”