Biomarker-targeted therapies: the answer to advancing equitable cancer care? 

GPI attended ASCO, held from 3rd to 7th June 2022, with this year's theme focusing on Advancing Equitable Cancer Care through Innovation. Here we discuss biomarker-targeted therapies related to market access and pricing.

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Biomarker-targeted therapies have revolutionised cancer care. Patients with specific biomarkers now have efficacious treatment options available to improve their survival and reduce the toxicities of chemotherapy. In parallel, patients without these biomarkers have limited treatment options. This inequity in cancer care has been the focus of oncology research, at ASCO.  


We ask: are restricted populations and personalised medicines the best direction to advance equitable cancer care through innovation? 

Clinical evidence and regulatory approval 

In many clinical trials, patient populations are stratified by biomarker. Significant improvements are often demonstrated in the biomarker-specific population but limited in the overall and non-biomarker population. Furthermore, upon regulatory review, survival data are normally immature, leaving decision-makers with limited evidence.  

Despite this, approval of broad oncology indications is common, regardless of patient biomarker status. Follow-up studies and real-world evidence contribute to additional understanding and label refining. Therefore, ultimately, patients without the specific biomarker have limited efficacious innovative treatment options, continuing the inequity in cancer care. 

Case study: detrimental efficacy for biomarker-negative populations 

Zejula (niraparib) demonstrated improved survival in patients with BRCA-positive ovarian cancer, but received a broader FDA indication for maintenance treatment in recurrent ovarian cancer.  However, a follow-up safety warning of a potential detrimental effect on survival in patients with no BRCA mutation was recently issued. This highlights that innovative treatment may only be applicable and effective in restricted patient populations. 


High development costs and applicability to a restricted patient population drives highly priced innovative biomarker-targeted oncology therapy. Therefore, affordability may further contribute to disparities in cancer care. Key discussion at ASCO included: 

  • Cost inequities in the US: The US has the highest cost of innovative biomarker-targeted oncology treatments globally, presenting insurance and out-of-pocket payment challenges. Certain populations, such as patients with low incomes or specific insurance plans, are unable to access the benefits of these therapies due to high cost.  
  • Affordability inequities between low-income and high-income countries: Low-income countries lack basic healthcare infrastructure and have limited access to well-established chemotherapies. Therefore, access and affordability of innovative biomarker therapies for restricted populations are not a priority, regardless of clinical benefit. 

To improve affordability and subsequent access of innovative biomarker therapies globally, several methods were presented at ASCO, including: 

  • Joint procurement approaches to ensure that affordability extends to low-income countries 
  • Legislation and data reporting to drive down prices, such as State Drug Price Transparency reporting rules as described in our previous blog 
  • Conduct of health technology assessments (HTA) to drive value-based decision making 


Although research within oncology is focussed on biomarker-targeted therapies and restricted populations, challenges with evidence availability and affordability continues to drive inequities in cancer care. As highlighted by the research at ASCO, steps to addressing these areas are in development. However, the feasibility of implementation within the US and globally remains to be demonstrated.   

By Kate Anstee, Senior Consultant, Global Pricing Innovations 

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