Aims: Current methodologies and guidelines for determining clinical effectiveness and cost-effectiveness may have limited applicability to the increasingly personalized nature of ‘omics’ treatment strategies. Using examples from oncology, the authors argue for the adaptation and tailoring of three existing methods for ensuring development and clinical use of multi-omics-guided therapies that are effective, safe and offer value for money.

Methods: Current development and assessment frameworks are first discussed, with a focus on early development and research prioritization, clinical study designs and economic evaluation methodologies. Unique issues requiring consideration in the development, clinical assessment and economic evaluation of multi-omics treatment strategies are then outlined. Finally, the adaptation of three existing methods to address the challenges in developing and evaluating multi-omics treatment strategies is proposed, with a discussion of their limitations and possible impediments to their implementation.

Results: We propose three approaches to modify current methodologies and discuss their respective applications for clinical development and reimbursement: 1) the use of value of information analysis by manufacturers to address difficulties in early identification of biomarkers by identifying evidence with the greatest uncertainty likely to affect investment decisions as well as use by reimbursement agencies to determine the value in further stratifying patients using ‘omics’ markers by quantifying the cost of ignoring ‘omics’ heterogeneity; 2) modified randomized controlled trial designs, specifically N-of-1 trials to collect clinical and economic evidence from micro-subgroups; and 3) stated preference methods such as discrete choice experiments to provide methods of determining the differential economic value among micro-subgroups.

Conclusions: There is a need to modify the use of conventional development processes, clinical study designs and economic evaluation methodologies to ensure the complete value of multi-omics treatments strategies are considered. A truly multi-disciplinary approach will be required to fully understand the reasons for inter-individual drug response heterogeneity and how these individual differences will affect the value assessment of developed therapeutic approaches.