Drug Assessment Framework Shows Promise for Identifying Cancer Drugs

Using a structured and elicit approach, researchers designed a criterion-based valuation framework to provide a transparent and consistent method with which to value and prioritize cancer drugs to facilitate the delivery of affordable cancer care, according to a report published in Cancer. 

“The [drug assessment framework; DAF] quantifies stakeholders’ expectations of meaningful advances in patient outcomes and is an important step toward the delivery of high-value cancer care,” the researchers wrote.

The authors recruited a multistakeholder group who identified and weighted key criteria to create the DAF. The final DAF included 10 criteria: overall survival, progression-free survival, response rate, quality of life, toxicity, unmet need, equity, feasibility, disease severity, and caregiver well-being. The first 5 represented approximately 64% of the total weight.

DAF scores ranged from 0 to 300, reflecting both the expected impact of the drug and the quality of supporting evidence. Construct validity assessed the degree to which DAF scores were associated with past pan-Canadian Oncology Drug Review (pCODR) funding recommendations and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) scores. 

When applied to the last 60 drugs (with reviewers blinded) reviewed by pCODR, those drugs with positive pCODR funding recommendations were found to have higher DAF scores compared to drugs not recommended (103 vs 63; Student t test P = .0007). DAF clinical benefit criteria were only mildly correlated with ESMO-MCBS scores (correlation coefficient, 0.33; 95% CI, 0.009-0.59). Sensitivity analyses did not vary the results. 

Outside of the fundamentals of clinical benefit and cost, stakeholders identified additional criteria (unmet need, equity, feasibility, disease severity, and caregiver wellbeing) as important in cancer drug decision-making processes. The DAF assessed both curative- and palliative-intent therapies, given that stakeholders ultimately valued the importance of benefit more than the intent of therapy.

“This derived score represents the overall impact of a new cancer treatment, and the quality of evidence used to generate the score,” the authors wrote. “This is the first version of the DAF, a dynamic tool that will be revised as public feedback is ascertained and the landscape of cancer treatment evolves.”

The results of the final calculated score can be used in conjunction with the incremental cost, incremental cost-effectiveness threshold, and/or budget impact analysis to inform clinical decision making and funding decision and establish explicit funding priorities, according to the authors. Additionally, the DAF can be applied to a group of drugs submitted for funding to help identify highly ranked drugs that, within the setting of favorable economic profiles, then can be prioritized for funding. This suggests that both private and publicly funded health care systems could benefit from multicriteria decision analysis (MCDA) options like DAF.

“The DAF can help decision makers prioritize the highest impact (highest scoring) drugs,” the researchers wrote. “Although the calculated score summarizes overall impact and quality of evidence, the decision-making deliberations and discussions are still necessary.” 

Researchers suggested that the entire framework for any MCDA be reviewed periodically to ensure the inclusion of geographic and temporal changes in stakeholder principles and values. Additionally, the authors noted that in order to implement MCDA, expert training of decision makers may be required to ensure the appropriate application of values assessment tools, avoiding possible individual cognitive biases. 

Reference:

Ezeife DA, Dionne F, Fares AF, et al. Value Assessment of Oncology Drugs Using a Weighted Criterion-Based Approach. Cancer. doi:10.1002/cncr.32639.