Methodological differences and the appropriate application of oncology value frameworks to assess clinical value

Background

A number of value frameworks have been developed recently as tools to evaluate new oncology products. The frameworks differ in stated objectives and methodology, and each uses a different set of criteria for evaluating products. We compare and contrast the frameworks, to identify the important differences and similarities between them, and conclude with the implications for clinical decision-making.

Methods

The methodologies of six value frameworks (ASCO, NCCN, ESMO, MSKCC, DrugAbacus, and ICER) were reviewed. Using the published scoring systems where available, we evaluated 33 recently approved oncology drugs by scoring them against individual framework criteria, using published data from pivotal trials. We compared results and identified the origins of the differences in scoring outcomes within the underlying methodologies, focusing on the clinical domains.

Results

The range of approaches to assessing clinical value, both in terms of assigning scores and the relative importance given to those scores, can produce considerable variability in the value assigned to a treatment in different value frameworks. Scoring frameworks are often complex and some important aspects remain subjective. Specific attributes, such as whether a treatment is considered to have curative potential or the pivotal trial is a single-arm Phase 2 study, have a significant bearing on value framework scores but the reasons for assigning this level of impact are not clear.

Conclusions

Understanding the methodology employed by a value framework to assess clinical value is critical to interpreting the scores of a product within it. The assessed clinical value of a treatment can depend on assumptions made during the assessment of efficacy and safety and the context in which the assessment takes place. To avoid inconsistencies in clinical decision-making and unintended consequences on incentives for innovation, the results of value frameworks should be used cautiously. Further evolution of value frameworks will be necessary to produce more transparent and robust tools for decision-makers.

Clinical trial identification

Legal entity responsible for the study

PRMA Consulting Ltd

Funding

PRMA Consulting Ltd

Disclosure

G. Monnickendam, K. Mortak, J. McKendrick: Employee of PRMA Consulting Ltd.