The frameworks that have been discussed (e.g., EGAPP, TEC) are designed to maximize internal validity but are not optimal with regard to feasibility or timeliness, which are important to the diagnostics industry and to patients. There is a need for methodologies to evaluate clinical utility that can achieve an acceptable balance of these elements, Tunis said, noting that the correct balance of validity with feasibility and timeliness is not solely a methodology issue. There is also a social judgment that must be made collectively by all stakeholders regarding the acceptable level of uncertainty.

A participant said that the goal in developing the Oncotype DX test for breast cancer, as well as with tests currently in development for colon cancer and prostate cancer, was to gather evidence that would be persuasive to both clinicians and to payers. Performing RCTs for diagnostics is not a necessity, and the length of time they take to produce outcomes data would render the test obsolete. Payer support is also required to ensure patient access.

The participant identified key questions in balancing stakeholder needs, including What are the risks and ramifications of being wrong? and, How comfortable are we with those risks? The further that studies deviate from the principles of the RCTs, the more that certainty declines. To move rapid evaluation forward, new data must be evaluated systematically as they emerge, and decision makers must be willing to stop coverage when it becomes clear that a product does not work as originally thought. It was noted that this is what already happens in many systems, such as the Ontario experience that was presented.1


One organizational model that can help address issues of funding, knowledge generation, and social change in the area of data sharing is a public-private, pre-competitive research partnership, said Aled Edwards of the Structural Genomics Consortium. Pre-competitive research is knowledge-generating research where data is openly shared and not encumbered by any restrictions on its use. For genetic tests, current precompetitive research is focused on generating hypotheses.

The Structural Genomics Consortium, founded in 2004, has 250 scientists working in three laboratories located at the University of Toronto, the University of Oxford, and the Karolinska Institute. Initially focused on studying the three-dimensional protein structure of drug targets, the consortium is now also working on pre-competitive medicinal chemistry. Thirty medicinal chemists from industry partners (including GlaxoSmithKline, Novartis, Eli Lilly and Company, and Merck) are generating new


Discussed by Levin in Chapter 3.

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