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Clinical utility: An imprecise construct at the heart of precision medicine

04 November 2015 Cameron Lockwood

In looking at the molecular diagnostics landscape historically, the industry's focus has been on establishing the analytical and clinical validity of diagnostic testing. Having recently completed a deep dive into the subject as part of our new study on precision medicine, what certainly rings true from our discussions with payers and other stakeholders across the major markets is that perceptions on the value of molecular testing will increasingly hinge on experimental demonstration of clinical utility. Part of the challenge for manufacturers, whether of stand-alone diagnostics or potentially those pursuing a test-treat strategy, stems from the lack of a uniform definition of clinical utility, let alone the evidence requirements for its evaluation.

In completing our research on the US market in particular, what I found interesting is the role that changing dynamics in the pharmacy benefit manager (PBM) space have played in this part of the equation. At one time, PBMs such as Medco were actively investing in personalised medicine programmes, designed to help payer clients optimise pharmacy benefits. For example, prior to its acquisition by Express Scripts in 2012, Medco had rolled out Genetics for Generics, a scheme to promote and validate patient use of generics through pharmacogenomic testing, and was generally offering testing services to clients through its Precision Health Solutions division. The organisation was also active in helping test developers in the design of clinical utility studies.

However, this latter activity was axed and pharmacogenomic initiatives scaled back following acquisition by Express Scripts, which has held a more conservative view on the personalised medicine market. For example, while Medco had previously promoted CYP2C19 testing for use of clopidogrel under its Genetics for Generics programme, Express Scripts did not recommend such testing prior to its acquisition of the latter.

Similarly, after its full-scale buy-out of Generation Health in 2012, CVS Caremark scaled back that subsidiary's work helping test developers with clinical utility and cost-effectiveness studies, as well as with payers in the design of utilisation and coverage policies for testing. While the former CEO of Generation Health, Rick Schatzberg, had intended to launch a new enterprise called MetaDiagnostic to carry on these activities, the entity does not appear to have materialised.

As an example of the sort of work that PBMs used to engage in, one can look to the early example of Roche Diagnostics' AmpliChip, the first FDA-cleared microarray assay. AmpliChip was cleared for CYP2D6 testing in 2004 and CYP2C19 testing in 2005. Both of these genes code for enzymes belonging to the cytochrome P450 system, which is important in the hepatic metabolism of a broad range of therapies ranging from antidepressants to beta blockers and proton pump inhibitors.

As an early entrant into the molecular diagnostics scene, AmpliChip is an example of a test that launched with minimal data on clinical utility, with Roche speaking out at the time against insurers holding test developers to evidence standards deemed to be out of sync with the diagnostics sector. However, the test did not fare well with payers, specifically because of the clinical utility question. Against this backdrop, in 2007, Medco, in partnership with LabCorp, launched a study of the utility of AmpliChip testing in patients receiving tamoxifen for breast cancer.

As it goes, since that time, many major insurers in the US continue to consider pharmacogenetic testing for tamoxifen as well as other agents investigational. But the point is that dynamics in the PBM market have probably slowed the evolution of precision medicine to some degree.

New start-up services offering decision-support tools to payers and physicians are beginning to enter the US marketplace. One example is Intervention Insights, a Waltham, Massachusetts based outfit providing what it calls a collaborative decision-support platform in molecular oncology. Its service supports payers in the design of evidence-based coverage policies for testing. However, a gap probably remains in the market for clinical utility studies, a reality contributing to the lack of a standardised evaluation framework in the US.

Going forward, we have seen a number of stakeholders such as Naomi Aronson, president of the Blue Cross and Blue Shield Association Center for Clinical Effectiveness, call for a patient-centred approach to clinical utility evaluation. In March 2015, the American College of Medical Genetics and Genomics issued a position statement arguing that clinical utility should not be limited to whether a test leads to an intervention that results in improved outcomes, but should also take into account factors such as the effects of receiving a diagnosis, patient management, and impact on both family members and broader society. Some of these ideas also came through in our discussions with UK payers.

The challenges facing precision medicine are plenty - the ongoing dialogue over clinical utility, and what platforms can best support industry to provide evidence thereof, will be an important part of the puzzle.

Here at IHS Life Sciences, we are looking forward to attending Virtue Insight's upcoming conference, Personalised Medicines & Biomarkers 2015, to be held on the 2nd and 3rd of December at the Pestana Chelsea Bridge Hotel in London. The conference will touch upon evolving strategies and developments in the personalised medicine space, incorporating a broad selection of themes ranging from clinical utility to partnerships and novel biomarker applications.

For more information on our new study, Pathways to Precision Medicine: Navigating Payer Needs and Healthcare Systems through Molecular Diagnostics, please click here.

Cameron Lockwood is a life sciences analyst for IHS
Posted 4 November 2015



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