Despite lots of headlines, an underappreciated storyline this year was the undercurrent surrounding the validity of medical tests:
—The Milwaukee Journal Sentinel published a long Watchdog Report focused primarily on quality issues with waived lab tests, which don’t require FDA approval.
—The headline of a shorter version in the Wall Street Journal asked whether lab testing is the Wild West.
—The Journal was responsible for tugging at a similar thread over the last few months and has unraveled many of the claims of the most well-known technical innovator in lab testing, Theranos.
—One of the other Theranos objectives has been support of consumer-driven lab tests, starting with legislation in Arizona. The billionaire investor Mark Cuban started a vigorous online debate when he suggested people have blood tests done quarterly to monitor their own health. The response to Cuban from many physicians was swift, with the majority seeming to point out the perils of false positives and false negatives, and the potential for overdiagnosis and overtreatment.
—The Atlantic asked about the clinical validity of genetics tests being used to make medical decisions, which makes the push for consumer genomics described in this “Signal” podcast all the more interesting.
Why do these stories matter for drug development? Particularly in oncology, similar tests are increasingly used to select patients for clinical trials of drugs as well as guide therapy for approved drugs. Although drug pricing dominated the storylines, lab test headlines are a symptom of the larger issue: selecting the proper patients is critical for the industry to demonstrate that a drug improves patient outcomes, which is the gold standard in the value and pricing argument. The analytical and clinical validity of medical tests could be viewed as the lynchpins to demonstrate value for a drug.
Excluding ongoing discoveries about the complexity of cancer biology, here are five medical testing stories to watch in 2016:
1. The FDA will push harder on more regulation. In late 2015, the agency published a report on 20 case studies showing potential dangers of lab-derived tests and a companion blog post on their goal to have more oversight. (A handful of the case studies were to guide cancer treatment decisions.)
2. To “improve the quality and accuracy of genomic tests”, in late 2015 the FDA launched the precision FDA initiative, an online portal for researchers to exchange information. As part of the platform, the FDA appears to be engaging the genomics community in work that will result in setting standards for these assays.
3. Immuno-oncology is one of the most competitive areas of drug development. For the PD-1 and PD-L1 checkpoint inhibitors alone, there are multiple drugs in clinical trials as well as two anti-PD1 antibodies, nivolumab and pembrolizumab, already approved by the FDA. Only one drug and indication, pembrolizumab in non-small cell lung cancer, has an associated companion diagnostic, meaning the test is written into the label of the drug. However, there are at least four assays to measure PD-L1 in varying stages of development. In 2015, the FDA, American Association for Cancer Research, and American Society of Clinical Oncology brought stakeholders together in a workshop that resulted in agreement to develop an information package to allow comparison of the analytic performance of the different assays. While we’ve seen this type of collaboration before*, the difference I see here is the fast timing relative to drug approval and the highly competitive nature of the checkpoint inhibitors.
4. Liquid biopsies are coming on strong. On top of analytical considerations, comparisons to the data for traditional biopsies will add to questions of what test provides the best actionable result.
5. Finally, the question we’ve all been waiting for an answer to: does selecting
