Refining Processes for the Co-Development of Genome-Based Therapeutics and Companion Diagnostic Tests Workshop Summary (2014) / Chapter Skim
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2 Regulatory Perspectives
2 Regulatory Perspectives
Pages 7-18
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Speakers discussed regulations from the perspective of FDA and of an entrepreneur with a focus on personalized medicine, in the context of the current co-development process for tests and drugs. Elizabeth 7
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In 1998 FDA approved the use of the drug erceptin in patients with breast cancer who tested positive for human H epidermal growth factor receptor 2 (HER2) , one of the earliest examples of a co-development companion diagnostic model before there was a formal process in place.
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Intended Use Erbitux therascreen KRAS Real-time qualitative PCR assay used for the RGQ PCR Kit detection of seven somatic mutations in the KRAS in colorectal cancer tissue. Erbitux, DAKO EGFR Qualitative immunohistochemical assay to identify Vectibix PharmDx Kit EGFR expression in normal and neoplastic tissues and as an aid in colorectal cancer tissue.
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Xalkori Vysis ALK Break FISH for ALK gene rearrangments in NSCLC Apart FISH Probe Kit tissue specimens. Zelboraf cobas 4800 BRAF Real-time PCR test for BRAF V600E mutation in V600 Mutation Test melanoma tissue.
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This is to account for the fact that better tests may be approved at a later date, a possibility that FDA wanted to account for, Mansfield said. As the policy states, "This will facilitate the development and use of more than one approved or cleared IVD companion diagnostic device of the type described in the labeling for the therapeutic product." Also, putting the test name in the label would essentially make the drug–test pair a combination product, which falls under a different category of regulation at FDA.
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Other non-IVD diagnostics are also being considered as companion diagnostics. An example is the recent approval of Exjade, a drug used to treat non-transfusion-dependent thalassemias and its companion radiological test to measure liver iron concentration.
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The most significant obstacles that need to be dealt with involve business development and reimbursement issues. "Drug development and test development are inherently difficult to coordinate," Frueh said.
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Yet the cost of the EML4-ALK companion diagnostic test that is required for the use of Xalkori is reimbursed at $128.48,1 and diagnostic companies are under pressure to reduce prices further. As a result, the disparity between drug and diagnostic prices is increasing, and the goals of drug development tend to dictate the goals of the test development in a co-development effort.
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In the case of molecular diagnostics, genomic information will point toward therapies for which no indication in a drug label exists, which is a much bigger regulatory challenge, Frueh said. "The sheer magnitude of the information that we'll find on the genetic and molecular level is going to far surpass our capacity to run clinical trials," he said.
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• Relative efficacy/effectiveness • Emphasis on RCT, • Active-controlled RCT • Cost-effectiveness/ • Emphasis on RCT, most often placebo- • Observational studies utility analyses most often active- and controlled • Cost-effectiveness/ • Budget impact placebo-controlled utility analyses analysis • Budget impact analysis • Active-controlled RCT • Adaptive Phase III–IV trials • Observational studies Assessors Assessment focus Studies/data • Meta-analysis FIGURE 2-1 Integrating regulatory agencies and payers for product development. NOTE: MA, marketing authorization; RCT, randomized controlled trial.
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Speaking for herself and not on behalf of FDA, Mansfield stated that medicine is heading toward next-generation sequencing, which is "the ideal place to be," she said. "When you get diagnosed, you get a test, and we have all the information on the table from a single test." In that case, the test may be quite general as opposed to being used as a companion diagnostic to provide usage information about a single targeted therapy.
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