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Reimbursement, New CDx Models Key for Driving Adoption of NGS in Clinic

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NEW YORK (GenomeWeb) – At this month's Business of Personalized Medicine Summit in South San Francisco, experts indicated that new models for reimbursement and companion diagnostic development are needed to truly tap the potential of using next-gen sequencing in oncology care.

In a presentation, Illumina's Chief Medical Officer Richard Klausner discussed the firm's plans to develop a universal oncology test, and how its Actionable Genome Consortium would define standards in oncology sequencing — two key ways the company is addressing the issues of reimbursement and bringing multi-analyte products through regulatory clearance, in order to drive adoption of sequencing in the clinic.

Meantime, analysts with investment banks said that in order for a diagnostic developers to attract investors, it is no longer enough to just demonstrate clinical utility of a test, but companies must demonstrate actionability and be able to make a case for reimbursement. Even representatives from pharmaceutical companies said that a change in how drugs and companion diagnostics are developed is necessary.

"True value is being able to demonstrate a positive outcome in patients," said Jamie Dananberg, executive vice president and head of therapeutic areas at Takeda Pharmaceuticals. "The only way to do that is to get the right medicines to the right patients."

New CDx model, standards

In August, Illumina struck a deal with AstraZeneca, Janssen Biotech, and Sanofi to develop a next-generation sequencing-based panel using its US Food and Drug Administration-cleared MiSeqDx system that would serve as a universal companion diagnostic and earlier this month said that it had founded an Actionable Genome Consortium with the Dana-Farber Cancer Institute, Fred Hutchinson Cancer Research Center, MD Anderson Cancer Center, and Memorial Sloan Kettering Cancer Center to define what constitutes the actionable cancer genome.

During a panel discussion Elaine Cheung, Illumina's associate director of business and corporate development, said that the goal of the universal oncology test is to "move away from the one-off product development efforts."

"There are about 800 targeted therapies in drug companies' pipelines, and it is not tenable to have 800 companion diagnostics," she added.

In his presentation, Klausner added that the goal of the collaboration with the three pharmaceutical companies would be not to develop a traditional companion diagnostic, but to develop what he described as a companion therapeutic.

He said the panel would include all the current genomic targets for which there are drugs available as well as targets for compounds that will go into the clinic within the next three years.

"We're coming to the end of the era of companion diagnostics," he said. "As we get more and more companion diagnostics and they don't neatly fall across traditional tissue-based and histology-based diagnoses of cancer, … we need a world not of companion diagnostics, but companion therapeutics."

In order to bring such a panel through regulatory clearance, Klausner said Illumina would like to work with the FDA to discuss the technical specifications and performance that the FDA would require of such a panel. The broad panel with all the targets would be marketed as an investigational-use only panel, he said, with narrower subsets brought through pre-market approval as the corresponding drug is approved. All that would need to be changed would be the software for analyzing the panel, he said.

Klausner added that the pharmaceutical companies also recognize the lack of andn need for standards around next-gen sequencing.

To address that problem, Illumina launched its Actionable Genome Consortium, which aims to tackle standards both in terms of technical performance standards for the entire process including sample prep, sequencing, and bioinformatics, as well as standards in terms of what in the genome is actionable as it relates to cancer.

"There are a lot of [oncology] panels out there, but they are a mixture of clinically actionable and research," he said. The Actionable Genome Consortium, which currently has around 40 members, Klausner said, plans to develop a set of recommendations that it will publish by the end of the year, he said.

In addition, the recommendations will also serve to guide the performance metrics of the universal oncology test being developed as part of the agreements with the pharmaceutical companies, Klausner said.

He said Illumina is also in discussions with the China FDA to create a consortium there to develop similar standards.

Value-based reimbursement

Creating standards around what is actionable in the cancer genome will also help drive reimbursement for such tests, and ultimately help change the reimbursement model, Klausner said. Reimbursement should be based on the "value for outcome and economics" of a test, he said.

He added that it is difficult to assess how panel tests that include markers that, while interesting for research purposes, do not yet have clinical actionability, should be reimbursed. But, standards that define the actionable genome, will help enable that type of value-based assessment.

Credit Suisse analyst, Vamil Divan, also said that there has been a "greater focus on reimbursement and the regulatory landscape" from investors. Previously, he said, it was enough for diagnostic test developers to demonstrate clinical utility, but now particularly with so much uncertainty around how the FDA will regulate NGS-based tests and whether they will be reimbursed, the focus has shifted.

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