Institute of Medicine (IOM) Report on Genome-based Therapeutics and Companion Diagnostics
Reporter: Aviva Lev-Ari PhD, RN
‘Viewpoint’ addresses IOM report on genome-based therapeutics and companion diagnostics
“Whenever a drug is developed that will target a pathway underlying disease, you need a test that says the patient will benefit,” explains Leonard. “The development of companion diagnostic tests to determine if a patient will respond to a specific drug is very complex and needs to be changed, but will require changes from many stakeholders, including test developers, pharmaceutical companies, the Food and Drug Administration, the Centers for Medicare and Medicaid Services, and other payers.”
The concept of what is called the FDA’s “Companion Diagnostic Pathway,” explains Leonard, allows for assessment of the test to predict a drug’s benefit, within the same clinical drug trial to assess the new drug’s effectiveness as a treatment. Cancer specialists are the primary users of these types of drugs and tests.
In their “Viewpoint” article, Leonard and colleagues Robert McCormack, Ph.D., and Joanne Armstrong, M.D., M.P.H., who represent the testing and medical insurance industry respectively, argue for stakeholders’ support of “a strategy to co-develop and co-submit to the FDA a diagnostic test (companion diagnostic [CoDx]) that specifically targets a drug to patients predicted to respond.” The authors share that “regulatory and business challenges . . . hamper the broader field of diagnostic testing” and not just the companion diagnostic pathway.
As an example of the current co-development CoDx environment, Leonard provides a potential scenario of a lung cancer patient for whom an oncologist may be considering several different drugs for treatment. “The pathologist has to do multiple separate CoDx tests, unless the institution does its own LDT that combines all the individual tests into a single test,” Leonard explains, which is done in some pathology laboratories, predominantly using next-generation genetic sequencing. This combined test reduces the number of tests required, can conserve tissue and produce more medically-relevant results. However, insurance payers will not always cover the breadth of the tests.
“A university-based lab can run a single test to assess 28 genes, but an insurance company might only cover three of the gene tests,” she admits.
The “Viewpoint” article addresses reimbursement and other economic obstacles, such as the investment environment for CoDx co-development and the costs associated with performing laboratory tests. The IOM Roundtable workshop explored these challenges, as well as discussed solutions to the companion diagnostic test co-development process.
“The goal was agreement on a unified plan, with every player doing their part,” says Leonard, adding that “This issue needs thinking outside the box, with the patients at the center of the discussion.”

More information: JAMA Online First “Viewpoint” link:jama.jamanetwork.com/article.aspx?articleID=1831289
SOURCE
http://medicalxpress.com/news/2014-02-viewpoint-iom-genome-based-therapeutics-companion.html
This is very insightful. There is no doubt that there is the bias you refer to. 42 years ago, when I was postdocing in biochemistry/enzymology before completing my residency in pathology, I knew that there were very influential mambers of the faculty, who also had large programs, and attracted exceptional students. My mentor, it was said (although he was a great writer), could draft a project on toilet paper and call the NIH. It can’t be true, but it was a time in our history preceding a great explosion. It is bizarre for me to read now about eNOS and iNOS, and about CaMKII-á, â, ã, ä – isoenzymes. They were overlooked during the search for the genome, so intermediary metabolism took a back seat. But the work on protein conformation, and on the mechanism of action of enzymes and ligand and coenzyme was just out there, and became more important with the research on signaling pathways. The work on the mechanism of pyridine nucleotide isoenzymes preceded the work by Burton Sobel on the MB isoenzyme in heart. The Vietnam War cut into the funding, and it has actually declined linearly since.
A few years later, I was an Associate Professor at a new Medical School and I submitted a proposal that was reviewed by the Chairman of Pharmacology, who was a former Director of NSF. He thought it was good enough. I was a pathologist and it went to a Biochemistry Review Committee. It was approved, but not funded. The verdict was that I would not be able to carry out the studies needed, and they would have approached it differently. A thousand young investigators are out there now with similar letters. I was told that the Department Chairmen have to build up their faculty. It’s harder now than then. So I filed for and received 3 patents based on my work at the suggestion of my brother-in-law. When I took it to Boehringer-Mannheim, they were actually clueless.