PharmExec Blog

Bringing Genetic Data into the Clinic

While Big Pharma frets over the costs and risks associated with exploratory endpoints in the clinic and the vagaries of companion diagnostic reimbursement, non-profit organizations like the Multiple Myeloma Research Foundation (MMRF) are stepping in to provide pharmacogenomic research for the next generation of cancer drugs.

Last week, the MMRF launched a 1,000-patient trial with newly diagnosed multiple myeloma patients, to study the relationship between a patient’s genetic profile and treatment outcome. The study, part of the MMRF’s “Personalized Medicine Initiative,” will track patients from initial diagnosis through the course of their treatment, over a minimum of five years. The study will “conduct sequential tissue sampling to identify how a patient’s molecular profile may affect his or her clinical progression and individual response to treatment.”

To what end? If pharmaceutical companies are reticent to integrate genomic data into the clinic, for the reasons above as well as regulatory issues around data validation and patient privacy, who will? Physicians will, according to Richard Resnick, CEO, GenomeQuest, and a former manager of bioinformatics software at Wyeth-Ayerst. “Physicians could potentially disrupt the value chain in pharmaceuticals, if [drug companies] don’t listen,” said Resnick, during a panel discussion yesterday at a Cambridge Healthtech Institute conference on next generation genome sequencing.

Brad Smith, VP, translational medicine at Quintiles, acknowledged that some Big Pharma companies are recognizing the importance of patient biomarkers in the early stages of drug discovery, but they aren’t making it a priority. “In our partnerships with pharma, biomarkers and lab tests are usually pretty far down at the bottom of the list,” said Smith during the panel discussion. “Suggesting a new endpoint adds risk to the trial protocol.”

What the pharmaceutical industry really needs to overcome cost concerns is a clear success story, said Iya Khalil, SVP and co-founder of GNS Healthcare, during the panel discussion. Despite the approvals last month of Roche/Daiichi-Sankyo’s Zelboraf (for melanoma) and Pfizer’s Xalkori (for lung cancer) – and their respective companion diagnostics – Khalil said the industry still needs a clear, end-to-end success story for expediting drug approval by using data collection, genomic sequencing and microRNA transcription, phenome measures including physiological outcomes, and finally, predictive analytical software. Khalil said the approval of Xalkori, originally a drug targeting the c-MET gene to prevent a certain type of protein expression in cancer cells, hinged on a serendipitous discovery of ALK inhibition. “Pfizer got lucky, good for them, but we know there’s a more rational way to do [discovery] that can work, there’s no reason why…you can’t profile these patients and study these things,” said Khalil. “The question is who is going to take the chance on that pilot that actually shows that this will work.”

At the conclusion of the “Genomics in Clinical Trials: Has the Time Come?” panel, Resnick was asked how many years it would take to have a full genome sequencing done for 50% of the US population. His estimate was 15 to 20 years.

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