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Gap Summit

By Nuala Moran

Staff Writer

CAMBRIDGE, U.K. – "I have trained a large dog to bite anyone who says the words 'personalized medicine.' We are a long way from that." So said George Poste, chief scientist at Complex Adaptive Systems and professor of health innovation at Arizona State University, opening a discussion on the state of play in the field, at the Gap Summit in Cambridge.

That is not to say there has been no progress in personalization in the 20 years that Poste has been talking about and been involved in this upcoming new era of medicine.

In terms of advances, he pointed to biomarkers that are beginning to predict individual response to a treatment. "This is an economic requirement, as well as a therapeutic one," Poste said.

There also has been headway in terms of environmental and behavioral aspects – for example, the ability to track adherence with taking medication and anticipating relapses in patients with an existing diagnosed condition.

"I'm a big fan of George and his dog," said Richard Klausner, founder and director of Juno Therapeutics Inc., and amongst other postings, director of the U.S. National Cancer Institute (NCI) from 1995 to 2001.

"What we want to do is not to complicate medicine more than we need to – there is no particular value in individualizing – the value is in getting it right," Klausner said.

That is borne out by "the real advent of cell therapy" in cancer immunotherapy using CAR T cells. "This is the first time we are using cells successfully, and I really believe we are largely curing people," said Klausner. "In 20 years at NCI, I never used the word cure; now I find myself using it."

However, unlike elsewhere in the development of precision medicine in cancer, genomics was largely irrelevant in developing CAR T therapy. "We didn't care about genetics – we are seeing cures with CAR T that eluded us with genomics," Klausner said.

The Gap Summit brings together luminaries of the biotech and pharma industry, such as Poste and Klausner, as well as Goran Ando, chair of Novo Nordisk A/S; Greg Winter, of monoclonal antibody fame; Trevor Mundel, president of global health at the Bill and Melinda Gates Foundation; and Paul Stoffels, chief scientific officer of Johnson & Johnson Inc. Also attending are 100 early stage researchers and young biotech entrepreneurs from around the world who will be future leaders of the industry.

The 100 future leaders, selected from thousands of applicants, put the old hands to the test in public debate. Over three days of discussion, the aim is to scope the gaps that must be addressed in seven areas that are critical to the development of the sector. In addition to personalized/precision medicine, those included drug affordability, R&D productivity and the funding gap faced by young startup companies.

If not personalized, then precision

If personalized medicine remains a distant prospect, precision medicine has arrived, said Mene Pangalos executive vice president of global business development at Astrazeneca plc. "It is not the future; it is here and now."

Within Astrazeneca, precision medicine is embedded in the 5Rs program the company put in place in 2011 in a bid to improve its R&D productivity. Amongst other aspects, that means focusing on finding the right drug targets, the right patients for clinical trials and the right commercial attributes so the final product will get reimbursed.

In 2005 to 2010, before the 5Rs program launched, Astrazeneca's R&D productivity stood at 4 percent, meaning 96 percent of programs it started ended in failure at some point. The industry average was 5 percent.

"We were lousy; the whole industry was lousy," Pangalos said. By putting the focus on precision medicine and the 5Rs, Astrazeneca's productivity has risen to 20 percent, as Pangalos and others reported in a paper in Nature Reviews Drug Discovery in January this year. Meanwhile, average productivity has fallen to 4 percent.

As a result of the precision approach, there has been "exciting progress" in cancer, and now the effect is beginning to be felt in other indications, including respiratory diseases and mental disorders, Pangalos said.

But even as precision medicine advances, it remains difficult to get payers to pay for the data inputs and reimburse the high-cost diagnostics that are required.

"Precision medicine is, without exception, based upon measurements. The fundamental problem is that the measurement side is the stepchild of drugs. So the question is how to capture the value that measurement gives to intervention," Klausner said. "There are more and more drugs whose value is tied to a diagnostic."

Pangalos said the industry will move on from offering a companion diagnostic for each drug, to the development of blood-based diagnostics across a panel of drugs. "The panels will represent several drugs from different companies and then the diagnostics will be paid for [by health care systems]."

Klausner agreed, saying in the future a prescription will be for a series of diagnostics and not for a drug. "It will say, 'You need to do the following tests and then treat with x or y'," he said.

The problem of reimbursement for companion diagnostics underlines how managing and interpreting the tsunami of data, not only from genomics tests, but all the other 'omics technologies, wearable devices, electronic health records, real-world evidence, and so on, stands in the way of the personalized medicine future.

"There are huge datasets to play for and we don't have the infrastructure in place to deal with them," Poste said. "We need to capture, standardize, validate the data, or we will still be languishing in the silos of today."

Rather than big data, what personalized medicine needs to move forward is smart data, Klausner suggested. "Perhaps the challenge is not the artificial intelligence fairy dust to get the wisdom from all the data. Maybe you want the smallest dataset needed to get the greatest value in terms of health care."

Published  April 18, 2018

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