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An Incentive to Partner for Children: Dr. David Williams Emphasizes the Value of Industry-Academic Collaboration around Children’s Health in 2017 ASAP BioPharma Conference Keynote

Posted By John W. DeWitt, Thursday, September 14, 2017

Dr. David Williams kicked off the ASAP BioPharma Conference, Sept. 13-15, 2017, at the Royal Sonesta Boston in Cambridge, Mass., with a forthright keynote making the case for why industry should pay attention to the early research and clinical trial capabilities of leading children’s hospitals—which, he argues, can find common ground with the for-profit objectives of biopharma, biotech, and information technology companies seeking new opportunities in healthcare. Williams is Boston Children’s Hospital’s chief research and scientific officer and senior vice president for research, as well as president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

 

Williams is a versatile fellow—he still insists on being a practicing physician, despite also being a researcher, senior administrator, international collaborator, entrepreneur, scientific journal editor, pediatrics professor at Harvard Medical, and more—and he says he embraces working in all those roles because they all advance the fight against childhood diseases, improve the quality of life for children, and help once-ill children become the healthy adults everyone hopes they grow up to be.

 

Yet, aside from the “right reasons” everyone agrees upon, it’s not an easy economic case to argue for biopharma to focus on children—especially rare pediatric diseases. Williams was blunt in his keynote. “Seventy percent of drugs we use in children have never been studied and are not FDA-approved for use in children.” Why? “There’s no incentive for drug companies to seek [pediatric] label use.”

 

But his keynote arguments—reflecting his newly established role as chief medical officer charged with magnifying the science and scientific partnerships at Boston Children’s Hospital—seem entirely undeterred by this tough reality.

 

“Many people are surprised at how many drugs have evolved out of Boston Children’s,” he told a packed room of life sciences partnering executives, noting that Boston Children’s ranks fifth among all hospitals in the US in number of licenses and/or options executed (48). (The leader, Mayo, has 96.) Statins got their start at BCH, for example, thanks research efforts prompted by a child’s unfortunate heart attack. But Dr. Williams is not just talking about successful licensing partnerships with pharma leaders—BCH is also a force to reckon with in startups.

“At times, it’s better not to take a license agreement with the standard royalty fee, but rather use discoveries as platform for startup companies. Moderna is a huge company now. I’m involved with Orchard Therapeutics. Alerion is a platform company formed in Germany.” He noted that three BCH spinouts made FierceBiotech’s Fierce 15—Moderna in 2013, Intellia Therapeutics in 2015, and Orchard in 2016.

 

The foundation of these achievements—and a major contributor to BCH’s success as America’s top-ranked children’s hospital—are the remarkable research credentials of the institution where Dr. Williams works, among them:

  • 600,000-plus visits a year
  • 40 clinical departments and 225 specialized clinical programs
  • 800 faculty members and 2,000 fellows
  •  The largest pediatric research program in world based on extramural research funding—more than $330 million in funding for numerous areas of research.

And more than one Nobel prize winner.

 

So why did Dr. Williams take the time to share BCH’s story with ASAP? Because BCH is serious about partnering—not just because the hospital has dedicated alliance managers, but more fundamentally, it recognizes collaboration as key to its success, past, present, and future more than ever. Williams described the organization’s vision going forward:

 

“Champion discovery around pediatric illnesses, deploy genomics into everyday applications, translate our wealth of research into more effective and precisely targeted therapies, and build more robust collaborations with biopharma,” he said. “We’re really ‘putting the gas pedal down’ on advanced experimental therapeutics. The basis for everything we do is discovery science.”

 

Again, why should biopharma companies and society more broadly care?

“In addition for doing it for the ‘right reasons,’ there are lots of economic and societal reasons for doing this work,” he says. “We have the rare cohorts of patients and experts with deep experience in rare diseases.”

 

At the beginning and at the end of the day, though, success for Dr. Williams and BCH means lives saved or extended. One oft-cited triumph is common childhood leukemia, once a near-certain killer, now defeated 90 percent of the time. These kids survive to become adults who lead profoundly better lives—and make a powerful impact on society as a result. “If we can prevent childhood progression [of many diseases] it will have enormous implications later on in adult life.”

Tags:  2017 ASAP BioPharma Conference  biopharma  Boston Children’s Hospital  collaboration  discovery  Dr. David Williams  entrepreneur  genomics  international collaborator  therapeutics 

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