"Golden Age" for Research

May 29, 2013



Declaring it a golden age for biomedical research, FDA Commissioner Margaret Hamburg appealed to the nearly 400 biotechnology industry leaders gathered for MassBio’s 2013 Annual Meeting for an increase in collaboration, noting that more progress needs to be made to effectively translate scientific discoveries into therapies, prevention or cures.

“As a nation, we must take a serious look at everything from intellectual property and patent policy to economic policy, including such things as access to capital, incentives, and tax policy, how we invest in science, our reimbursement policies and, of course, regulatory policy and regulatory reform,” said Hamburg, who served as a keynote speaker at the two-day event held at the Royal Sonesta Hotel in March. “As a result of our dialogues with industry – including MassBio – we have heard loud and clear that you want increased clarity, certainty and predictability about the FDA’s standards and expectations. I think I can say that we are striving to do our part. Success will require regulatory flexibility, advancing regulatory science and true collaboration among health professionals, industry, government, academia, and our global health partners.”

Hamburg shared examples of Massachusetts companies who have successfully navigated approval pathways, including the new expedited approval pathway. Of the 47 novel drugs and biologics the FDA approved in 2012 – the most in more than a decade – one out of three received fast track designation, 41 percent received priority review, and 10 percent were approved under the accelerated approval program. Those developed in Massachusetts included: Iclusig, which treats two rare forms of leukemia and was approved in just 2.6 months; the orphan drug Juxtapid, approved based on a single clinical trial of 29 patients; Linzess, a drug for irritable bowel syndrome; and Kalydeco, the cystic fibrosis drug, which was approved in 3.5 months.

“The FDA was able to review and approve many of these new therapies quickly by communicating with drug companies early in the development process, allowing flexible clinical trial design, and using priority review and other drug approval mechanisms to expedite approval,” said Hamburg. “As a result, the majority of these important products were made available to patients in the U.S. earlier than anywhere else in the world.”

John Crowley, Chairman & CEO of Amicus Therapeutics, also served as a keynote speaker at the annual meeting, sharing his experiences as a biotech executive, father and patient advocate.

“So much of what we do is about selling hope – not just hope for everyone who is invested today, but hope for so many people who don’t even know that they need it, that they’re going to be sick and that their lives are going to profoundly change,” said Crowley.

Crowley and his wife Aileen’s lives changed abruptly in 1998 when they were told that their daughter Megan (then 15 months old) and Patrick (just a few days old) had a rare genetic disorder, Pompe disease. Crowley left his job at Bristol-Myers Squibb and invested himself and his life savings in Novazyme Pharmaceuticals, a start-up company that was conducting research on a new experimental treatment for the disease. In one year, Novazyme went from an endowment of $37,000 to $27 million and was quickly acquired by Genzyme. Crowley was named head of Genzyme’s global Pompe program. He left the company in 2002 and in January 2003, Megan and Patrick received the enzyme replacement therapy through an experimental trial, which Crowley credits with saving his children’s lives. Megan is now 16 and Patrick is 15.

“Much of what we do is about extending and enhancing life – making that hourglass a lot bigger,” said Crowley. “As humans, all we really have and all that we’re pursuing is time. It’s essential to keep taking risk, if in the next 20 years we want to deliver the dream of all these early-stage ideas.”

The annual meeting also included discussions on healthcare reimbursement, what’s next in oncology research, the future of biosimilars, rare disease therapies and research resources sharing.

“These conversations go to the very heart of what we do and how we as an industry will operate into the future,” said MassBio President & CEO Robert K. Coughlin. “We must understand our role in a shifting economic and regulatory environment and attempt to answer these big, complicated questions. Our only way forward is to address them together.”

Josef von Rickenbach, Founder, CEO and Chairman of PAREXEL International, received the Henri A. Termeer Innovative Leadership Award for his commitment and contributions to the biotechnology industry at the closing awards luncheon. Von Rickenbach co-founded PAREXEL as a regulatory consulting company in 1982 and soon grew the organization into a pioneer in clinical research outsourcing. To make clinical development more effective for clients, he conceived a multidisciplinary and systems-oriented approach to outsourced clinical development, bringing efficiencies, scalability, and standardization to the process. Thirty years later, PAREXEL is a $1.4 billion global company and one of the top biopharmaceutical service organizations in the world.

Gloucester High School was named this year’s Joshua Boger Innovative School of the Year by the Massachusetts Biotechnology Education Foundation (MassBioEd) for inspiring students to explore life sciences careers through progressive biotechnology education and exemplary science career programming. In 2009, Gloucester High was selected as a BioTeach school, and in four years, students and faculty have actively participated in both teacher professional development and biotechnology lab experiences. The school has also creatively adapted BioTeach labs and equipment to establish new biotech summer camp programs for high school students.

“Gloucester High School has done a remarkable job fostering STEM education and encouraging students to pursue careers in biotechnology as they look toward their futures,” said Lance Hartford, Executive Director of MassBioEd.

“This recognition is an important step forward for Gloucester High School, as we work towards our goal of becoming a leader in STEM-related high school education,” said Principal Erik Anderson.

To recognize its commitment to growing the life sciences supercluster in Massachusetts, Massachusetts Biomedical Initiatives (MBI) of Worcester was honored with MassBio’s Leading Impact Award. MBI is the second organization to receive this award. Last year’s winner was Pfizer’s Center for Therapeutic Innovation.

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