At BIO convention, business boomed, and drug pricing loomed

Some 17,000 people descended on Boston during the first week of June for the annual meeting of the Biotechnology Innovation Organization (BIO). If the crowds were any indication, business is booming.

“This has been by far the most successful BIO convention we’ve ever had,” said John Maraganore, CEO of Alnylam Pharmaceuticals and chair of BIO. Attendees set a Guinness world record for the largest business-partnering event, with more than 45,000 meetings among people from across the globe.

“If you want to be in the business of biotechnology, you need to be here,” said Manu Nair, who came to Boston from the Oklahoma Medical Research Foundation.

Biotech investment is on track for a record year. “The first quarter was very strong for venture capital investing, and those trends seem durable,” said Glen Giovannetti, global biotechnology leader at the consulting firm EY. Cancer companies, especially immuno-oncology start-ups, are attracting a disproportionate amount of the capital, he added.

That influx of cash is allowing start-ups to go from first funding to an initial public stock offering faster than ever, Giovannetti said. And the second half of 2018 might bring some long-awaited activity from big pharma. “M&A should be really strong,” he said. “They have plenty of dry powder.”

But between bullish speeches, attendees grappled with several challenges confronting the industry, including the opioid crisis, the need to improve diversity, and the recently passed “right to try” law for the terminally ill. The issue that surfaced most often, however, was drug pricing.

“Justifying and supporting the value of our medicines is clearly the most important thing we have to do better at,” Maraganore said.

In a town hall meeting, Janet Woodcock, director of the U.S. FDA’s Center for Drug Evaluation & Research, said new medicines like gene therapies hold tremendous promise but are too expensive. The first commercial viral gene therapy costs $850,000 for a one-time treatment. “It isn’t sustainable,” Woodcock said.

Later in the meeting, FDA Commissioner Scott Gottlieb discussed the need for “more seamless transitions” between manufacturing therapies for research and commercial purposes—a challenge particularly important for cell and gene therapies.

Gottlieb admitted much is still unclear about “right to try,” a law designed to help terminally ill patients get access to experimental therapies. FDA already runs an expanded-access program to help patients receive such drugs, he said, and it will be up to drug companies to decide if they want to help patients through that program or the new law.