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“The world of biotechnology is in the midst of a wave of exciting research and clinical advances—from artificial intelligence-based drug discovery to cell and gene therapy, to precision genome editing and other newer technologies,” writes Genetic Engineering and Biotechnology News (GEN). “These advances are driving new therapies for patients and fueling recoveries for the markets that generate capital for the industry, from the venture capital that powers private startups, to the initial public offerings of more mature companies.”
On September 11, 2024, GEN’s flagship virtual event, “The State of Biotechnology 2024,” highlighted the “research and business leaders from industry and academia, [hosting them] to discuss the latest research developments, innovations, disruptive technologies and regulatory changes that will spur biotech forward to bigger and better things, for patients around the world.”
John F. Crowley, President & CEO of the Biotechnology Innovation Organization (BIO), gave a keynote about BIO’s work, how the current political landscape is affecting the industry, the Inflation Reduction Act (IRA), the BIOSECURE Act, and much more.
The political landscape and biotechnology
“[BIO is] the preeminent trade association in the world,” began Crowley, “promoting policies and advocating for biomedical research and the entire biotech innovation ecosystem that needs to happen to make newer and better medicines, as well as advancing all biotechnology, including agriculture and environmental technology.”
As such, BIO does not endorse any particular candidate or party; instead, it acts as a cross-partisan organization that works with politicians on both sides of the aisle.
“Our job right now is to be prepared for whichever leader may become President of the United States in January, and to work with either administration, because it’s so critically important,” said Crowley.
Biotech’s role in the U.S. economy, society, and international policy is not just a matter of health but international security, Crowley explains.
“I’m very encouraged—maybe more than I’ve been in many, many years—at the bipartisan, cross-partisan focus on biotechnology in Washington,” said Crowley. “A number of us for years have said this is a really important industry—arguably even the most important.” When the U.S. leads the world in biotechnological innovation, all nations are “better, safer and healthier,” he added.
What is needed now, he said, are policies that advance biotech.
How do we deal with the IRA?
“As we think about the IRA, there are parts of it that I think were good steps forward,” said Crowley. “The insulin caps, the Part D caps, the $2,000 caps, the elimination of the donut hole in the middle—I think those are good advancements that addressed market failures.”
“There were other parts of the IRA that were either uninformed or unintended in their consequences,” he continued. The industry is working to fix two major aspects of the legislation.
For one, small molecule drugs are given just nine years of protection from price controls, while biologics are given 13 years. “The majority of medicines made today are these small molecules,” explained Crowley. “Many of them cross the blood-brain barrier—so important for devastating neurologic diseases. They are also the medicines most likely to become generic and to have more competition. So it makes no sense to bias research and investment dollars.”
“And now what you’re starting to see is more and more funds going to the larger molecules. And that’s a shame because some really important programs are being slowed or stopped,” he explained.
Additionally: “There was a provision in there that you are exempt from the pricing control mechanisms of IRA if you have an orphan indication—so an orphan genetic rare disease,” explained Crowley. “But if you use a medicine, and you even just research and explore another rare disease with that medicine or technology, you lose that protection.”
“What that’s done now is create an incredible disincentive to research other uses of medicines, other indications of the disease states, and that, I don’t believe, was the intended consequence,” he noted.
The good news is that there are bipartisan bills introduced to address both challenges.
“Whether that can get done in the lame duck session of this Congress, we’ll see,” said Crowley. “I think it’s a very high bar, but we’re really focused on that for the next session, as well. So, more work to be done—and also education.”
The BIOSECURE Act
“The legislation itself specifically targets United States government contracts with a very limited number of biotechnology companies of concern that are based in China or believed to be potentially significantly influenced by the Chinese Communist Party,” Crowley said. “The legislation itself is very targeted.”
“But more than anything, what it is is a realization and a wake-up call that we have lost the resiliency of our entire manufacturing supply chain in the biotech, biopharmaceutical world,” he continued.
“Biotechnology is really important,” he continued. “What do we need to win? This is what we did with semiconductors. We’ve done it with cyber now with AI, and we need to do this with biotechnology.”
The power of the patient voice
The conversation touched on many points, including Crowley’s optimism around the Food and Drug Administration’s (FDA) creation of a Rare Disease Innovation Hub, as well as the Creating Hope Reauthorization Act, which would expand the FDA’s priority review voucher program to incentivize pharmaceutical companies to develop new drugs for children with rare pediatric diseases.
Crowley also spoke on the importance of better DEI standards across the industry—including how schools recruit and support STEM students.
Crowley ended the conversation by touting the importance of the patient’s voice.
As CEO at Amicus, he asked, “If you had a disease, or you were the mom or a dad of a child with a disease, what decision would you make? When would you start a program? When would you stop a program? Who would you hire? Who would you partner with? It’s a mindset, and [it’s one] that’s evolved, and that’s taken hold in the industry, from our smallest companies to our biggest. We need to do more of it. We need to do it in our government. And this is another role I think that BIO can play.”
