With grants from government sources looking less certain, partnerships between academic research centers and pharmaceutical companies are more important than ever to keep breakthrough hematology therapies in the pipeline, said Burt Adelman, MD, a hematologist who serves as executive vice president and chief medical officer for Dyax Inc.
Adelman, long affiliated with Brigham and Women’s Hospital in Boston, Massachusetts, told a gathering at the 55th American Society of Hematology (ASH) Annual Meeting and Exposition in New Orleans, held December 7-10, 2013, that traditional drug development models inside pharmaceutical companies lack the one thing that academic medical centers have in abundance: access to patients in real-world settings.
It is important, he said, for the best and brightest in academia to promote themselves to industry, and to not assume that pharmaceutical companies will find them. Try as they might to follow the literature, Adelman said, “We don’t know everyone, no matter how much exciting work you may have going on.”
And the pharmaceutical industry has hit a plateau of sorts, he said. In 2012, worldwide drug sales fell 1.6%; while 43 new drugs were approved by the US Food and Drug Administration (FDA) in 2012, the 13-year average has been 32. What is more alarming is that between 2013 and 2019, $230 billion of US drug sales are at risk due to patent expiration. “Pharma is facing the innovation stagnation dilemma,” he said.1
This means opportunities for partnerships with academic centers, who tend to bring more of the “breakthroughs” to the FDA, compared with industry, which tends to bring more of the “me too,” drugs.
At the same time, Adelman said, it is in academia’s interest to reach out to industry. The National Institutes of Health cut $1.55 billion from its FY 2013 budget; with the effects of the federal sequester continuing and no “grand bargain” in sight, things do not look promising for US government support for research.
In fact, the bipartisan budget agreement reached less than 2 weeks after the meeting drew this response from ASH:
“The passage of the bill is good news for the nation, with no looming government shutdown, but the demands of the sequester and constraints on NIH funding remain,” said Alan Lichtin, MD, chair of government affairs at ASH. Lichtin added that the institution where he works as a hematologist, the Cleveland Clinic, “is not immune to budget constraints [and] has experienced more voluntary retirements. With reimbursement rates going down, Cleveland Clinic has not been able to expand many of its research programs.”2
Who creates drugs? Adelman cited data from 1998 to 2007, which broke out new drugs from the following:
• Pharmaceutical companies, 147.2 new drugs, or 58%
• Biotech, 44.1 drugs, or 18%
• University transfer, 60.7 drugs; 24%
Adelman said he understands the reluctance of academic researchers to share ideas with industry; there is a fear that good work will be stolen, and the scholar will not reap the rewards. However, he said, as it stands, research institutions are not benefiting from the spending on healthcare, which in 2011 hit $2.7 trillion, including 10% on prescription drugs.
“Very little of that annual $268 billion goes back to investigators of academic institutions. It’s a big number to not be participating in,” he said.
And pharmaceutical companies maintain an enormous research effort: the total capital cost of the average new drug that comes to market is between $800 million and $1 billion; of course, that
includes the cost of the many attempts that fail. “There’s a less than 10% success rate across all therapeutic areas; the average development time is 10 years,” Adelman said. As he explained, researchers inside pharmaceutical companies are more risk-averse than those in academia with those kinds of statistics. Lack of efficacy, he said, is the main reason for drug failure, and often this is not discovered until the end of phase II or even phase III.
He cited statistics that said industry would continue to put $138 billion into research and development in 2014. Without enough new drugs coming to market, and more being spent on research, he said, “a cynic” would conclude that increased profits can only come from increased drug prices, both new and existing. Yet, there is pressure for that not to continue.
In the meantime, in hematology especially, the race is on to find better therapies to reduce the toxicity of older chemotherapies. “You cannot practice modern hematology without a prescription pad,” Adelman said. “We are constantly in need of new solutions to difficult problems.”
There are models in place for researchers to work either within their academic institution or directly with pharmaceutical companies to transfer technology. “The challenge is to figure out how to do this in a more reproducible way,” Adelman said. “If 24% of 252 drugs trace to academia, it is unlikely that 24% of the revenue is going to the institution.”
What does industry need?
“We need access to valued targets and technology. We need translational models; research tools. We need your patients, and we need your insights,” Dr Adelman said. There are also mundane needs, like access to diagnostic tools, such as MRIs, CT scans, and pathology labs.
Academics interested in sharing should work through their institutions to understand the rules, because most have a technology transfer office that can assist. Institutions can help figure out funding to grow the idea into a company, but they are not the only source: there are venture capital sources, foundations, angel investors. “Have a patent filed. Contact your colleagues in industry. Publish and speak at meetings and work the crowd.”
An important lesson: “An academic is rarely the best judge of IP value.”
1. Adelman B. Opening up drug development to everyone. Hematology Am Soc Hematol Edu Program. 2013;2013:311-315.
2. American Society of Hematology. Statement from the American Society of Hematology on Congressional Budget Deal [press release]. Washington, DC: ASH Newsroom; December 18, 2013. http://www.hematology.org/News/2013/11941.aspx. Accessed January 2, 2014.