On the Origin of New Drugs
Last week, I gave a talk at a course at Drew University in New Jersey. The presentation was about the misconceptions that exist around the role and contributions to medicine made by the pharmaceutical industry. During the Q&A session, a member of the audience asserted that academic institutions were responsible for roughly 50% of the new medicines that are approved annually. This is a provocative assertion – and completely false.
The topic of who is actually responsible for new medicines is not a new one. Worried that American taxpayers should be sharing more in the profits of the pharmaceutical industry, Congress commissioned a study in 2001 to determine which top-selling drugs had their origins in work done by the National Institutes of Health (NIH). The Department of Health and Human Services prepared the final report which showed that, of the 46 drugs that had annual sales of $500 million or more, only three were associated with Federal patent ties. The other 43 drugs were discovered and developed by the pharmaceutical industry with no Federal investment.
Of course, this study is a decade old. Critics of the industry are again challenging the premise that industry is the major driver for the discovery and development of new drugs. A recent article in Nature Biotechnology (June, 2011) entitled “Debate re-ignites on contribution of public research to drug development” provides some valuable data on this topic. Perhaps the most relevant information is taken from a paper that emanated from Boston University and was published in the New England Journal of Medicine (volume 364, 535 – 541, 2011). This work, which focused on “The Role of Public-Sector Research in the Discovery of Drugs and Vaccines,” shows that of the 1,541 drugs approved by the FDA from 1990 – 2009, nearly 10% were rooted in public sector research. Clearly, the public sector is making important contributions in this field. Yet, the fact remains that the private sector is still responsible for 90% of new medicines.
The NIH, as well as other academic institutions and research institutes, plays a critical role in funding important biomedical research that provides broad benefits not just to the pharmaceutical industry, but to society in general. Furthermore, the fact is that the vast majority of basic biological research is done in academia. But one must distinguish between important theoretical work and the application of this work in discovering and developing new medicines. Basic research is not drug discovery. The NIH does a great job in providing basic knowledge and hypotheses about the nature of living systems. However, it is the pharmaceutical industry, both large companies and small biotech firms, that discovers and tests the compounds to prove or disprove these medical hypotheses. Neither can work without the other. A successful academic-industry partnership is crucial in discovering new medicines.
Drug Truths 
Claiming that many drugs come directly from academia is certainly false. But claiming that those drugs discovered in pharma with no university patent ties are entirely the work of pharma R&D is highly misleading. If universities do the research that gave us the discovery of enzyme classes (say kinases), a description of the cell biological role of kinase signaling, genome sequencing to identify kinases in humans, massive clinical studies to find that certain cancers are driven by mutations in kinases, mouse models that describe the role of a kinase in a cancer, methods to solve the crystal structure of a kinase, and also a lot of the basic synthetic chemistry methodology involved in making drug-like molecules…and then a drug company decides to screen for small molecules that inhibit a kinase, develops one, and puts it through clinical trials–this is not the drug company doing all the work (and deserving all the credit). Yes, much of the crucial (and brutally expensive and risky) work is undertaken by the drug company. But to claim that publicly funded efforts had nothing to do with the development of kinase inhibitor X is false.
Lester Freamon
July 1, 2011 at 5:33 pm
I think that I gave significant credit to the NIH and Universities in this piece. The problem is with the popular misconception that pharmaceutical companies simply license compounds from academic institutions and then manufacture them. As the NEJM article shows, that’s not the case. The reason this concerns me is that new medicines come from the biotech and drug companies. The more these contibutions are belittled, the more difficult it will be for these companies to operate in an increasing financially constrained environment.
johnlamattina
July 1, 2011 at 6:58 pm
Dear Dr LaMattina: Working on the private sector, I agree with you, but…
What about “Rate of PSRI discovery” = 52%
“Public-Private Interaction and the Productivity of Pharmaceutical Research” Iain Cockburn, Rebecca Henderson – NBER Working Paper No. 6018 – April 1997 ( http://www.nber.org/papers/w6018 )
Public enabling discovery: 11 yes / 9 no / 1 n.a. (11/21 = 52%)
Public synthesis of compound: 1 yes / 20 no
Table (1) History of the Development of the 21 Drugs with “highest therapeutic impact” introduced between 1965 and 1992.
Best regards from Buenos Aires,
Natalio Casetta
marketing man
Natalio Casetta
October 21, 2011 at 3:51 pm