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Oncology outsourced: An emerging research model
In spite of advances in biology and medicine, cancer remains a major unmet clinical need. In the pharmaceutical and biotechnology worlds, cancer research is focused primarily on diagnostic tool development and target-based drug discovery. Diagnostic tests play an important role in patient stratification for treatment and for monitoring therapeutic benefit. The reductionist target-based drug discovery approach enables a more effective, personalized approach to clinical treatment.
Fortunately for us, the chances for meaningful advances in these areas are never higher than today. Over the past decade, we have witnessed the dawn of the 'omics era and the advent of Big Data. Emerging technologies like next-generation sequencing and innovative analytical tools such as network analysis have improved our understanding of basic cancer pathophysiology. What remains is translating this knowledge from the bench to the bedside.
Although academic and government research institutions and, increasingly, nonprofit research foundations, contribute to the search for new diagnostics and therapeutics, the bulk of cancer research is carried out by the pharmaceutical industry. In the past few years, however, multiple factors including decreased productivity, higher costs and the ongoing patent cliff have made drug research in the pharmaceutical industry unsustainable in its current format. As a consequence, newer models of science and business are replacing the fully integrated pharmaceutical company (FIPCO) model that is quickly becoming a relic of the past. Evolving technology has allowed and even driven this shift. The name of the game for drug research today is outsourcing.
Outsourcing of drug research
Pharmaceutical companies are adapting to the changing business dynamics by moving away from a vertically integrated model where company scientists do all of the research in company laboratories. Outsourcing has become the key to not only efficient technology development, but also the entire drug research process. This is true for all disciplines, but is nowhere more evident than in oncology, drug discovery and diagnostics, where the pharmaceutical industry has established relationships with thousands of external research partners. These days, many companies have become fully integrated pharmaceutical networks (FIPNets) comprised of company scientists working hand in hand with outside experts.
What are the benefits of this networked research approach? For one, it makes it easier to access research capabilities and experts on demand, thereby empowering cancer researchers. Other benefits include dramatically reduced costs, shortened development times, higher quality and improved productivity.
External research partnerships
Pharmaceutical companies have established three types of external research partnerships, each with its own unique benefits:
1. Pharmaceutical companies have long used the published fruits of academic research to help them in drug research. Today, pharmaceutical companies are actively partnering with universities. These companies get access to cutting-edge tools and technologies and an innovation mindset. The academics benefit from research funding and the ability to translate their basic research into clinical studies.
2. Pharmaceutical companies are turning to small and mid-sized biotechnology companies to access new technologies and fill their dwindling drug pipelines. Smaller companies are often nimble and more efficient than their larger cousins. These partnerships generate revenue for the small company and often lead to its outright purchase, such as the acquisition of MedImmune by AstraZeneca in 2007.
3. Pharmaceutical companies have established partnerships with tens of thousands of research vendors, including contract research organizations (CROs), contract manufacturing organizations (CMOs), non-governmental organizations (NGOs) and patient-advocacy groups. The relationships can be quite diverse, ranging from one-week, fee-for-service partnerships to multiyear, billion-dollar deals. As an example of the latter, a few years ago, Eli Lilly & Co. outsourced its drug safety research, in its entirety, to a single CRO.
Of the three types of partnerships, easy access to CROs is having the biggest impact on the pharmaceutical research landscape. Having thousands of external research providers eager to custom create research services, products or tools is game-changing for the pharmaceutical industry. This approach has enabled AstraZeneca to keep just 40 of 800 scientists in its neuroscience division and shut down most internal neuroscience laboratories, but still run the same number of neuroscience drug research programs.
Pharmaceutical companies fall into two research outsourcing camps. One camp prefers to outsource services to preferred vendors that are preselected in each research area, which means they routinely work with hundreds of small and large vendors at the same time.
The other camp prefers to outsource all or most services to a single large CRO, a so-called "full-service model." With thousands of new research tools being created every year at an ever-accelerating pace, however, it will become increasingly difficult for a single CRO, no matter how big, to offer every research tool required to run multiple drug research programs. Even companies that embrace the full-service model today will be forced to access a range of CROs if they want access to the most innovative and cutting-edge research technologies.
The almost 4,000 U.S. preclinical CROs are distributed widely across the country with CRO clusters in the expected pharma/biotech hot spots. For example, a 2010 study shows almost 150 CROs in the greater San Diego area alone that are collaborating with local drug research companies.
Research from virtual laboratories
Since it is no longer necessary to maintain expensive laboratories to run a drug research program, groundbreaking drug research is no longer restricted to the few big, wealthy organizations. Any talented individual with a computer and basic knowledge of spreadsheets, biology and statistics can run a drug research program or develop clinical diagnostic tests.
Take the example of Dr. Purvesh Khatri, a computer scientist working with Dr. Atul Butte at Stanford University. Purvesh accessed public genetic data on tumor samples from an online repository and used his knowledge of statistics and free online software to identify genes that were overexpressed in tumors compared to normal samples (a case/control study). He next used a CRO to obtain blood samples from 10 cancer patients and 10 healthy individuals, and then shipped the blood samples to yet another CRO for testing by ELISA. Using this virtual research approach, Purvesh identified biomarkers for three different cancer types within one year—without ever setting foot in a laboratory!
Purvesh's example is an encouraging step forward for do-it-yourself biologists with limited resources, but an unlimited drive and ideas waiting to be tested. As Butte says, "we say, 'outsource everything except the genius.'" Virtual research may represent a paradigm shift enabling highly efficient use of limited finances and manpower.
Outsourcing makes our scientific world flat; a citizen scientist with limited resources can now do projects previously restricted to a large pharmaceutical company with enormous resources.
Empowering citizen scientists
It's been more than 40 years since Richard Nixon first declared the "war on cancer." We have made progress, but with more than 7 million people dying every year from cancer, we still have a long way to go. Although troubling to some, it may be time for scientists to embrace the idea that we alone can't solve this problem fast enough. We may need help from our less well-trained brethren: the citizen scientist.
ClicktoCure is an excellent example of citizen science attacking the challenge of cancer. Created by Cancer Research UK, a charity dedicated to the cure of cancer through basic and translational research, ClicktoCure recruits citizen "soldiers" who examine colored sections of tumor images and analyze data using prompts provided on the website. Automated techniques can generate thousands upon thousands of images of tumor biopsies. The bottleneck is not having sufficient researchers to examine each and every image. The ClicktoCure program leverages the power of citizen scientists, asking volunteers to undertake the front line analysis of thousands of images of biopsy slides. Those that are flagged by volunteers are reviewed by experts. This crowdsourced project makes excellent use of citizen scientists eager to participate in meaningful research with tangible results. Cancer Research UK says it firmly believes it "will change the face of cancer research."
Citizen scientists are also important for the study of rare and orphan diseases, often neglected by the pharmaceutical industry because of small market size and low expected financial returns. Now that it is possible for a single highly talented person to use outsourcing to run an entire drug research program from a laptop computer, it is likely that there soon will be numerous examples of personalized drug discoveries carried out by patient advocates or even by patients themselves. With enough basic knowledge and resourcefulness, citizen scientists will be able to leverage outsourcing to develop specific, more effective drugs and diagnostic tests for rare cancers and other rare diseases.
The way we develop oncology diagnostics and therapeutics is changing rapidly. Though the shift to an outsourcing research model is being intensely debated within pharmaceutical and biotechnology companies, it is revolutionizing how virtual laboratories run by professional or citizen scientists can function. Empowering all scientists and lowering barriers to research innovation is the essence of this new research model.
Dr. Kevin Lustig is the co-founder and CEO of Assay Depot, an online marketplace that gives scientists the ability to access 8,000 global vendors to any research skill or service they need for their drug research project. In 2001, Lustig founded Kalypsys, a drug discovery company that raised more than $170 million in venture capital and put five drug candidates into human clinical trials. Prior to Kalypsys, Lustig directed lead discovery at Tularik, a biopharmaceutical company purchased by Amgen in 2004 for $2.5 billion. Kevin has an M.S. degree in biochemistry from the University of Missouri-Columbia and an A.B. degree in molecular and cell biology from Cornell University. He carried out postdoctoral work in cell biology at Harvard Medical School after receiving a Ph.D. degree in biochemistry from the University of California, San Francisco (UCSF). His research discoveries have been published in Science, Nature and other leading scientific journals, and he has been awarded eight technology patents.