One Fine Specimen: Where Biopharma Competitiveness Really Begins
By Kiran Ganda, MBA, Director of Marketing, iSpecimen
Competition amongst biotech and pharmaceutical companies is certainly nothing new, with firms working arduously to maintain solid pipelines, win disease state areas, and attract and retain top talent. An industry marked by mass consolidation of established organizations on one end, and seemingly ongoing entry of “next-generation” start-ups on the other, it is no surprise that differentiation and market share are top-of-mind for biopharma executives. Yet, far before there are new therapies to set a firm apart, or enough sales to garner Wall Street’s attention, there is a far more fundamental need that competitiveness will ultimately hinge on – and that is the need for high-quality, diversified, annotated human biospecimens. Without access to a ready supply of these specimens, particularly as precision medicine continues to reign supreme, today’s biopharma companies will falter, quite literally, getting off the bench.
Human biospecimens and corresponding data are critical to pre-clinical research, setting the stage for translational discovery that could give way to clinical trials and the next blockbuster drug. But, achieving access to quality human biospecimens that meet the right standards has long been a problem for biomedical scientists. Traditionally, researchers have gone to different labs or biobanks familiar to them, and asked for remaining or banked samples, but this process is time-consuming, laborious, and often doesn’t even result in the right types or amounts of biospecimens needed. Related information may be missing or scarce, and specimen integrity and patient consent, when required or desired, may not be intact. Alternatively, they may have engaged patients directly to obtain needed specimens, but this process of identifying and consenting patients has too been time-consuming and expensive.
With precision medicine surging, and ever-more targeted therapeutic approaches coming to light, such as customizing treatments based on patient manifestation of specific biomarkers, or guiding the immune system to fight cancer, a “door-to-door” approach of specimen search and selection will continue disappoint. Even seven years ago, a National Cancer Institute study found a striking 81% of researchers reported limiting the scope of their work due to a shortage of both the quality and quantity of human biospecimens. Fast forward to today, when this year alone a number of aggressive precision medicine initiatives have been announced, and it is clear a new system is needed if biopharma companies and associated researchers are to keep pace with this proliferation of research questions.
Not only are the right specimens from the right patients in higher demand than ever before, but also more diverse specimen types are needed than ever before. Many of the potentially transformational treatment research areas today, such as biomarker discovery, immunotherapy, or in-vitro tumor modeling, require coordinated sets of biofluids, solid tissues, and cells all from the same patients with often very specific inclusion and exclusion criteria around variables such as patient medical histories, treatments, procedures, test results, and family histories, to name a few. Gone are the days when a common researcher request was for a tube of blood with high cholesterol or a piece of fixed breast cancer tissue. A request of today is often as specific as matched sets of frozen cancerous lung tissue with corresponding normal adjacent tissue, plasma, and peripheral blood mononuclear cells (PBMCs) from patients with a history of (the rare) checkpoint inhibitor treatment and who have not responded.
So, what are biopharmaceutical researchers to do to ensure competitiveness from the start, faced with this need for high-quality, large-volume, data-rich human biospecimens at the bench? Through a combination of technology and associated data-mining, aggregation, and patient participation, efficient and adequate specimen procurement is possible. One lab or biobank cannot serve the needs of many, but the scope and scale associated with a collective network of hospitals, labs, biobanks, and other clinical institutions, many of which can produce the necessary de-identified patient and specimen data by way of electronic medical records (EMRs), sure can – particularly when technology is used to search across such a network for patients and specimens that precisely match researcher needs. This type of federated process allows for quick identification of large sample sets that can be procured by gathering specimens and data from across multiple organizations. Another capability that such a system allows, further down the research trajectory, is the identification of patients for potential clinical trials after bench research is complete. Further, this type of tech-driven solution can operationalize and simplify consent processes, which are increasingly more important to researchers, providers, and patients alike.
Thankfully for the advancement of science, while there will always be those who choose and have every right to opt out, studies have shown that the majority of patients are willing to donate their de-identified biospecimens to research,,,, with an August 2016 National Institutes of Health (NIH) study validating the same. As biopharmaceutical companies continue to push forth for market share and product differentiation, so too must they dedicate focus to differentiating drug development practices from the start – because the quality, quantity, and precision of specimens procured at the bench will drive the life cycle of drugs that make it to launch, and more importantly, real medical impact.
 Cancer Immunotherapy
 “The Cancer Human Biobank (caHUB): Advancing the Vision of Personalized Medicine” by Carolyn C. Compton, M.D., Ph.D., Director, Office of Biorepositories and Biospecimen Research, National Cancer Institute, presented at 2nd Annual Biospecimen Research Symposium, March 17, 2009.
 Examples include the National Institutes of Health (NIH) Precision Medicine Initiative, Joe Biden’s Cancer Moonshot, and the Parker Institute for Cancer Immunotherapy.
 Known formally as Matched Sets
 Report of the Public Responsibility in Medicine and Research (PRIM&R) Human Tissue/Specimen Banking Working Group, March 2007; Tool F “Patient Attitudes”
 “Public views on the donation and use of human biological samples in biomedical research: a mixed methods study”, BMJ Open, Celine Lewis, Margaret Clotworthy, Shona Hilton, Caroline Magee, Mark J Robertson, Lesley J Stubbins, Julie Corfield, August 7, 2013.
 “Cancer Patient Perceptions about Biobanking and Preferred Timing of Consent”, Biopreservation & Biobanking, Braun Kathryn L., Tsark JoAnn U., Powers Amy, Croom Kristen, Kim Robert, Gachupin Francine C., and Morris Paul, April 21, 2014
 “Patient Attitudes on the Use of Clinical Biospecimens for Medical Research – An independent, US-based population survey”, commissioned by iSpecimen Inc. and conducted by independent research firm Lab42, December 2015.
 “A Survey of U.S Adults’ Opinions about Conduct of a Nationwide Precision Medicine Initiative® Cohort Study of Genes and Environment”, PLOS One, David J. Kaufman, Rebecca Baker, Lauren C. Milner, Stephanie Devaney, Kathy L. Hudson, August 17, 2016, http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0160461