Biospecimen quality is critical to the success of clinical research. Selecting the right supplier is therefore of utmost importance.
Successful drug development begins with a deep understanding of disease biology. Too often, the root cause of late-stage clinical trial failures can be traced back to an incomplete—or even incorrect—understanding of disease drivers.
One prime example is Gilead’s experience with selonsertib in nonalcoholic steatohepatitis (NASH). Selonsertib, an inhibitor of the fibrosis-promoting protein ASK1, failed to beat placebo in two phase III trials. The disappointing results are likely rooted in targeting the disease phenotype rather than the underlying mechanisms of disease. A logical, but risky supposition that might have been avoided early on.
The challenge lies in elucidating the mechanistic biology of a disease and identifying targetable drivers, which can be like searching for a needle in a haystack. Biospecimens play a crucial role in this aspect of discovery by enabling a comparison between the target patient population and controls.
But the data derived from biospecimens is only as good as the samples and not all biospecimens are created equally. To optimize target identification and drug development, researchers need to put a robust biospecimens plan in place early in the research cycle.
What to look for when sourcing biospecimens
In this era of precision medicine, biospecimens are essential resources for bridging molecular insights and clinical information to uncover disease drivers and rational drug targets. High-quality, compliant biospecimens are critical for translational research and personalized care. To ensure that biospecimens are fit for their intended purpose, researchers should consider a number of variables that ultimately affect the quality and reliability of their data.
To learn more about the right biospecimens plan, we spoke with Jim Boushell, Vice President of Precision for Medicine’s Biospecimen Solutions, a global leader in biobanking and biospecimen solutions.
“Pharma researchers often struggle with finding good biospecimen and data sources for the therapeutic areas they work in,” said Boushell. “The result of that can be catastrophic for the industry in that the targets they identify and the drugs they develop against those targets ultimately fail because the basic science behind those targets was inadequate.”
Specimen quality is a given. The integrity of a specimen can be compromised by freeze-thaw cycles, the time elapsed since collection, and improper storage, transport, or chain of custody. To extract maximum value out of biospecimens, researchers should look for fresh, deeply phenotyped samples that come with data on disease severity, co-morbidities, past treatment response, and other clinical histories.
Specimen availability is also important. Large cohorts are needed to ascertain if a discovery is true across most patients with the disease of interest. Moreover, harmonized clinical and test data will be required across a sizable enough cohort to support analysis.
“Even if specimens are available, they are often not consented appropriately for the uses that researchers may intend, such as genomic analysis,” Boushell noted. “Working with a biospecimen solutions partner that understands how to manage the informed consent, collection, and accessibility of samples helps keep research moving forward.”
It is imperative for researchers to identify and understand the specimen types necessary for answering their scientific questions. They will typically need sample matrices that can be used to measure disease biology, matched controls to confirm potential drug targets are not present in healthy subjects, and matched specimen types, for example, serum, tumor tissue, and DNA from the same patient.
Increasingly, researchers are also recognizing the importance of biospecimen diversity. Having a diverse cohort is necessary for evaluating whether patients of various races and nationalities share the same disease biology and drug targets. Returning to the NASH example, Gilead is now investigating a combination approach based on the multiple pathways at play in the biology of the disease and the heterogeneity of the patient population.
Partnering with a knowledgeable biospecimen solutions provider
When selecting a biospecimen solutions provider, researchers may benefit from partnering with a provider that has the experience and infrastructure to rapidly build extensively characterized, fit-for-purpose specimen sets that have been consented for a broad range of applications.
“We obtain consent for every single subject or patient specimen we collect. Informing participants on what we are doing, why we are doing it, and how it can benefit other people and science at large is fundamental to our philosophy of ethical biospecimen sourcing,” said Boushell.
The provider should also have in-depth knowledge and understanding of the relevant industry, regulatory, and quality requirements, which may vary from country to country. True value is added by providers that understand not only specimen collection and management but also the science behind selecting the right samples at the right time in the research cycle.
Researchers may also want to inquire whether providers offer services beyond biospecimens, such as prospective collections, study kits, specialized laboratory and analysis services, and sample storage.
Meeting the demand for research-ready biospecimens
Precision Biospecimen Solutions, part of the Precision for Medicine global clinical research organization, has prospective collection capabilities across 100 research-ready clinical sites to maximize flexibility and sample freshness.
An established global leader in biospecimens, the company also offers custom assays to support biomarker development and validation and state-of-the-art biobanking facilities for secure storage, management, and distribution of future-use biospecimen assets.
Worldwide, pharmaceutical researchers are clamoring for well-curated, research-ready biospecimens to fuel the translation of laboratory discoveries into precision medicines.
With offices around the world and over 25 years of biospecimen-specific experience, Precision Biospecimen Solutions manages 25 million biospecimens and offers real-time sample processing in more than 55 countries.
Images via Precision for Medicine and Shutterstock.com