Biospecimen Research Will Increase Success in Clinical Trials

The efficient translation of discovery projects to clinical trials is necessary to improve productivity, decrease the risk of R&D projects, and ultimately deliver better medicines to patients faster.

Clinical and discovery groups in pharmaceutical and biotechnology companies are increasingly working together to share insights about the potential clinical impact of biomedical discoveries much earlier in the process.

Biomarkers are an essential part of such a translational effort and are critical to understanding human disease. Human biospecimens can provide a bridge between the early discovery and molecular mechanism of the disease and the clinical utility. Commonly, human biospecimens are used to:

• Identify and validate new biomarkers of the disease.

• Identify how diseases progress and vary.

• Group patients as more or less likely to respond to specific drugs.

• Develop screening tests to detect biomarkers that are associated with certain stages or subtypes of a disease.

In the discovery phase of a project, it’s important to use biospecimens that accurately reflect the phenotype of patients who will be involved in clinical trials later. For example, if they’re studying potential markers or drug responses for a disease that has several different subtypes, researchers should acquire specimens from patients with that specific subtype of the disease. Or if the trial revolves around certain responses in patients who exhibit early stages of a disease, the biospecimens should be taken from patients in those early stages.

There are typically three main areas that need to be considered when planning biospecimen collections: the characteristics of the biospecimen (biological), the characteristics of the patient (clinical), and the budget and timeline required (operational).

Unfortunately, it can be difficult for researchers in discovery and preclinical areas to apply a comprehensive understanding of patient clinical data, even when trying to find the right biospecimens.

Additionally, budgets and timelines are often hard to predict at the beginning of a project. Unlike an online search engine, however, there’s usually no single source of information to find out whether available specimens meet the exact criteria. Therefore, the procurement can be a slow, manual process of calls and emails between researchers and vendors trying to identify what kinds of biospecimens might be available. It can take days, weeks, or even months to obtain the necessary samples, depending on the specific requirements.  

Further complications arise when information gets lost in translation. A vendor may not ask for particulars, such as the necessary subtype of the disease in question, and the researcher might not know to specify. Such details, if ignored, can impact the biological significance of the discoveries made with the use of biospecimens, resulting in failure during the validation, translational, or clinical phases.  

The risks can be mitigated when researchers have clear, comprehensive clinical information, in addition to biological information, as early as the discovery and preclinical phases.

When researchers have reliable, transparent data on the biospecimens early on and can choose those that most accurately reflect the types of patients who will be enrolled in the clinical trial, the risks of failure are substantially reduced. 

Here are four areas to consider to accurately plan projects that require human biospecimens:

1. Utilizing clinical patient data in selecting biospecimens for research: Researchers should incorporate as much relevant clinical data and information about the patient (including the stage of the disease, co-morbidities, demographics, etc.) as they think might be relevant to discovery and validation. Doing so will ensure the biospecimens they receive accurately reflect the patients they’ll be treating later. More and more clinical data is becoming available due to the adoption of electronic health records, and there are now many federal and commercial initiatives to extract such data and make it useful and applicable for biomedical discoveries.   

2.  Prioritizing biospecimen characteristics that are the most important to the study: Pre-analytical variables related to biospecimen collection, processing, and storage can influence the results of experiments. Therefore, many biospecimen requests include tight constraints related to how the specimens must be collected. Unfortunately, if constraints are too numerous or include factors that are difficult for medical and laboratory personnel to control consistently, the result may be inadequate quantities or inconsistent quality. While tight requirements may be necessary for some projects, many investigators will benefit from pragmatically prioritizing biospecimen criteria to focus on quality factors that are consistently attainable. 

3. Patient consent: Federal and state regulations create stringent requirements for proper authorization from patients regarding the use of their biospecimens and data. Even when biospecimens are available, some projects may be unable to proceed if there are deficiencies in the original consent documentation. To optimize the value of biospecimen collection, consent forms should address key considerations such as future secondary use and the longitudinal collection of specimens and data.

4. Keeping biospecimen collection projects on time and on budget: The timeline for collecting biospecimens often paves a critical path for the whole project. Delays may increase the pharmaceutical company’s project costs by hundreds of thousands of dollars per day. Reliability and transparency around the timelines for biospecimen collection could improve project planning and reduce budget overruns. Providing researchers with upfront information regarding the relationship between timelines and specific request criteria could allow them to consider ways to modify the request in order to obtain specimens faster.

Access to detailed information, including clinical data, during discovery would enable researchers to obtain human biospecimens that most accurately represent the types of patients that are being studied and should substantially improve the chances for project success across the R&D enterprise.

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