Real-world data enables researchers to test hypotheses in advance of clinical trials
By Kate Torchilin, PhD, MBA
Healthcare is evolving toward precision medicine—an approach that uses information about a person’s genes, proteins, and environment to prevent, diagnose, and treat disease.1 Across the healthcare sector, every role is being affected by this trend, from researchers involved in drug development who can now study more specific subgroups of patients, to payers determining how to cover medications, to providers on the front lines of care, to patients themselves.
Clinical laboratories are also being affected, in several ways. The evolution toward precision medicine creates a unique opportunity for labs to elevate their role within the healthcare sector, and also to develop additional revenue streams.
A Growing Trend
Although targeted therapies have been an FDA priority since the 1990s, scientific and technological advances have played a major role in helping to advance precision medicine into mainstream clinical practice.2 As patients with the same disease or condition have been found to respond differently to various treatments, scientists have begun to use advanced technologies to better understand which patients respond to which kinds of treatments. In turn, drug companies have been able to begin developing treatments that are effective for those specific subpopulations.1
The precision medicine trend continues to grow. In 2014, nearly 20% of new drug therapies approved by FDA (eight out of 41) were personalized, or targeted, therapies.2 Meanwhile, advances in technology that enable the management of enormous data sets—including those resulting from the increasing adoption of electronic medical records (EMRs)—have made the promise of precision medicine a reality.
For patients, the shift to a personalized medicine approach offers significant benefits. Scientists are better able to understand diseases and make strides toward prevention, earlier diagnoses, and improved treatments; doctors can better predict which treatments will or won’t work for certain patients; and patients can be treated more effectively with fewer side effects.3,4
Historically, medicines have been developed to treat a single condition—in isolation from other diseases, or comorbidities, that could be affecting a patient’s health, and without regard to a patient’s genetic profile. When such medicines were tested through the clinical trial process to demonstrate that they were safe and effective, the patients included in the trials were selected according to specific inclusion and exclusion criteria that did not account for genetic variations and may have explicitly excluded patients with comorbidities. Yet, we now know that genetics and comorbidities can both potentially dictate how a patient responds to a particular treatment.
One reason that precision medicine is becoming a reality today is the rising capability of scientists and product developers to use real-world data collected and managed through EMRs. Real-world data is information about patients and their health that is gathered outside of the clinical trial process. It can include patient information amassed through other types of trials or registries, through case reports, medical histories, prescription information and, importantly, lab test results.5 Using the EMR structure, such information can now be recorded, stored, shared, and analyzed electronically.
Having access to massive databases with deidentified information about individual patients has enabled scientists to draw deep insights that would not have been obvious from the relatively small numbers of patients enrolled in clinical trials. Such databases have also enabled scientists to account for comorbidities and other relevant factors—including age, gender, and ethnicity—at a population level. Additionally, the EMR structure enables healthcare researchers to set up observational studies with patient consent and data-sharing authorization, so that the progress of a disease or treatment can be tracked in real time.
Real-World Data Studies
It is precisely because of the importance of real-world data to the rise of personalized medicine that clinical labs now have an opportunity to play a greater role in the drug development process than ever before. A large proportion of all EMRs consists of laboratory data, meaning that labs are handling huge amounts of patient information that constitutes the essential foundation for the development and success of precision medicine approaches.6
In the United States alone, clinical labs handle an estimated 13 billion tests each year, resulting in vast quantities of human biospecimens that remain after testing.7 While real-world data has an important role in helping researchers draw insights and develop theories for better drug development, human biospecimens remain critical for understanding the underlying biology of the disease. Many clinical labs have found additional opportunities to contribute to research—and increase revenue—by working with commercial enterprises that enable them to provide remnant biospecimens to research labs involved in drug development.
With access to both real-world data and human biospecimens, clinical labs are centrally positioned to help researchers test hypotheses in advance of setting up expensive and time-consuming clinical trials (see Figure 1). Real-world data—including which medications patients have taken, how long they have taken medications, their test results, and more—is critical to helping researchers identify cohorts of patients who might respond to a new therapy being developed. Meanwhile, the data-enriched specimens that clinical labs can provide offer drug developers additional detail to help deduce the conditions and traits that predict treatment success.
To facilitate the process of patient consent, some organizations and technology solutions gather consents in physician practices or hospitals, then work with labs to ensure that only specimens from consented patients are matched. The value of the lab’s role is further elevated when it can provide biospecimens that exactly match the criteria being tested by the researchers. Such studies help researchers assess whether there is enough evidence that a certain group of patients might respond to a candidate therapy to justify moving it forward in the process.
Leveraging the Lab’s Position
At this intersection of real-world data and patient specimens, clinical labs have a unique opportunity to serve as the conduit and interpreter of data-enriched biospecimens. They can also facilitate a better understanding of clinical pathways and treatment paradigms, including the clinician’s options for drug selection.
Most clinical lab professionals would agree that diagnostic tests are becoming more complicated, and that the results of such complex tests are often not readily understood by physicians or their staff. Clinical labs recognize this gap and are starting to provide educational materials to aid healthcare providers in understanding the relevance of certain results to treating the patient’s condition.
With access to data highlighting a patient’s genetics, comorbidities, and medical history, labs can provide even greater value to healthcare providers by presenting a patient’s results against the context of other patients with a similar profile, and by drawing high-level insights about populations of patients with a similar profile. In this way, clinical labs have an opportunity to differentiate their service offering and provide greater value to the healthcare providers who order lab testing.
Managing an Evolving Role
The opportunity to elevate the role of the clinical lab within the healthcare and biomedical sectors is enticing. Nevertheless, many clinical lab managers may wonder about the wisdom of adding to current workloads, which have continued to grow at an annual rate of 5% to 10% each year since the 1970s.8 Technological advances have helped to reduce strain on existing staff, but reduced reimbursement and a shortage of clinical lab employees have placed additional pressures for providing even core testing services.
Recognizing the value that clinical labs hold for advancing research, and the time constraints that impede the addition of ancillary services, a number of companies have developed businesses designed to facilitate connections among clinical labs, scientists, and product developers. Some facilitate the transfer of human biospecimens from clinical labs to research labs. Others help researchers define criteria for clinical trials based on deidentified data gathered from global sources.
Novaseek Research was started 3 years ago to marry these two approaches, connecting researchers with patient data and human biospecimens with the specific goal of advancing precision medicine. With an executive management team whose professional experience spans both the clinical lab and research sides of the equation, Novaseek was acutely aware of the need to seamlessly align with current workflows as well as with the EMR systems already in place.
Novaseek functions much like a large, virtual biobank, aggregating data from a network of participating hospitals and clinical laboratories (see Figure 2). Using proprietary informatics, Novaseek helps researchers find patients and biospecimens that meet clearly defined requirements, and enables clinical labs to repurpose for research specimens that might otherwise be discarded. (For more information, see “An Easy Route for Advancing Medical Research.”)
Novaseek filters through streams of data about the thousands of clinical samples that flow through its partners’ laboratories each day, and enables researchers to request data and specimens that satisfy their specific criteria, including the donor’s diagnosis, lab values, medication history, and so on. The criteria are then compared to data for available specimens at clinical partner sites, where hospital or laboratory personnel can secure samples that satisfy the criteria.
As the field of medicine evolves to focus increasingly on precision medicine, the work of clinical lab professionals will gain added importance. The results of clinical lab testing will not only provide information critical to the healthcare management of the patient being tested, they will also contribute to our understanding of disease and inform the development of treatments for future patients with similar profiles.
Kate Torchilin, PhD, MBA, is CEO of Novaseek Research, Cambridge, Mass. For further information contact CLP chief editor Steve Halasey via [email protected].
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