Does Precision Medicine Live Up to the Hype?
It’s no exaggeration to say that precision medicine is the future of drug development, particularly for rare diseases and cancer. When a drug targets a specific biomarker to fight disease at its source, it provides a complex but powerful goal: targeting a precise mechanism of action in a subpopulation of individuals with a disease. This approach could have a much higher probability of extending life or facilitating disease remission in comparison to currently approved treatments that were developed through one-size-fits-all clinical trials. Being able to tell a patient that there are clinical trials that are tailored to their physiology and genetic makeup offers a new level of hope that traditional approaches could not provide. Precision medicine offers clear, tangible benchmarks to gauge success, making this area of drug development attractive to sponsors.
Can precision medicine improve cancer treatments?
Nearly everyone is connected to someone who has dealt with cancer. Millions of patients around the world struggle with one of these diseases, which historically have been difficult (if not impossible) to cure. Because of this, hearing "you have cancer" can feel like a death sentence. The reality is we must continue to identify differential mechanisms of action and their interdependencies to find targets we can harness to bring about a cure. Decoding the genome in 2003 allowed scientists to accelerate precision-based research, which is now gaining significant traction due to increased funding and commercial success of immune checkpoint inhibitors — among other drugs.
Precision medicine is thus the frontier of oncology research, as it has the potential to treat without employing the "scorched earth" methods of radiation and chemotherapy that damage ancillary systems in the struggle to rid the body of cancer. Now that scientists have identified many cancer genes, the potential for targeted therapies is rapidly expanding. However, researching these drugs in patients with the disease presents challenges that are much bigger than genetic modification. The disease itself and its treatments affect the body in myriad ways, and teasing out how exactly the drug interacts with the cancer apart from other factors can be challenging. Despite these obstacles, sponsors are eager to accelerate precision medicine drug development because of its unique capabilities.
What is the urgency?
Given the promise of these therapeutics, the race to develop precision medicine is measured by the lives that could be saved. Patients in clinical trials often extend their lives by months or years, highlighting the impact of these therapeutics. HIV treatments are an example of how effective precision medicine can be. Thanks to therapies that target specific biomarkers and mechanisms of action, the complicated virus that was once a death sentence is now curable in some instances.
In addition, when scientists research individual precision drugs, they learn more about the disease’s mechanisms to inform future studies. Many hard-to-treat diseases require a combination of targeted drugs; and the more biomarkers are studied, the closer scientists get to unlocking the right combination of targeted therapeutics that can put a disease into complete remission.
However, the research is intricate because biomarker pathways work like highways in the body. When one is activated, hundreds or even thousands of other interactions are affected, like exits closing, rerouting, or opening due to construction. If a precision-based drug targets a particular cell membrane protein or upregulates a gene, the ensuing observations through biomarker testing and safety analysis allow scientists to learn more about the complex mechanisms of the human body and the nature of the disease.
Why is development difficult?
Running a precision medicine clinical trial is logistically challenging. First, the assays are complicated and require a unique, highly technical skillset and instrumentation. It is difficult to find the partners to perform this testing on a global scale while simultaneously ensuring their ability to quickly and properly handle the sample processing, discrepancy reconciliation, and assay data reporting.
In addition, the samples collected to support these biomarker assays often have narrow stability windows and challenging collection, processing, and storage requirements. Keeping them viable throughout their journey requires multifunctional expertise and an exact handle on sample metadata and preanalytical variables that give you insight into your samples and the viability of the assay testing. This also means companies coordinating those logistics must be equipped to meet local regulations to transport biomaterials through customs.
The reality of today’s clinical trial logistics means that separate data sets are being created at separate facilities that receive, process, and/or test your samples. Sample-related data drives clinical trials, but the average trial collects data from several unique locations, not including the clinical sites where the sample journey starts. Each facility has its own databases and processes, and sponsors must reconcile the information received across disparate platforms to ensure the integrity of sample metadata and assay data.
Risk management mechanisms must be employed to govern biospecimens across their entire lifecycle to ensure the highest level of data integrity and demonstrate proper chain of custody. Unfortunately, many drug developers still rely on outdated methodologies—such as paper records or Excel sheets—that are inadequate for any clinical trial’s samples, but especially precision medicine research. In addition, most technologies focus on aggregating data after samples have been collected, processed, and even tested to tell a story of what happened. Given the great need for these treatments, finding advanced technological solutions to control and manage samples and their data from the moment of origination and throughout the complete sample lifecycle is paramount. Better sample data management is the key to unlocking precision medicine’s potential.
Effective sample management is also crucial to safeguarding patient care in studies involving patients with life-threatening or debilitating conditions like cancer. Sponsors should consider sample management solutions before enrollment begins and employ an agile, real-time platform that adapts to the complex nuances of today’s clinical trials. To learn more about streamlining sample management for precision medicine, check out Slope’s white paper on Ensuring Patient Centricity In Oncology Trial Sample Management.
About Slope
Slope is on a mission to transform the biospecimen lifecycle by providing sponsors with immediate access to biospecimen data and insights needed to mitigate risk and inform critical study decisions. We offer a singular platform, Biospecimen360™, for the traceability of lab kits, patient samples, and the data that results from those samples — uniting sponsors, research sites, labs, and other third-party vendors and data systems. Our software orchestrates the entire biospecimen lifecycle from kitting to sample collection to arrival at its final destination through chain of custody tracking.
Slope captures sample metadata from sites as they perform study activities, and the platform integrates with other stakeholders and data systems, including EDC, LIMS, IRT/RTSM, kitters, couriers, and more. Sponsors can leverage real-time data to meet critical study KPIs, ensure site compliance and vendor oversight, and perform reconciliation on the entirety of their sample metadata.
