Characteristics of a Well-Designed Clinical Trial

By Michelle Churchman, PhD

Clinical Trial Design in the Age of Precision Medicine

The next generation of cancer care is being designed with the understanding that cancer is a genetic illness, and no two patients’ diseases are exactly alike. To combat cancer on a global scale, it is becoming increasingly clear that a combination of therapies targeted to individual genomic characteristics is the most effective strategy. This approach—known as precision medicine—is already underway and yielding effective results. For example, cancers that were once broadly classified are now being stratified by the presence or absence of any array of diverse cancer biomarkers, allowing for the development of more precision therapies, such as immunotherapies and kinase inhibitors, with very specific molecular targets.

As researchers continue to uncover the genomic signatures that underpin specific cancers’ development, spread, and response to treatment, an increasing number of clinical trials will be needed to test the efficacy of newly developed targeted therapies. To provide the greatest number of cancer patients with life-saving intervention as quickly as possible, it will be critical to streamline the process by which clinical trials are organized and executed while still adhering to the strictest protocols. Here, we’ll explore what constitutes a well-designed clinical trial and how oncology-focused bioinformatics companies, such as M2GEN, are helping to accelerate the discovery and development of new treatments.

Characteristics of a Well-Designed Clinical Trial

Similar to robust scientific experiments, clinical trials must be well controlled to deliver reliable data that can be used for a new drug application (NDA). The ultimate purpose of a clinical trial or interventional study is to determine the effectiveness and long-term safety and side effects of a newly developed drug or medical device. To achieve this, the study should maintain control of all variables and be easy to replicate.

The Food and Drug Administration (FDA) provides up-to-date regulations that clearly outline the requirements for a well-designed clinical trial. These include:

• Clearly stated objectives as to the purpose of the clinical trial
• A summary of the methods and means of analysis being utilized
• Adequate data to indicate due diligence was performed in selecting participants for the clinical trial
• The use of a control group that receives a placebo with standard of care treatment, different dosage, alternative treatment, or no treatment to compare data against
• Measures taken to minimize bias in all aspects of the clinical trial including, but not limited to, the random assignment of participants to control and experimental groups
• Well-defined and reliable methods of assessment that explain the variables measured, methods of observation, and criteria used to assess responses
• An analysis of the results of the study that adequately describes and assesses the results of treatment with the drug or medical device

Overcoming Challenges in Clinical Trial Design

Bringing a new cancer therapy to market is a time- and cost-intensive process. In fact, from discovery to approval, it takes an average of 10 years and costs hundreds of millions of dollars. However, there are many efforts underway to accelerate the discovery, development, and testing of new cancer therapies by addressing some of the hurdles that have traditionally impeded these processes.

For example, one challenge associated with developing robust clinical trials has been identifying an adequate number of qualified patient participants. There is often a narrow set of criteria, such as the presence of specific cancer biomarkers, that need to be satisfied to qualify an individual for a clinical trial. Modern data-sharing networks are making it easier for researchers to identify the right candidates. The Oncology Research Information Exchange Network^® (ORIEN^®), which has formed their own ORIEN Clinical Trial Network (OCTN), facilitated by M2GEN is a prime example. By providing researchers with access to longitudinal clinical and genomic information from hundreds of thousands of de-identified patients who have agreed to be contacted, we are helping to streamline the process to identify suitable candidates that are less likely to fail screening for enrollment in certain clinical trials, thus saving valuable time and money to accrue the right patients upfront to enter the funnel of potential participants. Additionally, our flexible data solutions can be used to create in silico external control arms.

Another issue that has hindered clinical trials is the establishment of research silos. Often, top cancer centers are set up to separate care providers and researchers. There are certainly benefits to this approach, including that it provides researchers and physicians a higher degree of freedom. However, it can also result in poor communication and, most significantly, missed opportunities.

Effective clinical trial design depends on the synergy yielded from active collaboration between key stakeholders, including patients, physicians, and researchers. This unified approach is something M2GEN is helping to facilitate as well.

Our partnership with the nation’s leading cancer centers, as well as patient advocates, has established a model for data and knowledge sharing to enhance collaborations that provide clear benefits in expediting the development and time-to-market for novel life-saving therapies. We are bridging the gap between discovery and patient care by eliminating roadblocks and fostering alliances that drive innovation. Our platforms allow professionals at every stage of cancer research, prevention, diagnosis, and treatment to more easily cooperate and realize the full potential of their research and initiatives.

For more information about how M2GEN is helping to advance and streamline all processes related to the discovery, development, and testing of novel cancer therapies, contact us to schedule a demonstration of our data solutions, including our flagship Avatar platform.