Clinical Research: Phase 1 - Phase 4

5 Essential Factors for Navigating Early Stage Trials

Bringing a novel drug to market can be a long, perilous journey down the clinical testing pipeline, taking upwards of 10 to 15 years. Maintaining research and development productivity while navigating the ever-changing regulatory landscape, the choppy waters of today’s reimbursement environments, and the rising tide of clinical trial costs is increasingly challenging. Medicines that fail to make it through human trials litter the banks of the pipeline. Some trials sink before ever leaving the harbor.

Pretrial preparation can calm the waters, and addressing certain factors can increase the probability of successful transitions to later-stage trials. Successful navigators know they must chart a clear course to their destination, secure a harbor, and deliver a safe, commercially viable product that consumers need. Likewise, when bringing a drug to market, success relies on choosing the right target, tissue, safety profile, patient population, and commercial potential.

1. Target

Gather information that establishes a clear link between the target and the disease. Information can be in the form of direct evidence, a well-validated animal model or preclinical study data, or a description of the biological underpinning of the disease or target. The best evidence of linkage between target and disease may be validated efficacy biomarkers that predict patient responses to specific drugs. These biomarkers can help guide the investigation to strengthen or invalidate the study’s scientific hypothesis.

2. Tissue

Show that the target organ is reactive to the candidate drug. The drug’s chances of success increase greatly if pharmacokinetics / pharmacodynamics modeling shows sufficient bioavailability of the compound and pharmacological activity in the target tissue. Understanding the PK/PD relationship can also help guide the therapeutic concentration range under review.

3. Safety profile

Developing an appropriate safety profile is essential, as the smallest wave at the beginning of a project can become a tsunami in time, causing project delays or even closures. While safety signals may be unclear or difficult to translate from preclinical models to humans, applying robust safety standards prevents molecules that are likely to fail from advancing too far in clinical trials.

4. Patients

Test the compound of interest in the correct population. Confidence in patient selection correlates with molecule progression to later-stage clinical trials, and projects with clear patient stratification plans are more likely to be successful. Current scientific understanding of the disease should help target the optimal patient population to shape development plans. Predictive biomarkers, known markers of disease stratification, or a series of potential biomarkers monitored throughout the trial can help identify and stratify patients.

5. Commercial potential

Use the molecule’s commercial potential to guide project development. Without a clear commercial need, your new medicine is dead in the water. Be sure to research current and future standards of care thoroughly to ensure that your new medicine adheres to tomorrow’s standards for efficacy and safety.

Addressing these five factors pretrial can increase the likelihood of successfully navigating to later-stage trials. For more information on maximizing success of your drug trials, read our white paper, Maximizing Success in Early Stage Oncology Trials.