Glossary Definition: Preclinical Development

What is preclinical development?

Preclinical development aims to determine safe dosages and processes for treatments, drugs, or pharmaceutical procedures. This happens after preclinical animal studies are complete, and before human trials start.

It’s a four-part process that includes drug discovery and development as well as assessing the drug pharmacology, also known as ADME (absorption, distribution, metabolism, and excretion), and toxicology. In other words, how the drug works and whether it’s safe.

Once the formulation is complete, the drug or treatment moves on to manufacturing. Prior to moving any medication into clinical trials, batches are tested for purity and quality using the assays that are created during preclinical development.

What happens during preclinical development?

During preclinical development, researchers use lab-grown human cells and computer models to test potential drugs. They’ll study the efficacy and side effects of medication, based on how the human cells react to treatment. They use this data to create templates for procedures to make the drug that will scale to clinical and then commercial manufacturing.

After receiving a drug candidate, preclinical process development groups figure out how to produce it at high enough quantities to support clinical studies. They surface this information in a template to lock down the process, and then transfer it to clinical teams for pilot studies.

During this stage, failure rates are high and any therapeutics that researchers determine are potentially harmful, do not work as intended, or damage human cells do not move on to clinical trials. Another cause of failure rates is when teams cannot figure out a way to produce the drug at scale.

Preclinical vs clinical: What’s the difference?

Preclinical development is all about collecting and analyzing data from animal testing and human cells to determine if drugs should be moved onto human trials. In contrast, clinical manufacturing involves testing therapies on human subjects. As such, they only begin after the preclinical trials determine the therapies to be safe and potentially effective.

Why is preclinical research important?

Preclinical research is important because it prevents therapies, treatments, or procedures from being prematurely tested on human subjects. Preclinical studies vet drugs for their safety and efficacy. This is crucial because preclinical studies have, on average, a 31% success rate. This means in the vast majority of cases, these drugs are determined to be unsafe, ineffective, or a combination of the two. As a result, they will not progress to human trials.

Ultimately, the goal of preclinical development is to determine safe dosages for drugs, processes, and treatments. To accomplish this, it requires extensive testing on both lab-grown human cells, and animals.

During this stage, researchers consider factors such as:

  • Formulation stability
  • Toxicity levels 
  • Dosage frequency
  • Routes of administration
  • Solubility

Based on their findings, researchers will then determine the best path forward. Approximately one-third of the time, preclinical drugs move on to clinical trials.