From laboratory to clinic: how preclinical development determines the success of a medicine

The path from a promising molecular discovery to a successful medicine is long, costly and uncertain. Only a small percentage of drug candidates ultimately reach the market. Although considerable attention is devoted to clinical trials, the foundation for success is established much earlier, during the preclinical development phase.

Preclinical development provides the bridge between fundamental research and first-in-human use. This phase determines not only whether a drug candidate is sufficiently effective and safe to progress, but more importantly whether the underlying scientific hypothesis is strong enough to justify substantial investment in clinical research. Preclinical development is therefore far more than a collection of laboratory activities. It is a strategic decision-making process through which risks are identified, assumptions are validated and the likelihood of clinical success is increased.

From data to decision-making

A common misconception is that the purpose of preclinical research is to generate as much data as possible. In reality, this phase is about generating the right data: information that supports decision-making and reduces uncertainty.

Each step in the development pathway should answer fundamental questions:

Is the mechanism of action sufficiently substantiated?
Are the pharmacological effects reproducible?
Is the safety profile acceptable?
Are the anticipated clinical benefits realistic?
Is the candidate suitable for further development?

The quality of these answers ultimately determines whether an organisation can invest in the next phase of development with confidence.

Translational science as a critical success factor

One of the greatest challenges in preclinical development is translating laboratory findings into clinical practice. Results that appear promising in vitro or in animal models are not always predictive of efficacy in humans.

Leading pharmaceutical and biotechnology organisations are therefore investing increasingly in translational science. Combining biomarker development, advanced disease models, pharmacokinetic analyses and integrated data analytics provides a more realistic understanding of a drug candidate’s potential clinical performance.

The more effectively this translation is achieved, the lower the risk that fundamental limitations will emerge only during costly clinical trials.

Risk reduction begins before the first patient

Every clinical trial represents a substantial investment of time, resources and capacity. At the same time, each study carries risks for patients, investors and development organisations.

The greatest value of preclinical development therefore lies in the early identification of risk.

A robust preclinical strategy evaluates areas including:

Potential toxicological risks;
Pharmacokinetic properties;
Optimal dosing strategies;
Manufacturing and formulation challenges;
Potential regulatory considerations.

The earlier these uncertainties are identified, the better equipped organisations are to make well-informed go/no-go decisions.

The power of integrated collaboration

Preclinical development is no longer an isolated research process. Its success depends on collaboration across a wide range of disciplines.

Pharmacologists, toxicologists, drug metabolism and pharmacokinetics specialists, chemistry, manufacturing and controls experts, bioanalytical scientists, regulatory affairs professionals, quality specialists and project managers all make essential contributions to the development of a drug candidate.

Organisations that bring this expertise together at an early stage identify risks sooner, make decisions more rapidly and prevent delays during subsequent development phases. Preclinical development is therefore becoming an increasingly integrated decision-making process that brings together science, strategy and operational excellence.

The value of experience

As medicines become more complex, including cell and gene therapies, RNA technologies and precision medicine, the complexity of preclinical development also increases.

Successful programmes are distinguished not only by scientific excellence, but particularly by experienced professionals who understand which decisions matter at each stage. They recognise risks before they become apparent, understand regulatory expectations and connect different disciplines effectively.

This combination of deep subject-matter expertise and broad development experience is becoming increasingly decisive to the success of innovative drug development.

A strategic investment in future success

Preclinical development is sometimes regarded merely as a necessary step on the path to clinical research. In reality, it is one of the most decisive phases of the entire development pathway.

Organisations that invest in high-quality preclinical expertise, strong scientific evidence and integrated decision-making not only increase the likelihood of successful clinical trials, but also improve the efficiency of their research and development portfolio as a whole.

The success of a medicine therefore does not begin with the first patient, but with the quality of the decisions made beforehand. Preclinical development provides the strategic foundation on which successful innovation in the life sciences is built.

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