Preclinical vs Clinical Evidence

Understanding the crucial differences in peptide research stages.

The Research Hierarchy

Peptide research progresses through distinct stages, each providing different types of evidence. Understanding these stages is essential for interpreting what research findings actually mean.

Preclinical Research

Preclinical research occurs before human testing and includes:

In Vitro Studies

Research conducted in test tubes or cell cultures. These studies:

  • Investigate molecular mechanisms in controlled environments
  • Cannot capture whole-organism effects
  • May not reflect what happens in living systems
  • Are useful for understanding basic biology

Animal Studies

Research conducted in animal models. These studies:

  • Provide whole-organism data
  • May not translate directly to humans
  • Use species with different biology (often rodents)
  • Can explore safety margins not ethical in humans
Most peptide research is preclinical. Animal or cell culture findings do not prove human effects.

Clinical Research

Clinical research involves human subjects and follows a phased approach:

Phase 1

First-in-human studies focusing on safety:

  • Small numbers of healthy volunteers (typically 20-100)
  • Primary goal: safety and tolerability
  • Secondary: pharmacokinetics (how the body processes the compound)
  • Not designed to show efficacy

Phase 2

Early efficacy studies in target population:

  • Larger groups (100-300 participants)
  • Studies in people with relevant conditions
  • Explores dosing and initial efficacy signals
  • Still relatively small for detecting rare effects

Phase 3

Pivotal trials for regulatory approval:

  • Large, multi-center studies (hundreds to thousands)
  • Randomized, controlled, often double-blind
  • Designed to definitively demonstrate efficacy and safety
  • Required for drug approval in most jurisdictions

Phase 4

Post-market surveillance:

  • Ongoing monitoring after approval
  • Detects rare adverse effects not seen in trials
  • Studies long-term outcomes
  • May lead to label changes or withdrawals

The Translation Problem

Why don't preclinical findings always translate to humans?

  • Species differences — Mice and humans have different biology
  • Dosing differences — What works in rodents may not scale
  • Endpoint differences — Measured outcomes may not be equivalent
  • Complexity — Human conditions involve factors not modeled in animals
  • Publication bias — Failed translations often aren't published

Estimates suggest only about 10% of compounds that show promise in preclinical research eventually receive approval for human use.

Interpreting Research Stages

When evaluating peptide claims, consider:

  • What stage of research supports the claim?
  • Has any human research been conducted?
  • How well do the research subjects match the claim being made?
  • Is the evidence being over-extrapolated?

Be especially skeptical when preclinical findings are presented as proof of human effects.