Phases I-IV_Explanation

Preclinical Development

  • Toxicology – Science of toxins, the poisoning and the treatment of poisoning
  • Pharmacology – science of interaction between substances and living organisms
  • pharmacodynamic (PD) – Science of the effect of substances in the organism
  • pharmacokinetics (PK) – Set of processes, a substance is subject to in the body.
  • pharmacogenetics / pharmacogenomics (PG) – Study of genetic variation, which result in a different response of individuals to medicinal products. Application of DNA-based genotyping with the aim to develop pharmaceutical agents for specific populations.

Clinical Trials Phase I

  • first use of the potential future medicinal product in humans after first effects on animals are known
  • normally healthy volunteers (except for certain indications, participants already suffering from target disease)
  • Investigation: how the medicinal product acts on the body and how it is absorbed by the body
  • Absorption, distribution, metabolism and excretion
  • different doses to determine the acceptable dose range
  • Short-term application
  • open studies
  • normally no more than 20 participants
  • focus on tolerance

Clinical Trials Phase II

The early phase II (Phase IIa):

  • therapy concept (Proof of Concept)
  • carried out on small groups of patients
  • open studies
  • focus on: tolerance, efficacy and safety
  • Patients with target disease
  • up to 400 participants

The late phase II (phase II b):

  • Controlled Style: comparison group available (standard treatment and / or placebo)
  • efficacy and safety: The objective of the Phase II trials is reached when there are very clear ideas as to which symptoms, in which patients and in what dosage the medicinal product is effective and well tolerated.

Clinical Trials Phase III

  • proof of therapeutic efficacy and safety in larger patient groups
  • Controlled comparison: new medicinal product vs. Placebo or standard therapy
  • Often multicenter studies
  • After completion of Phase III, the decision on a marketing authorization is made
  • Dosage and duration adjusted to real application in order to obtain statistically significant information.
  • Typically, 200 to 10,000 patients
  • safety and efficacy, “Pivotal” studies (key studies)
  • Studies to support the MAA
  • registration studies
  • obtaining demographic data
  • Randomized, comparative studies