Description
The investigational use of drugs involves administering pharmaceutical compounds not yet approved by regulatory authorities.
These drugs are evaluated in clinical trials to assess safety, efficacy, dosage, and side effects, aiming to gather data for potential approval.
Principles
Scientific Methodology: Rigorous study design, control groups, and statistical analysis ensure valid and reliable data.
Ethics: Trials adhere to ethical principles—respect, beneficence, and justice—with informed consent and harm minimization.
Regulatory Compliance: Compliance with FDA, EMA, or equivalent regulations ensures participant safety and data integrity.
Good Clinical Practice (GCP): Trials follow international standards for ethical and scientific quality in human research.
Classification of Investigational Use of Drugs
Investigational drugs can be classified based on:
1) Development Phase:
Phase I: Assess safety and dosage in a small group of healthy volunteers or patients.
Phase II: Evaluate efficacy and side effects in a larger patient group.
Phase III: Confirm effectiveness, monitor adverse reactions, and collect information for drug labeling.
Phase IV: Post-marketing studies to delineate additional information, including the drug's risks, benefits, and optimal use.
2) Type of Agent:
New Chemical Entities (NCEs): Compounds that have not been previously studied in humans.
Biologics: Products derived from living organisms, including vaccines and monoclonal antibodies.
Gene Therapies: Treatments that involve modifying the genetic material within cells.
3) Therapeutic Area:
Categorized by the medical condition they aim to treat, such as oncology, cardiology, or neurology.