Curious about where the journey of a new medicine begins? Let’s explore the process together!
Identification of Unmet Need – The process begins with recognizing diseases where existing treatments are insufficient, aiming for better efficacy, safety, or patient compliance.
Target Identification & Validation – Biological targets (e.g., enzymes, receptors, genes) where drug needs to act are identified and confirmed to play a critical role in the disease.
Hit Identification & Lead Generation – Using assays and screening methods (e.g., high-throughput, fragment-based), compounds (“hits”) are found. These are optimized into lead compounds with improved potency, selectivity, and drug-like properties.
Lead Optimization – Leads are chemically modified to improve pharmacokinetics, safety, and efficacy while reducing toxicity.
Preclinical Development – Selected candidates undergo in vitro and in vivo studies to evaluate pharmacology, toxicology, ADME, and formulation before human testing.
INDA (Investigational New Drug Application) is filed in order to initiate clinical studies/trials.
Clinical Development – Conducted in phases:
-
Phase I: Small group, safety, tolerability, pharmacokinetics.
-
Phase II: Patients, efficacy, dosing, continued safety.
-
Phase III: Large population, confirm efficacy, compare with standard care, detect less common side effects.
NDA (New Drug Application) is filed to various regulatory bodies (FDA, CDSCO etc.) for approval of marketing.
Marketing & Post-Marketing Surveillance (Phase IV) – Drug is launched; real-world safety, effectiveness, and rare adverse events are monitored, sometimes leading to label updates or withdrawal.
This process is the reason why branded drugs are more costlier than the generic ones. Do you know when a company is eligible to make a generic version of a branded drug 
MBH/AB