Integrated Drug Discovery Process in BioNome

Drug designing is a magnificent inventive process in the development of novel therapeutics against the biological target. There are five major steps in the drug development process.

  1. Discovery and Development
  2. Preclinical Research
  3. Clinical Development
  4. FDA Review
  5. FDA Post-Market Safety Monitoring.

Step 1: Discovery and Development

Target Identification and Validation: Target identification means finding a gene or protein which plays a vital role in disease-causing. The therapeutic character of the target is recorded. The drug target must be safe, usable, effective, and capable of clinical and commercial use. Target validation is done by using different techniques like cell-based models, functional analysis of genes, signaling pathways analysis, and in-vitro genetic manipulation.

Hit Discovery Process: Compound screening assays are developed after the validation of the target.

Assay Development and Screening: Assays are test systems that analyze the effects of the new drug-like compound at the biochemical, molecular, and cellular levels. High Throughput Screening (HTS) rapidly conducts millions of genetic, chemical, and pharmacological tests reducing the time by using robotics, data processing software, and tools and sensors. It helps to identify active compounds or genes which affect human molecules.

Hit to Lead: In this process, small molecule hits resulting from HTS are evaluated and optimized into lead compounds. These compounds are then further moved to lead the optimization process.

Lead Optimization: In this process, lead compounds are synthesized and modified to increase efficiency and decrease side effects. It conducts experiments using animal models and ADMET analysis tools.

Step 2: Preclinical Research

In vivo, In vitro, and Ex Vitro Assays: In vivo assays includes the development of new Drug using animal models such as mice or rat. In vitro research conducted in research laboratories. Ex vivo assays use cells or tissues from a non-living animal.

ADME: Adsorption, Distribution, Metabolism, and Excretion is a process of analysis of how a new drug affects or reacts with the body. It involves a mathematical explanation of each effect.

Drug Delivery: It includes oral, topical, membrane, intravenous, and inhalation. This system is used for the controlled delivery of the new drug. This aims to prevent the drug to affect healthy cells or tissues and is still effective to inhibit the effects of the disease.

Formulation optimization and Improving Bioavailability: This process is ongoing during all pre-clinical and clinical stages. It helps to ensure that the drug reaches the right place in time at the proper concentration.

IND application: IND applications are submitted to FDA before starting clinical trials. If FDA responded positively to the drug, then only the developer may start the clinical trials.

Step 3: Clinical Drug Development Process

Phase I (Healthy Volunteer Study): In this process, the drug is tested for the first time on humans. Researchers and Scientists ensure the safety levels and side effects for a safe dosage range on 100 volunteers.

Phase II and Phase III (Studies in Patient population): In these phases, drug safety and efficiency are tested on 100-500 patients. Analysis of the optimum dose helps to create schedules for the next dose and risks are also recorded. In phase III drug is given to 1000-5000 patients with medication labeling, instructions, and precautions for proper use of the drug.

Dose escalation. Single ascending and multiple-dose studies: Proper dose determines the efficiency of the drug and clinical trials determine the best dose for the treatment.

Pharmacokinetic Analysis: This is an experimental trial to determine the behaviour of a new drug in the human body. Through compartmental modeling, the volume of distribution, and terminal half-life are determined.

Bioanalytical method development and Validation: It detects analytes and metabolites to determine their efficiency and safety. The complete bioanalytical assay includes sample collection, clean-up, analysis, and detection.

Step 4: FDA Review

            Once a new drug is formulated for its safety and efficiency, and the records from clinical trials are forwarded for FDA review. Then FDA reviews and decides whether to approve the drug or not and the application of the drug must be submitted by the developer.

NDA Application: An NDA abbreviated new drug application (ANDA) is submitted to FDA after clinical trials. FDA reviews the study data and then decides whether grant permission or not for the drug.

Reasons for Drug Failure:

  1. Toxicity
  2. Inefficiency
  3. Poor Bioavailability
  4. Inadequate drug performance

Step 5: Post-Market Monitoring

            Even after drug approval and manufacturing, it is required that drug companies continue monitoring the safety of drugs using the FDA Adverse Event Reporting System (FAERS) database. It helps FDA to implement a post-marketing safety surveillance program. Manufacturers, health professionals, and consumers can report problems with approved drugs.

Computer-Aided Drug Discovery (CADD)

            To overcome the challenges faced by the traditional drug discovery approaches, CADD is employed by drug development and pharmaceutical companies. CADD is an essential tool to reduce the failure from preliminary screening till the final phase of drug discovery and helps in reducing trials and saving time. In the current era, CADD tools are being used as a virtual shortcut to analyze, develop, and identify drug-like molecules. The use of computational tools and experimental findings has an important role in the novel drug development process. It also reduces the total cost of drug discovery without affecting accuracy. The most common CADD-based approaches are structure-based drug design (SBDD), ligand-based drug design (LBDD), and sequence-based approaches.


  1. Advances in Bioinformatics by Vijay Singh and Ajay Kumar.