The document provides a comprehensive overview of the drug development journey, detailing stages from drug discovery to regulatory approval. It highlights the importance and challenges of drug development, explains the processes of target identification and optimization, as well as preclinical and clinical study phases. The document concludes with an emphasis on regulatory submission and post-approval activities critical for ensuring drug safety and efficacy in the market.

1. New Drug Discovery and
Development
Overview of the Drug Development Journey
SUBMITTED TO
MO SUHEB ANSARI
FACULTY OF PHARMACY
SUBMITTED BY
NEHA GUPTA
B.PHARM 4th YEAR

2. CONTENT
✧ Introduction
✧ Drug Discovery - Target Identification
✧ Drug Discovery - Lead Compound Identification and Optimization
✧ Pre clinical Studies
✧ Non-Clinical Activities
✧ Clinical Studies - Phase I
✧ Clinical Studies - Phase II
✧ Clinical Studies - Phase III
✧ Regulatory Submission and Approval

3. Introduction
✧ From Discovery to Market:
✧ Emphasizing Process Scope: This phrase highlights that the drug development
journey encompasses numerous stages, from initial discovery in the laboratory to
making the drug available to patients in the market.
✧ Importance:
✧ Crucial Nature of Drug Development: This section underscores the significance of
drug development in advancing medical treatments, improving patient outcomes,
and addressing unmet medical needs.
✧ Challenges in Drug Development: It acknowledges the complexities and hurdles
involved in drug development, such as scientific uncertainties, regulatory
requirements, and financial investments.

4. Drug Discovery - Target Identification
✧ Identifying Biological Targets:
✧ Crucial First Step: Identifying the specific molecules, such as proteins or genes, that play a role
in a particular disease or condition. These targets serve as the focus for developing drugs to
treat the ailment.
✧ Methods:
✧ Advanced Techniques: Employing sophisticated methodologies like genetic studies and
bioinformatics to analyze vast amounts of biological data. These techniques help researchers
pinpoint potential drug targets with greater precision and efficiency.
✧ Example: HER2 in Breast Cancer:
✧ Real-World Application: Highlighting a concrete example of successful target identification.
HER2 (human epidermal growth factor receptor 2) is a protein that is overexpressed in some
breast cancers. Drugs like trastuzumab (Herceptin) target HER2-positive breast cancer cells,
demonstrating the therapeutic potential of targeting specific biological markers in disease

5. Drug Discovery - Lead Compound
Identification and Optimization
✧ High-Throughput Screening:
✧ Quick Identification: High-throughput screening is a method used to rapidly test thousands of
compounds to identify those with potential therapeutic activity against a target. It accelerates the
process of finding lead compounds for further development.
✧ Computational Methods:
✧ Predictive Software: Computational methods involve using computer algorithms and models to
predict the interactions between compounds and their target molecules. This allows researchers
to prioritize compounds for experimental testing based on their predicted efficacy and safety
profiles.
✧ Lead Optimization:
✧ Enhancing Compounds: Lead optimization is the process of modifying lead compounds to
improve their potency, selectivity, and pharmacokinetic properties while minimizing adverse

6. Preclinical Studies
✧ Pharmacokinetics (ADME):
✧ UnderstandingDrugBehavior: Beforedrugs can betested in humans, preclinical studies
evaluatehow they behavein thebody. This includes how they areabsorbed into the
bloodstream, distributed to tissues, metabolized (broken down), and excreted from thebody.
Understanding theseprocesses helps determineappropriatedosages and potential side
effects.
✧ Pharmacodynamics:
✧ AssessingDrugEffects: Preclinical studies also investigatehow drugs exert their effects on
thebody. This involves understanding thedrug's mechanisms of action, such as how it
interacts with specificreceptors or enzymes. By studying pharmacodynamics, researchers can
assess theeffectiveness of thedrug in treating thetargeted condition.
✧ Toxicology:
✧ EnsuringSafety: Toxicology studies areessential to ensurethat thedrug is safefor human
use. Thesestudies evaluatethepotential toxicity of thedrug at various doses and exposure
levels. Researchers assess theeffects of thedrug on organs, tissues, and biological systems to
identify any adversereactions or harmful effects. Thegoal is to establish safedosageranges

7. Non-Clinical Activities
✧ Manufacturing and Formulation:
✧ Production Methods:This involves developing efficient methods for manufacturing the drug
on a large scale. It includes processes such as synthesis, purification, and formulation.
✧ Dosage Form: Determining the form in which the drug will be administered to patients, such
as pills, injections, topical creams, or liquid solutions. This decision is based on factors like
stability, bioavailability, and patient preference.
✧ Regulatory Compliance:
✧ Meeting Standards: Ensuring that all stages of drug development comply with strict
regulatory standards set by agencies like the FDA (Food and Drug Administration) or EMA
(European Medicines Agency).
✧ Documentation and Reporting: Maintaining comprehensive documentation of all
development activities and submitting regulatory documents to demonstrate compliance with
guidelines and regulations.

8. Clinical Studies - Phase I
✧ Safety and Tolerability:
✧ First Human Tests: Phase I clinical trials are the initial stage of testing in humans
after successful preclinical studies. They focus primarily on evaluating the safety
and tolerability of the drug in humans.
✧ Monitoring Side Effects: Researchers closely monitor participants for any adverse
reactions or side effects to determine the safety profile of the drug.
✧ Small Group of Healthy Volunteers:
✧ Participant Selection: Phase I trials typically involve a small group of healthy
volunteers who do not have the condition being treated by the drug.
✧ Healthy Population: By testing in healthy individuals first, researchers can establish
baseline safety data and identify any potential safety concerns before testing in
patients with the target disease.

9. Clinical Studies - Phase II
✧ Efficacy and Side Effects:
✧ Testing on Patients: Phase II clinical trials involve administering the drug to patients
who have the target disease or condition to assess its efficacy.
✧ Outcome Measures: Researchers evaluate whether the drug produces the desired
therapeutic effects and identify any potential side effects or adverse reactions.
✧ Refine Dosage:
✧ Optimizing Treatment: Phase II trials also aim to determine the most effective dosage
of the drug with the least incidence of side effects.
✧ Dose-Finding Studies: Various doses of the drug may be tested to establish the
optimal balance between efficacy and safety.
✧ Data Analysis: Researchers analyze the results to refine the dosing regimen for
subsequent phases of clinical testing.

10. Clinical Studies - Phase III
✧ Confirm Effectiveness:
✧ Large-Scale Trials: PhaseIII clinical trials areconducted on a larger scalewith a moreextensivepatient
population.
✧ EnsuringEfficacy: Theprimary objectiveis to confirm theeffectiveness of thedrug in treating thetarget
diseaseor condition under real-world conditions.
✧ Statistical Significance: Researchers userigorous statistical methods to analyzedata and demonstrate
thedrug's efficacy compared to standard treatments or a placebo.
✧ MonitorAdverse Reactions:
✧ Long-Term Safety: PhaseIII trials also aim to monitor for any long-term or raresideeffects that may not
havebeen detected in earlier phases.
✧ Safety Profile: Researchers closely monitor participants for adversereactions throughout thetrial to
ensurethedrug's safety.
✧ Risk-Benefit Assessment: Data from PhaseIII trials help regulatory authorities and healthcare
professionals makeinformed decisions about thedrug's approval and use, considering its overall safety

11. Regulatory Submission and Approval
✧ New Drug Application (NDA):
✧ Comprehensive Submission: The NDA is a detailed dossier containing all preclinical, clinical, and manufacturing data related to the drug.
✧ Regulatory Authorities: It is submitted to regulatory agencies such as the FDA in the United States or the EMA in Europe for review and approval.
✧ Approval Process:
✧ Regulatory Review: Regulatory bodies carefully evaluate the NDA to ensure the drug's safety, efficacy, and quality meet regulatory standards.
✧ Clinical Data Evaluation: The review process includes a thorough examination of clinical trial data, manufacturing processes, labeling, and risk
management plans.
✧ Decision Making: Based on the review, regulatory authorities make a decision to approve, reject, or request additional information about the drug.
✧ Timeline:
✧ Submission: The timeline begins when the NDA is submitted to the regulatory agency.
✧ Review Period: The review process typically takes several months to a year, during which regulatory authorities assess the submitted data.
✧ Approval: If the drug meets regulatory requirements, it is granted approval for marketing and sale.
✧ Post-Approval Activities: After approval, post-marketing surveillance and additional studies may be required to monitor the drug's safety and efficacy
in real-world settings.

12. THANK YOU

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