The 5 Phases of Drug Development | Rondaxe CMC Consulting

Phases of Drug Development


Drug development has become exponentially more difficult over the last 40 years; it now requires preclinical studies, investigational new drug (IND) filings, and full clinical testing before receiving FDA marketing clearance. In general, new drug applications (NDAs) and biologics license applications (BLAs) are thoroughly evaluated before approval and then resubmitted to regulatory authorities for post-marketing research. The ultimate objective is to provide patients with more effective and safer therapies as quickly as possible after a complete medical review.


There are 5 essential milestones (phases) in the drug development process in the United States, each having stages within it. We will explore each of these phases and stages in detail to fully grasp the whole process.

Phase 1: Discovery and Development

New drugs are discovered through drug discovery. Since the sequencing of human DNA was implemented, reverse pharmacology has identified cures for existing ailments. Researchers then searched chemical libraries for small medicinal compounds, natural products or plant extracts.


Today's drug development incorporates hit screening, medicinal chemistry and hit optimization to limit adverse effects (increasing affinity and selectivity). This phase improves efficacy, potency, metabolic stability (half-life) and oral bioavailability.

Phase 2: Preclinical Research

Preclinical research determines the drug's effectiveness and safety after a lead chemical has been identified. The drug's researchers discover:


  • Information on absorption, distribution, metabolization and excretion

  • Benefits which may accrue and respective modes of action

  • The optimal dose and mode of delivery

  • Consequences and adverse events

  • Effects on certain racial, ethnic or gender groupings

  • Interactions with other therapies

  • Effectiveness in comparison to peer group medications


Preclinical studies examine the drug's effectiveness, toxicity and pharmacokinetics in non-human individuals. Scientists undertake unlimited dose studies in vitro and in vivo.

Phase 3: Clinical Research and Development

After preclinical research, researchers proceed to clinical medication development including clinical trials and volunteer studies.

Study Design Complexity, Cost and Implementation Issues

Trials must be safe, effective and conducted within the drug development budget. To be successful, this arduous approach requires a large number of volunteers. Contextual concerns and financial considerations can impact trials conducted during this period.

Phase 4: FDA Inspection

Upon the completion of clinical trials, the new medicine is submitted to the FDA for a comprehensive examination. The FDA now evaluates and approves or denies the drug developer's drug application.

Regulatory Approval Timeline

Depending on the application and patient need, a new drug's regulatory approval timeframe may be normal, breakthrough, expedited or priority. A year may be necessary if a standard review is applied; a breakthrough or expedited approval is quicker.

Phase 5: FDA Post-Market Safety Monitoring

The FDA mandates drug manufacturers to monitor medication safety using the FDA Adverse Event Reporting System (FAERS). Manufacturers, doctors, and patients use this service to report concerns with licensed pharmaceuticals.


Drug Development Concepts

Other Relevant Drug Development Concepts

Drug Master File

This contains private information on facilities, techniques or commodities used in producing, processing, packing and storing human drugs.

Drugs for Pediatric Use

Pediatric drugs are indicated for use in children and adolescents under 21. The AMA may make exceptions if a doctor and family agree on their use by an older adult.


Drugs for Veterinary Use

Veterinary drugs are meant for animals, pets and cattle. However, several veterinary medications are developed before becoming human or animal pharmaceuticals.

Small Molecule vs. Biologics

Small molecules have several biological activities. Large molecules (biologics) are therapeutic proteins. Small molecules signal cells; large molecular pharmaceuticals have around 1,300 amino acids.

Process Scale-up Differences and Issues

Changing procedures for various batch sizes can bring unanticipated challenges. Using the correct pharmaceutical equipment and identifying important process parameters can assist (CPPs).


Medication development aims to identify and supply new treatments which can enhance our health and well-being. New medication development is a highly regulated, complex procedure requiring highly skilled medical researchers. Human and animal clinical trial participants must be treated with the highest care.


To learn more about the drug development process, visit Rondaxe online or phone us in New York at (315) 469-2800 today.



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