PCL 201 Stages in the Development of a New Drug Outline

Clinical Trials

• A protocol is carefully designed to balance the potential benefits and risks to participants, and answer specific research questions.
• A protocol describes the goal of the study, who is eligible to take part, protections against risks, details about tests and procedures, and how long the trial is expected to last.

Informed Consent

• Informed consent is a process of providing key information about a research study before deciding to participate.
• The research team explains the study details, provides an Informed Consent document, and explains risks and potential benefits.
• Participants can decide whether to sign the document and can leave the study at any time.

Phases of Clinical Trials

• Phase 0: A small study with fewer than 15 people to test a very small dose of medication.
• Phase III: A large-scale study with 1000-3000 people to confirm the drug's effectiveness, monitor side effects, and compare it with standard treatments.

New Drug Application (NDA)

• A New Drug Application (NDA) expresses the full story of a drug molecule, verifying its safety and effectiveness for its proposed use.
• The developer must include all data, reports, and analysis, as well as proposed labeling, safety updates, and drug abuse information.

FDA Review

• The FDA reviews the NDA to decide if it is complete and acceptable.
• If the NDA is incomplete, the FDA can refuse the application.

Pharmaceutical Formulation and Development

• Pharmaceutical formulation is a stage of drug development that characterizes the physicochemical properties of Active Pharmaceutical Ingredients (APIs) to produce a bioavailable, stable, and optimal dosage form.

Preclinical Research

• Preclinical research is conducted on animal models to determine whether a drug has the potential to cause serious harm to humans.
• The two types of preclinical research are in vitro and in vivo experiments.

Investigational New Drug Application (INDA)

• An INDA is a request for authorization from the FDA to administer an investigational drug or biological product to humans.
• If the INDA submission is accepted, the product is further forwarded to clinical research studies.

Clinical Research

• Clinical trials are research studies that test new medical treatments or new ways of using existing treatments to see if they will be beneficial or detrimental to humans.
• Clinical trial phases are steps in the research to determine if an intervention would be beneficial or detrimental to humans.

Types of Clinical Trials

• Treatment trials: Test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
• Prevention trials: Look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning.
• Diagnostic trials: Conducted to find better tests or procedures for diagnosing a particular disease or condition.
• Screening trials: Test the best way to detect certain diseases or health conditions.
• Quality of life trials: Explore ways to improve comfort and the quality of life of individuals with chronic illness.

Clinical Trial Protocols

• A clinical trial follows a plan known as a protocol, which describes the goal of the study, who is eligible to take part, and protections against risks.

Stages of Drug Development

• The stages of drug development include target identification, target validation, lead identification, lead optimization, product characterization, formulation and development, preclinical research, INDA, clinical trials, NDA, FDA review, and FDA approval.