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Pharmaceutical Development and Review Process


Title: Pharmaceutical Development and Review Process Author: Alvero, Ruben Last modified by: Michelle Kobryn Created Date: 5/4/2014 3:50:37 PM Document presentation ... – PowerPoint PPT presentation

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Title: Pharmaceutical Development and Review Process

Pharmaceutical Development and Review Process
APGO Interaction with Industry A Medical
Student Guide
Rev. 10/21/2014
  • Learn the processes involved in drug discovery
    and development in the United States
  • Understand the role of the Food and Drug
    Administration (FDA) in the drug development and
    review process in the US
  • Define the phases involved in FDA drug approval

Overview-Research and Development Process (RD)
  • Development of new drugs is a complex and costly
  • It takes an average of 9 years and 850 million
    to take a chemical entity from the lab to the
    pharmacy shelf
  • RD involves discovery, preclinical studies and
  • One out of every 1,000 compounds that begin
    preclinical studies will ultimately be marketed

Brief Flow Diagram of the Drug Approval Process
A History of Federal Oversight of the Drug
Development Process
  • The Food and Drug Administration (FDA) is the
    Federal agency that is required by law to review
    and approve all new drugs in the US.
  • The 1906 Food and Drugs Act prohibited
    mislabeling but did not require pre-market
    approval of drugs.
  • The 1938 Food, Drugs and Cosmetics Act required
    submission of evidence of a drugs safety before
    it could be marketed.

The Role of the FDA
  • The FDA reviews and evaluates new drugs based on
    evidence presented from the clinical research
    studies performed by the drug sponsor-typically a
    pharmaceutical company
  • The Center for Drug Evaluation and Research
    (CDER) is the largest of the FDAs five centers
    and is responsible for prescription and
    over-the-counter (OTC) drug safety and efficacy

Preclinical Studies
  • Synthesis and purification of the new drug
  • Pharmacology of the new drug
  • Pharmacokinetics absorption, distribution,
    metabolism, excretion, half-life
  • Pharmacodynamics mechanism of action and
    estimates of therapeutic effects
  • Toxicology including carcinogenicity,
    mutagenicity and teratogenicity
  • Safety studies on animals

Definitions-Investigational New Drug (IND)
  • New drug applications (NDAs) require clinical
    trials using the candidate chemical compound for
    safety and efficacy, usually in centers in
    multiple states
  • Federal law requires that a drug be the subject
    of a marketing application before transporting
    across state lines
  • The IND is the process by which an exemption to
    the law is obtained
  • Studies in humans can only begin after IND is
    reviewed and approved by the FDA and an
    institutional review board (IRB)

Overview of Clinical Study Phases
  • Phase 1 Safety and tolerability studies on
    healthy volunteers
  • Phase 2 Clinical studies to demonstrate proof of
    concept and dose findings
  • Phase 3 Efficacy and safety studies on large
    number of subjects
  • NDA regulatory review
  • Phase 4 Post-marketing safety studies

Phase 1 Clinical Studies
  • Typically 20-80 healthy volunteers
  • Emphasis on drug safety
  • Identify major side-effects, metabolism, routes
    of excretion
  • Duration about 1 year
  • Sufficient information about pharmacokinetics and
    effects to permit design of well-controlled Phase
    2 studies
  • About 70 that make it to this phase will pass

Phase 2 Clinical Studies
  • Typically involves 100-300 individuals who have
    the target disease
  • Emphasis on effectiveness
  • Closely monitored, can evaluate short-term
    side-effects and risks
  • Patients receiving the drug are compared to
    similar patients receiving a placebo or another
  • Duration about 2 years
  • About 33 of drugs will pass this phase

Phase 3 Clinical Studies
  • Typically involves 1,000-3,000 patients
  • Emphasis on safety and effectiveness
  • Investigates different populations and dosages as
    well as combination with other drugs
  • Extrapolation to a general population
  • Acquire data used for physician labeling
  • Duration about 3 years
  • 25-30 pass this phase

The Role of the Institutional Review Board (IRB)
  • IRBs ensure the rights and welfare of people
    participating in clinical trials, both before and
    during trial participation
  • IRBs make sure that participants are fully
    informed and have given written consent before
    participating in studies
  • IRBs are located in hospitals and research centers

Definition-New Drug Application (NDA)
  • Starting with the 1938 and subsequent
    (increasingly demanding) amendments, all the data
    gathered during the animal studies and human
    clinical trials of an Investigational New Drug
    (IND) are required to become part of the NDA
  • While varying by type of compound, an NDA can
    consist of as many as 15 different sections

NDA (contd)
  • Pre-NDA period FDA and drug sponsors meet
  • Submission of NDA Formal step asking FDA to
    consider approving a drug for marketing
  • FDA has 1 year to decide whether it will file the
    NDA for approval consideration
  • If filed, a review team is assigned to evaluation
    the new drug

FDA Approval
  • FDA review team evaluates the research on safety
    and effectiveness
  • Labeling information reviewed
  • Inspection of production facilities
  • Decision and justification letter
  • Not approvable
  • Approvable
  • Approval

FDA Approval (contd)
  • Not approvable Lists deficiencies in application
    and why it cannot be approved
  • Approvable Ultimately can be approved and lists
    deficiencies that can be corrected, including
    labeling changes and requests for post-approval
  • Approval Drug is approved

Phase 4 Clinical Studies
  • Post-market surveillance of the drug to
    continually assess safety of the drug
  • May include incidence and severity of rare
    adverse reactions, cost-effectiveness analyses,
    comparative trials, and quality of life studies

/ucm289601.htm Accessed 7/9/2014
/ucm289601.htm Accessed 7/9/2014
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