New Drug Development Flashcards

1
Q

What is a drug product?

A

Drug (components)

Drug products: systems of mixtures: API and Excipients

(Basic) Dosage Forms

(Advanced) Drug Delivery Systems

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2
Q

What are Basic Dosage Forms?

A

Liquid Dosage Forms:

Solutions

Collodial Dispersions

Coarse Dispersions (Suspensions and emulsions)

Semisolids

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3
Q

What is the definition of a drug?

A

A drug: articles intended for use in the diagnosis, cure, mitigation, treatment or prevention of disease in man or other animals (CFR)

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4
Q

What is “new” drug?

A

A new drug is not recognized among qualified experts

A new drug can be a

  • New chemical entitiy (NCE)
  • New Formulation
  • New combination
  • New proposed used for an established drug
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5
Q

FDA Drug Classification System

A

Type 1: New molecular entity; not marketed in US

Type 2: New ester, salt, or other derivatives of an approved active moiety

Type 3: New Formulation of a drug marketed in US

Type 4: New combination of two or more compounds

Type 5: New Manufacturer of drug marketed in US

Type 6: New therapeutic indication for an approved

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6
Q

How is a new drug named?

A
  • Empirical formula and systematic chemical name
  • Code name by the inventor
  • Nonproprietary (generic) name: Aspirin
  • Proprietary name
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7
Q

What is the roadmap of NDDDA?

A
  1. Drug Discovery (& design)
  2. Drug Development
    1. Preclinical studies: preformulation & formulation, animal testing, process development
  3. Investigational New Drug Application (IND)
    1. Phase 1
    2. Phase II (a,b)
    3. Phase III
  4. New Drug Application (NDA)
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8
Q

What are the preformulation studies?

A

Preformulation studies

  • Chemical characterization structure MW
  • Physical characterization
    • pKa, pH and ionic properties
    • Drug solubility
    • Partition coefficient
    • Rheology and/or powder properties (micrometrics)
    • Physical form -polymorphism
  • Stability (chemically, physical and biological)
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9
Q

IND application Submission

A
  • Application for the first human clinical trial
  • General Content of the
    • Contact information
    • Investigation plan
    • Chemistry Manufacture and Control (CMC) information
    • Pharmacology and toxicology information
    • Clinical protocol
    • Institutional review board (IRB) approval
    • Investigator brochure
  • The FDA must reply in 30 days
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10
Q

What are the three phases of clinical investigations?

A

Phase I

  • 20~100
  • First in man
  • Safety
  • tolerability
  • ADME (pharmacokinetics, PK)

Phase II

  • ~hundreds
  • Controlled
  • Effectiveness: proof of concept
  • Dose ranging and determination
  • Safety
  • Additional PK

Phase III

  • Hundreds to thousands
  • Usually controlled
  • Usually replicated
  • Primary data to support marketing approval in FDA
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11
Q

What is the clinical trial design?

A
  • Controlled Trial - investigational drug is compared to another agent
    • Placebo controlled
    • Positive controlled - investigational drug is compared to another active drug or comparative agent (comparator)
  • Blinded studies - identities of IND and placebo/other agent are not revealed to certain participants
    • Main advantage = decreases bias
    • Single blind study - patient unaware of administered agent
    • Double blind - both patient and clinician are unaware of administered agent
  • Parallel and crossover
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12
Q

What is involved in New Drug application (NDA)

A
  • General Content of the NDA submission
    • Product information: name, DF, strength
    • Statement and summary
    • CMC
    • Non-clincal pharmacology and toxicology
    • Human Pharmacokinetics and bioavailability
    • Clinical data
    • IRB compliance
    • Common Technical Document Format
  • Drug Product Labeling
  • FDA review And Action letters
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13
Q

What is the formulation development during the process

A
  • Preformulation study: Physical and chemical characterization
  • Initial formulation with the considerations of: preformuation data, proper dose, proper dosage form and route of administration
  • Formulation during the clinical trial
    • Phase 1: simple formulations with multiple strength (e.g. sapsule)
    • Phase 2: developed formulations for finalizing
    • Phase 3: final formulation is determined and used
  • Control materials: placebo and comparator products
  • cGMP (current good manufacture practice) quality
  • Formulation design
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14
Q

What is the drug product labeling?

A
  • Description
  • Clinical pharmacology
  • Indication and usage
  • Contra-indications
  • Warnings
  • Precautions
  • Adverse reactions
  • Drug abuse and dependences
  • Over-dosage
  • Dosage administration
  • How supplied
  • For packaging label, container label, package inserts and others (company literature, promotion materials)
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15
Q

What are the other ND application routes?

A
  • Supplement New Drug Application (SNDA)
    • The sponsor want to make a change on an approved NDA
  • Abbreviated New Drug Application (ANDA)
    • Generic copies
  • Biologics license application (BLA)
    • biological products
  • New Animal Drug Appliaction (NADA)
  • Medical Device
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16
Q

What are the “Phase 4” studies and Post marketing surveillance?

A

“Phase 4” - controlled clinical investigations which help contribute to the understanding of a drugs mechanism/scope of action

Help indicate:

  • New therapeutic uses
  • Need for additional strengths, dosage forms, ROA’s
  • Additional side effects, serious reactions, and drug interactions
17
Q

What is regulatory Science?

A

An emerging field & new FDA initiative

It is the science of the assemessment and evaluation of safety, effectiveness, potency, quality and performance of a product

To develop new mehtods, assays, standards and models that will help development, review and approval of medical products

18
Q
A