2 - Drug Approval Process (Part 1) Flashcards Preview

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Flashcards in 2 - Drug Approval Process (Part 1) Deck (18):

Describe the "drug discovery to market" portion of drug development process

-Molecule discovery
-Pre-clinical trials
-Clinical trials (phase 1, 2, 3)
-Therapeutic Products Directorate (TPD)
-Notice of Compliance (NOC, NOC/C)


Describe the "drug pricing and access" portion of drug development process

-CADTH Common Drug Review (CDR)
-Phase 4 trials, ADR Surveillance
-Formulary Decision (provincial, hospital)


What is involved in Pre-Clinical Trials ?

-physicochemical characterization, impurity analysis, formulation, stability testing

Efficacy screening:
-in vitro, in vivo models

ADME profiling

-Cytotoxicity, hepatotoxicity, CV safety pharmacology, maximum tolerated dose, etc.


Describe a Phase 1 Clinical Trial

-Primarily to establish safe dosing range in humans
-Pharmacologic profiles
-Normal, healthy volunteers (n = 20-100)
-1 year


Describe a Phase 2 Clinical Trial

-Pilot efficacy trials in small population with the indicated condition
-Select optimal dose, observe for AEs
-Volunteer patients (n = 100-150)
-2-3 years


Describe a Phase 3 Clinical Trial

-Extensive clinical trials in large populations with the indicated condition (n = hundreds to thousands)
-Goal to verify efficacy of the drug by comparing the new drug to a control group (placebo or comparator) - demonstrate magnitude of benefit is not due to chance
-1-5+ years depending on indications
-Also intended to establish safety in target patient population

-Sample size of phase 3 trials are designed to have enough power to detect difference in the clinical outcomes of interest, NOT adverse events
-Severe, life-threatening ADEs (ex. Steven Johnson's, QT prolongation) occur far more less frequently than the clinical outcomes


Know difference between type 1 and 2 errors man



Just because it's gone through phase 3 trials doesn't mean it's ____. This is where we need phase 4 (surveillance of real-world effectiveness)



Describe phase 4 clinical trials

-Post-marketing trials of effectiveness and safety
-Surveillance of real-world effectiveness and long-term safety
-New indications for use
-Special disease-state and populations (ex. renal impairment, geriatric and paediatric populations, genetic subtypes)
-Drug interaction studies
-Pharmacoeconomic studies


Describe Efficacy vs. Effectiveness

-the actual therapeutic benefit that a drug has (controlled environment)
-frequency contact with clinicians
-100% compliance

Effectiveness: In real world (not controlled)
-Each patient has other factors that may contribute


Describe the Therapeutic Products Directorate (TPD)

The national authority in Canada that regulates, evaluates and monitors the safety, efficacy and quality of therapeutic and diagnostic products available to Canadians

In the drug licensing process, TPD has regulatory authority in the following 3 stages:
-Clinical trial authorization
-Drug submission reviews


How is TPD applied to conduct a clinical trial

-informed consent phase 1
-good clinical practices
-primary safety and dosing range in humans
-controlled environment - administration and evaluation monitored
-drug limited to clinical investigators named on trial



Notice of compliance


Describe NOC (Notice of compliance)

-Permits the sponsor to market drug
-Indicates the drug's official approval in Canada



Drug product database


Describe Timeliness of drug licensing process in Canada

remains a criticism - numerous steps taken to improve access to drugs for patients


Describe the Priority Review Process

-Faster review possible for promising drug products for life threatening/severely debilitating illnesses (i.e. cancer, AIDS, parkinson's)
-Effective treatment, of a disease or condition for which no drug is presently marketed in Canada
-OR, a significant increase in efficacy and/or significant decrease in risk such that the overall benefit/risk profile is improved over existing therapies for a disease or condition that is not adequately managed by a drug marketed in Canada


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