New Drug Design and Approval Flashcards
About how long does it take to bring a drug to market
15-20 years
Process of discover
- Can come from dicovery of a new physiologic pathway
- Can come from newly discovered molecular compounds that are related to or developed from existing drugs
- Can be from study of existing treatments and observation and anticipated side effects at could potentially have therapeutic applications in a different disease
- Can be from development of new technologies not previously in existence.
Process of development
Once a compound has been identified for development, experiments must be conducted to better characterize the compound and determine the following:
Mechanisms of absorption, distribution, metabolism, and excretion
Potential benefits and mechanisms of action
Optimal dosage
Optimal route of administration
Side effects, toxicity, and potential drug interactions
Affects in specific populations
Effectiveness compared to similar drugs or drugs for the same disorder
What is LD50
This is the amount of the drug that is required to kill half of the animals in the test group
What is the therapeutic index
The difference between the dosage that produces a therapeutic effet and the dosage that produces a toxic effect
What is the first step of the clinical research phase
Investigational New Drug process
In the IND application drug developers must include:
Animal study data and toxicity
Manufacturing information
Clinical protocols a.k.a. study plans for phase 1-3 studies
Data from any prior human research
Information about the investigator’s credentials
Two outcomes of IND process
Approval to begin clinical trials, OR
Clinical hold to delay or stop the investigation. The FDA can place a clinical hold for specific reasons including:
Participants are exposed to unreasonable or significant risk
Investigators are not qualified
Materials for the volunteer participants are misleading
The application does not include enough information about the trials risks
A clinical hold is RARE
What goes into study design
Who qualifies to participate
How many people will be part of the study
How long the city will last
Whether there will be a control group and other ways to limit research bias
How the drug will be given to patients and at what dosage
What assessments will be conducted and what data will be collected
How the data will be reviewed and analyzed
What is a phase 1 trial
the purpose of phase 1 studies is to establish the safety of various dosing levels in humans.
In most cases, 20 to 80 healthy volunteers or people with the disease/condition participate in Phase 1, emphasize safety
What is phase 2 trial
The purpose of phase 2 studies is to establish safety and side effects with prolonged use, over the period of time that would be typical for treatment of the disorder of interest.
What is a phase 3 trial
phase 3 studies are the first study phase that attempts to demonstrate whether or not a new drug offers a treatment benefit to a specific population of patients. These are sometimes also described as “pivotal studies.”
What is the NDA
New Drug Application
An NDA includes all animal and human data and analyses of the data as well as information about how the drug behaves in and how it is manufactured. This differs from the IND, which was the application to the FDA to receive approval to begin trials in humans.
What is a phase 4 trial
Phase 4 trials are an important component of drug monitoring, in addition to the FDA requirement that the drug manufacturer submit periodic safety updates. Phase 4 trials are carried out in several thousand volunteers who have the disease or condition of interest. If you’ve ever been to a doctor’s office or hospital and then asked if you wanted to be enrolled in a study of a drug, that study was a Phase 4 study. Phase 4 trials are only carried out once the drug or device has been approved by the FDA during the post market safety monitoring. Any new safety concerns that come up in Phase 4 trials must be reported to the FDA.
What is the Accelerated Approval program
specifically for drugs that meet an unmet medical need or treat a fatal or devastating disease.
The changes to the process allow for manufacturers to submit the application in parts as soon as they have sufficient experimental information becomes available aka “Rolling submission”
What is the Prescription Drug User Fee Act
This legislation, passed in 1992, allows companies to pay extra money that is used to increase the FDA resources applied to review of a specific drug. In return for the increased FDA resources, the FDA agrees to quicker time frames for the FDA side of the review and approval timeline.
Drug Review Steps Simplified (13)
