mod 5

On average, how much does it cost to make a new drug?

$1.3 billion

How many phases are there for human testing?

4 phases

What is the gold standard for testing?

RCTs (Randomized controlled trials)

What is the first round of testing?

preclinical trials

What are pre-clinical trials?

Medicine/drug trials done on animals, cells or tissues

What is determined during pre-clinical trials?

cancer causing, gene mutations, or fertility issues

What section of the drug label includes nonclinical toxicology information?

13.1

What comes after pre-clinical trials?

IND (investigational new drug application)

Where does the IND go?

to FDA's Center for Drug Evaluation and Research (CDER)

What does the IND include?

results of pre-clinical testing and proposal for human testing

What is the goal of CDER?

to protect volunteers who participate in clinical trials from unreasonable and significant risk

What are the two possibilities of the result of CDER review?

-FDA indicates proposed clinical studies are safe to proceed

-FDA issuance of "clinical hold" - no clinical studies can be conducted

What is phase 1 of clinical trials?

the first time humans take the drug

How many people are normally in phase 1 of clinical trials?

20-100

In Phase 1, are the volunteers usually healthy or not healthy?

healthy

What are the three things we're looking for in phase 1 of clinical trials?

-is the drug safe?

-what are the side effects?

-How does the drug work in the body?

What are pharmacodynamics?

what a drug does to the body

What is pharmacokinetics?

what the body does to a drug

What percent of drugs move on to phase 2?

around 60%

How many volunteers enter phase 2 of clinical trials?

100-500

What is the purpose of phase 2?

-is the drug safe?

-how effective is the drug?

-how much drug should be taken, how often?

What is the main purpose of phase 2?

to test effectiveness

What is an indication?

-for the treatment, prevention, mitigation, cure, or diagnosis

of a disease or condition in age group

How many drugs make it to phase 3 of clinical trials?

around 60%

What are the goals of phase 3 of clinical trials?

-is the drug safe?

-how effective is the drug?

-is there a benefit or risk to a specific population?

What is the most expensive phase of clinical trials?

phase 3

What is the biggest goal of phase 3?

asking how it compares to another drug

What happens after phase 3 of clinical trials?

NDA or BLA

What does the manufacturer submit after phase 3?

-new drug application (NDA) for conventional/chemical drugs

-Biologics license Application (BLA) for therapeutic biologics

What does CDER do?

-review all human and animal data, including adverse effects that occurred during clinical trials

-review drug labeling-- some labeling is intended for HCPs and some for patients

What is phase 4 of clinical trials?

post marketing surveillance

What are the objectives of phase 4 studies?

study the drug's safety and effectiveness in real life scenarios to study long-term risks and benefits

-discover any side effects that did not show up in clinical trials

How does a new indication for approval come about?

the manufacturer must submit a supplemental new drug application (sNDA)

What phases need to be conducted for the approval of a new indication?

phase 2 and phase 3

Is a prescriber acting within the law to write for a use that is not an indication?

yes, as long as there is reasonable scientific evidence that the drug is effective for that use