The Research Process

Question 1

Drug Research & Discovery

Answer 1

TYPICALLY RESEARCHERS DISCOVER NEW DRUG THROUGH:

. New info into a disease process that allow researchers to design a product to stop or reverse the effects of the disease.

. Many test of find beneficial effects against large number of diseases.

. Existing treatments that have unanticipated effects

. New technologies that provide new ways to target medical products to specific site w/in the body or to manipulate genetic material

ONCE A PROMISING COMPOUND IS IDENTIFIED, THEY CONDUCT EXPERIMENTS TO GET INFO ON

. How it’s absorbed, distributed, metabolize, and excreted

. It’s potential benefits and process of action

. Best dosage

. The best way to give the drug (mouth/injection)

. Side effect or AE that can be referred to as toxicity

. How it affects different groups of people (gender, race, or ethnicity) differently

. How it interacts with other drugs and treatments

. It’s effectiveness as compared w/similar drugs

RESEARCH USUALLY INVOLVES YEARS OF EXPERIENCE IN ANIMALS AND HUMAN CELLS

. In Vitro - biological properties done in a test tube
. In Vivo - living organisms, animals, humans, etc

Question 2

Pre-clinical Research

Answer 2

. Before testing in humans, extensive pre-clinical or lab research is required.

. If this stage of testing is successful, the Sponsor then provides this data to the FDA requesting approval to begin testing in humans. This is called an Investigational New Drug (IND) application

. If approved by the FDA, testing in humans begins. This is done through a formally written and approval PROTOCOL.

Question 3

The Investigational New Drug Process

IND

Answer 3

. Drug developers, or sponsors, must submit an IND app to FDA before beginning clinical research.

. Animal study data and toxicity (side effects that cause great harm) data

. Manufacturing info

. Clinical protocols (study plan) for studies to be conducted

. Data from any prior human research

. Info about the investigator

Question 4

FDA Approval

Answer 4

The FDA review team has 30 days to review the original IND submission a s respond to IND app in 1 or 2 ways:

. Approval to begin clinic trials.

. Clinical hold to delay or stop the investigation. FDA can place a clinical hold for specific reasons, including:

. Subjects are exposed to unreasonable or huge risk
. Investigators are not qualified.
. Material for volunteers are misleading.
. IND doesn’t include enough info about trial’s risks.

Question 5

Clinical Trial Design

Answer 5

. Before a Clinical Trial begins, researchers review prior info about the drug to develop research questions and objectives.

. Then they decide

. Who qualifies to participate (selection in/exclusion criteria)
. How many people will be in the study
. How long it will last
. Will there be a control group or other ways to limit bias
. How the drug will be given to the patients and at what dosage
. What assessments will be conducted, when, and what data will be collected
. How the data will be reviewed and analyze