Clinical Trials

Clinical TrialNew Drug Development Timeline

Source: FDA/Center for Drug Evaluation and Researchwww.fda.gov/Drugs/DevelopmentApprovalProcess/SmallBusinessAssistance/ucm053131.htm

Clinical TrialsResearch and Development

R&D involves initial synthesis and analysis of a promising pharmaceutical OR development and analysis of a biopharmaceutical produced in living cells.

On upcoming slides, the word “drug” applies both to pharmaceuticals and to biopharmaceuticals.

Clinical TrialsInvestigational New Drug

Application The application to the FDA to request permission to begin human testing is called an Investigational New Drug application or IND.

The IND permits the use of an investigational new drug for the sole purpose of conducting clinical trials.

Clinical TrialsPhase 1

Drug is tested for its interaction with the human system.

How is the drug absorbed How is the drug distributed in the body How is the drug metabolized by the body

Trials usually involve normal, healthy volunteers and take about a year to complete.

Clinical TrialsPhase 2

Pilot student to begin to define the effectiveness and safety of the drug in patients with the disease or condition to be treated, diagnosed or prevented.

Testing the various doses of the drug and dosing regimens

Clinical Trials

Phase 3 Expanded clinical trials Designed to …Gather additional evidence of effectiveness

for specific interactionsBetter understand safety and drug-related

adverse effects

Clinical TrialsPhase 4

Studies that occur after a drug has received approval from the U.S. Food and Drug Administration to be marketed

Performed to determine the incidence of adverse reactions

Determine the long-term effect of the drugTo study a patient population not previously

studiedFor marketing comparisons against other

products and users

Clinical TrialsTesting in Humans

For example, of 100 drugs for which investigational new drug applications are submitted to the FDA, about 70 percent will clear Phase 1 and go on to Phase 2. About 33 of the original 100 drugs will clear Phase 2 and go on to Phase 3. About 25 to 30 of the original 100 drugs will clear Phase 3 and go on to Phase 4. Finally, on average, 20 of the original 100 drugs ultimately will be approved for marketing. www.centerwatch.com/clinical-trials/overview.aspx

Clinical TrialsNew Drug Application (NDA)

Submitted to the FDA if Phase 1, 2 and 3 trials indicate the drug is safe and effective

Comprehensive statement with information about the drug’s chemical structure, scientific rationale and purpose of the drug therapy, preclinical and other laboratory results, all human clinical testing data, drug formulation and production details, and proposed labeling

1000s of pages…which are submitted electronically.

FDA has 60 to conduct a preliminary review and decide if it has enough information to proceed with the NDA review

FDA required to make a decision within 180 days of the date the NDA is submitted

FDA and company can create a mutual agreement to lengthen the time frame

NDA decision-making process can take anywhere from 2 months to 7 years — the average time is 2 years

May require minor or major changes of the NDA or additional clinical studies and may inspect the production, testing and packaging facilities to ensure they are compliant with regulations

Clinical TrialsNew Drug Application (NDA)

Clinical TrialsPost-marketing surveillance

Monitor the ongoing safety of marketed drugs by reassessing drug risk based on …New data collected after the drug is marketed By recommending ways of trying to most

appropriately manage that risk Includes adverse reaction reporting by the medical

community of the pharmaceutical company that markets the drugs

Periodic sampling and testing of the drug Periodic inspections of the manufacturing and

distribution process