Clinical Studies

During this phase, the results of the preclinical tests are submitted to the monitoring agency (i.e., FDA) for review before the drug can be tested in humans. An investigational new drug (IND) application is filed and must be approved before any human testing begins. Approximately one in five new IND request makes it to the marketplace.

Phase 1: Safety studies. This phase begins trials in humans that test a compound for safety, tolerance, and pharmacokinetics. The Phase 1 trials usually employ normal (i.e., healthy) volunteers and may expose up to 50 individuals to the drug. For most therapeutic biologics and any known toxic compounds, such as anti-cancer agents, only patients with the targeted illness would be used.

Phase 2: Efficacy studies. These are the first studies (usually) to define efficacy. In general, 100 to 300 patients enter into various closely monitored clinical trials during this phase. Dose and dosing regimens are assessed for magnitude and duration of effect during this phase. Some companies further differentiate this phase into Phase 2A for studies designed to set dosing and Phase 2B for studies designed to determine efficacy.

Phase 3: Extensive clinical testing. These are expanded controlled and uncontrolled clinical trials intended to gather additional evidence of effectiveness for specific indications and to better understand safety and drug-related adverse effects. Phase 3 trials are usually large multicenter trials, which collect substantial safety experience and may include specialized studies needed for labeling (e.g., pediatric or elderly, comparative agents). Phase 3 trials normally include between 1,000 and 3,000 patients.

Phase 4: Postapproval studies. This phase is activated or requested only by the FDA when a drug is approved for marketing. There are many reasons for performing Phase 4 studies, including:

To elucidate the incidence of adverse reactions.

Large scale, long-term studies to determine the effect of a drug on morbidity or mortality.

To study a patient population not previously studied (e.g., children).

Marketing-oriented comparison studies against competitor products.

Project Management Made Easy

Project Management Made Easy

What you need to know about… Project Management Made Easy! Project management consists of more than just a large building project and can encompass small projects as well. No matter what the size of your project, you need to have some sort of project management. How you manage your project has everything to do with its outcome.

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