Clinical Trials

Testing is multi-phased, tightly controlled, and may involve a large number of patients, and each phase can take several months to several years for completion. Trials are designed to assess the safety and effectiveness of a new product.

• Phase I is made up of short-term tests on a limited population of test subjects, generally about 20-50 healthy volunteers. The purpose of these trials is to evaluate the safety and dosages still further than was managed in the pre-clinical tests, and to assess the pharmacological and toxicological effect of the compound in humans.
   
• Phase II involves still relatively small-scale tests, though they are longer in duration and involve a slightly larger test population, generally around 100-300 volunteers. What the R&D teams are looking for in this phase are things like optimal dosage levels, effectiveness, and potential side effects.
   
• Phase III has the largest test population, typically 1000-3000 volunteers. In this phase, the effectiveness and side effects of longer-term use across a larger user base is studied. It is the results of this phase that will deliver the information used by the FDA for approval.

Like the preclinical testing, the clinical trials must also be conducted in controlled situations. The final results from the testing period must be analyzed in the same way and under the same conditions as the first results even if they occur years apart. It is the engineer who will ensure the facilities and equipment work consistently and reliably and that the compounds produced are consistent.

Once the sponsor has conducted sufficient trials to demonstrate the safety and efficacy of the drug, they submit that information—along with information on manufacturing specifications, drug stability and bioavailability, and suggested packaging and labeling—as a "new drug application" (NDA) to the FDA.