American patients have access to the safest and most advanced healthcare and pharmaceutical system in the world.1 But, to be able to market a drug treatment for therapeutic use in the United States, U.S. Food and Drug Administration (FDA) approval is required. Other countries have similar regulators, but most fall short of the United States FDA standards. Before granting an approval, the FDA requires that sponsors like Personalized Stem Cells, Inc. conduct escalating stages of clinical trials to demonstrate the safety and efficacy of their drug.
In addition to clinical trial results, drug developers must also submit information about clinical pharmacology, toxicology, microbiology, chemistry, and manufacturing. Planned product labeling that provides information about the product on its final packaging is also required to assure that a complete profile of safety and efficacy information is communicated to health insurers, physicians, and the public. The bulk of a company’s drug development time involves the three stages of clinical testing before a submission.
Clinical trials are conducted by a trial sponsor, typically a biotechnology and pharmaceutical company. It is never the FDA. The FDA is solely responsible for advancing public health by helping to deliver the most efficacious, safe, and affordable drugs to the public, as patients require the most accurate, clinically relevant and science supported information when choosing medicines to maintain or improve their health. Human trials governed by the FDA are completely voluntary, and studies conducted in people are designed to answer a variety of scientific questions about the safety and effectiveness of a drug.
Because of their high standards, the regulatory approval of new drugs by the FDA is a long and complex process and often requires multiple cycles of review. There are three distinct phases to complete in the clinical trial process before a sponsor can submit their drug therapy to the FDA for consideration to be sold in the United States. Each phase has its own purpose to ensure that a treatment is safe and effective for use by the public. A Phase I study is performed in healthy volunteers or people with the disease or condition to help determine safety and dosage. A Phase II study is performed in a larger patient population of people with the disease or condition to look at efficacy and side effects. And a Phase III study is performed in an even larger patient population to determine efficacy and monitoring of adverse reactions.2
In summary, for the vast majority of new drug applications the FDA requires rigorous safety and efficacy evidence to decide which drugs will be allowed to be marketed in the United States. While their numerous review steps can and do delay patient access, this thoroughness plays an important role in the FDA’s charter to protect the nation’s patients by studying and measuring as many, if not all of the benefits and risks before any new drug is approved for sale.