Atrium Health Navicent Infectious Disease

FDA-Approved Clinical Trials

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The U.S. FDA's Interest in Clinical Research

The seed for the Federal Drug Administration and its consumer protection mandate was planted in the 1848 U.S. Patent Office, Agricultural Division, but the FDA did not become overtly involved with consumer protection until 1906 with the passage of the Pure Food and Drugs Act. Harvey Washington Wiley, Chief Chemist of the Bureau of Chemistry (U.S. Department of Agriculture) was an advocate for consumer protection at the time and his Chief Chemist position morphed into the Commissioner of Food and Drugs, hence the clear line of FDA development.

Mandate to Safeguard the Public

The 1938 U.S. Food, Drug, and Cosmetic Act gave the FDA responsibility for evaluation of drugs for safety before marketing using pre-clinical and clinical test results. Drug Amendments in 1962 stipulated that new drugs must be safe and efficacious. In 2007 criteria to assure study transparency further delineated FDA responsibilities in Section 801 of the Food and Drug Administration Amendments Act. From these roots a century and a half ago, the FDA has developed a study protocol to safeguard the introduction of any new drug, biologic, device, or experimental protocol into the medical treatment marketplace.

Defining Clinical Research

Computer simulations and modern projections of drug efficacy are technically, not researched. To know how medications or devices really work in human beings, there needs to be human experimentation. This is why clinical research is required. Research (aka studies, trials, etc.) requires an evaluation of the medications or devices in people using an investigational process. That process is delineated in a study protocol developed by the researcher or manufacturer specifying subjects, study period, control groups, dosage and delivery method, assessment of results being studied, etc. The research process is phased from early studies (aka Phase 1) to late studies (aka Phase 3).

Study Size

There has been a move towards the use of large study groups in order to tease out clinical findings that would be obscured if smaller groups were used. The FDA has also been proactive in encouraging diversity in study groups, e.g., using age, sex, ethnicity, etc. as reasons for inclusion in study groups. Such diversity adds extra evaluation data affecting all demographics in the health care population. In addition, the agency has expedited its review process to cut development times for new products. For example, such protocol management has reduced total study time for "Investigational Device Exemption" by 75% in recent years.

Clinical Trials

Recognition and registration of FDA clinical studies begin with animal studies to rule out any suspicious toxic issues in a new medicine in the early phases. Later "FDA-approved clinical trials" commence, utilizing human test subjects to evaluate treatment strategies. Drugs, medical devices, or treatment regimens (use of biologics, vaccines, gene modifications, blood products, etc.) to heal or mitigate disease processes are all evaluated with the clinical trial methodology. New products cannot be introduced to the U.S. public without these FDA-approved clinical trials or studies aimed at both discovery of safety and efficacy in new product development. Formal recognition identifies the FDA's oversight of the ongoing study.

Testing Safety

Human subjects participate in trials only with full understanding of risks inherent in FDA-approved clinical trials. The sponsor of the clinical trial or the trial's principal investigator must officially register trials. Either must meet requirements spelled out in Section 801 of the Food and Drug Administration Amendments Act (aka FDAAA 801) which detail specifics and mandates study transparency. Studies that do not investigate drugs, biologics, or devices do not require registration.

Generic Drug Clinical Trials

Innovation drives development and is dependent on clinical trials. The FDA has developed a priority list for its clinical trials annually. The Generic Drug User Fee Amendments (GDUFA) of 2012 established priorities for generic drugs, to evaluate the post-market efficacy, equivalence, components, therapies, and evaluation tools. The 2016 priority list reflects public input from 2015, including FDA desires for establishing equivalence methods for evaluation and standards for drugs, monitoring systematically generics in the marketplace, etc. The FDA seeks to define effective generics in the current climate of interest in generics because of their cost effectiveness.

Atrium Health Navicent Infectious Disease Clinical Trials

Working jointly with the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), the FDA released a roadmap for industry to target development of medical answers for specific maladies. The publication, Guidance for Industry: Expedited Programs for Serious Conditions, Drugs and Biologics, identifies serious conditions, existing therapies available, unmet medical needs by category, and proposes how to expedite specific programs using a fast track protocol. The plan delineates a breakthrough therapy designation, and specifies clinical trials to meet the designations. Moreover, it advocates an accelerated approval process to meet specific goals. The document sets forth priority processing, including both nonclinical and clinical inspection requirements for studies.

Consumer Access to Research Studies

For those seeking involvement with studies or additional information, the U.S. National Institutes of Health provides information for specific studies by a single word or specific phrase of interest on one of its websites entitled accessible by entering the address "" in the computer browser of choice. The phrase entered may be a disease, a medicine, or a symptom. Active studies and recruiting information for new study subjects come up upon entry of phrase or word in the website information box.