Patient Resources

Dug Approval Process

Most drugs that go through pre-clinical (animal) testing never make it to undergo human testing and be reviewed by the FDA. All the drugs that make it to human testing phase must undergo FDA’s rigorous evaluation process, which scrutinizes everything about the drug; from the design of clinical trials to the severity of side effects to the conditions under which the drug is manufactured.

Stages of Drug Development and Review

INVESTIGATIONAL NEW DRUG APPLICATION (IND) FDA comes into play when a Pharmaceutical Company which is also considered a Sponsor submits an IND to the agency (FDA). The Sponsors, research institutions, and all other organizations that are responsible for marketing the drug, must show the result of the pre-clinical (animal) testing that has been done in the laboratory to FDA and what they propose to do regarding human testing. At this point, the FDA makes the decision whether it is reasonably safe to move forward with testing the drug on humans.
CLINICAL TRIALS Clinical trial is the stage of drug development after the pre-clinical stage. The drug studies in humans can only begin after an Investigational New Drug (IND) is reviewed by the FDA and a local Institutional Review Board (IRB), a panel of non-scientists and scientists in hospitals and research institutions that oversees clinical trials.

IRB is responsible for the approval of the clinical trial protocols, which describe the objectives of the study, the type of people who may participate in the clinical trial, the schedule of tests and procedures, the medications and dosages to be studied, the length of the study, and other details. IRBs ensures that the study is acceptable, IRB ensures that study participants are fully informed of their risks, and that researchers take appropriate steps to protect patients from harm. IRB ensures that the study participants give their consent to participate in the study and their safety is primarily protected.

Phase I: During phase I, the investigational drug or biologic is given to humans for the first time. Studies are usually conducted with healthy volunteers between 20 to 100 participants. Although, in certain cases, patients with the target disease are studied. The type of subject depends on the nature of the disease and the expected toxicity of the investigational drug. It would be unethical, for example, to give healthy subjects a toxic investigational drug meant to treat one of the cancers. The purpose of phase I studies is to determine the metabolic and pharmacologic action of the investigational drug in humans, assess the adverse effects associated with different doses, and to perhaps get an indication of whether or not there is any evidence of efficacy. Phase I studies are usually done in special testing facilities designed for Phase I.

Phase II: When the appropriate phase I studies have been completed and sufficient safety data are in hand, phase II studies are then initiated. Phase II studies are rigid, well-controlled studies in a relatively small homogenous patient population, usually no more than a few hundred subjects in total. These subjects have target disease, but no other confounding illness. For controlled trials, patients receiving the drug are compared with similar patients receiving a different treatment, usually a placebo or a different drug. While the focus of phase II studies is primarily efficacy, they also assess safety. Safety continues to be evaluated, and short-term side effects are studied. Typically, the number of subjects in Phase II studies ranges from a few dozen to about 300.

Phase III: studies are only initiated if the data generated in phase I and II have a satisfactory safety profile and there is sufficient evidence of efficacy. The purpose of phase III studies is to demonstrate the safety and efficacy needed to assess the risk/benefit relationship for the intended use of the investigational drug and to provide adequate data for the product package insert.  Phase III studies are expanded, controlled studies in large patient populations (often thousands of patients) that represent the types of patients the compound is intended to treat after it is marketed. These studies gather more information about safety and effectiveness, studying different populations and different dosages and using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people.

Phase IIIb: A sponsor will frequently have some studies that are still active at the time they file the NDA for a new compound. There are also studies that may be initiated and conducted while the NDA is pending approval. These studies are known as phase IIIb studies. The purpose of these studies may be to gather additional safety data, or to gather information on additional indications for the drug, or to assess its use in special patient populations such as geriatric patients.

Phase IV: studies are done after the approval of the NDA, often to determine additional information about the safety or efficacy profile of the compound. They consist of studies:

  1. Required as a condition of approval by the FDA
  2. Long-term safety studies required by the FDA
  3. Conducted to study the compound in comparison with other marketed products
  4. Designed to familiarize physicians with the compound

NEW DRUG APPLICATION (NDA): The NDA is a formal request to be allowed to market a drug. The sponsor submits the NDA to the FDA at the time that the primary efficacy studies (phase III) are complete. The company is essentially telling the FDA that it has completed the necessary safety and efficacy requirements needed for approval. This signals the end of phase III, although there are likely to be some studies still in progress.

When an NDA comes in, the FDA has 60 days to decide whether to file it so that it can be reviewed. FDA can refuse to file an application that is incomplete. For example, some required studies may be missing. In accordance with the Prescription Drug User Fee Act (PDUFA), the FDA’s Center for Drug Evaluation and Research (CDER) expects to review and act on at least 90 percent of NDAs no later than ten months after the applications were received for standard reviews and six months for priority reviews. Priority reviews are granted by the FDA after an application has been submitted and are intended for products that address unmet medical needs.

The Tufts Center for the Study of Drug Development in Boston estimates that about 1 in 5 drugs that enter clinical testing ultimately are approved by the FDA. How often the FDA meets with a drug sponsor varies, but the two most common meeting points are at the end of Phase II clinical trials and pre-NDA–right before a new drug application is submitted.

The information contained in “The Drug Approval Process” was compiled from several FDA sources.

Medical Disorders

Below are detailed descriptions of various Medical Disorders that Our Doctors are specialized in;

Cardiology/Vascular Diseases

These are diseases that have to do with structure and function of the heart and blood vessels. Studies in these areas include: heart failure, coronary artery disease, high cholesterol, blood clots, circulation disorders, Hypertension, and others.

Endocrinology

Field relating to hormone-manufacturing glands such as the pituitary, thyroid, parathyroid, and adrenal glands, as well as the ovary and testis, the placenta, and the pancreas. Studies in this area include: diabetes and diabetes-related disorders, diet and nutrition, hormone-replacement therapy, menopause, obesity, and others.

Gastroenterology
This involves the study of gastrointestinal organs and diseases relating to them. This includes any part of the digestive tract from mouth to anus, liver, biliary tract, and the pancreas. Studies in this area include: constipation, Crohn’s disease, diarrhea, gall bladder disease, heartburn, hemorrhoids, Irritable Bowel Syndrome (IBS), ulcers, liver disease, stomach cancer, and others.

Hematology

Field regarding blood, blood-forming tissues, and the diseases associated with them. Studies in this area include: anemia, blood clots, bone marrow transplant, leukemia, platelet disorders, red-cell disorders, T-cell lymphoma, vitamin deficiencies, white-cell disorders, and others.

Immunology/Infectious Diseases

This involves the diseases affecting the defense mechanisms of the body. Studies in this area include: AIDS, auto-immune diseases, bacterial infections, chronic fatigue syndrome, common cold, genital herpes, genital warts, hepatitis, HIV infections, immuno-suppressives, influenza, lyme disease, meningitis, parasite and protozoan infections, strep throat, vaccines, viral infections, and others.

 

Musculoskeletal

This involves the field having to do with the muscles and the bones of the body. Studies in this area include: aging, bone density, bone fractures, chronic back pain, hip replacement, osteoarthritis, osteoporosis, rheumatoid arthritis, spinal cord injuries, and others.

Nephrology/Urology

The studies and the treatment of diseases of the kidney and the urinary tract. Studies in these areas include: bladder cancer, impotence, kidney disease, kidney stones, mastectomy, nocturia, renal cell carcinoma, urinary tract infections, and others.

Neurology/CNS

This is the field concerning the nervous system, especially the brain, peripheral nerves, and spinal cord. Studies in this field include: Alzheimer’s disease, Attention Deficit Hyperactivity Disorder (ADHD), Carpal Tunnel Syndrome, Huntington’s Disease, dementia, memory loss, migraine headaches, multiple sclerosis, muscular dystrophy, Parkinson’s Disease, strokes, Tourette’s Syndrome, and others.

Pulmonary/Respiratory Diseases

This includes diseases having to do with the lungs and/or breathing. Studies in these areas include: Acute Respiratory Distress Syndrome (ARDS), allergy, asthma, bronchitis, cystic fibrosis, emphysema, lung disease, pneumonia, sinus infections, smoking cessation, Chronic Obstructive Pulmonary Disease (COPD) and others.

Rheumatology/Pain Management

This field is related to joints, tendons, muscles, ligaments, and associated structures. Studies in this area include: arthritis, osteoarthritis, psoriasis, rheumatic fever, rheumatoid arthritis, and others.

If you are interested in participating in any of our studies, please complete the “contact us” form or feel free to contact us at 770-305-6464 or 770-305-6466 for further questions.

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