What Are Clinical Trials

What Are Clinical Trials?

Bioscience Americas ...Solutions for a Healthier World.

What Are Clinical Trials?

A critical part in the process of bringing new therapies to patients is clinical development, the study of potential new therapies in humans.

It typically takes 12 years from the moment of discovery in the lab for a potential new therapy to gain approval for use and to reach patients. Clinical development is the most time intensive and expensive part of this research and development continuum accounting for approximately 45 to 75 percent of the $50 million average cost of bringing a new therapy to market.

Trials conducted during clinical development generate thousands of pieces of data and are generally used by regulatory agencies, such as The U.S. Food and Drug Administration (FDA) to determine approval for use of the therapy and in which medical indications it can be used.

Helping Patients

Clinical trials involve hundreds to many thousands of volunteer trial participants. Every trial is governed using well-defined ethical rules and standards to protect participant safety.

For patients suffering from a condition that is currently untreatable, participation in a clinical trial may provide access to potential new therapies. While participating, volunteers obtain closely controlled care in research medical facilities, in addition to helping others by contributing to medical research.

Phases of Development

Clinical Development Process

An experimental therapy is first tested in the laboratory and in animal studies. After this “pre-clinical” testing, and only if shown to meet certain safety criteria and shown to have value as a potential new therapy, the therapy advances to clinical testing in humans. The Phases of Clinical Development (please select a Phase below to view).

Phase I

Phase 1 clinical trials mark the first time an experimental therapy is administered to humans. Phase 1 trials usually focus on ensuring the therapy is safe to use in people, rather than how effective it may be as a treatment for a given disease. During this phase, escalating doses of the experimental therapy are given to a small number of study participants so that researchers can measure the body’s response, including how it is absorbed, its duration in the blood stream and which dosage levels are safe and well tolerated.

Phase II

Phase 2 trials generally assess the effectiveness of an experimental therapy at treating a specific illness or medical condition. Information about the experimental therapy’s safety, side effects and potential risks are also collected. In this phase, researchers work to determine the most effective dosages for the experimental therapy and the most appropriate method of delivery, such as tablets, extended release capsules, infusions or injections. Phase 2 clinical trials involve a larger number of trial participants, typically up to several hundred, who usually have the medical condition that the experimental therapy is intended to treat.

Phase III

Phase 3 trials test the results of earlier trials in much larger groups of people and gather additional information on the effectiveness and safety of the experimental therapy. This phase will usually involve several hundred to several thousand participants across multiple study locations. These trials are often randomized, where participants are arbitrarily allocated to receive the experimental therapy, placebo or another therapy (a “comparator”), and “double-blinded,” in which neither the investigator nor the participant are aware if the therapy given is the true experimental therapy, placebo (medication with no active ingredients) or another therapy (a “comparator”). Phase 3 trials generally provide the primary basis for the benefit-risk assessment for the new therapy and much of the core information about the therapy that is analyzed for inclusion in final labeling if approved by the regulatory authority.

Registration

Filing an application for registration with the country’s health regulatory authority is the next step in bringing a potential new therapy to patients. In the U.S., a New Drug Application (NDA) is filed with the U.S. Food and Drug Administration (FDA). In Europe, a Market Authorization Application (MAA) is filed with the European Agency for the Evaluation of Medicinal Products (EMEA). A description of the therapy’s manufacturing process along with quality data and trial results from all pre-clinical and clinical trials are provided in order to demonstrate the safety and effectiveness of the new therapy. If approval is granted, the new therapy can then be sold for use as detailed in the final approval label granted by the regulatory authority.

Phase IV

Phase 4 trials — also called “post-marketing studies” — are conducted after regulatory approval and are critical to informing the ongoing use of the therapy. Through such trials, researchers collect additional information about longer-term risks, benefits and optimal use. These trials often involve thousands of participants and may continue for many years.

Notice: The information provided on this page is for general information only and is subject to the Bioscience Terms Of Use It is not intended to provide medical advice or guidance, and should not be used for that purpose. Medical use of any product should be strictly in accordance with the approved prescribing information.

WHO / WHAT REGULATES THE GIOSTAR Colombia/BIOSCIENCE AMERICAS STEM CELL TREATMENT PROTOCOL AND THERAPY?

There are two aspects to consider regarding the use of stem cell therapies in the U.S. and potential host countries outside of the U.S.

The first is the existing legal structure surrounding research and secondly, the regulatory framework regarding application of that research to treat patients.

The Global Institute of Stem Cell Therapy and Research (GIOSTAR Colombia) adheres to the guidelines promulgated by the U.S. Food and Drug Administration (FDA) under the U.S. Department of Health and Human Services, Code of Federal Regulation, Part 1271 entitled “Regulation of Human Cells, Tissues, and Cellular and Tissue Based Products”.

The FDA makes a clear distinction of minimally manipulated cells and autologous transplantation from other cell types when it states in Part 1271, that ‘you are not required to comply with the requirements of this part if you are an establishment that removes human cells, tissues and cellular and tissue-based products from an individual and implants such products into the same individual during the same surgical procedure’. What this implies is that minimally manipulated and autologous cell therapy should not have regulations that are in place for other human cells, tissues, tissue based products and drugs.

GIOSTAR Colombia follows Part 1271 for adult autologous stem cell transplant.

Adult HSC (Hematopoietic Stem Cell) transplants under practice and approved by US FDA over the last 30 years include the following examples of diseases:

Malignant:

  • Acute myeloid leukemia (AML)
  • Chronic myeloid leukemia (CML)
  • Acute lymphoblastic leukemia (ALL)
  • Hodgkin lymphoma (relapsed, refractory)
  • Non-Hodgkin (relapsed or refractory) lymphoma
  • Neuroblastoma
  • Ewing sarcoma
  • Multiple Myeloma
  • Myelodysplastic syndromes
  • Gliomas, other solid tumors

Non-malignant

  • Thalassemia
  • Sickle Cell Anemia
  • Aplastic anemia
  • Fanconi anemia
  • Immune deficiency syndromes
  • Inborn errors of metabolism

Regarding patient application in our host countries, as indicated in a previous e-mail, prohibitions are generally in place regulating the use of embryonic stem cell. GIOSTAR Colombia does not conduct research or treat patients with embryonic stem cells.

As currently applied in Colombia, for example, patient therapies will be administered under FDA Part 1271 guidelines, as noted above, operating under the authority and license of the attending physician and associated medical professionals.