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FromHopeToCures.org

FromHopeToCures.org

Glossary of Terms

The glossary below is a resource for understanding terms and phrases used across the health care spectrum. From scientific definitions and emerging fields of medicine to processes and regulatory jargon, it provides simple and straightforward explanations helping you better understand America’s biopharmaceutical industry.

 A

  • Accelerated approval

    A review pathway by which the U.S. Food and Drug Administration expedites approval for medicines that address a serious condition or fill an unmet medical need, based on a surrogate or an intermediate clinical endpoint.

  • Adaptive Clinical Trial

    A promising approach to clinical trials that allow for pre-planned, well-defined changes to modify aspects of the study (e.g., dosing, sample size, patient population) as it is under way, without undermining the validity and integrity of the trial.

  • Antibody Drug Conjugates

    Antibodies are immune system proteins that recognize specific targets for the immune system to attack, such as viruses, bacteria, or tumor cells. Antibody drug conjugates include an antibody, a linker, and a chemotherapy drug. The antibody targets and binds to a cancer cell, delivering the chemotherapy directly to its target while leaving more healthy cells unharmed.

 B

  • Best Pharmaceuticals for Children Act

    An Act that allows biopharmaceutical companies to receive an additional six months of exclusivity upon the completion and submission of pediatric studies that meet the terms of a written request from FDA.

  • Biologics

    Biologics are medicines made from living cells through highly complex manufacturing processes. They are used to prevent, treat, diagnose or cure a variety of serious and chronic illnesses including cancer, chronic kidney disease, autoimmune disorders and infectious diseases.

  • Biologics License Application (BLA)

    An application proposing that the FDA approve a new medicine for sale and marketing in the U.S. that contains the product’s whole story including what happened during the clinical tests, what the ingredients of the medicine are, the results of the animal studies, how the medicine behaves in the body, and how it is manufactured, processed and packaged.

  • Biomarker

    A biomarker, shorthand for “biological marker,” is a measure or physical sign that can be used to determine how the body is functioning.

  • Biosimilar

    A biosimilar is exactly what its name implies — it is a biologic that is “similar” to another biologic drug already approved by the FDA, known as a reference product. It may not have any clinically meaningful differences in terms of safety and effectiveness from the reference product.

  • Breakthrough therapy

    A process designation designed to expedite the development and review of drugs that treat a serious condition and where preliminary clinical evidence indicates that the medicine may demonstrate substantial improvement over available therapies.  

 C

  • Clinical Trial Protocol

    A clinical trial protocol is the blueprint for a study and describes how a clinical trial will be conducted (the objective(s), design, methodology, statistical considerations and structure of a clinical trial). It ensures the safety of the trial participants and quality of the data that will be collected and analyzed. Whether it will be to test a new drug, device or procedure, or is only observational in nature, every study begins with the protocol.

  • Clinical Trials

    A clinical trial is a study that is carefully designed to test the benefits and risks of a specific medical treatment or intervention, such as a new drug or a behavior change (e.g., diet). 

  • Clinical Trials – Phase 1

    Phase 1 clinical trials test the candidate medicine in a small group (e.g., 100 or less) of healthy volunteers to assess the compound’s safety and how it is best metabolized or processed in the body.

  • Clinical Trials – Phase 2

    Phase 2 clinical trials involve a somewhat larger group of patient volunteers (100 to 500) living with the disease or condition the compound is designed to target. In addition to examining the compound’s safety and possible short-term side effects, phase II trials also evaluate the compound’s effectiveness and identify optimal dosing.

  • Clinical Trials – Phase 3

    Phase III clinical trials test the compound in a much larger group of patients (usually in the thousands). The purpose of these studies is to generate a wealth of statistically significant information about the safety and efficacy of a candidate medicine to determine the overall benefit–risk ratio.

  • Continuous Manufacturing

    Continuous manufacturing is an alternative process to batch manufacturing for medicines to improve scalability, increase capacity and enhance quality. It involves raw materials continuously feeding raw materials into a process train while finished product materials are continuously removed at the other end.

 D

  • Data Exclusivity

    Prohibits third parties for a set period of time from using or relying upon an innovator’s valuable clinical data to obtain FDA approval for their product.

  • Drug Target

    A molecular structure in the body that, when it interacts with a potential drug compound, produces a clinical effect (treatment or prevention of a disease, for example).

 F

  • Fast track

    A designation from the FDA that facilitates early and frequent communication between the FDA and a drug company to expedite development and review of drugs that treat serious conditions and fill an unmet medical need.

 I

  • Immunotherapy

    Immunotherapy is used in some cancer treatments by harnessing the body’s immune system to eliminate cancer or slow its growth and ability to spread; rather than kill cancer cells directly as traditional tools like radiation or chemotherapy do.

  • Institutional Review Board

    An institutional review board (IRB) is an independent group composed of physicians, researchers, and members of the general public to ensure clinical trials are ethical and the rights and welfare of participants are protected at all times. Furthermore, the IRB ensures research risks are minimized and reasonable in relation to potential benefits.

  • Intellectual Property (IP) protections

    Incentives for individuals and companies to invest the significant time, effort, and dollars needed to develop new medicines.

  • Investigational New drug application (IND)

    An application filed with the FDA, containing all preclinical testing results and plans for clinical development, in order to demonstrate drug safety before initiating clinical studies in humans.

 N

  • New Drug Application (NDA)

    An application proposing that the FDA approve a new medicine for sale and marketing in the U.S. that contains the product’s whole story including what happened during the clinical tests, what the ingredients of the medicine are, the results of the animal studies, how the medicine behaves in the body, and how it is manufactured, processed and packaged.

 O

  • Orphan Drug Act

    The Orphan Drug Act of 1983 created economic incentives to promote the development of new treatments for rare diseases, defined generally as those conditions affecting fewer than 200,000 people in the United States. The Act has been a great success, as demonstrated by the nearly 500 orphan drugs approved since its passage, with 233 of those approvals in the last decade alone. In contrast, the FDA had approved fewer than 10 orphan drugs in all of the 1970s before the Act was passed. Moreover, research demonstrates that most of the recent approvals have been for diseases with fewer than 10,000 patients demonstrating the research community’s commitment to meeting the needs of small patient populations.

 P

  • Patents

    Patents grant inventors the exclusive right with respect to their inventions for a set period of time before others may copy and sell it.

  • Patient-Focused Drug Development

    Patient-focused drug development incorporates the patient’s voice in the development and the U.S. Food and Drug Administration’s evaluation of new medicines.

  • Personalized Medicine

    Personalized medicine — sometimes referred to as precision or individualized medicine —is an emerging field of medicine that uses diagnostic tools to identify specific biological markers, often genetic, to help assess which medical treatments and procedures will be best for each patient.

  • Pharmacodynamics

    The impact an investigational compound has on various functions within the body.

  • Post-Approval Research

    Post-approval research is data produced following the FDA approval of a medicine. Also known as real-world evidence, it is produced in the practice of medicine and helps physicians, patients, payers, health authorities and biopharmaceutical companies better understand the medicine and its appropriate use.

  • Prescription Drug User Fee Act

    The Prescription Drug User Fee Act (PDUFA) provides the U.S. Food and Drug Administration with necessary resources to meet performance goals for the regulatory review of new medicines.

  • Priority Review

    A designation that accelerates the FDA’s evaluation of an application for approval for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.

 S

  • Surrogate Endpoints

    A surrogate endpoint is a biomarker (or other measure) that is expected to predict clinical benefit and can thus be substituted for a clinical endpoint.

 U

  • U.S. Food and Drug Administration

    The U.S. Food and Drug Administration (FDA) is responsible for protecting the public health by assuring the safety, efficacy and security of medicines and biologics, among other products. FDA also helps to speed innovations that make medicines more effective, safer and more affordable and by helping the public get the accurate, science-based information they need to maintain and improve their health.