Abbreviated New Drug Application (ANDA)
Termed “abbreviated” because preclinical (animal) and clinical (human) data to establish safety and effectiveness are not required. Instead, generic applicants must scientifically demonstrate that their product is bioequivalent (i.e. performs in the same manner as the innovator drug).
This is characterized as any undesirable experience associated with the use of a medical product in a patient.
A committee that provides FDA with independent advice from outside experts on issues related to human and veterinary drugs, vaccines and other biological products, medical devices, and food.
A written communication to an applicant from FDA approving an application or an abbreviated application.
Seeking foundational knowledge to gain new insights into biology of the disease.
A biologic is a medicine that is manufactured in a living system such as a microorganism or animal cells. Most biologics are very large, complex molecules and include proteins, blood products, vaccines, and cellular therapies.
Biologics License Application (BLA)
A formal presentation of data collected by drug sponsor supporting the safety and effectiveness of an investigational biologic agent.
A biological product that is highly similar to an FDA-approved biological product, known as a reference product, and has no clinically meaningful differences in terms of safety and effectiveness.
The process of keeping study participants, those involved with their management, and those collecting and analyzing clinical data unaware of the assigned treatment, so that they should not be influenced by that knowledge.
Breakthrough Therapy Designation
FDA designates certain drugs as “breakthrough therapies” that show promising early clinical trial results and that are intended to treat serious diseases. The breakthrough designation is intended to speed the development of drugs that show the most promise in improving the treatment of the disease.
These are outcomes that directly measures the therapeutic effect of a drug, such as how a patient feels, functions, or survives. An example is the incidence of stroke in a trial for cardiovascular disease drug.
A research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.
Complete Response Letter
A written communication to an applicant from FDA usually describing all of the deficiencies that the agency has identified in an application or abbreviated application that must be satisfactorily addressed before it can be approved.
In a double-blind trial, only the study pharmacist knows what study medication a participant is receiving; the participants, doctors, nurses, and other clinical trial staff are not informed.
A new drug’s ability to demonstrate the ability to improve at least one of the following: the way a patient feels, functions, or survives. If a new drug can demonstrate that it produces at least one of these three outcomes, it can be said to provide clinical benefit.
The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.
An event or outcome that can be measured objectively to determine whether the intervention being studied is beneficial. Some examples of endpoints are survival, improvements in quality of life, relief of symptoms, duration of response, and disappearance of the tumor.
A drug that is comparable to an FDA-approved drug, known as a reference product, in dosage form, strength, route of administration, quality and performance characteristics, and intended use.
Informed consent is used by researchers to explain the clinical trial to potential volunteers. Its purpose is to protect the participant. It is used when somebody who is interested in participating first asks about the study and it continues throughout the study, until the study ends. The research team will review the details of the trial with the potential participant and will answer any questions. This information is also written in a document, known as the informed consent form, which is designed to be clear and easy to understand. If a person decides to enroll in a clinical trial, they will sign the informed consent form to acknowledge that they understand the details of the trial and consent to participating. The informed consent form is not a contract and the participant can withdraw from the trial at any time, and for any reason. Informed consent is used by researchers to explain the clinical trial to potential volunteers. Its purpose is to protect the rights and welfare of the participant.
It is used when somebody who is interested in participating first asks about the study and it continues throughout the study, until the study ends.
The research team will review the details of the trial with the potential participant and will answer any questions. This information is also written in a document, known as the informed consent form, which is designed to be clear and easy to understand.
If a person decides to enroll in a clinical trial, they will sign the informed consent form to acknowledge that they understand the details of the trial and consent to participating. The informed consent form is not a contract and the participant can withdraw from the trial at any time, and for any reason.
Institutional Review Board (IRB)
An IRB (also known as an independent ethics committee (IEC), ethical review board (ERB), or research ethics board (REB)) is a group of doctors, scientists, advocates, researchers, and members of the community that has been formally designated to review and monitor all research involving humans. IRBs are in place to provide ethical oversight and to minimize risk to participants. An IRB is in place to protect the rights and welfare of human participants in a clinical trial and became a key, established piece of medical oversight because of the Tuskegee study.
Investigational New Drug Application (IND)
A request for authorization from the FDA to administer an investigational treatment to humans.
This study is performed outside a living organism, typically in a test tube or culture dish.
New Drug Application (NDA)
A formal presentation of data collected by drug sponsor supporting the safety and effectiveness of an investigational small molecule drug.
Off-Label Drug Use
This occurs when prescribing a medicine for an indication not specified on the drug label.
Patient-Focused Drug Development Meetings
A series of disease-focused meetings held at FDA, featuring patients who provide their experience with a disease and their perspective on new treatments.
Phases of Clinical Trials
Clinical trials are conducted in “phases.” The trials at each phase have a different purpose and help researchers answer different questions.
A placebo is a pill or liquid that looks like the new treatment but does not have any treatment value from active ingredients.
These are studies or clinical trials that a drug sponsor has agreed to conduct, but that are not required by a statute or regulation. These may include studies to assess drug response in specific subpopulations, evaluation of new doses, or further characterization of rare adverse events.
These include studies and clinical trials that sponsors are required to conduct under one or more statutes or regulations. These may include studies to assess drug response in specific subpopulations, evaluation of new doses, or further characterization of rare adverse events.
A system in which manufacturers of medical products are required to demonstrate the safety and effectiveness of a product before it can be sold to consumers.
Prescription Drug User Fee Act (PDUFA)
1992 law that created “user fee” program, wherein the drug sponsor or applicant provides funds to hire additional FDA reviewers in return for assurances of timely reviews of new drug applications.
A Principal Investigator is a doctor who leads the clinical research team and, along with the other members of the research team, regularly monitors study participants’ health to determine the study’s safety and effectiveness.
Document that describes how a trial will be conducted, including the objective(s), design, methodology, statistical considerations and organization, and ensures the safety of the trial subjects and integrity of the data collected.
Randomized Controlled Trial (RCT)
A study in which patients are divided by chance, or randomly, into separate groups that compare different drugs, treatments, or other interventions. Randomly dividing people into groups means that the groups will be similar and that the effects of the treatments they receive can be compared more fairly. In most cases, one group will receive the drug being tested and the other group will receive the current standard of care treatment in that disease area.
The science of developing new tools, standards, and approaches to assess the safety, effectiveness, quality, toxicity, public health impact, or performance of FDA regulated products.
Request for More Information
A letter sent to an applicant during an application review to request further information or clarification that is needed or would be helpful to allow completion of the review.
A drug sponsor can submit separate sections of its drug application for review by FDA, rather than waiting until every section of the application is completed. When rolling review is not in effect, review usually begins only when the drug sponsor has submitted the entire application to FDA.
Closely related to toxicity, this refers to a drug’s potential to cause harm to a patient. While extremely toxic drugs are rarely approved for use, analyzing a drug’s safety is more complicated than determining whether or not it has any harmful side effects.
Regulators must decide whether a drug’s benefits outweigh its potential harms. If a mild painkiller has the potential to cause long-term liver damage, for example, that would likely be deemed an unreasonable risk.
On the other hand, a third-line cancer drug with the same potential side effect might be considered reasonably safe, due to the severity of the condition being treated.
A process designed to determine individual’s eligibility for participation in a clinical research study.
A research tool and FDA program to actively identify drug-induced adverse events from electronic healthcare data.
Serious Adverse Event
An adverse event that is life-threatening, requires hospitalization or extended hospital stay, results in ongoing or significant incapacity, causes congenital anomalies or birth defects, or results in death.
Single Arm Trials
In this type of trial, the test drug is not usually compared to a control. All the participants will receive the experimental therapy.
Small molecule drugs
Most common medicines are small molecule drugs, meaning they are typically manufactured through chemical synthesis, which means that they generally have well-defined chemical structures. By contrast, biologics are much more complex.
Supplemental New Drug Application (sNDA)
After receiving FDA approval for a new drug, sponsors may seek approval for additional indications.
An endpoint reasonable likely to predict how a treatment impacts how patients feel, function, or survive. In cancer, tumor shrinkage might be a surrogate endpoint that can be measured before survival outcomes.
Can be defined as how well the patients’ behavior corresponds with the prescribed professional medical advice. Patients actions include attending follow-up appointments, engaging in preventative care, adhering to medical regimens.