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Glossary of Terms

Active clinical trial
A trial open for enrollment of patients

Active, not recruiting (clinical trial)
The study is ongoing but new participants are not currently being enrolled

Adverse event
A medical problem that happens during treatment with a drug or other therapy, which may be mild, moderate, or severe and which may or may not be associated with the therapy

Alternative treatment options
Any other comparable or satisfactory treatments approved to diagnose, monitor or treat the disease

Approved for marketing
Therapy has been approved by the Food and Drug Administration for sale in the U.S. 

Biologics
Products that replicate natural substances such as enzymes, antibodies or hormones in the body, made from a variety of natural resources

Clinical trials
Research study that tests how well new medical approaches work in people - including new methods of prevention, diagnosis or treatment of a disease

Completed trial
The study has ended and participants are no longer being examined or treated in the study

Convened IRB meeting
Usually, a formal meeting with a majority of the Institutional Review Board members present

Disease
A health condition with specific characteristics or symptoms

EAP
Expanded Access Program

Efficacy 
The effectiveness of the medicine or other therapy

Eligibility criteria
The specific requirements that must be met for participation in a clinical trial

Eligibility requirements
The specific requirements that must be met for participation in a clinical trial (like age group or gender, e.g.)

Exclusion criteria
Defined factors that prevent participation in a trial

Existing standard of care
The treatment that medical professionals consider at the time of the study to be the most prevalent and best available treatment

Expanded Access
The use of investigational treatments not yet approved by the FDA, to treat patients with serious or life-threatening diseases or conditions, who have exhausted other treatment options and who cannot participate in a clinical trial

FDA
A U.S. government agency that protects and promotes the public health by making sure medical products are safe and effective

Group Expanded Access Programs
Program that provides a defined group of people with access to an investigational treatment when there is enough evidence that they may benefit from it

Inclusion criteria
Requirements for participation in a clinical trial, like gender, age, diagnosis, e.g.

Informed consent
Explanation by researchers of procedures, potential benefits and risks and alternative treatment options (if they exist), as well as a clear explanation of your rights as a patient in a clinical trial and of how your privacy will be protected. Agreement is essential to participate in the trial

Institutional Review Boards
Group formally designated by a medical institution to review and approve research involving people

Intermediate-sized expanded access program
Expanded access use for more than single patients and less than large populations

Investigational
Therapy being studied for safety and effectiveness, not yet approved by FDA

Investigational New Drug Application (IND)
A request for authorization from the FDA to administer an investigational drug or biological product to humans

Investigator 
Person responsible for the conduct of the clinical trial at the trial site

Large-size expanded access program
Treatment use of an investigational therapy for a larger patient population, as defined by FDA

Letter of authorization
Required documentation from pharmaceutical company granting access to their investigational treatment

Life-threatening disease or condition
Likely to result in premature death or death within months without early treatment

Marketing approval
FDA permission for commercial marketing of product

Off-label use
Prescription by physician for a use not studied or approved by FDA. Also called unapproved use of an approved product

Outcomes
Results of the treatment, including patient response, and all adverse events

Path to market
Drug development and approval process

Patient advocacy group
Organization supporting groups of patients

Placebo
An inactive substance or treatment that looks the same as, and is given the same way as, an active drug or treatment being tested

Protocol
A detailed plan of a scientific or medical experiment, treatment, or procedure. In clinical trials, it is a description of what the study is designed to determine and how it will be done.

Quality of life
Health status or health-related functioning and well-being

Regulatory body
Formal group with review and approval responsibilities, like FDA or an Institutional Review Board

Risk-benefit balance
The potential risks of a therapy compared to potential benefits, including symptom relief and slowing of disease progression

Serious adverse event
A drug reaction or side effect that can result in death, can be life- threatening or can result in hospitalization, a persistent or significant incapacity or substantial disruption of ability to conduct normal life functions, or a congenital abnormality/birth defect.

Serious disease or condition
Has substantial impact on day-to-day functioning

Single-patient expanded access
Treatment use of an investigational therapy for an individual patient with a serious or life-threatening disease or condition who has exhausted other treatment options or is unable to participate in a clinical trial or group expanded access program if available.

Sponsor
An entity such as a company developing and testing a drug and seeking FDA approval to market

Therapies
Treatments or medical interventions like a drug, biologic or device

Treatment
A therapy or medical intervention like a drug, biologic or device

Treatment plan
Detailed plan with information about a patient's disease, the goal of treatment, options and possible side effects and expected length of treatment

Treatment product disposition
Returning or destroying unused drugs

Unexpected adverse event
Negative effect compared to described or predicted outcome

21st Century Cures Act
Legislation including requirement that pharmaceutical companies publicly provide their policies and procedures for processing requests for expanded access

 

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