Clinical trial

A research study involving human volunteers (also called participants) that is intended to add to medical knowledge. The participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. The assignments are determined by the study's protocol. Participants may receive diagnostic, therapeutic, or other types of interventions.3

Expanded access/Compassionate use (not considered clinical trials)

“Expanded access” within this site means the availability of emicizumab prior to its approval outside of a clinical trial for intermediate to large populations who meet certain criteria in accordance with local health authority requirements.4 “Compassionate use” within this site means the availability of emicizumab prior to its approval on a case-by-case basis to individual patients, following a request from their physician, who have a serious or life-threatening disease or condition, are not eligible for, or are unable to participate in an ongoing clinical trial, have exhausted all therapies typically used to treat the disease and are no longer responsive to, or able to tolerate alternative treatments, and have no other approved treatment options.1

Postmarketing

Postmarketing generally refers to the time after a drug or biological product has been approved by a country’s regulatory authority. See FAQ for additional information.5

Postmarketing adverse event reports

A postmarketing adverse event is defined on this site to mean one which occurred after regulatory approval, meets spontaneous adverse event reporting criteria, and is not due to a drug received in a clinical trial, or through expanded access or compassionate use.

References

1.

Data on file. Genentech, Inc. March 2018

3.

US National Library of Medicine. ClinicalTrials.gov. Glossary of Common Site Terms. https://clinicaltrials.gov/ct2/about-studies/glossary. Accessed March 2018

4.

US Food and Drug Administration. Expanded Access (Compassionate Use) https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm. Accessed March 2018; 6. Jarow JP et al. Ther Innov Regul Sci. 2016;50(6):705-709

5.

US Food and Drug Administration. Postmarketing Requirements and Commitments: Reports https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Post-marketingPhaseIVCommitments/ucm064436.htm. Accessed March 2018