Biomedical Engineering Reference
In-Depth Information
the FDA publishes the Summary Basis of Approval (SBA) and most
of the other reports and letters generated during the approval
process on the “drugs@fda” page of its website [
2
].
Drugs that treat conditions of particular concern may receive one of
several special designations that affect how the drug is approved
and/or regulated [
25
,
26
].
Orphan product designation
. This designation is granted to drugs
intended to treat uncommon diseases (those affecting
3.2.1 Special FDA
Regulatory Designations
200,000
people in the US) and was developed to help motivate pharma-
ceutical companies to develop drugs for markets that might
otherwise be considered too small to be profitable. Orphan drug
status does not change the regulatory requirements that the drug
must meet for approval, but it may result in the manufacturer
being allowed to waive certain fees, or being granted additional
marketing exclusivity or
<
eligibility
for drug development
grants [
26
,
27
].
Fast track designation
. This designation is granted to drugs
intended to treat a serious or life-threatening condition (e.g.,
cancer, diabetes, AIDS, and Alzheimer's) and fill an important
unmet medical need (e.g., improved efficacy or safety, decreasing
the toxicity of another treatment, or allowing earlier disease diag-
nosis). It allows the drug sponsor to access various mechanisms
designed to accelerate the drug approval process, such as more
frequent and interactive communication between the sponsor and
the FDA and the ability to request accelerated approval (see below)
or submit a “rolling submission.” A rolling submission allows the
sponsor to submit each section of the NDA as it is completed
rather than requiring the entire application to be complete before
submission. This allows the preclinical and quality portions of the
application to be reviewed while the clinical studies are being
finalized [
25
,
26
].
Priority review
. A priority designation is granted at the time of NDA
submission to those drugs intended to treat a disease for which
there is no satisfactory therapy, or to drugs that offer a significant
improvement over existing therapies. Drugs that receive a priority
designation are reviewed within 6 months rather than the usual 10
months. It is not unusual for a fast track designation during the
development process to be followed by a priority designation dur-
ing the review process [
25
,
26
].
Accelerated approval regulation
. This regulation allows the approval
of a drug based on a surrogate endpoint rather than a final clinical
endpoint. It is reserved for those drugs intended to treat a serious
disease and to fill an unmet medical need, and is important when
the impact of treatment on the final clinical outcome (e.g.,
