32 Special US Regulatory

Procedures: Emergency and

Compassionate INDs and

Accelerated Product Approvals

Anthony W. Fox

32.1 Introduction

ThespecialtypesofINDandNewDrugApplication
(NDA) probably represent the greatest differences
in regulatory practice between Europe and the Uni-
ted States. These differences reside not only in the
particular procedures themselves but also in the
philosophy of regulatory authorities. Emergency
INDs, Treatment INDs and accelerated approvals
are essentially of United States interest, and the
Code of Federal Regulations, Title 21, Chapter I
(21CFR) is where most of these rules are published
(the orphan drug regulations may, perhaps, also be
seen as a special type of IND or NDA, and are
described elsewhere in this book). It is probably
fair to say that these procedures have created quite
a revolution in the US drug approval process, and
have helped drug developers. Their careful and
gradual introduction has not damaged the public
health. This chapter covers the following topics:

Emergency INDs

‘Compassionate Use’: The Treatment IND

Acceleratedapprovals: seriousandlife-threatening
diseases

Accelerated approvals: ANDAs and generic

drugs

It should be noted that an ‘investigator’s IND’, or

‘physician’s IND’, is not a specific practice defined

by regulation, and that these are orthodox INDs. It

is true, however, that these IND submissions are

usually smaller than those from pharmaceutical

companies (see Section 32.3 below).

32.2 Emergency INDs

The IND in the United States is based legally on the

notion that Food and Drug Administration (FDA)

permission is needed to convey investigational (i.e.

unapproved) drugs across state or international

boundaries. This defines the jurisdiction of the

Federal government in comparison to the state

governments in all matters of commerce, not just

drug development. The FDA imposes control over

this process by requiring information of appropri-

ate quantity and quality, before granting its permis-

sion. Much of the documentation is judged by how

well it supports a proposed clinical protocol;

the latter is one of the most important pieces of

information that FDA quite properly demands.

Principles and Practice of Pharmaceutical Medicine, 2nd Edition Edited by L. D. Edwards, A. J. Fletcher, A. W. Fox and P. D. Stonier
#2007 John Wiley & Sons, Ltd ISBN: 978-0-470-09313-9