Other Ways in Which a Drug May Be Sold: Clinical Trials, Special Access, Extraordinary Use, Urgent Public Health Need, and Shortages

• Author: T. Nessim Abu-Zahra/Simon Carvalho/David Edwards
• Pages: 95-112



SEVEN

Other Ways in Which a Drug May Be

Sold: Clinical Trials, Special Access,

Extraordinary Use, Urgent Public

Health Need, and Shortages

A. INTRODUCTION

Most drugs are made generally available for sale by way of the drug iden-

tication number (DIN) and notice of compliance (NOC) issuance path-

ways described in Chapter , or under Part  of the Natural Health Products

Regulations (NHPRs) (see Chapter ). ese types of market authorization

permit the marketing of a product. However, there are several other author-

ization pathways that may permit the lawful sale of a drug for more limited

purposes under the Food and Drugs Act (FDA). ese alternative pathways

serve a number of dierent public health and safety objectives, including to

permit the study of drugs in humans (the clinical trials regime) and enable

the sale of drugs in emergency circumstances (the Special Access, Urgent

Public Health Need, Extraordinary Use and Shortages regulations). As dis-

cussed below, in some cases these schemes are part of the DIN/NOC regimes,

and in other cases they are distinct from it.

is chapter will explain the following:

• the regulation of drugs sold for the purpose of clinical trials under

Division  of the Food and Drug Regulations (FDRs)

 SOR/-.

 RSC , c F-.

 SOR/-.

The Regulation of Drugs in Canada

• the rules in Division  of the FDRs regarding special access to

unapproved new drugs for emergency treatment of a patient

• the rules in Division  of the FDRs for drugs for urgent public health

need or for exceptional import in response to a shortage

• the rules in Division  of the FDRs providing for special access to

an unapproved drug for public health or military health emergencies

B. CLINICAL TRIALS

A clinical trial is dened in section  of the FDA as a study involving human

subjects for the purpose of discovering or verifying the eects of a drug,

medical device, or food for a special dietary purpose. Trials involve a sponsor

(the person who designs and controls the study), an investigator (the person

who conducts the research), and a research subject (the person who is being

studied under the care of the investigator).

A convention has developed to describe clinical trials involving humans

as falling into one of four phases depending on the design of the study and

where the drug is in development. Phase I studies are generally conducted

in healthy volunteers and seek to determine the pharmacokinetics/pharma-

cological actions of the drug and the side eects associated with increasing

doses. Phase II trials seek to evaluate the preliminary ecacy of the drug

in patients with a medical condition of interest and to determine the side

eects and risks associated with the drug. Phase III trials, which tend to be

much larger studies, are conducted after preliminary evidence suggesting

that the ecacy of the drug has been demonstrated. As described in Chap-

ter , a brand-name manufacturer wishing to introduce a new drug to the

Canadian market will typically rely on Phase III clinical trials to obtain an

NOC. Finally, Phase IV studies are conducted using an authorized drug to

further study its use for one of its approved indications.

In , the FDA was amended to add a prohibition against conducting

a clinical trial without an authorization, as well as adding a power to make

 CRC c , ss C.. & C...

 SOR/-.

 SOR/-.

 See, for example, Health Canada, “Guidance Document for Clinical Trial Sponsors:

Clinical Trial Applications” (eective  May ), online (pdf): Government of

Canada www.canada.ca/content/dam/hc-sc/migration/hc-sc/dhp-mps/alt_formats/

pdf/prodpharma/applic-demande/guide-ld/clini/ctdcta_ctddec-eng.pdf.