[Code of Federal Regulations]
[Title 21, Volume 5]
[Revised as of April 1, 2001]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR312.84]

[Page 89]
 
                        TITLE 21--FOOD AND DRUGS
 
CHAPTER I--FOOD AND DRUG ADMINISTRATION, DEPARTMENT OF HEALTH AND HUMAN 
                           SERVICES--Continued
 
PART 312--INVESTIGATIONAL NEW DRUG APPLICATION--Table of Contents
 
    Subpart E--Drugs Intended to Treat Life-threatening and Severely-
                         debilitating Illnesses
 
Sec. 312.84  Risk-benefit analysis in review of marketing applications for drugs to treat life-threatening and severely-debilitating illnesses.

    (a) FDA's application of the statutory standards for marketing 
approval shall recognize the need for a medical risk-benefit judgment in 
making the final decision on approvability. As part of this evaluation, 
consistent with the statement of purpose in Sec. 312.80, FDA will 
consider whether the benefits of the drug outweigh the known and 
potential risks of the drug and the need to answer remaining questions 
about risks and benefits of the drug, taking into consideration the 
severity of the disease and the absence of satisfactory alternative 
therapy.
    (b) In making decisions on whether to grant marketing approval for 
products that have been the subject of an end-of-phase 1 meeting under 
Sec. 312.82, FDA will usually seek the advice of outside expert 
scientific consultants or advisory committees. Upon the filing of such a 
marketing application under Sec. 314.101 or part 601 of this chapter, 
FDA will notify the members of the relevant standing advisory committee 
of the application's filing and its availability for review.
    (c) If FDA concludes that the data presented are not sufficient for 
marketing approval, FDA will issue (for a drug) a not approvable letter 
pursuant to Sec. 314.120 of this chapter, or (for a biologic) a 
deficiencies letter consistent with the biological product licensing 
procedures. Such letter, in describing the deficiencies in the 
application, will address why the results of the research design agreed 
to under Sec. 312.82, or in subsequent meetings, have not provided 
sufficient evidence for marketing approval. Such letter will also 
describe any recommendations made by the advisory committee regarding 
the application.
    (d) Marketing applications submitted under the procedures contained 
in this section will be subject to the requirements and procedures 
contained in part 314 or part 600 of this chapter, as well as those in 
this subpart.