[Code of Federal Regulations]
[Title 21, Volume 7]
[Revised as of April 1, 2008]
From the U.S. Government Printing Office via GPO Access
[CITE: 21CFR601.41]

[Page 40]
 
                        TITLE 21--FOOD AND DRUGS
 
CHAPTER I--FOOD AND DRUG ADMINISTRATION, DEPARTMENT OF HEALTH AND HUMAN 
                          SERVICES (CONTINUED)
 
PART 601_LICENSING--Table of Contents
 
  Subpart E_Accelerated Approval of Biological Products for Serious or 
                       Life-Threatening Illnesses
 
Sec. 601.41  Approval based on a surrogate endpoint or on an effect on a 

clinical endpoint other than survival or irreversible morbidity.

    FDA may grant marketing approval for a biological product on the 
basis of adequate and well-controlled clinical trials establishing that 
the biological product has an effect on a surrogate endpoint that is 
reasonably likely, based on epidemiologic, therapeutic, 
pathophysiologic, or other evidence, to predict clinical benefit or on 
the basis of an effect on a clinical endpoint other than survival or 
irreversible morbidity. Approval under this section will be subject to 
the requirement that the applicant study the biological product further, 
to verify and describe its clinical benefit, where there is uncertainty 
as to the relation of the surrogate endpoint to clinical benefit, or of 
the observed clinical benefit to ultimate outcome. Postmarketing studies 
would usually be studies already underway. When required to be 
conducted, such studies must also be adequate and well-controlled. The 
applicant shall carry out any such studies with due diligence.