Recombinant DNA Advisory Committee - 01/14/93 
approval for the compassionate use of drugs under much different circumstances. Drug 
use in this country is not necessarily regulated on the basis of efficacy in experimental 
situations or in compassionate use situations. For example, there is widespread use of a 
variety of drugs for the treatment of acquired immunodeficiency syndrome (AIDS) that 
have not been proven to be efficacious in the treatment of this disease. A balance has 
been struck between the right of an individual patient to have access to an experimental 
drug or therapy and the concerns of the community about safety. Dr. Healy's decision is 
consistent with the way that compassionate drug use is being used for diseases such as 
AIDS. 
FDA STATEMENT-DR. WOODCOCK 
Dr. Janet Woodcock, Director of the Office of Therapeutics of the Center for Biologies 
Evaluation and Research, stated her intention to clarify some of the questions that have 
arisen during the course of this discussion regarding the FDA's review of gene therapy. 
The vectors that are used for gene therapy are classified as investigational biological 
products. Therefore, vectors used for human gene therapy come under statutory 
authority for regulating investigational products, investigational drugs, and biologies. The 
FDA has a defined set of regulations that are followed for the approval of human 
protocols. A 2-page FDA document was distributed summarizing single patient use and 
emergency use situations. 
Dr. Woodcock stated that the RAC plays an important complementary role to the FDA. 
The FDA focuses on manufacturing, i.e., the production of these experimental 
biologicals. The FDA does not always provide the opportunity for open public 
discussion that the RAC permits. 
The FDA's review of any investigation protocol in humans includes an assurance by the 
investigator and an understanding that any applicable regulations, policies, or approvals, 
have been obtained and followed including IRB, IBC, and RAC approval, if appropriate. 
There has been increasing pressure from patients, patient advocacy groups, and the 
general public, to increase access to experimental therapies earlier in the investigational 
stages. Traditionally, this access has been limited to drugs that were late in the 
development cycle. However, over the last 5 years, especially in human 
immunodeficiency virus related illnesses and cancer, there has been increasing pressure 
and response by the FDA to provide access to patients earlier in drug development. The 
FDA provides these therapies with the understanding that patients may be exposing 
themselves to the increased risk of toxicity. In addition, these patients may be deferring 
themselves from another more effective therapy that might be available. 
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