Recombinant DNA Advisory Committee - 01/14/93 
for compassionate plea exemption to the procedures of these NIH Guidelines, see Section 
III-A, or request for any such emergency use of gene therapy in a human subject or subjects, 
shall be directed to the NIH RAC in the manner prescribed in these NIH Guidelines, see 
Section III-A, and shall comply with all public notice procedures. 
Ms. Matthews stated that if there is any diminution of the public access to RAC 
deliberations in contravention of the existing law, the Foundation on Economic Trends 
would file a law suit against the NIH. 
X. STATEMENT-DR, HENRY MILLER 
Dr. Walters called on Dr. Henry Miller of the FDA to present his statement to the RAC. 
In the years since Asilomar, recombinant DNA has been defined and circumscribed as 
an area that requires special attention and regulation. Superimposing this additional 
regulation has introduced inevitable delays and regulatory disincentives for many 
activities involving recombinant DNA There are essentially four layers of regulation for 
gene therapy: IBC approval, IRB approval, NIH approval, and FDA approval. 
In much of the rest of the Federal government, there has been a move to stay away from 
imposing a separate, discreet, regulatory, paradigm for recombinant DNA If Dr. 
Royston's compassionate request had been for a biologic other than gene therapy, the 
protocol would not have required evaluation by his IBC or the RAC. There may be 
subsets of gene therapy that may be relegated only to the usual, very comprehensive, 
very meticulous, methods of evaluation that can be accomplished by the local IRB and 
the FDA. The RAC should identify some of these rationally circumscribed subsets of 
gene therapy protocols. 
XI. STATEMENT-HUSBAND OF DR. ROYSTON'S PATIENT 
The husband of Dr. Royston's patient introduced himself to the members of the RAC. 
He added that he did not come to this meeting to be critical of the RAC or to try to 
bury it. As a point of clarification, the patient's husband stated that the informed 
consent document that was dated August 10, 1992, granted permission to Dr. Royston to 
grow his wife's tumor cells in culture. The informed consent document did not grant 
permission for the gene therapy protocol. His wife was granted a compassionate plea 
exemption, both by the FDA and the NIH. Therefore, he is not trying to obtain anything 
additional for her. What Dr. Healy did was not only thoughtful and compassionate, but 
clearly legal. 
He stated that he is a lawyer and is quite knowledgeable in the biology of his wife's 
disease. He became deeply involved in his wife's therapy, i.e., radiation treatments, in 
January 1992. He was aware of the RAC through the Federal Register but was not able 
Recombinant DNA Research, Volume 17 
[29] 
