Recombinant DMA Advisory Committee - 01/14/93 
she is not authorized to discuss this specific case in detail. 
Dr. Leventhal inquired whether each autologous tumor cell preparation constitutes a lot. 
Dr. Woodcock said that the requirements for testing autologous cells are less stringent 
than for other biologies because of technical limitations. 
Ms. Meyers stated that she is a public member on this committee who represents 
individuals with rare diseases, the majority of whom are as desperate as Dr. Royston's 
patient. She expressed concern about the criteria that the FDA uses to grant single 
patient INDs. Patients with AIDS and cancer are granted permission routinely to take 
drugs that are not approved and that are in the early stages of testing. However, the 
FDA stopped the production of a drug that was being produced by an investigator at 
Johns Hopkins University because of failure to pass good manufacturing practices. This 
drug, which was routinely manufactured for years, has kept hundreds of children alive 
who were diagnosed with a rare disease. Apparently, what is true for certain politically 
important groups is not true of the politically unimportant groups. 
Ms. Meyers stated that her recommendation is that the RAC should send a letter, which 
includes recommendations on policy, to the incoming administration. There is no reason 
that the RAC should be forced into taking any action on this issue prior to January 20. 
Ms. Meyers explained that there is an enormous difference between drugs and gene 
therapy. If a patient is adversely affected by a drug, only that patient is usually affected. 
With gene therapy, nothing is known about the long-term affects to the patient, health 
care workers, the patient's spouse, or the next generation. 
Ms. Meyers explained that the Foundation for Economic Trends has gone to court in 
order to ensure that gene therapy deliberations are held in public, because society has a 
great stake in the outcome. However, the RAC is being urged to turn away from the 
public and allow these decisions to occur in private. 
Ms. Meyers said that Dr. Royston's patient is not an inpatient. Therefore, she is allowed 
to go out into the public following treatment. The FDA approval of this protocol is 
analogous to approving an envelope without knowing the message of the letter. The 
members of the RAC are the experts that know what that message should be and the 
potential danger that can occur if that message is incorrect. The RAC was completely 
cut out of this approval process. 
Ms. Meyers explained that Dr. Royston's protocol was deferred in 1991 because he was 
not prepared. The RAC told Dr. Royston to perform additional experiments. In 1991, 
Dr. Royston did not even have a patient in mind; he wanted to rush at that point. Dr. 
Royston said in November 1991 that he would resubmit his protocol in 3 months; he 
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