Biomedical Engineering Reference
In-Depth Information
1996, and the ongoing debate about both the continuation and the
proper role of RAC, the OTC deficiency protocol simply disappeared
from public view. Here are several questions about the Penn protocol for
which there were no clear answers in the years 1996 through 1999:
•
Had the FDA given Penn approval to proceed with the OTC deficiency
protocol in response to Penn's Investigational New Drug Application?
•
Had the design of the study been changed after public RAC review?
•
Had the consent form been changed after RAC review?
•
Had the clinical trial been initiated?
•
If so, had any serious adverse events occurred?
In fairness to the NIH, I should note that the new guidelines published
in the
Federal Register
on October 31, 1997 did require researchers to
report to the NIH and RAC all post-RAC-review changes and serious
adverse events.
There was also more general evidence that RAC's role had been weak-
ened and the national oversight system was less effective from 1996 on.
There was no annual audit of the human gene transfer field conducted
in 1996, 1997, 1998, or 1999. Thus, policy makers, the public, and
researchers around the world lost the kind of comprehensive overview
that RAC had provided in June 1995. Such an audit would have been
difficult to conduct during these years for at least three reasons. No
senior person (PhD or MD) was appointed to direct the staff that served
RAC for more than two years after the former director's departure at
the end of June 1996. Second, in 1995, the FDA had withdrawn its agree-
ment to cooperate with the NIH and RAC in developing a public, online
database to track serious adverse events in human gene transfer trials;
NIH efforts to create such a database alone proceeded slowly and had
not borne fruit four years later. Third, as noted above, the size of RAC
was reduced from twenty-five to fifteen members early in 1997; thus, the
shared workload that allowed the 1995 audit to be performed was more
difficult to achieve.
In 1998 and 1999, the refusal by one researcher and one company to
provide public disclosure of serious adverse events in their gene transfer
trials was symptomatic of additional problems in the public oversight
system. In preparation for their September 1999 meeting, RAC members
were asked to sign a confidentiality agreement stating that they would
