FDA Issues Rules to Speed Drug Testing

On October 19 the U.S. Food and Drug Administration announced new regulations to speed the development and commercial release of some drugs. Initial reaction by AIDS organizations has been mostly negative (see The New York Times, October 20, page 1). Project Inform expressed limited support.
The rules essentially codify what was already done with AZT. They ask drug companies to work more closely with the FDA to design an expanded phase II (efficacy study), after which drugs could be released without going through a phase III (larger efficacy study). But there are no commitments to use the new procedures in any particular situations; and some have questioned whether the FDA has enough money or staff to handle accelerated approvals.
These rules were adopted after months of bitter infighting behind the scenes. Powerful FDA officials who never wanted this reform may try to sabotage it.
The previous "treatment IND" (now called "treatment protocol") remains as before. In theory those rules permit the early release of treatment for life-threatening or severely debilitating illness; in practice they have almost never been
used since they were announced over a year ago.
The current new rules do not change the real intentions and operations of the agency concerning early release of drugs before full commercial approval. The FDA still adamantly refuses to allow early release through the treatment protocol (treatment IND) unless it believes that the data supports approval for full commercial marketing and only paperwork remains. The treatment protocol can be used only to get the drug to patients during the time of this considerable paperwork; it is only a steppingstone toward commercial release. The FDA sees no drug as eligible for commercial release (and therefore none eligible for a treatment protocol) at this time.
For reasons outlined in "Beyond an Unreasonable Doubt", below, we believe that this new FDA ruling will do nothing for AIDS in the foreseeable future, probably nothing for at least two years. Yet it may be an important long-range step toward improving U.S. drug regulation.
Martin Delaney, co-founder of Project Inform, called the rules "only a small step, not the whole solution to the problems we have been addressing for the last three years... Nevertheless, they stake out new philosophical ground for the agency, acknowledging for the first time that drugs must be evaluated flexibly, that the rules should be different when a life-threatening illness is involved, and that approval should be based on a risk-benefit analysis rather than rigid, standard rules."
For a copy of the new rule, which will appear in the Code of Federal Regulations, 21 CFR 312 as a new Subpart E, "Drugs Intended to Treat Life-Threatening and Severely-Debilitating Illnesses", send a self-addressed stamped envelope to AIDS Treatment News, P.O. Box 411256, San Francisco, CA 94141. Ask for the FDA rule.
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