AIDS TREATMENT NEWS Issue #206, September 2, 1994
John S. James

[The meeting will be at the Holiday Inn Plaza Ballroom, 8777 Georgia Ave., Silver Spring, Maryland, starting at 8:30 a.m. on September 12 and September 13; all parts of this meeting are open to the public, and there might be times to make a brief statement to the Committee even without prior arrangement. Also, written statements can be submitted after the meeting. Address them to Antiviral Advisory Committee, c/o Lee Zwanziger, Ph.D., Advisors and Consultants Staff (HFD-9), Parklawn Building, Room 8B45, 5600 Fishers Lane, Rockville, MD 20857; or fax them to 301/443-0699. For more information, call Dr. Zwanziger at 301/443-5455.]

The following is an outline of our talk to the Committee:

* While everybody agrees that better data is needed, what AIDS Treatment News is hearing from people with HIV disease is the strong, widespread feeling that access remains most important to them, and must not be sacrificed. This is true whether or not people are highly educated about treatments. When conventional treatments are failing, no special education is needed to know that other possibilities have shown promise, and to want to try them.

* Parallel track and other pre-approval access programs cannot replace accelerated approval, as companies are increasingly refusing or unable to pay for them.

* The concerns around accelerated approval of ddC and other AIDS drugs reflect the inadequacy of the drugs, not fundamental flaws in the approval process.

* Within the research and medical communities, there is a rapidly growing consensus that plasma RNA is a much better indicator of antiviral effect than the measurements we have had in the past. This new marker will almost certainly improve future decisions about antivirals. Accelerated- approval decisions will not be based on any one marker, however, but will reflect a judgment involving all information available. We should avoid premature decisions or doctrines which would limit flexibility and prevent timely and effective use of new technologies as professional consensus develops.

For example, it may be tempting to impose the doctrine that whenever accelerated approval is given, it must be in the context of a development plan that will eventually use clinical endpoints to prove or disprove the drug's superiority to standard treatment. In some cases, this approach may make sense; in other cases, different ways of organizing post-marketing studies may be more effective in obtaining the information physicians need to optimize use of the drug. And the human costs of clinical-endpoint trials (which tend to keep people on protocols that don't work for them so that body counts can be obtained) are often underestimated. These costs are highest for marginal drugs, where the resulting information is of least value.

* A major problem in drug development is that most credible treatment possibilities do not even begin clinical research, because the cost barriers to eventual approval are too high. This is especially tragic in view of the historical experience that important medical advances are seldom predictable -- meaning that the few drugs which do get big-corporate backing are not the cream of the crop, but closer to a random selection. Critical treatment advances may be quietly lost, if they do not look good ahead of time and therefore never get the chance to begin developing credibility. Accelerated approval can reduce cost barriers by deferring major expenses until after there is an income stream, encouraging early testing by making the ultimate development path more feasible.

* In addition, the movement toward individualized therapy will make accelerated approval more important than it has been in the past, as physicians and patients rely less on averages and more on viral and other measurements for rapid indications of which treatments are working for which people.

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