Gay Men's Health Crisis Treatment Issues, Vol. 5, No. 6 - August 30, 1991
David Barr

Bristol-Myers Squibb, maker of the drug, has made ddI available to patients through an expanded access program developed by the company, the FDA, and AIDS activists. Through this program, the drug has reached over 20,000 people during the past two years. FDA approval will make ddI available to many more people who might benefit from it and will allow doctors to prescribe the drug in combination with AZT. There is, however, no conclusive evidence so far showing that AZT in combination with ddI is better than either drug alone.

The most prevalent side effects of ddI are pancreatitis, peripheral neuropathy and gastrointestinal (GI) problems. The GI difficulties are most probably related to the buffer used and should be resolved when Bristol releases the new formulation of the drug.

This is the first time that an AIDS treatment for approval based solely on the improvement of T4 counts. Most of the data presented to the committee were from phase I trials and from the expanded access program. Therefore, the amount of data was not sufficient to reach a standard worthy of drug approval. However, supplementary data were presented from an interim analysis of ACTG trial #117, a phase Ii study comparing ddI with AZT in patients on long-term AZT. These data showed a higher sustained T4 cell rise in ddI patients as compared to patients taking AZT, and proved instrumental to the committee's decision. The continuing ACTG ddI trials will eventually offer a better picture about the drug's effectiveness and clinical outcomes.

Participants in the committee meeting were uncomfortable with the small amount of useful data presented. However, the importance of ddI's approval was apparent to those involved. While we would all like more and better data to help in making our treatment decisions, the reality is that patients and physicians have had to use insufficient data to make every single treatment decision since the first day of the epidemic. Unfortunately, we do not have time to wait until better data are available. The drugs that look most promising must be made available as soon as possible in order to provide patients and doctors with choices. At the same time, we must ensure that researchers continue to collect solid data, in ways which meet both the immediate and future needs of people with AIDS.

The development of ddI should be seen as the model for all AIDS drugs in the future. Expanded access programs, the use of surrogate markers for approval and an expeditious approval process must be the status quo in developing treatments for life- threatening illnesses. Further, community involvement is important at every step to facilitate a drug development process that best meets the needs of patients. Some aspects of the approval process for ddI went well, and others did not. We must learn from our mistakes and move on. FDA should approve ddI soon. There is still no word on the price of the drug.

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