CONGRESSIONAL HEARING ON PARALLEL TRACK, JULY 20

On July 20, the U. S. House of Representatives Subcommittee on Health and the Environment, chaired by Henry A. Waxman (D- Los Angeles) held a public hearing on the "parallel track proposal for clinical drug development." The first panel was James Mason, Assistant Secretary for Health, testifying for the government, with FDA Commissioner Frank Young, NIAID director Anthony Fauci, and National Cancer Institute Director Samuel Broder available for questions. The second panel was Jim Eigo of the Treatment and Data Committee of ACT UP/New York, and Martin Delaney of Project Inform, representing AIDS advocacy organizations. The third panel was John Petricciani, vice president of the Pharmaceutical Manufacturers' Association. We did not attend this hearing but have received copies of the prepared text submitted in advance by most of the speakers.

The opening statement by Congressman Waxman is an excellent neutral summary of the issues as they are conventionally viewed, so we reproduce it in full:

"Today's hearing is on the controversial and critical issue of release of experimental drugs. The hearing is
specifically about recent proposals referred to as the parallel track for drug development.

"Food and Drug Administration law does not allow drugs to be sold until they are proven to be safe and effective.
The same law prohibits distribution in any manner of drugs without permission of the FDA. This law was adopted to protect consumers from dangerous products and from snake-oil remedies. It was intended to establish a straightforward policy as to when manufacturers can profit from -- and when consumers can take -- drugs whose effectiveness is unknown.

"Exceptions have been carved out of the general rule. Drugs whose safety and effectiveness are unknown may be distributed for research purposes. Drugs whose safety is proven, but whose effectiveness is undemonstrated, may be distributed outside of strict medical trials under various labels -- as Treatment Research or as Compassionate Use or as Open Protocols.

"But the law and its exceptions have run head on into the AIDS epidemic. Thousands of Americans find themselves without useful approved treatment and with steadily declining health. A handful can get into controlled trials, but most can just read about drugs they cannot get.

"During the course of the epidemic, this rationing has been scientifically and morally justified as necessary to
the conduct of trials and appropriate for quickly meeting the needs of future Americans with AIDS. We have lived with
a policy of limited distribution today so that we will have adequate information for tomorrow.

"But now, many people -- patients, their families, and researchers -- are questioning this policy. They argue that scientific trials do not require that everyone else be denied access to potential therapies; indeed, they say, the trials are better conducted with willing volunteers rather than desperate ones. And, they continue, it is, therefore, morally wrong to withhold promising drugs from patients with nothing else to turn to.

"From this questioning has grown the Parallel Track, a proposal that drugs be available for some distribution when they are known to be safe and when trials for effectiveness are fully enrolled. Doctors treating patients who receive such drugs would be asked to provide information back to researchers, thus forming a source of information that is parallel to the formal trials.

"This is an important proposal. It could change ground rules on research, clinical care, markets, and insurance.
It could also provide access to drugs -- the good ones and the worthless ones- -long before data are available. If it works it could revolutionize drug development. If it fails it could cripple AIDS research for some time.

"Today's hearing is to form a record of the proposal, its reach and its limits as well as the questions that remain unanswered. I hope that with this hearing we can begin to form a consensus as to how AIDS drug development will proceed."

Congresswoman Nancy Pelosi (D-San Francisco) gave specific examples of problems with the current system:

"Each month, I hold an open meeting in my district for people with symptomatic HIV infection and their advocates. The group is very knowledgeable about HIV-related drugs and their potential uses. Their top priority is access to
potentially lifesaving experimental drugs. Their stories are compelling.

"One young man participated in a NIH-sponsored phase I trial of DDC at Stanford. From his perspective, the drug worked well. He felt subjectively much better and his laboratory tests indicated a decline in p24 antigen level (a measure of viral activity) and limited increases in T-cell count (a measure of immune functioning). After 13 weeks, he received a phone call telling him that his participation in the study was no longer needed. Since then he has started AZT several times but has had difficulty tolerating even half of the standard dose. Although he has not yet received a formal AIDS diagnosis, as of last month his T-cell count dropped to 18. He is receiving no anti-retroviral therapy. He and his treating physician want him to try DDI or to go back on DDC. Given his history of prior use of AZT and DDC, he is a poor candidate for other clinical trials. Why can't this man and his physician be given access to either DDC or DDI?

"Another man from this group has been on AZT for just under two years. He did very well on this drug. However, in the last two months, his T-cell level has declined from over 300 to around 130. He is also beginning to experience symptoms which he has not had in the last two years, including disabling levels of fatigue. He and his physician believe it is time for him to switch to another antiretroviral drug. Why can't this man and his physician be given access to DDI? "

We have not seen Dr. Mason's testimony. But Jim Eigo of ACT UP/New York's Treatment and Data Committee summarized the status of the parallel track after the hearings. According to his summary:

* Dr. Mason's testimony showed that the Public Health Service is committed to some form of parallel track -- but that what parallel track means is so far undecided. The Public Health Service asked that an FDA advisory committee, to include AIDS advocates, report to Dr. Mason by August 21 and prepare recommendations on the parallel track.

* DDI will not be available under the "parallel track", at least not immediately, but it will be available under the "treatment IND" for people who are AZT-intolerant. However, FDA commissioner Frank Young admitted that Bristol-Myers has not yet applied for a treatment IND.

* The day after the hearing, Jim Eigo "argued with Dr. Ellen
Cooper of FDA that DDI has to be made available under compassionate use to at least two other groups of people: those too sick to abide the rigors of the DDI clinical trials and those who can't go off medication that the DDI trials forbid. She said she believed that we could work that out."

* Parallel-track drugs will be open label (meaning that patients will know what they are getting, instead of being randomly assigned to two or more treatments and not told which).

* Parallel-track drugs will be judged individually -- probably by an FDA advisory committee with AIDS-community representation. Drugs must show "some indication of efficacy." Persons receiving these drugs will sign informed-consent forms.

* There must be some way to prevent "secondary transfer" (i.e. an underground market) of drugs obtained under the parallel track.

On July 31 we spoke with Dr. Mason's office and learned:

* Dr. Mason wanted to emphasize that setting up the parallel track must not interfere with or delay access to DDI.

* The committee to advise Dr. Mason by August 21 has not yet been formed. However, it will probably consist of an FDA antiviral advisory committee which already exists, plus AIDS advocates brought in as consultants.