The Politics of Access

Drug regulation is one arena where the sheer effect of activism would be hard to dispute. Activists were not the only ones calling for change in the FDA, but they were the key players in pushing for the approval of AIDS drugs at an earlier stage in the drug development pipeline. And although some procedures allowing early access to experimental therapies were already on the FDA's books, others, such as expanded access and accelerated approval, are new to medicine as a result of AIDS, and have resulted in the provision of such therapies to much larger groups of patients than had been the case in the past. (Expanded access has since been instituted for drugs treating other diseases, such as Alzheimer's, while a drug for multiple sclerosis has received accelerated approval.^[20] This book has not focused on drugs that treat or prevent opportunistic infections, but it is perhaps there that the significance of activist efforts has been felt most keenly: earlier access to anti-infective drugs, though not without risk, has meant a longer life and better quality of life for many people with AIDS and HIV.^[21] In addition, activist efforts (for better or worse) propelled the adoption of interpretative mechanisms such as surrogate markers that, in turn, have hastened the approval process. Absent the AIDS activists, CD4 counts would not have been accepted as a surrogate marker of treatment efficacy in 1991; without the adoption of the CD4 marker, the AZT/ddC combination would not have been licensed in 1992.

Finally, activists have insisted on broadening the demographic characteristics of clinical trial participants, hence broadening access to experimental therapies. In fiscal year 1988, 82 percent of the new

subjects recruited into ACTG trials were white. By 1994, only 56 percent were white (26 percent were African-American, 16 percent were Hispanic/Latino, and 2 percent were "other"). Over the same period, the ratio of men to women in trials was reduced from about thirteen to one to about five to one.^[22] While activists cannot take all the credit for the demographic diversification of trials, there can be little doubt that politicization of the issue by activists brought about a climate in which change became perceived as a necessity. The minutes of the ACTG Executive Committee meetings recorded Daniel Hoth's exhortations to investigators to diversify trials: on one occasion Hoth noted that the ACTG was "under a great deal of political pressure to address this issue successfully"; on another, he described a phone conversation with the assistant secretary of health, "who had called to ask what the ACTG was doing to increase minority participation."^[23] These new emphases at NIAID were matched by changes at the FDA: in March 1993 agency officials announced that, instead of excluding women of childbearing age from trials, they would henceforth insist on their inclusion in nearly all new drug applications. "We now believe that there are ways to protect the fetus and to include women in studies at the same time," David Kessler told the press.^[24]

Beyond the very real concerns about risks to patients, the key criticism of expedited access to experimental therapies has focused on the potential threat to the research process. If, for example, patients were able to obtain drugs like ddI and ddC through expanded access programs, why would they bother to sign up for long-term clinical trials? This criticism ignores the other reasons why patients might enroll in trials—for example, altruism or the desire to obtain access to high-quality medical care and intensive monitoring of their medical condition. But in the end, as Anthony Fauci notes, "the proof of the pudding is that we were right. [Expanded access] hasn't hampered anything"; research subjects have enrolled and the trials have still been completed.^[25]