The Hazards of Misguided Compassion

The plight of patients with human immunodeficiency virus (HIV) infection is heart-rending, and so the patients, their families, and doctors want to be free of restrictions on the availability of drugs. Compassion combined with lobbying has led to exemptions from the usual way of evaluating the safety and efficacy of new treatments, so that drugs to treat HIV infection are available outside of randomized trials, and treatment decisions must be based on smaller randomized trials, which lack statistical power, and on nonrandom trials that may be biased. Through misguided compassion, the medical community may never learn enough about new drugs for HIV infection to use them properly and to understand whether they do more good than harm.

The plight of the many patients with human immunodeficiency virus (HIV) infection is heart-rending to all who care for them. The desire of the patient, the patient's family, and the doctor to do something can be overwhelming. Frustration about the lack of a preventive vaccine or curative treatments has led to demands that new therapies for the acquired immunodeficiency syndrome (AIDS) be exempted from the usual process of rigorous scrutiny used in the evaluation of new drugs. Compassion combined with heavy lobbying pressure by activists has led to the granting of exemptions. What changes in drug testing have been granted and how do the new policies affect the evaluation of treatments for HIV infection?

Early release of drugs has been an important demand of activists [1]. Because of the desperate cry for new therapies, drugs have been rushed to market even before they …