Many health care systems include programs that allow patients in exceptional circumstances to access medical interventions of as yet unproven benefit. In this article we consider the ethical justifications for—and demands on—these special access programs (SAPs). SAPs have a compassionate basis: They give patients with limited options the opportunity to try interventions that are not yet approved by standard regulatory processes. But while they signal that health care systems can and will respond to individual suffering, SAPs have several disadvantages, including (...) the potential to undermine regulatory and knowledge-generation structures that constitute significant public goods. The “balance” between these considerations depends in part on how broadly SAPs are used, but also on whether SAPs can be made to contribute to the generation of knowledge about the effects of health interventions. We argue that patients should usually be required to contribute outcome data while using SAPs. (shrink)

Researchers and ethicists have long been concerned about the expectations for direct medical benefit expressed by participants in early phase clinical trials. Early work on the issue considered the possibility that participants misunderstand the purpose of clinical research or that they are misinformed about the prospects for medical benefit from these trials. Recently, however, attention has turned to the possibility that research participants are simply expressing optimism or hope about their participation in these trials. The ethical significance of this therapeutic (...) optimism remains unclear. This paper argues that there are two distinct phenomena that can be associated with the term ‘therapeutic optimism’—one is ethically benign and the other is potentially worrisome. Distinguishing these two phenomena is crucial for understanding the nature and ethical significance of therapeutic optimism. The failure to draw a distinction between these phenomena also helps to explain why different writers on the topic often speak past one another. (shrink)

Drug-resistant tuberculosis has a high mortality rate. Most medicines used to treat it are poorly tested and have terrible side effects. Activists have campaigned for patients with drug-resistant TB to have access to experimental drugs, particularly one called bedaquiline, before these have been approved by regulatory authorities such as the Food and Drug Administration in the United States and the Medicines Control Council in South Africa. Some activists have also campaigned for bedaquiline to be approved by regulatory authorities before testing (...) of the drug is completed. These campaigns raise ethical concerns about whether patients should be offered experimental, unapproved, medicines for the treatment of life-threatening illnesses, and if authorities should approve drugs for life-threatening illnesses when vital questions about safety and efficacy remain outstanding. (shrink)

The year 2014 brought a new development in the bioethics “laboratory of the states.” Five states adopted “right to try” laws intended to promote terminally ill patients' access to investigational drugs. Many more state legislatures are now considering such laws. The campaign for right to try laws is the latest move in an ongoing effort to give seriously ill patients access to drugs whose safety and effectiveness remain largely unknown. Although scientists and policy‐makers oppose the right to try approach, it (...) has proven quite popular among state legislators and the public. (shrink)