Oversight of Right‐to‐Try and Expanded Access Requests for Off‐Trial Access to Investigational Drugs

Abstract

For decades, the U.S. Food and Drug Administration (FDA) has provided an “expanded access” pathway that allows patients who meet qualifying conditions to gain access outside a clinical trial to an investigational medical product being tested to see if it is safe and effective for a specific use. The Right to Try (RTT) Act, enacted in 2018, created a second mechanism for off‐trial, or non‐trial, access to investigational drugs. In contrast to the expanded access pathway, the federal RTT pathway does not require the involvement of the FDA or an institutional review board (IRB). Given that physicians, drug manufacturers, and medical institutions now have a choice whether to assist individual patients through the expanded access or the federal RTT pathway, we review the differences between these options and discuss the benefits and burdens of IRB involvement in requests to access interventions through the pathways. We also suggest ways in which IRB oversight may be further improved.

Compassionate Use of Investigational Drug During Emergency Conditions and Associated Ethical Aspects, Challenges, and Benefits

“…In 2018, a second alternative option was approved for CU of drugs which are under Phase I clinical trial. In this case, no authorization is required from the FDA or IRB, only the patients have to pay only the costs for investigational drugs or devices to the respective companies [20]. It was the last stage in the steps of introducing Right To-try laws in individual states.…”

Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special article: compassionate use and clinical trial on CAR-T cells

“…Albeit, in May 2018, the American Congress promulgated the law Right to Try Act, which permits the patient to request the compassionate use of a product, prescinding the FDA proceedings, as the American society considered as just the individual's endeavor to gain access to an experimental product without the interference of a state agent. 9 The objective of this law is to facilitate patient access to experimental medications. In this case, it is also recommended that consent be obtained from the institution's CEM, despite not being obligatory, but the solicitation should be based on at least a phase I clinical study.…”

“…Critics of the law Right to Try fear that its implementation might discourage patients from participating in clinical trials, as they could be allocated to the control group which receives the placebo, in addition to the attribution load imposed on them (exams and return visits to attend to the necessities of the study), as well as by the pharmaceutical industry for sponsoring them. 9 Another concern refers to the risks for the patient that the use of an experimental product could entail, as the requisition of its use would not have been scrutinized by the agency, whose staff boasts qualified professionals to analyze each case. Moreover, the attending physician would not have the commitment to inform the agency of eventual adverse effects related to the use of the product.…”

Understanding the challenges and ethical aspects of compassionate use of drugs in emergency situations

“…Important features of compassionate use in different countries are provided in Table 2 . [ 2 4 19 20 21 26 27 ]…”