Testing times ahead

Abstract

The research and testing of drugs and devices within the rigid three-tier clinical trial framework is designed to ensure both the safety and efficacy of a drug or device as a prerequisite for marketing authorisation from the regulatory body. Under this criterion, experimental drugs can be given only to people participating in a clinical trial; under the jurisdiction of the US Food and Drug Administration (FDA), however, there are several very different mechanisms to grant access to experimental treatments when the standard of care may be inadequate or even non-existent.1http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/ucm051345.htm (accessed July 22).Google Scholar Such early or extended access options include emergency use, compassionate use, off-label use, and humanitarian exemptions.2DiPALS Study Group CollaboratorsSafety and efficacy of diaphragm pacing in patients with respiratory insufficiency due to amyotrophic lateral sclerosis (DiPALS): a multicentre, open-label, randomised controlled trial.Lancet Neurol. 2015; 14: 883-892Google Scholar Although such applications can provide accelerated access for patients, in the USA this process is administered by the FDA, and although efforts have been made to streamline applications, many think that the process is still overly bureaucratic. This may, in some part, have contributed to the proliferation of right-to-try legislation across many US states.3FDA to simplify ‘Compassionate Use’ drug applications.Medscape. Feb 04, 2015; (accessed July 22).Google Scholar Under this legislation, terminally ill patients who have exhausted other available treatments will have a means to apply, through their physician, to the manufacturer of drugs that have passed phase 1 of a clinical trial, bypassing the need for federal approval and moving the decision to grant access onto the pharmaceutical company. Following on from individual state laws, on July 9, 2015, the Right to Try Act of 2015, which aims to prohibit federal government agencies from taking action to prevent terminally ill patients from accessing experimental drugs, was introduced to US Congress.4States move to give terminally ill ‘right-to-try’ drugs.USA Today. Feb 20, 2015; Google Scholar, 5http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2015/07/the-right-to-try-act-of-2015-a-serious-challenge-to-fda-control-of-expanded-access-.html (accessed July 22).Google Scholar Understandably, these legislatures have divided commentators on issues of patient rights and the undermining of the FDA's authority. These arguments aside, the eventual outcomes of these deliberations will doubtless have a huge effect on patients, physicians, federal agencies, and pharmaceutical companies alike.