"Right to Try" laws.

Readers of the journal should be aware of developments in the long-standing efforts by groups opposed to US Food and Drug Administration (FDA) regulation of drugs. Recently, the States of Colorado and Louisiana have approved legislation that gives patients suffering from life-threatening illnesses the right to have access to experimental drugs that have passed phase I testing, and other states such as Arizona and Missouri may soon follow suit. These bills seek to allow access to experimental drugs to patients who have exhausted all available treatment options. The bill does not require the manufacturer to make a drug available to an eligible patient, but it does allow those manufacturers who elect to take advantage of the law to charge the patient for the costs of the drug, and it does not mandate coverage by health insurers. In short, it allows drug manufacturers to market their drugs prior to the efficacy testing needed for FDA approval. Society members may recall a very similar US federal bill that was developed nearly a decade ago, sponsored by Senators Brownback and Inhofe, entitled the ‘‘Access, Compassion, Care and Ethics for Seriously Ill Patients Act.’’ This bill also sought to permit marketing of drugs after phase I testing. At the time, the Society prepared an extended Position Paper opposing this legislation, describing the history of clinical trials and the reasons why controlled trials are essential to ensure that marketed drugs are safe and effective, and laying out the likely adverse consequences for the public health of any change in the law that would remove efficacy testing of drugs from the jurisdiction of the FDA. That bill never made it through the congressional committee system and as a result never came up for a vote. By contrast, the Right to Try legislation has already been approved in Colorado and Louisiana, and it sets up a potential conflict between state and federal laws on this issue. The provision to permit access to experimental agents for seriously ill patients exists in the current federal law. This system involves a petition by the treating physician attesting to the opinion that the risks to the patient do not outweigh the potential benefits, and the petition must be approved by an Institutional Review Board. That is, the system is closely monitored to protect the interests of the patient. Also, adverse events and outcomes must be provided to the FDA, ensuring that the data are recorded. Nonetheless, there have been long-standing concerns that these hurdles make access difficult. The new laws would appear to be designed to largely remove such oversight. FDA regulation of drugs has been controversial ever since the founding of the agency and especially since the requirement for proven efficacy became the law of the land (USA) in 1962 via the Kefauver–Harris Amendments. We suspect that most members of the Society for Clinical Trials, and indeed the readership of this journal, support these laws that enshrine the need for evidence-based medicine, and prevent the marketing of untested agents to exceptionally vulnerable patients. However, the arguments that support the restriction of access of drugs to patients until FDA approval are complex and hard to articulate convincingly to members of the public unfamiliar with the rationale for clinical trials and the need for careful evaluation of new drugs before they become available outside of clinical trials. Indeed, a similar debate has been taking place in the United Kingdom where the so-called Saatchi Bill, sponsored by Lord Saatchi and motivated by the recent death of his wife to cancer, seeks to make it easier for doctors to prescribe untested drugs, focusing primarily on removal of the threat of litigation. These legislative efforts, although they are phrased in the context of patients with life-threatening disease, really challenge at a fundamental level our current systems of drug evaluation and approval. As interested parties, it is important for us to be aware of legislation like this that pursues the availability of drugs untested for efficacy outside of the normal regulatory processes. It is critical that any system for expanded access of treatments for patients with life-threatening diseases or with conditions for which no alternative treatments exist have safeguards in place.