US President Donald Trump speaks before signing the 'Right To Try Act' on May 30, 2018. (Photo by... [+] Nicholas Kamm/AFP) By Martina Rozumberkova Pharmaceutical and biotechnology companies need to start preparing for the compliance and reporting requirements of a controversial new law. US President Donald Trump on May 30 signed into law the Right to Try Act (H.R.5247), which will allow eligible patients, through drug manufacturers, to seek access to drugs that have not yet been approved by the Food and Drug Administration (FDA). In order to be eligible, a patient must be diagnosed with a disease or condition that they are likely to die from within a matter of months, or that causes significant irreversible morbidity likely to lead to a severely premature death. The new federal law specifies that eligible patients will be able to use drugs that have completed phase-one clinical trials and are currently the subject of an active FDA application or undergoing clinical trials. Prior to this new legislation, more than 38 states already had laws allowing certain patients access to experimental treatments. Additionally, the FDA already had a pathway in place for granting expedited access to experimental drug treatments for patients with terminal illnesses. However, under the existing regime, only about 3% of terminally ill patients have historically been able to gain access to experimental drug treatments in clinical trials. This bill is designed to greatly expand that number. The Right to Try Act was not met with unanimous approval. Prior to its passing, the bill was stalled in the House due to concerns for patient safety. Shortly after the Right to Try Act was signed, an updated version was introduced that added additional patient protections, including requirements to more swiftly report adverse events to the FDA. Immediately after the House of Representatives passed the Act, the National Organization for Rare Disorders, an organization supporting more than 30 million patients with rare diseases, issued a statement to the House stating, “we have strongly opposed all Right to Try legislation, as we do not believe that it will safely and genuinely increase access to investigational therapies outside of clinical trials.” Regardless of any lingering controversy, pharmaceutical and biotechnology companies will need to start preparing for the compliance requirements that this legislation brings. While the details of those requirements have not yet been fully disclosed, known requirements include annual reporting to the FDA when a patient receives an unapproved drug through the new alternative pathway and real-time reporting of adverse events. The Right to Try Act also requires the FDA to report on its website the use of drugs pursuant to this bill as data is reported by the manufacturers. We will learn more about the new requirements as they are finalized in the coming months, but one thing is clear today: the Right to Try Act will bring additional compliance and reporting requirements to pharmaceutical and biotech companies whose drugs fall within the purview of the new law. Martina Rozumberkova is a Director for Compliance, Forensics and Intelligence at Control Risks, the global specialist risk consultancy. She is based in Boston.
Pharmaceutical companies face new compliance and reporting requirements after recently passed federal legislation in the US means eligible patients can apply to access drugs that have completed phase one clinical trials and are the subject of an active FDA (Food and Drug Administration) application. The Right to Try Act lets eligible patients, through drugmakers, seek access to treatments not yet approved by the Food and Drug Administration if they are likely to die within months or face a significantly premature death from an illness or condition. Before the act, 38 states had laws that allowed patients to try experimental treatments.