HJBR Sep/Oct 2020

HEALTHCARE JOURNAL OF BATON ROUGE I  SEP / OCT 2020 21 under compassionate use. Under the federal Right to Try law, there is not a requirement to secure prior approval from the FDA or to obtain review from an IRB, thereby making the process of obtaining the therapeutic less burdensome for the local site. Remov- ing barriers to access will typically speed up the process of obtaining an investigational agent, and allow the patient to start treat- ment sooner. However, under Right to Try there is no mechanism in place for oversight about drug safety since no approval is grant- ed from either the FDAor an IRB. Clinical trials are extremely important to gain further knowledge about therapeutics and their effectiveness and safety in popula- tions of patients. That being said, many indi- vidual patients are not candidates for clinical trials due to rigorous rules about who may or may not participate, distance of clinical trial sites from a patient’s home, or avail- ability of new therapies. The Right to Try act now allows for terminally ill patients to access promising new therapeutics without participating in a clinical trial. Many termi- nally ill patients are too sick to participate in a clinical trial, or do not have the luxury of time to wait for FDA drug approval, which can take 5–10 years from initial clinical trial study initiation. What are the pros and cons of Right to Try? Zatarain Right to Try does not require per- mission of the FDAprior to obtaining a ther- apeutic from a drug manufacturer. There may be less paperwork to process prior to obtaining the therapeutic, often translating into a patient getting started more quickly on the medication. However, the drug man- ufacturers are not obligated to grant access of a requested medication to a patient, de- spite this new law. The law also protects the prescribing physician and drug manufactur- er from liability associated with choosing to provide or not provide the medication to a patient. Expanded access has been around a lot longer. What are the pros and cons of this option? Zatarain The expanded access program has been available since the 1970s, with reform over the decades. It involves FDA oversight to ensure safety and reporting of adverse events of therapeutics, as well as local institutional review boards (IRBs) to comply with adequate monitoring while on the therapeutic. Although there is more pa- perwork involved, and several layers of ap- proval from multiple entities, this provides the safest route for accessing a therapeutic outside of a clinical trial. Thankfully, nearly 99 percent of single patient expanded access applications were approved by the FDAfrom 2010–2015, allowing more patients access to promising agents. The patient’s physician starts by contacting the drug manufacturer of the desired therapeutic to determine if it qualifies as expanded access, also known as compassionate use. Similar to the Right to Try act, drug manufacturers are not obli- gated to grant access of a requested medica- tion through expanded access programs. A protocol is then developed by the physician, with input from the drug manufacturer, to follow similar guiding principles as would be used in an active clinical trial to monitor for side effects. Informed consent is required to educate patients on potential risks associ- ated with utilizing the therapeutic. Once the application is approved by the FDA, the drug manufacturer ships medication to the treat- ing physician for dispensation to the patient. Knox While the process of obtaining an investigational therapeutic through com- passionate use may be more cumbersome than Right to Try, the FDA has set up Project Facilitate to help sites navigate the process successfully. Project Facilitate was designed