Loyola University Chicago School of Law, JD 2021
The Federal Food, Drug, and Cosmetic (FDC) Act of 1938 requires pharmaceutical drugs to provide evidence of their safety before they are allowed on the market. As such, pharmaceutical companies submit applications to the Federal Drug Administration for approval. There are situations, however, in which patients seek to receive access to a particular pharmaceutical drug before the FDA approval process is complete. This blog will explore the various pathways to pre-approval access in addition to recent legislation and legal considerations for such pathways, in addition to the principles and common obstacles that pharmaceutical companies face within such pathways.
Expanded access, sometimes used interchangeably with the phrase “compassionate use”, allows participants who cannot participate in clinical trials to receive access to the drug(s) and/or other biomedical products before FDA approval. Groups of individuals – with their physicians’ approval – may receive treatment via the expanded access program. However, the final decision on whether to grant access to such patients rests upon the pharmaceutical company. If only a single individual, rather than a group of patients, is seeking access, then the company must submit the request for the FDA to review prior to granting access to the drug. In addition, an institutional review board (“IRB”) historically has had to review the request and ensure that informed consent is in place. Beginning in 2018, FDA commissioner Scott Gottlieb starter to require physicians to receive approval from only one IRB member instead of the entire board in order to process requests more efficiently. Patients may apply for expanded access during any phase of the FDA approval process. The FDA approval process includes the development phase, pre-clinical phase, clinical trial phase, submitting the application for FDA review, and then finally, the FDA post-market safety monitoring phase.
In 2018, President Trump signed the Right to Try Act. Right-to-Try is an alternative pathway to pre-approval access that differs from expanded access/compassionate use. Patients may only seek access via right-to-try after Phase I of the clinical trial is complete, and the drug in question has to be in active development (and heading towards the next phase/FDA approval). Right-to-try encompasses pharmaceutical drugs, but not other products such as vaccines or biomedical devices. Similar to expanded access, informed consent is required as well in right-to-try situations, though the liability shield is much stronger in right-to-try cases.
Ethical and legal considerations
Patients who seek alternative pathways to pharmaceutical products before FDA approval are almost always severely ill, and may even be facing certain death. It is thus important for pharmaceutical companies to not provide false hope and to manage expectations.
In addition, it would be best practice to provide as much information as possible within the informed consent each patient is required to sign prior to participating, detailing inclusion and exclusion criteria for pre-approval access, potential side effects, the data available on efficacy thus far, and perhaps even presenting alternatives to seeking pre-approval access. It is also important to emphasize the completely voluntary nature of pre-approval access, and ensure that the patient(s) understand that termination of treatment is possible and allowable.
With the rise of individual social media campaign advocacy, it is important for companies to ensure a fair pre-approval access program while maintaining public relations. All responses to such campaigns ought to be tempered with compassionate language in addition to explaining the reasons for the company’s decision (while maintaining patient privacy). It would, after all, be unfair to grant access on the basis of the vigor of one’s social media campaign.
The MRT Center of Brigham and Women’s Hospital and Harvard states in their publication, “Principles of Post-Trial Responsibilities,” that “provision of continued access must be fair and not inadvertently advantage some and harm others.” It is thus important to establish a uniform policy ahead of time that sets the criteria on granting continued access to the treatment. Companies would not want to be in a situation where it appears unjust in granting continued access to some patients and not others.
Additionally, it is highly important to collect data both during treatment as well as after treatment, as such data will provide further insight into the efficacy and safety of the treatments. Should a treatment be shown to not be effective, or have highly questionable results, it may be in the best interest of pharmaceutical companies to not continue access to such treatment in order to preserve the company’s reputation and standing in the pharmaceutical industry.