As health sciences researchers conduct clinical trials to develop safe and effective therapies and vaccines for COVID-19, two nontrial pre-approval access pathways - expanded access and right-to-try - are becoming more widely-known to the general public.
A study published in Regenerative Medicine - written by University of Minnesota Associate Professor Leigh Turner, Simon Fraser University Professor Jeremy Snyder, and New York University Assistant Professor Alison Bateman-House - suggests that most patients engaged in online crowdfunding and seeking nontrial preapproval access to investigational medical products obtain them using the expanded access regulatory pathway rather than through what is known as "the right-to-try."
The federal Right To Try (RTT) Act became law in 2018. It allows for individuals with life-threatening diseases to access investigational medical products if they:
- are unable to access these types of products through clinical trials cleared by the U.S. Food and Drug Administration (FDA); and
- have exhausted use of FDA-approved therapies; or
- their legally authorized representative provide informed consent concerning the investigational product they wish to receive.
"Supporters, including President Trump, promote the right-to-try option as an important pathway for obtaining access to investigational medical products," said Turner, who is with the U of M's Center for Bioethics. "To date, however, few individuals appear to be obtaining access to such products on a right-to-try basis. Right-to-try has been promoted as a faster and less bureaucratic alternative to expanded access.
"However, our research findings suggest that when it comes to crowdfunding campaigns on GoFundMe, references to right-to-try often reflect a poor understanding of nontrial preapproval access. In reality, at least according to the crowdfunding campaigns we examined, expanded access is the route by which individuals more commonly access investigational products outside clinical trials."
Expanded access, while also providing access to investigational medical products provided outside clinical trial context, differs significantly from the right-to-try pathway in that it requires FDA and institutional review board (IRB) oversight. The FDA and the IRB review submitted requests and decide if an investigational medical product should be provided on an expanded access basis. President Trump, for example, recently obtained access to an investigational product for COVID-19 - a monoclonal antibody "cocktail" - on an expanded access basis.
The experimental interventions allowed under the RTT Act and through an expanded access basis are not commonly covered by insurers. As a result, many individuals engage in online crowdfunding activity to cover the often substantial costs associated with accessing investigational interventions provided outside clinical trials.
Turner and his co-authors identified 79 GoFundMe campaigns referencing right-to-try and 115 campaigns referencing expanded access between April 2019 and April 2020. These campaigns also discussed seeking experimental medical interventions in the U.S.
When restricting analysis to campaigns initiated in 2018 and later, which is around the time the RTT Act was made into law, the researchers identified:
- 26 campaigns referencing expanded access as the access pathway to experimental interventions; of which, 21 described being able to obtain such products on an expanded access basis;
- 29 campaigns referencing right-to-try; of which, just one campaign clearly described the campaign recipient being granted access to an investigational product on a right-to-try basis; and
- two campaigns that referenced both expanded access and "right-to-try."
Through this study, researchers found that:
- GoFundMe campaigns which identified right-to-try had a general interest in accessing investigational medical products, while campaigns mentioning expanded access commonly identified a specific investigational product to be made available by a particular sponsor and were actively using this pathway;
- for individuals engaged in online crowdfunding activity, right-to-try - while an indication of interest in accessing investigational medical products - is little used in practice and does not appear to be solving a problem related to accessing experimental medical interventions;
- expanded access critics state the pathway is too restrictive and bureaucratic; however, the campaigns analyzed did not indicate that right-to-try removes those barriers or is a more practical pathway to navigate than the expanded access option;
- the GoFundMe campaigns revealed that cost continues to be a barrier for nontrial pre-approval pathways, including both right-to-try and expanded access; and
- the GoFundMe campaigns which discussed right-to-try were, in general, poorly informed about how the pathway works and treated it more as a slogan for accessing experimental medical interventions than as a practical, effective means of obtaining access to investigational products.
It is important to note that the campaigns on GoFundMe list donation goals and funds received but they may not accurately reflect costs associated with seeking access to investigational medical products pursued via expanded access pathway or a right-to-try option. Furthermore, data collected on crowdfunding sites does not necessarily reflect experiences of individuals who seek access to investigational medical products administered outside clinical trials but do not engage in crowdfunding activities.
Acknowledging these limitations, the study does suggest that, in practice, expanded access provides meaningful access to investigational medical products whereas right-to-try is more rhetorical slogan than practical option.
