According to the 21st Century Cures Act, US-based pharma companies with drugs at the Phase II or Phase III development stage are required to publicly display their stance on expanded access programs. But many companies appear not to comply, according to a Clinical Trials Arena analysis.

Expanded access, also known as compassionate use, allows certain patients with serious disease to access experimental drugs outside of the clinical trial setting. The 21st Century Cures Act, which became US law in December 2016, requires companies that offer expanded access to make their policy for evaluating and responding to requests “public and readily available.” The law does not require companies to offer expanded access, but they must also make this position publicly visible.

Overall, only 51% of qualified US drug companies appear to fully comply with these expanded access visibility requirements, a Clinical Trials Arena analysis finds. An additional 10% of companies appeared to partially comply, defined as meeting some but not all requirements of displaying their process for evaluating and responding to expanded access requests.

Around 56% of public companies—but just 24% of private companies—appeared to fully comply with expanded access visibility requirements. The analysis is based on identifying qualifying companies through GlobalData’s Clinical Trial Database and analyzing apparent compliance through the FDA’s Reagan-Udall Expanded Access database and a standardized keyword search of company websites (detailed methodology notes at bottom of article).

The US expanded access pathway is a type of managed access program (MAP), which have different types of regulations depending on the geographic area. To successfully implement MAPs, experts say sponsors must collaborate with vendors and multiple stakeholders in unison, especially in Europe where requirements vary widely by individual member states.

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Low visibility hinders awareness

The goal of the FDA’s expanded access pathway is to offer access to experimental drugs for patients with ultra-rare or life-threatening diseases who would not otherwise have access to clinical trials, explains Alison Bateman-House, PhD, a bioethicist researching expanded access at New York University. But whenever possible, both FDA and sponsors still prefer patients to access experimental treatments through traditional clinical studies.

If a company offers expanded access, it must specify who to contact regarding expanded access, what information is necessary for a request, and how long it typically takes for a response, according to the 21st Century Cures Act. However, the FDA does not widely enforce this law, and many smaller companies feel they lack the resources to run an expanded access pathway, Bateman-House has told Clinical Trials Arena.

Public, US-based drug companies that appeared to fully or partially comply with expanded access visibility requirements had, on average, more employees and higher revenues than companies that did not comply. Fully or partially compliant companies had an average annual revenue of $2.48 billion, compared to $533 million for companies that appeared not to comply, according to the Clinical Trials Arena analysis, which also uses publicly available data compiled in GlobalData’s Companies Database.

The lack of expanded access visibility among many US companies could contribute to the overall lack of awareness that has hindered the pathway’s use, Bateman-House says. While many patients have heard of clinical trials, far fewer are aware of expanded access pathways and procedures. “The biggest challenge with expanded access is not many people are familiar with it,” Bateman-House explains. “In many cases, patients feel like they have to start from scratch.”

Expanded access protocols decline

Expanded access often entails granting individual patients access to treatments, but it can also take the form of registered expanded access protocols. Though distinct from clinical trials, expanded access protocols and resulting data can form part of an experimental drug’s IND application, according to the FDA.

For example, the FDA authorized multiple expanded access protocols for experimental drugs during the Covid-19 pandemic. These include a US government-sponsored expanded access protocol for remdesivir (NCT04302766) and an investigator-sponsored protocol for convalescent plasma (NCT04338360).

More recently, however, the number of expanded access protocols registered with has been on the decline, according to GlobalData’s Clinical Trials Database. The number of registered expanded access protocols, measured as a percentage of total trial initiations, dropped from 0.66% in 2019 to just 0.05% in 2022, though there appears to be a slight uptick in expanded access protocols among planned trials in 2023.

Expanded access and the road ahead

As awareness of ultra-rare diseases and personalized medicine approaches gains prominence, regulators are working hard to stay in front. “The FDA has been ahead of the curve compared to most regulators with expanded access,” Bateman-House says. “Most scientists and physicians think the FDA does a good job balancing patient autonomy and the need for standards of safety and potential efficacy.”

That said, expanded access pathways are not without their challenges. Already, international bodies have convened to explore their potential for misuse, and there are concerns that expanded access could limit the pool of clinical trial participants.

Still, expanded access in the US continues to struggle with a lack of visibility. Once more drug companies comply with existing regulations, more patients could become aware of expanded access pathways, including both their challenges and potential benefits.

Methodology: The analysis of expanded access visibility compliance includes several hundred US-based companies with drugs at the Phase II or Phase III stage, thus qualifying for the 21st Century Cures Act’s expanded access visibility requirements, identified through GlobalData’s Clinical Trials Database. This list was cross-referenced with the FDA’s Reagan-Udall Expanded Access database, which the FDA states meets expanded access visibility requirements, as well as a standardized keyword search in each company website. The analysis only reflects apparent compliance, as it is possible some companies meet expanded access visibility requirements without appearing in the standardized search.