Transparency Lacking in Drug Manufacturer Expanded Medication Access Policies

A recent analysis found that relatively few drug manufacturers share rules and regulations for granting patients access to experimental drugs, which may pose a challenge if the 21^st Century Cures Act passes as is.

Approximately 19% of pharmaceutical and biotechnology companies publicly post their policies about compassionate use on their websites, according to an analysis conducted by Avalere Health.

Compassionate use, also called expanded access, is the process that allows patients to access investigational drugs and products outside of ongoing or completed clinical trials. While the FDA prefers patients to use experimental drugs within the controlled environment, sometimes enrollment is not possible due to inclusion criteria or lack of ongoing trials.

Compassionate use applies to investigational devices and drugs, including biologic therapies. In the analysis, Avalere reviewed 100 websites of publically traded pharmaceutical and biotechnology companies to determine if they listed their compassionate use policies, and how much information was given.

Large companies (market cap of over $10 billion) comprised 25% of the groups examined, and medium-sized companies (market cap of $1.5 to $10 billion) made up 28%. Small companies (market cap of less than $1.5 billion) comprised 47%.

They discovered that 52% of large companies, 14% of medium-sized companies, and 4% of small companies posted their policies on their websites.

“Patients need to understand what potential medicines in clinical development are available to them and be able to request access easily and fairly,” said Brenda Huneycutt, vice president at Avalere Health. “Accessible compassionate use policies posted on company websites reduces the burden on patients to obtain this information.”

The legislation considered in the 21^st Century Cures Act would require more transparency, and will likely effect these companies not publicly posting their compassionate use policies, Avalere reported. If passed as is, these companies would have 60 days to comply with new transparency requirements.

Researchers at Avalere then examined the companies who posted their policy to determine which would already be in compliance with the potential new legislation. They found that 17 of 19 companies included contact information to facilitate the requests for the drug.

Researchers also discovered that 75% of the companies who posted their policies were easy to find, meaning that it came up as the first or second item on a website search, Avalere reported.

Only 1 company had a public list of procedures that are needed for making a request. The company had a list of the information that the physician must provide, but did not list contact information.

A majority of companies also listed criteria that the company would review to determine whether they would approve the request, according to Avalere. Only 3 out of 19 companies listed information about the length of time they expect will be needed to acknowledge receipt of compassionate use receipts.

“As patients continue to demand increased access to investigational products, it’s important for manufacturers to understand the complex nuances and substantial reputational risks they can face with compassionate use cases,” said Gillian Woollett, senior vice president at Avalere Health. “If current policy proposals are passed as is, manufacturers will need to prepare carefully yet act quickly to meet the new requirements.”