SummaryProposed Requirements in 21st Century Cures Act Would Increase Transparency Requirements
A new analysis from Avalere finds that only 19 percent of pharmaceutical and biotechnology companies publicly post their compassionate use policies on their web sites. Compassionate use is a process that enables patients to request access to investigational products outside of a clinical trial before the product is approved by the Food & Drug Administration. Current legislation being considered as part of the 21st Century Cures Act would require increased transparency.
Avalere recently reviewed the websites of 100 publically traded pharmaceutical and biotechnology companies to assess the availability and elements of their posted compassionate use policies. Avalere found that large companies were the most likely to post their compassionate use policies with 52 percent of large companies sharing their policies. Fourteen percent of medium-size companies posted their policies, followed by four percent for small companies (Figure 1).
“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 a vice president at Avalere Health. “Accessible compassionate use policies posted on company websites reduces the burden on patients to obtain this information,” Huneycutt continued.
21st Century Cures Implications
If the compassionate use policy provision in the 21st Century Cures Act passed as currently drafted, companies would have 60 days from enactment to comply with the policy transparency requirements. Currently, there are four different requirements in the bill for transparency of compassionate use policies. Avalere examined the 19 companies which posted their policies to see who may comply with the different requirements. For two out of the four requirements, less than five of the 19 companies may meet the requirements outlined in the bill.
“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.”
The analysis also examined how easily accessible the polices were to find, in addition to naming conventions for the posted policies, procedures for making requests, and timing information for either acknowledgment or response to a request. Avalere found that of the companies that did post their policies, the majority of them– almost 75 percent — were easy to find (i.e., they came up as a first or second hit in the website search bar or can be found directly from the main page).
Avalere’s research also found that only one company posted information about procedures for making compassionate use requests. In addition, few companies provided information about the length of time that the manufacturer or distributor anticipates will be necessary to acknowledge the receipt of such requests.
We determined the list of 100 companies by identifying the top 100 pharmaceutical or biotechnology companies from Google Finance’s list of healthcare companies ranked by market cap which appeared from the information included on their websites to be developing investigational drugs or biologics. We separated the 100 companies based on market cap size into “large”, which we defined as a market cap of more than $10 billion US dollars, “medium”, which we defined as a market cap of from $1.5 to $10 billion US dollars, or “small”, which we defined as a market cap of less than $1.5 billion US dollars. Large companies made up 25 percent of the analysis group. Medium-sized companies represented 28 percent of the companies, and small companies made up the remaining 47 percent.
We searched each company website to determine if a posted compassionate use policy (as defined above) existed. Each identified policy’s accessibility was characterized as “easy” if the policy came up as either the first or second “hit” on a search (in the website search bar) for either term “compassionate use” or “expanded access”. If a website search for those two terms did not yield a compassionate use policy as the first two hits, we looked through the whole website section by section and if the policy was explicitly listed in a drop-down menu on the home page it’s accessibility was characterized as “easy” and if it was not listed in a drop-down menu on the home page, i.e., we had to click through sections/menus, it was characterized as “medium difficulty”. We did not use general internet searches (e.g., Google) to search for company compassionate use policies.
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