The Reagan-Udall Foundation for the FDA (RUF), a non-profit organization created to help the FDA accomplish its mission, is in the process of developing an online Navigator to help patients and physicians understand the processes and programs associated with expanded access (EA).
According to the FDA, “expanded access, sometimes called ‘compassionate use,’ is the use outside of a “well-controlled” clinical trial of an investigational medical product (i.e., one that has not been approved by FDA).” Almost all compassionate use requests that FDA receives are approved. However, the difficulty is not receiving approval. Rather, many patients run into trouble with the fact that the patient’s treating physician and the drug maker must agree to proceed with a compassionate use application. Because this sometimes creates an issue for those seeking early access, many patient advocates are asking “the FDA to make it easier for terminally ill patients to access potentially helpful new drugs,” says an article from BioPharma Dive.
In order to solve this issue, RUF proposes to develop an online navigator, which would provide details regarding FDA policies and points of contact, as well as additional information concerning the compassionate use request process.
According to RUF’s project proposal, the Navigator will provide educational content, including information on:
RUF notes that its Navigator will not address ethics, policy, or advocacy. Furthermore, it will have no effect on decisions regarding eligibility and access.
In creating the Navigator, RUF’s goal is to increase the transparency and accessibility of information, thus increasing the overall understanding of compassionate use programs. RUF explicitly states that its goal is not to encourage or discourage people from requesting individual patient access. “By increasing the understanding of EA and the individual patient access request process, RUF hopes to achieve the following outcomes:
On Monday, May 16th, the organization held a public workshop in which it sought feedback on its proposed informational database. The workshop was intended for patients, patient advocacy groups, healthcare providers, drug manufacturers, Institutional Review Boards (IRBs), and representatives from academia. In holding this workshop, RUF’s goal was “to discuss how best to disseminate information on the EA request process.”
For additional information, visit RUF’s website.
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