by Mark Corbett, Senior Vice President, Clinigen Global Access Programs

Around the world, countless patients with a high degree of unmet medical needs are in urgent need of new therapeutic alternatives. For many of these patients, access to new innovative medicines via the well-defined clinical trial process or through commercial channels is not an option and the wait for approval and commercialization may simply be too long, and in some cases, deadly.

In response, a number of industry experts are collaborating on an ISPE concept paper that describes an option for these patients and drug developers. Whether termed compassionate use, named patient use, expanded access or early access, there are existing regulatory mechanisms in many countries around the world that may enable patients in these situations to gain access to medicines that are in clinical development, are unlicensed in a specific market but licensed elsewhere, or are in the Marketing Authorization Application (MAA) process. Access programs afford companies the opportunity to meet ethical obligations and can offer patients with an unmet medical need a potentially life-saving, life-enhancing or life-extending treatment option, ahead of commercial availability.

The ISPE concept paper, available electronically this summer, will describe these access programs, the regulations governing them, and best practices for designing and implementing as well as ensuring patient safety and minimizing risk.

Greater awareness of these programs and how they work is essential for all stakeholders.  Similarly, an understanding of when these programs can and cannot be used (and why) is critical when the need presents itself.

If your organization is currently conducting or considering an access program, I welcome your perspective.