What Key Elements Should A Company Consider When Setting Up An Expanded-Access Program For An Experimental Drug Candidate?

THERE ARE MANY EXAMPLES WHERE EA PROGRAMS HAVE BEEN LIFESAVING and have provided important information about an experimental medicine. But not all experimental medicines work, especially when their activity is not fully understood. Corporate leaders need to consider the impact, potentially positive and negative, on the development program for their experimental medicine and on the lives of patients. What risks are the company willing to take in the development program? If complications in an EA program slow down the approval timeline, even for only a month, that delay could mean that many future patients must wait an extra month to receive therapy. Who is advocating for those future patients? These questions are difficult to address when faced with an individual, often a child, who has an immediate need. Yet corporate leadership must balance the near-term needs of that individual versus the longer-term needs of the population.

KEN MOCH, President, Euclidean Life Science Advisors and faculty affiliate at the Division of Medical Ethics at NYU.