The question of whether patients with life-threatening disorders should be able to access treatment with investigational, unapproved, and unproven medications is a complex one, and I’m not going to try to get into all the details here.
What is interesting, however, is an initiative announced yesterday, by the Janssen Pharmaceutical division of the Johnson & Johnson group of companies. In collaboration with the New York University School of Medicine, Janssen is setting up an external “Compassionate-Use Advisory Committee” — made up of physicians, ethicists, and patient advocates — that will help them to assess such applications. Two senior staff members of Johnson & Johnson discuss the initiative in this video on YouTube.
While the U.S. Food and Drug Administration (FDA) does have to give approval for any such clinical use of an investigational drug, this is normally a formality. The FDA has approved 99% of all such applications over the past few years. So the decision to permit such use of an investigational agent is really a matter for the drug’s developer.
Strategically, this looks like a really smart move on the part of Johnson & Johnson. There are long-standing questions about when and why patients with truly life-threatening disorders should not be able to gain such access to such investigational agents — if there is reason to believe they might have activity. However, there are also problems when small, start-up biotechnology companies are approached and asked to provide access to early stage drugs, starting with the fact that all supplies of such drugs are usually committed to planned clinical trials designed to achieve product approval for specific indications over time, and such companies have limited resources.
By taking a clear position on this issue, seeking guidance from an independent advisory panel, and going public with this information, J&J is setting a leadership example that others may be willing to follow.