Last week, the Wall Street Journal’s Pharmalot blog reported that Rep. Michael McCaul (R-Tx) plans to introduce legislation to reform expanded access. (Expanded access, or compassionate use, refers to the use of an investigational drug outside of a clinical trial, when the purpose is to treat a patient’s serious or life-threatening condition, rather than to study the drug.)
If you’re familiar with FDA’s expanded access regulations and their history, you might be underwhelmed by this news. There have been many efforts aimed at reforming expanded access – from litigation to proposed federal legislation to recently enacted state legislation – which generally have focused on the government allowing patients access earlier in drug development and with fewer safeguards (or obstacles, depending on your perspective). Usually, these proposals haven’t focused on what many identify as the real barrier to access: industry’s understandable reluctance to provide unapproved drugs outside of clinical trials.*
Rep. McCaul’s proposal, however, sounds somewhat different than past reform efforts.
In a white paper – which, according to Pharmalot, Rep. McCaul recently submitted to the House Energy and Commerce Committee’s 21st Century Cures Initiative – Rep. McCaul makes 4 recommendations for reforming expanded access:
- Incentivize industry to have clear and publicly accessible expanded access policies
- When drug companies deny requests for expanded access, ensure that patients know why the request was denied
- Require industry to track requests for expanded access and report that information to FDA, and
- Establish a task force to make recommendations for further improvement.
The stories about terminally-ill patients and their families seeking access are often heart-wrenching—the desire to do everything possible when you or your loved one is facing a terminal, incurable illness is entirely understandable. That said, I’m a bit skeptical of the value of spending time and resources on reforming expanded access. It’s not clear to me that our current expanded access system is underperforming, and I fear that the focus on reforming expanded access may distract from what will ultimately help the most patients—encouraging and hastening the development of treatments that are shown to be safe and effective.
The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.