Compassionate use of drugs and medical devices in the U.S., the European Union and Japan

open access

..... In this paper, we assess the current compassionate use, or expanded access-related mechanisms, of the US, the EU and Japan in regard to drugs, medical devices and biologics, including human cells and tissue products, and discuss the benefits and issues of these mechanisms. The purpose of these mechanisms is principally to save patients with life-threatening condition. However, the information obtained after the compassionate use is potentially useful to facilitate marketing authorization. In fact, the data from compassionate use cases are employed in some approval review reports to indicate the product safety.....
 

Conclusion

There are various compassionate use-associated mechanisms for both drugs and medical devices in the US and the EU. The main purpose of these mechanisms is to save patients with life-threatening conditions with no other approved alternative therapy options. Another important benefit could be to provide safety and efficacy information to support future market authorization. However, there are some cases in which companies decline requests from patients to provide unapproved products because they want to avoid the risk of the use of products before confirming their efficacy and safety in clinical trials. In these cases, companies are criticized for not providing the products to patients with life-threatening conditions [31] and [32]. We propose comprehensive mechanisms that consider the risks and benefits to the patients interested in using unapproved products and the companies providing the products.

Conflicts of interest

Kenichiro Tsuyuki and Dr. Kazuo Yano are employees of Medtronic Japan Co., Ltd.

Dr. Masayuki Yamato is a shareholder of CellSeed Inc.

Keywords

• Compassionate use;
• Expanded access;
• Life-threatening;
• Serious disease;
• Clinical trial;
• Unapproved products