This article discusses the scope and purpose of the special designation for regenerative medicine advanced therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT and provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.   Introduction Section 3033 of the 21st Century Cures Act, titl...

This article discusses the scope and purpose of the special designation for Regenerative Medicine Advanced Therapies (RMAT) created by the passage of the 21 st Century Cures Act . The authors explain the benefits expected to be realized with RMAT, such as keeping the US globally competitive in the field. They provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.   ...

This article reviews the latest revision of Japan’s Pharmaceuticals, Medical Devices and other Therapeutic Products (PMD) Act and outlines the history and benefits of the SAKIGAKE designation system. The authors share details about the criteria for SAKIGAKE designation and the procedures and timing of SAKIGAKE designation.   PMD Act Revision History   Prior to 2013, the main regulation for medicines in Japan was called the Pharmaceutical Affairs Law or PAL ....

This article discusses the history, reasoning, legal decisions and subsequent regulatory requirements behind Japan's bid to become a world leader in regenerative medicine. The authors outline the process for seeking new regenerative product approvals through an accelerated regulatory pathway and spell out the oversight role played by Japan's regulatory authorities to enable new regenerative, cell-based therapies to be made available to the public in Japan as soon as possi...

Studying the history of regulatory approvals can provide insights into regulator expectations for new products. The authors undertook an investigation to examine the data sets on which previous US Food and Drug Administration (FDA) approvals for melanoma treatment products were based to determine whether trends have changed in the level of evidence provided. Melanoma was chosen due to its relevance to ongoing projects by the authors. Methodology The authors obtai...