Snapshot

The Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program pathway has been developed by the of United States.

This pathway can be used where certain CBER- and CDER-regulated products (novel drug and biological products) that are intended to treat a rare disease. .

This pathway facilitates activities during development.

It is an abridged review (a reliance pathway).

The purpose of the START Pilot Program is to further accelerate the pace of development of novel drug and biological products that are intended to address an unmet medical need as a treatment for a rare disease. The pilot is designed to be milestone-driven (i.e., to facilitate the progression of a development program to pivotal clinical study stage or the pre-BLA or pre-NDA meeting stage) where product development programs selected would benefit from enhanced communications with FDA. Participation in the pilot will be considered concluded when the development program has reached a significant regulatory milestone such as initiation of the pivotal clinical study stage or the pre-BLA or pre-NDA meeting stage as agreed upon with the sponsor. Pilot participants will be selected based on demonstrated development program readiness. The START Pilot Program is intended to provide a mechanism for addressing clinical development issues that otherwise would delay or prevent a promising novel drug or biological product from progressing to the pivotal clinical trial stage or pre-BLA/pre-NDA meeting stage. FDA intends to select participant CBER and CDER INDs based on the criteria outlined. FDA will make its determination of participants following the close of the application period. FDA intends to issue a letter to notify each sponsor of FDA's decision on sponsor requests to participate within 90 days of the application deadline.