Patient Access Overview

A key priority for ASGCT since the first approvals of ex vivo and in vivo gene therapies in 2017 is advocating for patient access to approved therapies. To facilitate access to these transformative treatments, the Society supports:

• Access to genetic testing and screening to expedite diagnosis of genetic conditions that may be treatable by gene therapy.

• Payment policies that facilitate patient access, including full coverage and sufficient reimbursement of providers for gene therapies, as well as novel payment models that address the unique nature of these treatments.

• Global access to gene therapy through outreach to partners in low- and middle-income countries.

• Empowerment of patients, their families, and other advocates through the provision of accurate, reliable, and accessible information about gene and cell therapies, including a clinical trials finder. 

ASGCT collaborates with a number of other entities related to patient access issues, including as a member of the Sickle Cell Disease Coalition.