Key Advances in Gene Therapy and the Promise for Patients

ASGCT Staff - November 19, 2018

ASGCT partnered with the Bipartisan Policy Center and the Alliance for Regenerative Medicine to identify six key areas to focus our work as part of a leadership summit representing patients, industry, scientists, payers, the investment community, as well as federal government officials, including FDA’s CBER Director Peter Marks.

ASGCT Executive Director David Barrett has co-authored a blog post with the Bipartisan Policy Center and the Alliance for Regenerative Medicine following a leadership summit representing patients, industry, scientists, payers, the investment community, and federal regulators last week.

The summit identified six key point towards which all three organizations have committed their efforts. As laid out in the post, those key points are as follows:

  • New value-based payment models that take into account the unique nature of gene therapies (a potentially one-time durable therapy vs. ongoing treatment of symptoms);
  • Mechanisms to capture and assess both short- and long-term outcomes to advance the science and assess the durability of treatments;
  • Increased international standards and requirements convergence;
  • Greater collaboration and coordination among academic scientists and industry to facilitate bringing new discoveries to patients more rapidly;
  • Increased public education on the promise of gene therapies; and
  • Further exploration of ways to support the development and delivery of gene-based therapies for those with neurodegenerative diseases.

All three organizations will release a report based on the insights of the meeting and follow-up research next month.

Read the Full Post on BipartisanPolicy.org