The unsung public policy innovators paving the path to cures

Patients with diseases like hemophilia, spinal muscular atrophy and β-thalassemia that could be cured with gene or cell therapies probably don’t know it, but their fates depend as much on the success of public policy innovations brainstormed around conference tables as on research conducted in labs and clinics.

“We’re living in a time where the opportunity from science to alter or cure disease is firmly at hand,” former FDA Commissioner Scott Gottlieb said in a speech on May 10. “Scientific challenges remain, but our biggest obstacles may be policy.”
The most difficult challenge, he said, “may be our inability to devise coverage schemes that can enable the efficient, and when appropriate the rapid, adoption of these innovations; allow for a return on capital that maintains investment in these high-risk endeavors; and most important, enables equal access to a cure regardless of a person’s wealth.”...