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.”
Operating out of the limelight, often under Chatham House rules, teams based at the Massachusetts Institute of Technology and Duke University are taking on these challenges.
They have launched separate, complementary initiatives to serve as brain trusts for the cures ecosystem, convening key players and driving them to conceive of policies that are making it possible to rapidly transform scientific advances into medicines.
Programs at the two universities have pioneered models that achieve the kind of collaboration among diverse stakeholders that public-private partnerships aspire to, without the governance hassles that can limit the effectiveness of PPPs. And they have earned the trust of stakeholders who may be reluctant to engage with think tanks that are associated with ideologies or partisan politics.
MIT’s NEW Drug Development