4:17 PM
 | 
May 10, 2019
 |  BioCentury  |  Politics, Policy & Law

The unsung public policy innovators paving the path to cures

How NEWDIGS, Duke-Margolis’ public policy innovation is enabling curative therapies

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 ParadIGmS (NEWDIGS) program and the Margolis Center for Health Policy at Duke bring academics and thought leaders together with executives, patient advocates and government officials to go beyond 30,000-foot observations and dive into ground-level details to identify and overcome obstacles to the development and commercialization of potentially curative therapies.

“Patients are in dire need of treatments,”

Hans-Georg Eichler, EMA

Both the Duke-Margolis Center and NEWDIGS are funded by a mix of foundations and non-profits, academic institutions, government, and industry.

The development and launch plans for the gene therapy Zynteglo from bluebird bio, for example, are being shaped by work conducted at NEWDIGS and at the Duke-Margolis Center.

If it lives up to expectations, Zynteglo will transform the lives of patients with β-thalassemia, freeing them from lifelong transfusion dependence and a host of associated medical problems. Its success will be attributable to unheralded policy innovations developed at Duke-Margolis and MIT as much as to the decades of scientific work on which it is built.

One set of policy innovations incubated at NEWDIGS has shaped the way bluebird is investigating and demonstrating Zynteglo’s safety and efficacy. Formalized as the Adaptive Pathways initiative by EMA, this process involves reaching consensus among patients, regulators, physicians and payers about a minimum dataset required for approval, coupled with commitments to continue rigorously collecting data and potentially modifying indications and pricing for long after the therapy is approved.

Participation in EMA’s Adaptive Pathways pilot project helped bluebird craft a development plan that rocketed Zynteglo through the review process in 150 days, the shortest review time to date for an Advanced Therapy Medicinal...

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