Policy Summit

Crucial Patient Access Issues Take Center Stage at the Policy Summit

Max G. Bronstein, M.P.P. - September 17, 2020

ASGCT Government Relations Committee member Max Bronstein provides a preview of some of the issues that speakers will discuss at next week's Policy Summit.

We are living through a remarkable time in human history, where biotechnology innovation is moving faster than ever and is poised to deliver life-changing and life-saving treatments to patients. The promise is perhaps most pronounced at the bleeding edge of cell and gene therapy, with dozens of disease-modifying therapeutics in late stage development. But regardless of these scientific achievements, the ultimate metric will be whether patients gain access and, therefore, benefit from these novel medicines.

In essence, FDA approval is not the finish line for these products, but rather, marks the next stage in the long march to ensure that timely patient access is a reality. But getting there requires stakeholders in the drug development ecosystem to have an understanding of the current policy paradigm and insight into how it could make or break a drug development program.

Take for example, our nation’s newborn screening system - a hallmark of public health that helps ensure babies with rare and, in some cases, deadly conditions are diagnosed as early as possible after birth. Leveraging this public health asset can be a key part of ensuring the earliest possible access to life saving medicines, but how does one convince 50 state departments of public health to implement screening for a new disease? How do you develop a screening assay and partner with leaders in the public and academic sectors to launch a pilot program? These are exactly the type of questions that will be explored in the newborn screening discussion.

With the election heating up, drug pricing has returned as a hot-button issue with changes coming down the pike that could have implications for cell and gene therapy for decades to come. Recently, the Centers for Medicare and Medicaid Services (CMS) have announced a new proposed rule that could allow for substantial payment model innovations, which could dramatically alter the patient access landscape, especially for potentially curative, single-administration therapeutics. Models such as payment-over-time or value-based payments have already begun to be deployed in the U.S. and the EU. We will have the benefit of learning about these new developments, and what we may expect in the future, during the panel highlighting real world experiences on gene and cell therapy access.

For better or worse, the future of the field of gene and cell therapy is intimately entwined with the policy decisions that will be made at the state and federal levels. Building an understanding of the policy environment, its implications for research and development, and forming connections with policymakers themselves is a vital part of ensuring a bright, healthy future for our field and for the patients who are relying on us.

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Max G. Bronstein leads MGB Consulting and has held positions at Audentes Therapeutics, the EveryLife Foundation for Rare Diseases, and is the Founder of the Journal of Science Policy & Governance. He is a member of the ASGCT Government Relations Committee.

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