Market Access Uncertainties Impacting Commercial Uptake of Gene Therapies

This article was published in the Pharmaceutical Executive which can be accessed via https://www.pharmexec.com/view/market-access-uncertainties-impacting-commercial-uptake-of-gene-therapies

Overview

While gene therapies continue to revolutionize healthcare, uncertainty from commercialization issues still looms large.

Gene therapies will revolutionize healthcare by offering potential curative treatments for patients dealing with genetic diseases. Recently approved gene therapies are showing significant promise in saving or enhancing patient lives. Today, the development pipeline has hundreds of gene therapies that work by “replacing a disease-causing gene or inserting a new or corrected gene or directly editing genome (in vitro) in patients.” Despite the enormous market opportunity this new sector presents, many challenges remain for gene therapy—the science is complex, treatments are costly, the supply chain is tenuous, and the regulatory pathway is not as straightforward as conventional therapies.

The approach to market access requires a radical rethinking to ensure that patients who could benefit from these gene therapies have timely access. There is a legitimate concern that the sizable one-time upfront payment for these expensive therapies might pose an undue burden on the healthcare system. However, given that the lifetime cost of treating many genetic conditions is significantly higher than the one-time cost of these gene therapies, it is essential to evaluate long-term clinical and cost-effectiveness tradeoffs associated with these therapies. As the sector grows, manufacturers must explore new commercialization models to collaborate with providers, payers, and policymakers to optimize market access to these therapies. This article will examine key commercialization uncertainties that may hamper the successful adoption of exciting novel gene therapies.

Authors: Subbarao Jayanthi, Managing Partner


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Featured in the Pharmaceutical Executive in Oct 2020

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