Headlines

A RACE FOR INNOVATION: Regulatory Initiatives to Bolster Cell and Gene Therapy Development

A RACE FOR INNOVATION: Regulatory Initiatives to Bolster Cell and Gene Therapy Development

Cell and gene therapies represent the cutting edge of biopharmaceutical development, leveraging innovative technologies to address medical conditions which often have no other existing disease-modifying therapeutic options. These groundbreaking assets garnered tremendous attention initially but have since seen sizeable slowdowns in funding due to cases of concerning adverse event profiles, high price tags, and macroeconomic trends. However, recent regulatory movements may generate renewed enthusiasm for these therapeutics due to published guidance that could ease the development and commercialization process.

In this white paper, we explore the implications of recently adopted regulatory policies on biopharma organizations that are developing cell and gene therapies. Specifically, we discuss recent FDA approvals and guidance to help streamline and accelerate the approval process for cell and gene therapies, CMS proposals for outcomes-based pricing, and the impact of the Inflation Reduction Act of 2022 (IRA) and implications for cell and gene therapy drug pricing. We conclude that these measures may create new opportunities for cell and gene therapy development, particularly in rare diseases and for programs with orphan drug designations.

A RACE FOR INNOVATION

I acknowledge and agree to the use of my contact information to receive messages about offerings by Questex, its brands, affiliates and/or third-party partners, consistent with Questex Privacy Policy