As the science of cell and gene therapies (CGT) reaches unprecedented heights, equitable access to these lifesaving treatments must keep pace, said John F. Crowley, President and CEO of the Biotechnology Innovation Organization (BIO), during a White House Cell and Gene Therapy Forum.

“Innovation without access for all isn’t enough,” Crowley emphasized while moderating a panel discussion at the event. “The science is hard enough, so let’s work to increase access.”

The forum brought together leading voices in biotech, healthcare, and policy to address the barriers preventing widespread availability of CGTs. Attendees identified key strategies for expanding access, including:

• Supporting the Centers for Medicare & Medicaid Services (CMS) Cell and Gene Therapy Access Model while leveraging state-level innovations.
• Reforming the Employee Retirement Income Security Act (ERISA) to enhance coverage options.
• Advocating for the passage of the Rare Pediatric Priority Review Voucher (PPRV) Act and other supportive legislation.

BIO has long championed initiatives to address the economic challenges of CGTs, particularly their high upfront costs. Crowley reiterated BIO’s call for value-based payment models to ensure these therapies, which significantly reduce long-term healthcare costs, can reach more patients.

“Cell and gene therapies offer transformative potential for conditions like cancer, sickle cell disease, and other rare disorders,” Crowley said. “The science has progressed unbelievably, but the true challenge lies in ensuring these breakthroughs benefit everyone who needs them.”

Honoring Biotech Leaders: National Medal of Technology and Innovation

The biotech sector was further recognized at the White House on January 3, when President Joe Biden presented the 2024 National Medal of Technology and Innovation to three biotech trailblazers:

• Jennifer A. Doudna (Innovative Genomics Institute): For her groundbreaking development of CRISPR-Cas9 gene editing technology, enabling advancements in agriculture, health research, and treatments for diseases like cancer and sickle cell disease.
• Moderna, Inc.: For harnessing mRNA vaccine technology to combat COVID-19, saving millions of lives, and opening new frontiers in immunology.
• Pfizer Inc.: For leveraging mRNA technology to deploy a lifesaving COVID-19 vaccine, cementing America’s leadership in innovation.

The Future of Cell and Gene Therapy: A Call to Action

Rare Disease Congressional Caucus Co-Chair Sen. Amy Klobuchar (D-MN), a speaker at the forum, echoed Crowley’s call for policies that balance innovation with accessibility.

“These therapies have the power to change the course of diseases and improve countless lives,” Klobuchar said. “We must ensure they’re available to all who need them.”

Crowley closed the forum with a rallying message: “The progress we’ve made in science is extraordinary, but our mission is not complete until every patient has access to the cures and treatments they deserve.”