Spurring innovation in the biotech industry will save lives

By John Crowley

This is a lightly edited excerpt of testimony recently provided to the U.S. Senate’s Health, Education, Labor, and Pensions hearing "The Future of Biotech: Maintaining U.S. Competitiveness and Delivering Lifesaving Cures to Patients.”

Twenty-five years ago, my two youngest children, Megan and Patrick, were diagnosed with Pompe disease, a rare and fatal neuromuscular disorder. At the time, there was no treatment — no hope beyond comfort care. I left my job, went back to graduate school, and eventually helped start a small biotech company dedicated to developing an enzyme replacement therapy.

That therapy — born from the ingenuity and perseverance of American scientists, and approved by the FDA — gave my children a chance at life. Today, Megan and Patrick are living full, meaningful lives.

That experience taught me two things I will never forget: first, that science can save lives, and second, the biotech ecosystem is complex, interdependent and each piece is critical for success.

So, when I speak today about America’s biotechnology ecosystem and our global competitiveness, I speak not only as a CEO, but as a father who owes everything to innovation and the people who make it possible.

The current biotech landscape

Nearly 50 years ago, the biotechnology industry was born in the United States with groundbreaking discoveries by American companies, including the development of synthetic insulin. Today, this sector generates over $3 trillion in annual economic output, employs nearly 2.3 million Americans and supports 8 million additional jobs across the country

Maintaining America’s dominance in biotechnology is now a national security imperative, particularly in the face of aggressive efforts by China to dominate global health technologies. The bipartisan National Security Commission on Emerging Biotechnology (NSCEB) has warned that China is rapidly closing the innovation gap and may soon overtake the United States as the global leader. The numbers are telling: As of March 2025, China accounts for 30% of global clinical trial starts, nearly matching the U.S. share of 35%. In 2024, China’s innovative drug assets represented 32% of the global biopharma pipeline, up from just 4% a decade earlier. To confront these threats and secure America’s biotech future, we must take proactive, strategic action. 

Strengthening America’s biotech future: Spur innovation and address unmet needs for the most vulnerable through Congressional action

Rare disease drug development faces many barriers. Just 5% of the 10,000 known rare diseases have an approved treatment on the market.  The Rare Pediatric Disease Priority Review Voucher (RPD PRV) program was created by Congress in 2012 to spur treatments that offer hope to children with few or no treatment options and to provide uniquely American incentives for biotech investment. To date, 53 PRVs have been awarded across 39 rare pediatric diseases. Prior to the creation of the RPD PRV, only 3 of those 39 diseases had any FDA-approved treatment. Seventy-four percent of qualifying drugs that were awarded an RPD PRV are first in disease, indicating that trailblazing innovation is underway and has already improved treatment options for children.

The United States has led the world in biotechnology for decades — not by chance, but by design. A science-driven, well-resourced and globally respected biotechnology ecosystem is essential not only to provide Americans with health and hope but also to provide national security.  

Read the full testimony here.

John Crowley is the President & CEO of the Biotechnology Innovation Organization. 

_{*The opinions expressed in this column are those of the author and do not necessarily reflect the views of HealthPlatform.News.}