This year marks roughly fifty years since the modern biotechnology era began — an era defined by recombinant DNA, monoclonal antibodies, genomics, and platform technologies once belonging to science fiction. Biotech has transformed what it means to diagnose, treat, and prevent disease. Faith in that innovation is a promise made to patients, and momentum has carried it forward.
It comes at a particularly incidental time. The U.S. administration just put an industry veteran with an interest in accelerating clinical trials as the new head of the FDA Center for Biologics Evaluation and Research (CBER), along with an acting Commissioner who has committed to clearing the decks for speedier outcomes. An appetite for change is evident in the FDA; the mood has shifted towards speed and efficiency.
FDA systems have incorporated AI more deeply to expedite reviews, and rules are proposed for AI’s use in evidence collection. Changes to the pivotal trial rules may reduce time and cost. The National Priority Voucher Program (CNPV) has resulted in seven key approvals within just about a year. Though still young, the program has provided faster approvals and encouraged deeper coordination and speed by industry, setting the stage for faster access for Americans.
Biotech’s achievements are clear. Formerly fatal conditions are now manageable or curable. Sickle Cell Disease can be functionally cured. Research in HIV/AIDS has reduced mortality nearly to the general population and generated countless insights. CAR-T therapies evolved from HIV treatment into a therapy for otherwise fatal cancers and a potentially transformative treatment across the autoimmune space. Neurogenerative diseases linked to vision loss and weakness, such as NMO and MOGAD, have been differentiated, allowing for targeted therapy and the opportunity to evaluate risks in populations such as pregnant women.
Yet, advancing today’s pipeline hinges on aging systems. An analysis of FDA-approved drugs between 2010 and 2020, found that on average, 9.1 years were spent from first human trial to approval. The health implications of pediatric disease are lifelong; yet these trials average 239 days from startup packet to first patient consented. A patient diagnosed with Colorectal Cancer likewise cannot wait, facing increased mortality risks every four weeks treatment is delayed. A 2022 analysis of fast-track approvals found that 42% of confirmatory studies either initiated after a year or never at all. While a 2023 analysis of 2542 surgical trials registered on ClinicalTrials.gov found that between 2010 and 2014, 1 in 5 were completed on time and fewer than half met enrollment targets.
What is needed to meet the moment?
An aggressive and speedy reauthorization of PDUFA that includes a thoughtful approaches to a new era of clinical trial reforms.
The reauthorization of the Prescription Drug User Fee Act (PDUFA) may provide opportunity to carry the momentum further, to support FDA goals for the use of adaptive and platform trial designs, real-world evidence, decentralized and digital tools, and support the reshoring of phase I trials.
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Industry buy-in is essential, however. The CNPV has already shown what is possible when clinical trials are treated as an opportunity during discovery, rather than a downstream obligation. AI tools may provide efficiencies translating EHR data, optimizing study documents, and supplementing data. Rare disease consortia, such as the NCI ETCTN, may connect experienced sites and disparate populations. Adaptive trial platforms linked to novel models, such as organoids, may be the new engines translating insights from the bench.
The fiftieth anniversary of biotech is an appropriate moment for both reflection and resolve; the next fifty years will bring even more. Progress will mean building on momentum now and ensuring that innovation fulfills its promise. After fifty years of extraordinary science, the measure of success will be making time work for patients rather than against them.
Andrew Barnhill is the Chief Policy Officer of IQVIA, the world’s largest clinical research organization, and a faculty member at the NYU Wagner School of Public Service. Abe Sinay-Smith is Clinical Research Manager at the NYU Grossman School of Medicine.