Accelerating Cures in the Modern Era
The most significant legislative change to the FDA in a generation is accelerating medical breakthroughs while ensuring safety and efficacy.
When the 21st Century Cures Act was signed into law in December 2016, it marked a paradigm shift in how the U.S. Food and Drug Administration approaches medical product development. Designed to help accelerate medical product development and bring new innovations to patients faster and more efficiently, the law represents the most significant modernization of FDA's regulatory framework in decades 1 .
This article explores how the FDA is transforming to keep pace with scientific advances, embracing new tools and approaches that maintain the gold standard of safety while getting breakthrough treatments to those who need them most.
The 21st Century Cures Act emerged from a critical realization: the pace of medical innovation was accelerating, but regulatory processes risked becoming a bottleneck. The law provides FDA with new authorities and resources to modernize clinical trial designs, incorporate patient perspectives, and leverage cutting-edge science 1 .
An expedited pathway for certain eligible biologics products, including cell therapies and tissue engineering 1 .
Accelerates the review of innovative medical devices that provide more effective treatment for life-threatening conditions 1 .
Allows FDA to consider data from actual patient experiences alongside traditional clinical trials 1 .
Systematically incorporates patient perspectives into the development process 5 .
These initiatives reflect a fundamental shift from the FDA as solely a gatekeeper to a collaborative partner in innovation.
The Cures Act's impact is evident throughout the drug development lifecycle. In 2025 alone, FDA has approved numerous novel drugs addressing previously untreatable conditions 2 .
| Drug Name | Approved | Treatment For |
|---|---|---|
| Rhapsido (remibrutinib) | Sept 30, 2025 | Chronic spontaneous urticaria in adults 2 |
| Palsonify (paltusotine) | Sept 25, 2025 | Acromegaly in adults with inadequate surgical response 2 |
| Inluriyo (imlunestrant) | Sept 25, 2025 | Advanced or metastatic breast cancer with specific mutations 2 |
| Forzinity (elamipretide) | Sept 19, 2025 | Muscle strength improvement in Barth syndrome 2 |
| Modeyso (dordaviprone) | Aug 6, 2025 | Diffuse midline glioma with H3 K27M mutation 2 |
Performance standards that make the drug review process more organized and integrated when multiple offices are involved 8 .
Ensures all professional viewpoints are fully expressed and understood before decisions are made, creating a more collaborative review environment 8 .
One of the most transformative areas of FDA's modernization effort involves alternative methods—testing strategies that reduce or replace animal use while maintaining rigorous safety standards 3 .
Often called "organs-on-chips," these are microscale cell culture platforms that model functional features of specific human organs 3 .
Computer simulations and artificial intelligence models that can predict biological responses 3 .
Using scaffolds and cells to form biologically active tissues for testing 3 .
Evaluating multiple aspects of cell and tissue responses to study the whole organism 3 .
FDA's regulatory framework now permits and encourages these new approaches when they're backed by strong science and produce valid data. The agency actively conducts research to advance these methods, recognizing they can provide more timely and predictive information while reducing animal testing 3 .
The Innovative Science and Technology Approaches for New Drugs (ISTAND) pilot program represents the cutting edge of FDA's embrace of new tools. In a landmark decision, ISTAND recently accepted its first organ-on-a-chip technology designed to predict human drug-induced liver injury (DILI) 9 .
Researchers create tiny channels and chambers on a clear polymer chip, typically about the size of a USB drive.
Human liver cells are introduced into the chip's chambers, recreating the tissue architecture of a human liver.
Microscopic channels mimic blood flow, delivering nutrients and test compounds to the cells.
Sensors continuously measure cell health, metabolic activity, and injury markers.
Potential drug candidates are introduced to assess their toxic effects on the liver cells.
Early validation studies have demonstrated that these liver chips can successfully predict drug-induced liver injury with greater accuracy than traditional animal models. The technology can detect subtle signs of toxicity that might be missed in conventional testing.
| Method | Advantages | Limitations |
|---|---|---|
| Animal Testing | Established regulatory acceptance; Whole-body responses | Species differences; Ethical concerns; Cost and time |
| Traditional Cell Cultures | Human cells; Controlled environment; High-throughput | Simplified systems; Lack tissue complexity |
| Organs-on-Chips | Human-relevant data; Complex tissue architecture; Real-time monitoring | Emerging technology; Standardization needed; Higher cost |
This acceptance marks a pivotal moment—the first time a non-animal, human-based microphysiological system has been recognized as a potential standalone tool for regulatory decision-making about drug safety 9 .
Behind every medical breakthrough lies a suite of essential research tools. Here are key reagents and their functions in modern drug development research:
| Reagent Category | Examples | Primary Function |
|---|---|---|
| Buffer Solutions | Phosphate-Buffered Saline (PBS), Tris Buffer | Maintain stable pH and conditions for biological reactions 4 |
| Staining Reagents | Gram Stain, Crystal Violet | Differentiate cell types and visualize microscopic structures 4 |
| Biochemical Reagents | Kovacs' Reagent, Oxidase Reagent | Identify specific metabolic pathways and bacterial properties 4 |
| Immunological Reagents | Bovine Serum Albumin (BSA), Anti-Human Globulin | Block non-specific binding and detect antibodies in immunoassays 4 |
| Cell Culture Reagents | Trypsin-EDTA, Physiological Saline | Detach cells from surfaces and maintain cell viability 4 |
As science continues to advance, FDA is preparing for even more transformative changes. The agency is actively monitoring emerging fields like:
FDA has published a comprehensive paper outlining how its centers are working together on AI development and use across the medical product lifecycle 9 .
FDA continues to develop frameworks for using real-world data from clinical practice to support regulatory decisions 5 .
Exploring complex innovative designs that can make clinical research more efficient and patient-focused 5 .
| Initiative | Purpose | Status |
|---|---|---|
| ISTAND Program | Qualify innovative drug development tools beyond traditional pathways | Active; first organ-on-chip accepted in 2024 9 |
| Sentinel Initiative | Create a national electronic system for monitoring medical product safety | Ongoing; enhances post-market safety surveillance 8 |
| Rare Disease Cures Accelerator | Facilitate cooperative approaches and standardized platforms for rare diseases | Developing common resources for rare disease research 8 |
The 21st Century Cures Act represents more than just regulatory changes—it embodies a fundamental shift in how we approach medical product development.
By embracing patient perspectives, leveraging real-world evidence, and adopting innovative tools like organs-on-chips, the FDA is transforming to keep pace with scientific advancement while maintaining its gold standard for safety and efficacy.
This modernization benefits everyone: patients gain faster access to breakthrough treatments, researchers can utilize more predictive testing methods, and the public health system becomes more responsive to emerging challenges. As FDA Commissioner Dr. Scott Gottlieb noted in the early implementation of the Cures Act, these changes help ensure that "medical discoveries can move from the laboratory to consumers more efficiently" 1 .
The future of medicine is being shaped not just in laboratories, but in the regulatory frameworks that ensure innovations reach the patients who need them—safely, efficiently, and effectively.