Can Patients Trust a Drug Designed By AI?
Can we trust AI-designed therapeutics? Dr. Jo Varshney says: “Yes, and here’s why.”
In traditional drug discovery, it can take an entire decade and in excess of a billion dollars to successfully bring a drug to market. Despite the best efforts of scientists, around 90% of proposed drugs fail at the clinical trial stage.
AI technology presents a new era for drug discovery – one where drugs can be tested and refined in silico before reaching human trials, resulting in dramatic time and cost savings.
While this technological advancement is of great excitement to scientists and those involved in the drug discovery pipeline, patients may naturally have some concerns when they hear of “AI-designed therapeutics”.
To learn more about how AI is shaping drug development and the importance of prioritizing transparency, safety and building patient trust, Technology Networks spoke with Dr. Jo Varshney, CEO and founder of VeriSIM Life. At VeriSIM Life, Varshney leverages her experience in computational pharmacology to lead the team developing their BIOiSIM® platform, an AI-driven mechanistic modeling system designed to predict drug behavior in human biology.
How is AI and machine learning being applied in the drug discovery space?
Artificial intelligence and machine learning are redefining how medicines are designed, developed and delivered – from the earliest stages of discovery to clinical translation. One of the greatest challenges in biopharma has always been predicting how a potential therapy will behave in the human body. Traditional models rely on limited datasets and animal studies that too often fail to translate.
At VeriSIM Life, we go far beyond discovery. Our BIOiSIM® platform integrates data and knowledge spanning chemistry, multiomics, physiology and clinical outcomes into dynamic, mechanistic simulations that model how drugs interact with living systems. This allows us not only to discover better molecules but to develop them more intelligently – predicting human dosage, toxicity, efficacy and even patient variability before a clinical trial begins.
By simulating biology itself, we can test hypotheses, optimize formulations and identify risks long before they become costly failures – reducing time, expense and the ethical burden of animal testing. The result is a smarter, faster and more predictive path from idea to impact.
In essence, AI/ML is not just transforming drug discovery – it’s transforming drug development. It enables us to move from reactive science to proactive design, where every decision is informed by data, every model reflects biology and every step brings us closer to safer, more effective and more humane medicines for patients.
What does the increased adoption of AI in drug development mean for pharmaceutical companies? What about clinicians and patients?
For pharmaceutical companies, AI means making smarter bets. Instead of pouring billions into programs that fail late, companies can now identify risks earlier, prioritize the most promising assets and optimize trial design and dosing with far greater precision. This shift transforms R&D from a costly gamble into a data-driven discipline, accelerating timelines, reducing waste and enhancing capital efficiency across the portfolio.
For clinicians, AI brings confidence. Therapies entering the clinic are better characterized, with deeper insight into safety, efficacy and variability across patient populations. It moves medicine from assumption to prediction – empowering physicians with evidence that reflects real-world biology, not just trial averages.
And for patients, the impact is profound. AI shortens the time to breakthrough treatments, minimizes exposure to ineffective or unsafe drugs and opens the door to therapies tailored to their biology. It transforms access, safety and trust, ensuring innovation reaches the people who need it most.
Ultimately, the power of AI in drug development is not only about efficiency – it’s about equity. By making the process more predictive and transparent, we can bring better medicines to more patients more quickly and with the confidence that science and technology are working together for the greater good.
VeriSIM Life’s BIOiSIM platform uses AI to drive mechanistic modeling and inform drug discovery efforts, helping partners to bring new drugs to market more quickly. Can you tell us a little more about this platform and how it works?
You can think of BIOiSIM as a 'flight simulator for drug development'. It’s a mechanistic modeling system powered by AI that captures the complexity of human biology, including chemistry, organ systems, genomics and disease progression to predict how drugs will interact with the body.
Researchers can simulate dosing, safety and efficacy across different patient types in silico, dramatically reducing time and cost. It’s like a flight simulator for scientists, de-risking the journey before takeoff.
Some patients may have concerns about the safety and efficacy of an AI-designed therapeutic. What would you say to these concerns?
It’s completely natural for patients to ask, “Can I trust a drug designed with AI?” The answer is yes, and here’s why.
Every AI-designed therapeutic must meet the same rigorous safety, efficacy and regulatory standards as any traditional drug. Nothing about AI changes the fact that a therapy must pass preclinical testing, multiple phases of clinical trials and a comprehensive US Food and Drug Administration review before reaching patients. The difference is that AI doesn’t bypass these safeguards; it enhances them.
At VeriSIM Life, we utilize AI to enhance the safety net: our predictive simulations identify potential safety or efficacy issues well before human trials commence. That means fewer late-stage failures, smarter trial designs and therapies that are more likely to perform as intended once they reach people.
Patients should feel confident that AI isn’t replacing scientific rigor – it’s reinforcing it. It enables researchers to make better, data-driven decisions, design safer dosing strategies and continuously refine insights as new data emerges.
In short, while a therapy may be “AI-designed,” its approval still rests on the same scientific rigor, regulatory oversight and ethical responsibility that have always protected patients; only now, these standards are strengthened by technology that helps make medicine safer, faster and more humane.
Cybersecurity and data privacy are core issues for pharmaceutical companies. How are these values protected when using AI-driven systems?
Cybersecurity and data privacy are not just compliance checkboxes. They are the foundation of trust in modern drug development. In an era where AI depends on massive volumes of biomedical and proprietary data, protecting that information is absolutely nonnegotiable.
At VeriSIM Life, we treat data protection as a fundamental principle. Every dataset, whether preclinical, clinical or partner-provided, is handled with the highest security standards. That means end to end encryption, strict access controls and continuous monitoring to identify and neutralize threats in real time. We conduct regular third-party security audits and maintain compliance with global frameworks.
Our AI systems are also designed to minimize exposure from the start. We primarily work with anonymized and de-identified datasets, ensuring that sensitive patient information remains protected at all times.
Ultimately, cybersecurity and privacy are not just technical safeguards. They are enablers of innovation. By protecting the integrity of our data and the confidentiality of our partners, we enable pharmaceutical companies, clinicians and researchers to utilize AI with complete confidence. Responsible AI can only thrive when it is built on a foundation of security, transparency and trust.
What else can the pharma industry do to ensure patient trust?
Transparency is absolutely essential in the pharmaceutical industry, especially as we depend more on AI and other advanced technologies to develop new therapies. Patients, clinicians and regulators must have confidence not only in the safety and effectiveness of treatments, but also in the integrity of the processes and data that shape them. Transparency builds that confidence by making decisions understandable, methods open to scrutiny and limitations clearly communicated.
At VeriSIM Life, transparency is built into our technology through explainable AI. Our systems are designed not only to show what a model predicts, but also why. By revealing the biological and mechanistic reasoning behind each prediction, we make AI-driven insights interpretable to scientists, regulators and clinicians. This approach strengthens validation, improves decision-making and ensures that trust is earned through clarity, not abstraction.
True trust goes beyond transparency alone. Companies should proactively share data from preclinical and clinical research, communicate clearly about how AI is being used and collaborate with independent researchers and patient advocacy groups. Just as important is maintaining high ethical standards, protecting patient data and demonstrating through action that patient welfare always comes before commercial interest.
By combining openness with explainable AI, we ensure that innovation is not only effective but also understandable, accountable and worthy of patient trust. Firms that lead this way will drive the next generation of responsible and sustainable progress in medicine.