Abstract

The conventional drug development pipeline—from target discovery through clinical trials to regulatory approval—traditionally spans more than a decade and costs billions of dollars. In the face of urgent medical needs, such as pandemics, rare diseases, and antimicrobial resistance, accelerated strategies have become critical. This article reviews innovations and regulatory reforms that promise to compress timelines, reduce costs, and improve success rates while maintaining scientific rigour. The discussion highlights applications of artificial intelligence, adaptive trial designs, digital twins, real-world data, decentralized trials, and drug repurposing. It further considers mRNA vaccines and CRISPR-based drug discovery as transformative paradigms. Finally, it discusses regulatory pathways, equity challenges, and ethical implications shaping the future of drug development.

1. Introduction

Drug development is one of the most resource-intensive endeavours in modern science. The average cost of bringing a new therapy to market is estimated at USD 1–2 billion, with timelines often exceeding 12 years and clinical attrition rates above 85%1. This slow pace is incompatible with urgent needs such as global pandemics, rising cancer incidence, and antimicrobial resistance. To address these gaps, new strategies—technological, methodological, and regulatory—are being designed to fast-track innovation without compromising safety.

2. Artificial Intelligence and Machine Learning

Artificial intelligence (AI) and machine learning (ML) are revolutionising early-stage research. AI algorithms enable target identification, de novo molecular design, protein structure prediction, and virtual screening of compound libraries2. For example, DeepMind’s AlphaFold has transformed protein structure modelling, significantly accelerating drug discovery3. ML also supports trial optimisation by predicting patient responses, identifying biomarkers, and minimising recruitment bottlenecks. AI-driven platforms such as BenevolentAI and In-silico Medicine demonstrate how computational approaches can generate viable candidates within months compared with years in traditional pipelines.

3. Adaptive Trial Designs

Conventional randomised controlled trials (RCTs) are often rigid and inefficient. Adaptive trial designs allow modification of parameters—such as dose, sample size, or treatment arms—based on interim analyses. This reduces wasted resources and accelerates decision-making?. Platform trials, such as RECOVERY during COVID-19, illustrate the utility of adaptive frameworks in testing multiple therapies simultaneously under a master protocol?. Regulators now increasingly endorse adaptive designs, provided robust statistical controls are applied to maintain validity.

4. Digital Twins and Real-World Data

Digital twin models—virtual replicas of human systems—offer unprecedented potential for in silico testing of interventions. These computational simulations enable pre-clinical exploration of drug effects under diverse scenarios, thereby refining hypotheses before trials commence?. In parallel, real-world data (RWD) derived from electronic health records, wearables, and patient registries is reshaping post-market surveillance and trial augmentation. Regulatory agencies, including the US FDA and EMA, are developing frameworks for using RWD in decision-making11,12, although concerns about data quality, privacy, and interoperability remain.

5. Decentralized Clinical Trials

Decentralised clinical trials (DCTs) leverage telemedicine, home delivery of investigational products, and remote monitoring to overcome geographic and logistical barriers. DCTs became prominent during the COVID-19 pandemic, with regulators issuing guidance to sustain research continuity?. Beyond improving convenience and diversity in recruitment, DCTs reduce dropout rates and enable real-time data capture. However, infrastructure disparities and cybersecurity issues require ongoing attention before widespread implementation.

6. Drug Repurposing

Drug repurposing—applying existing drugs for new indications—remains one of the most efficient strategies to accelerate therapeutic availability. Repurposed drugs bypass early safety testing, saving years in development. The discovery of dexamethasone’s efficacy in severe COVID-19 cases is a notable example?. Computational screening and network pharmacology approaches are expanding repurposing opportunities across oncology, neurology, and infectious diseases?.

7. Expedited Regulatory Pathways

Regulatory agencies now offer accelerated frameworks such as the FDA’s Fast Track, Breakthrough Therapy, and Accelerated Approval programmes. The EMA provides PRIME (PRIority MEdicines) and conditional approvals12. These mechanisms allow earlier patient access while requiring confirmatory evidence. While essential during public health emergencies, expedited approvals raise concerns about long-term safety monitoring and post-market commitments1?. Harmonisation between global agencies remains a pressing challenge for multinational development programmes.

8.  mRNA Vaccines as a Model for Rapid Therapeutics

The COVID-19 pandemic highlighted the transformative potential of messenger RNA (mRNA) technologies. Unlike traditional vaccines, mRNA vaccines can be designed within weeks once the viral sequence is available1?. Their rapid scalability, modular design, and adaptability position mRNA platforms as models for broader drug development. Beyond infectious diseases, trials are underway for mRNA-based cancer vaccines and personalised immunotherapies1?. The ability to bypass lengthy cell-culture processes shortens timelines dramatically, but challenges such as cold-chain requirements and global accessibility remain key barriers.

9. CRISPR and Gene Editing in Drug Discovery

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology has reshaped functional genomics and therapeutic innovation. CRISPR enables high-throughput gene editing to validate drug targets, model diseases, and even directly treat genetic disorders1?. CRISPR-based screens accelerate early discovery, identifying vulnerabilities in cancer and antimicrobial resistance pathways. Clinical trials of CRISPR-based therapies, including those targeting sickle cell disease, illustrate its promise for precision medicine1?. Nevertheless, ethical concerns, off-target effects, and regulatory hurdles demand careful oversight before widespread adoption.

10. Discussion

Recent advances in AI, adaptive designs, DCTs, and repurposing illustrate how scientific and regulatory innovation can compress development timelines. Yet challenges persist. Data quality and reproducibility remain critical barriers for AI and RWD integration. Adaptive trials demand advanced statistical expertise, and DCTs face issues of digital literacy and inequity. Moreover, balancing speed with safety remains the central ethical dilemma13,1?.

The discussion must also acknowledge the role of global equity. While wealthy nations can leverage AI platforms, decentralised trials, and regulatory fast-tracking, many low- and middle-income countries (LMICs) still struggle with basic infrastructure and access to innovative therapeutics. Addressing these disparities requires multilateral collaboration, technology transfer, and harmonised regulatory frameworks that prevent innovation gaps1?. Ethical considerations—particularly around patient privacy in digital trials and the use of real-world data—remain central. Thus, any acceleration strategy must integrate principles of equity, transparency, and global accessibility.

Future research must explore the use of programmable virtual humans, enhanced patient-centric trial methodologies, and federated AI models that allow privacy-preserving data analysis across borders. Regulatory harmonisation for RWD, stronger post-market surveillance, and global capacity-building initiatives will be essential. By embedding equity, ethics, and transparency into innovation, the promise of faster drug development can be realised without sacrificing trust or safety.

11. Conclusion

The convergence of computational power, innovative trial methodologies, repurposing strategies, mRNA platforms, CRISPR-based discovery, and regulatory reforms defines a new era in drug development. These approaches are not merely incremental but transformative, promising to reduce the average timeline of drug development from over a decade to a few years. However, this acceleration must be accompanied by rigorous safeguards, global equity, and sustainable infrastructures to ensure that innovation translates into real health gains worldwide.

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