2024 was a transformative year in pharmacy and therapeutics, marked by groundbreaking advances that promise lasting impacts on healthcare. Dr João Gonçalves PhD presents his pick for this year’s top five pharmacy innovations, illustrating how they set new standards for patient care and pharmaceutical development.
The key pharmacy innovations witnessed throughout 2024 reflect a growing shift toward precision medicine, affordability and rapid therapeutic development.
mRNA vaccines: from Covid-19 and beyond
The rapid success of messenger ribonucleic acid (mRNA) vaccines during the Covid-19 pandemic set the stage for broader applications, and in 2024, mRNA technology expanded significantly. Researchers have now adapted mRNA vaccines to target a range of other infectious diseases such as influenza, HIV and Zika.
One of the year’s most exciting innovations includes tailored mRNA vaccine trials to target patient-specific mutations in certain malignancies, creating a personalised immunotherapy approach. The scalability of mRNA enables rapid vaccine production and reduces costs, making it more accessible across healthcare systems.1,2
With the flexibility to quickly design and deploy new vaccines, mRNA technology is proving its worth beyond Covid-19, showing promise for rapid responses to global health threats and for patient-specific cancer care.3,4
Artificial intelligence in innovative drug discovery
The use of artificial intelligence (AI) has rapidly progressed in the field of drug discovery, and 2024 marked a turning point with its real-world applications. Leading pharmaceutical companies now use machine learning and deep learning to reduce drug development timelines significantly. AI models can screen vast libraries of compounds, identify promising candidates and predict toxicological effects far faster than traditional methods.
This year, AI played a pivotal role in discovering a new class of antibiotics, crucial as antibiotic resistance continues to be a global health challenge.5 Deep learning algorithms were used to sift through millions of compounds, identifying those with high efficacy against resistant strains.
AI is accelerating drug discovery and improving precision in patient care. By analysing biological data, AI can predict how different patient populations might respond to a given drug, reducing the time and costs associated with trial and error in prescribing.
This year’s advancements underscore AI’s potential to become a staple in drug development, making personalised therapies feasible and affordable.
Biosimilars for autoimmune diseases: expanding access
The continued high demand for biologic drugs to treat autoimmune diseases has led to growing healthcare costs, and biosimilars are stepping in as a cost-effective solution.6 This year saw an unprecedented increase in the adoption of biosimilars, such as adalimumab and infliximab, used to treat rheumatoid arthritis and Crohn’s disease.
A major study in 2024 led by pharmacist David Young confirmed that switching from original biologics to biosimilars did not compromise patient outcomes. This has encouraged more healthcare systems to adopt biosimilars, particularly in regions where high biologic costs have historically limited access.
Patients switching to biosimilars experience comparable results, making life-changing treatments more affordable and improving patient adherence. As more biosimilars gain regulatory approval, healthcare systems stand to save significantly, which could allow for better allocation of resources toward innovative therapies.
Innovations in pharmacogenomics and personalised medicine
Pharmacogenomics – the study of how genes affect a patient’s response to drugs – has been increasingly integrated into clinical practice in 2024. This year, genetic testing has shown to be invaluable in fields including cardiology, psychiatry and oncology, where drug responses can vary widely among individuals. By considering a patient’s unique genetic profile, pharmacists and clinicians can choose safer and more effective treatments, improving outcomes and reducing adverse effects.7–9
Pharmacogenomics embodies the promise of personalised medicine. By minimising trial-and-error prescribing, pharmacogenomics helps reduce hospitalisations, enhances patient satisfaction and opens the door to more precise, effective treatment options. As genetic testing becomes more accessible, it is set to become an essential component of pharmacy and routine care, transforming the patient experience.
CRISPR-Cas9 gene editing for rare diseases
CRISPR-Cas9 gene editing reached new heights in 2024, with early-phase trials showing promising results for rare genetic diseases. This innovative technology allows for targeted editing of disease-causing genes, offering potential cures where conventional treatments have been limited or ineffective.10
Patients with sickle cell disease and beta-thalassaemia were among those who first benefited from CRISPR treatments, with results indicating long-term improvements and reduced disease symptoms.11
Gene editing applications are also expanding into other genetic conditions, with trials now targeting metabolic and neurodegenerative diseases.12 In these cases, correcting genetic mutations at the molecular level has shown promise in preclinical studies, with several therapies progressing toward clinical trials.
CRISPR’s potential to ‘cure’ genetic diseases is sparking excitement, but it also raises ethical questions, particularly around accessibility, affordability and long-term safety.
As CRISPR trials advance, the technology offers hope for patients with conditions once considered untreatable. Its potential to provide one-time treatments instead of lifelong therapies could redefine healthcare for rare diseases.
While ethical and regulatory discussions are ongoing, CRISPR is changing the narrative around genetic disorders and transforming our understanding of what’s possible in medicine.
Conclusion
These innovations, which are central to the work of pharmacy, address critical challenges in modern healthcare, from the expanded applications of mRNA vaccines and CRISPR gene editing to AI-driven drug discovery and the cost-saving promise of biosimilars. The advancement of pharmacogenomics also adds a crucial layer of personalisation to patient care, allowing for optimised, targeted treatment.
Author
João Gonçalves PharmD PhD
Faculty of Pharmacy, University of Lisbon, Portugal
References
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- Hsieh CL et al. Prefusion-stabilized SARS-CoV-2 S2-only antigen provides protection against SARS-CoV-2 challenge. Nat Commun 2024;15:1553.
- Bitounis D et al. Strategies to reduce the risks of mRNA drug and vaccine toxicity. Nat Rev Drug Discov 2024 Apr;23(4):281–300.
- Huang X et al. Unlocking the Therapeutic Applicability of LNP-mRNA: Chemistry, Formulation, and Clinical Strategies. Research (Wash D C) 2024 Jun 18;7:0370.
- Swanson K et al. Generative AI for designing and validating easily synthesizable and structural novel antibiotics. Nat Machine Intelligence 2024;6:338-53.
- Hanauer SB et al. Subcutaneous Infliximab (CT-P13 SC) as Maintenance Therapy for Inflammatory Bowel Disease: Two Randomized Phase 3 Trials (LIBERTY). Gastroenterology 2024 Oct;167(5):919–33.
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- Ferwerda ME et al. Pharmacogenetic educational needs and the role of pharmacogenetics in primary care: a focus group study with multiple perspectives. Front Pharmacol 2024 Jul 23;15:1404370.
- Davis DJ, Yeddula SGR. CRISPR Advancements for Human Health. Mo Med 2024 Mar-Apr;121(2):170–6.
- Greco F, Cosentino M, Marino F. The Italian breakthrough in CRISPR trials for rare diseases: a focus on beta-thalassemia and sickle cell disease treatment. Front Med (Lausanne) 2024 Feb 15;11:1356578.
- Deneault, E. Recent Therapeutic Gene Editing Applications to Genetic Disorders. Curr Issues Mol Biol 2024;46:4147–85.
