The intersection of pharmacists and biotechnology—particularly in gene therapy and pharmacogenomics—is transforming health care. These advancements enable pharmacists to increasingly customize treatments based on genetic profiles, enhancing drug efficacy and safety. However, challenges persist, particularly the high costs associated with gene therapies and the ethical dilemmas surrounding pharmacogenomics. As health care evolves, pharmacists must navigate these obstacles to realize the benefits of these innovations while addressing the financial and ethical complexities of this emerging field.
Gene therapy is an innovative medical approach that involves modifying or replacing defective genes to treat or prevent genetic disorders.1 A significant advancement in this field is the development of CRISPR (clustered regularly interspaced short palindromic repeats)-Cas9, a gene-editing technology that allows for precise alterations in a patient’s DNA sequence. CRISPR-Cas9 has shown promise in correcting mutations responsible for various genetic conditions, including sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Notably, the first CRISPR-based medicine, exagamglogene autotemcel (exa-cel; Casgevy; Vertex Pharmaceuticals Incorporated) was approved in 2023 for treating SCD and TDT, marking a milestone in gene therapy applications.2
As gene therapies become more integrated into clinical practice, pharmacists will be crucial in managing these protocols. Their responsibilities include ensuring the proper storage, preparation, and administration of gene therapy products. Pharmacists are also essential for providing education to health care providers and patients as well as monitoring for potential adverse effects (AEs).
Although gene therapy has the potential to cure diseases, the high cost of treatment makes access extremely difficult.3 Pharmacists play an impactful role in spreading awareness about potential therapeutic options and preventing barriers to patient access.
The limited accessibility for patients is a significant challenge associated with gene therapy.4 The high costs arise from the significant development and research required to create these technologies. Additionally, gene therapy is often associated with being a treatment option for rare or ultrarare diseases, imposing a high cost for individual treatment as there is a reduced population of patients who would receive the treatment.
Gene therapies come with expensive prices for patients, and many health plans either do not cover the already-approved gene therapies on the market or place restrictions on the number of patients who may be treated with the therapy per year.5
Pharmacists have a duty to balance these promising therapies with the financial burden on patients and health care systems. Because formulary decisions incorporate the clinical benefits and long-term impacts of these high-cost treatments, it is important for pharmacists to be involved in helping health care centers weigh the benefits and financial feasibility of gene therapies.
Pharmacogenomics, the study of genetic variations that influence drug response, is rapidly reshaping modern pharmacotherapy. With advancements in biotechnology, pharmacists are at the forefront of incorporating genetic information into clinical practice, offering personalized medicine that improves therapeutic outcomes and minimizes AEs. Integrating pharmacogenomic data into clinical workflows allows pharmacists to tailor drug regimens based on a patient’s genetic profile, enhancing the efficacy of treatments such as oncology drugs and psychiatric medications.6
About the Authors
Nicole Aldover, PharmD, RPh, is a global medical communications-global medical affairs PharmD fellow at Alexion Pharmaceuticals in affiliation with Massachusetts College of Pharmacy and Health Sciences.
Satyaharshini Reddy, PharmD, is a global medical communications-global medical affairs PharmD fellow at Alexion Pharmaceuticals in affiliation with Massachusetts College of Pharmacy and Health Sciences.
Zahra Arsalan, PharmD, RPh, is a global medical communications-global medical affairs PharmD fellow at Alexion Pharmaceuticals in affiliation with Massachusetts College of Pharmacy and Health Sciences.
Sawyer Patrick, PharmD, RPh, is a US medical affairs fellow at Alexion Pharmaceuticals in affiliation with Massachusetts College of Pharmacy and Health Sciences.
Although pharmacogenomics holds significant promise for personalized medicine, it also raises several ethical implications. Access to pharmacogenomic testing can be unequal, particularly in low-resource settings where such technologies may be unavailable or unaffordable, exacerbating existing disparities in health care.7 These advancements in pharmacogenomics must be distributed fairly, ensuring all patient populations benefit, regardless of socioeconomic status.
Additionally, the use of genetic data in pharmacogenomics necessitates stringent safeguards to protect patient privacy.8 There is a concern that genetic information could be misused by insurers or employers, leading to discrimination based on genetic predispositions. Therefore, robust legal frameworks and ethical guidelines must be established to secure patient confidentiality while fostering trust in pharmacogenomic innovations. Addressing these ethical considerations is crucial for realizing the full potential of pharmacogenomics in a socially responsible manner.
Pharmacists can play a crucial role in mitigating the ethical implications of pharmacogenomics by educating and guiding patients and health care providers, advocating for privacy protections and equitable access to personalized medicine, and supporting inclusive research to ensure fair distribution of benefits across all communities.9
In conclusion, the intersection of pharmacists and biotechnology, particularly in gene therapy and pharmacogenomics, holds extraordinary potential for personalized medicine. Pharmacists are integral to optimizing therapies based on genetic insights to improve treatment outcomes; however, the high cost of gene therapies and the ethical considerations surrounding pharmacogenomics may present substantial hurdles. As the field continues to advance, pharmacists must remain key contributors in navigating these complexities, ensuring that innovations in medicine are accessible and ethically sound for all patients.
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