The Vital Role of Education in Patient Advocacy: A Gene Therapy Perspective

March 27, 2024

Gene therapy promises to revolutionize medicine, particularly for rare diseases, approximately 80% of which are genetic in origin, and most are monogenic/Mendelian. 2023 was a landmark year for cell and gene therapies approved by the FDA, and significant progress continues in 2024 across drug pipelines spanning multiple therapeutic areas.

Read more:

Changing What’s Possible with Cell and Gene Therapies

The 2024 NEXT Report: Rewriting the Rules

However, as this groundbreaking field progresses, it’s crucial to emphasize the indispensable role of patient education, particularly within the context of clinical trials. Understanding the world of cell and gene therapy-based treatments will enhance patients’ ability to make informed decisions about participating in these trials. This is an example of education driving action.

Education cannot be divorced from advocacy. Education empowers individuals living with rare diseases to not only understand the molecular basis of their condition but to then feel confident to engage in informed conversations with their healthcare team, including conversations about the feasibility of a gene-based treatment and risk/benefits of gene therapy-based clinical trials. Education amplifies effective advocacy by equipping patients and families to:

Understand the Science

Gene therapy involves manipulating genes to treat diseases, a concept that can seem daunting to patients that are unfamiliar with molecular biology. Patient engagement efforts need to focus on disease-state education and providing accessible explanations of the science behind gene therapy, clarifying its goals, mechanisms, and potential benefits. 

Learn more about gene replacement and gene editing therapies:

View session from the Global Genes 2023 RARE Advocacy Summit

Make Informed Decisions

Participation in gene therapy clinical trials is a significant decision that requires informed consent. Patients must comprehend the nature of the treatment, including potential risks, benefits, and uncertainties. Educating patients about the trial protocol, its objectives, and what participation entails empowers them to make informed choices about their healthcare journey.

Resources like the toolkit below provide information and guidance for anyone considering participating in a clinical trial.

Download toolkit

Manage Expectations

While gene therapy holds promise, it’s not a guaranteed cure. Patient education should foster realistic expectations about the therapy’s limitations, possible side effects, and the likelihood of success. Managing expectations also helps mitigate disappointment and ensures patients approach the trial with a balanced perspective.

Examine Ethical Considerations

Ethical principles underpin gene therapy research, encompassing issues such as privacy, equity, and consent. Patient education plays a vital role in fostering understanding of these ethical considerations, promoting transparency, and fostering trust between researchers and participants. 

Learn more about making informed and shared decisions about clinical trial participation.

Empower Advocacy

Well-informed patients are empowered advocates for their own health and for the advancement of gene therapy research. By educating people about their rights, the significance of their contributions to science, and the broader societal implications of gene therapy, we foster a community of engaged stakeholders driving progress in healthcare.

Patient education is not merely a peripheral aspect of gene therapy clinical trials (or any clinical trial, for that matter) but an integral component essential for ensuring ethical conduct, informed decision-making, and meaningful patient engagement. By recognizing patient education as an integral part of advocacy, we uphold the values of transparency, autonomy, and respect, and ensure that gene therapy research and clinical trial recruitment is conducted responsibly and equitably.

About the Author

Shruti Mitkus, Ph.D. is the Director of Genetic Education and Navigation at Global Genes. She is a molecular geneticist-turned-patient advocate who has worked extensively as a research scientist and in genetic diagnostics for rare diseases. She leads Global Genes’ patient navigation service (RARE Concierge) as well as directs educational programming and initiatives. Shruti uses her expertise in genetics and medical communication to help rare disease patients and their families understand their disease and feel empowered to make evidence-based decisions about their care.

Visit RARE Concierge

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