Patients Are at the Heart of Cell and Gene Therapy

In the rare disease community of 7,000+ diseases, where only a few hundred have treatments, cell and gene therapies can be a lifeline—and the only hope. So, this is an exciting time in medicine. However, while many of these therapies hold great promise, they also carry substantial challenges and risks, and there is a gap in awareness and understanding of cell and gene medicine. It is critical that patients are well informed, that their reservations are addressed, and that their expectations are managed —not everyone will be viable candidates who are able to get these therapies.

Research studies show that one of the biggest concerns people have about gene therapy is not having appropriate information or being able to make an informed decision. These technologies are very new and are advancing at an unprecedented pace. The science is creating a lot of hope for potentially curative therapies but there are also so many unanswered questions. Easy-to-find, easy-to-understand, and trustworthy resources are imperative for patients to make informed decisions.

If you are an organization content with patient-centric approaches, you are behind. Today it is about patient-driven drug development. Patients are driving drug development more than ever before, bringing desirable assets to research partners, including clinical trial data, registries, natural history studies, electronic health records (EHRs), and genetic test results such as whole genome sequencing. Many patient organizations are also funding and investing in research.

At Global Genes, we equip the rare disease community with reliable information to help them make healthcare decisions. Regarding cell and gene medicine, the questions patients most frequently ask are about:

• Immunogenicity: Is this my only chance to try a gene therapy? What if it is not effective? What if I have antibodies that attack the vector that is used?
• Durability: Is this a cure? How long will the effect of the therapy last? Will I need additional treatments?
• Long-term follow-up following treatment: How long will I need to participate in these studies? What is involved in being tracked/monitored long term?
• Access: Will these drugs be available in time to help me? What do these drugs cost? Will my insurance help cover the cost? How can I help payers and policymakers understand the value of these therapies?

Education and patient focus are two important keys for society to accept cell and gene therapies, as well as:

• Affordable genetic testing, including patient assistance and resources.
• Rare disease patient participation in natural history studies and disease registries, which can help us understand disease progression and treatment potential (providers and researchers can be contributing data to these studies whenever possible with consent from patients).

At Global Genes, we’re hopeful and optimistic about the transformative potential for cell and gene therapies in rare disease, and we encourage the entire healthcare ecosystem to continue to work tirelessly toward solutions to turn hope into reality for these patients and families.

Susan Brisendine, VP, Engagement, Programs, Global Genes