Aligning innovation with patient needs in gene and cell therapy development

Gene and cell therapies are providing new treatment for those with rare diseases. These therapies offer hope for long-term solutions—and even potential cures—for millions of people worldwide. However, for these treatments to truly make a difference, they must align with patient values, perspectives, and access needs.

Our latest whitepaper, "Gene and cell therapies in rare diseases: Aligning innovation with patient needs," dives deep into the current state of gene and cell therapies, exploring critical advancements, patient perceptions, and actionable strategies for creating more inclusive, accessible treatments. Because while the science is advancing at a rapid pace, major hurdles remain—including high costs, limited patient understanding, and unequal access. 

Patient perspectives on cell and gene therapies

Gene and cell therapies are transforming rare disease treatment by targeting underlying genetic causes. Gene therapy modifies DNA to correct mutations, while cell therapy replaces or repairs damaged cells. These approaches offer long-term solutions for diseases caused by single gene mutations.

Successful therapies must prioritize patient experiences and needs. Clear information about treatment benefits, risks, and long-term effects is essential. High costs restrict access, especially in lower-income regions where diseases like SCD are prevalent. Involving patients in development and offering options like at-home testing can improve accessibility and satisfaction.

Stakeholder recommendations

The whitepaper expands on the following recommendations for relevant stakeholders.

For Pharma and Biotech:

• Communicate risks and benefits clearly.
• Implement multi-stage consent processes.
• Provide personalized support systems.

For Policymakers:

• Incentivize therapy development through frameworks like Orphan Drug Designation.
• Promote equitable access by viewing therapies as long-term healthcare investments.

For Patient Advocacy Groups:

• Amplify patient voices in therapy design.
• Collaborate on patient-reported outcome measures.

Gene and cell therapies offer new hope for rare disease patients. A patient-centric approach focusing on affordability, accessibility, and education is crucial. Collaboration among researchers, policymakers, and advocates will ensure these therapies reach those in need, advancing effective treatment and management of rare diseases.