Importance: Cell and gene therapies are revolutionizing the treatment landscape for children and adults with rare diseases and can be life-changing for patients and their families. Successful implementation of these new therapies into clinical practice depends on their accessibility and affordability, particularly through publicly funded Medicaid agencies, which cover many children and adults with rare diseases.
Objective: To provide a framework to broadly assess cell and gene therapies, evaluate payment options, and ensure equitable access through the lens of publicly funded Medicaid programs.
Evidence review: This review draws on peer-reviewed articles, federal reports, and other relevant publications as well as the expertise of chief medical officers and medical directors of state Medicaid agencies across 5 diverse states.
Findings: Twenty-nine articles and other references provide the foundation for this review. The recommendations presented focus on thoughtful implementation of cell and gene therapies, including policy recommendations in the domains of safety, effectiveness, population health, access, and budget.
Conclusions and relevance: Proposed health care policy changes are intended to balance innovation, affordability, and equitable access for children and adults with rare diseases.