Activity ID
14031Expires
January 13, 2028Format Type
Journal-basedCME Credit
1Fee
$30CME Provider: JAMA Pediatrics
Description of CME Course
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.
Disclaimers
1. This activity is accredited by the American Medical Association.
2. This activity is free to AMA members.
ABMS Member Board Approvals by Type
ABMS Lifelong Learning CME Activity
Allergy and Immunology
Anesthesiology
Colon and Rectal Surgery
Family Medicine
Medical Genetics and Genomics
Nuclear Medicine
Ophthalmology
Pathology
Physical Medicine and Rehabilitation
Plastic Surgery
Preventive Medicine
Psychiatry and Neurology
Radiology
Thoracic Surgery
Urology
Commercial Support?
NoNOTE: If a Member Board has not deemed this activity for MOC approval as an accredited CME activity, this activity may count toward an ABMS Member Board’s general CME requirement. Please refer directly to your Member Board’s MOC Part II Lifelong Learning and Self-Assessment Program Requirements.
Educational Objectives
To identify the key insights or developments described in this article
Keywords
Health Care Economics, Insurance, Payment, Genetics and Genomics, Pediatrics
Competencies
Medical Knowledge
CME Credit Type
AMA PRA Category 1 Credit
DOI
10.1001/jamapediatrics.2024.5100
