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Publication
The cost of biotech innovation: Exploring research and development costs of cell and gene therapies
Molecular Therapy - Methods & Clinical Development -
Publication
The Landscape of Cellular and Gene Therapy Products: Authorization, Discontinuations, and Cost
Human Gene Therapy Clinical Development -
Video
Value and Values in Payment for Gene Therapies
The ELSI Friday Forum was held on May 12, 2023. Advances in gene therapies for monogenic disorders have brought hope of curative treatment. Since the Food and Drug Administration first approved a…- gene therapies
- cost of gene therapies
- payment
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Collection
Paying for Cures: The Ethics and Economics of Gene Therapies for Rare Diseases
Rare diseases, most of which have a genetic basis, collectively affect an estimated 300 million individuals worldwide and are associated with significant morbidity and mortality. Although less than…- gene therapy
- sickle cell disease
- health economics
- nusinersen
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Publication
Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions
Molecular Therapy - Methods & Clinical DevelopmentAllen and colleagues describe how discrepancies between the legal requirement to cover all FDA-approved drugs according to their medically accepted indications and the coverage policies of 16… -
Publication
Gene therapies: The challenge of super-high-cost treatments and how to pay for them
Regenerative Medicine -
Publication
Value-based pricing for emerging gene therapies: The economic case for a higher cost-effectiveness threshold
Journal of Managed Care & Specialty Pharmacy -
Publication
Infrastructuring precision medicine: Making gene therapies for rare diseases workable in practice
Social Science in MedicineWadmann and colleagues describe the work required to fit the first two clinically available in-vivo gene therapies into the publicly funded Danish healthcare system. -
Grant Abstract
From Research to Clinic: Addressing Ethical and Policy Challenges Facing the Gene Therapy Ecosystem
PROJECT NARRATIVE The translational gene therapy ecosystem faces several interrelated challenges: 1) obtaining sufficient evidence of safety/efficacy from clinical trials to justify regulatory… -
Publication
Equitable access to gene therapy: A call to action for the American Society of Gene and Cell Therapy
Molecular Therapy