Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States
Gene therapies hold the promise of delivering groundbreaking improvements in health outcomes for previously intractable diseases. These therapies possess unique characteristics that differ from traditional small molecule or biologic treatments, posing new challenges for healthcare stakeholders. Wh...
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| Format: | Article |
| Language: | English |
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Becaris Publishing Limited
2024-11-01
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| Series: | Journal of Comparative Effectiveness Research |
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| _version_ | 1850220012127649792 |
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| author | Tyler D Wagner Lisabeth Buelt Kimberly Westrich Jonathan D Campbell |
| author_facet | Tyler D Wagner Lisabeth Buelt Kimberly Westrich Jonathan D Campbell |
| author_sort | Tyler D Wagner |
| collection | DOAJ |
| description | Gene therapies hold the promise of delivering groundbreaking improvements in health outcomes for
previously intractable diseases. These therapies possess unique characteristics that differ from traditional
small molecule or biologic treatments, posing new challenges for healthcare stakeholders. While previous
analyses have predominantly focused on the payment challenges associated with emerging gene
therapies, a more holistic examination reveals numerous obstacles that currently hinder optimal patient
access. In the United States (US), these include production and distribution issues, logistical and treatment
administration challenges and coverage and reimbursement limitations imposed by US healthcare payers.
Several opportunities exist to address these challenges and improve patient access, including the use of
appropriate value assessment methods, the development of innovative payment solutions, the removal
of inappropriate access barriers imposed by payers and improving education and awareness regarding
treatment availability, benefits and risks. Moving forward, we must collectively strive for comprehensive
policy and market solutions that are sensitive to the uniqueness of gene therapies to ensure their full
potential is realized and these treatments are made available to those in need. |
| format | Article |
| id | doaj-art-6c95e5fd45f746ab9802ccd905e789db |
| institution | OA Journals |
| issn | 2042-6313 |
| language | English |
| publishDate | 2024-11-01 |
| publisher | Becaris Publishing Limited |
| record_format | Article |
| series | Journal of Comparative Effectiveness Research |
| spelling | doaj-art-6c95e5fd45f746ab9802ccd905e789db2025-08-20T02:07:12ZengBecaris Publishing LimitedJournal of Comparative Effectiveness Research2042-63132024-11-01131210.57264/cer-2024-0098Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United StatesTyler D Wagner0https://orcid.org/0000-0001-7248-4890Lisabeth Buelt1Kimberly Westrich2https://orcid.org/0000-0001-9392-7670Jonathan D Campbell3https://orcid.org/0000-0001-6539-1258National Pharmaceutical Council, Washington DC 20006, USANational Pharmaceutical Council, Washington DC 20006, USANational Pharmaceutical Council, Washington DC 20006, USANational Pharmaceutical Council, Washington DC 20006, USAGene therapies hold the promise of delivering groundbreaking improvements in health outcomes for previously intractable diseases. These therapies possess unique characteristics that differ from traditional small molecule or biologic treatments, posing new challenges for healthcare stakeholders. While previous analyses have predominantly focused on the payment challenges associated with emerging gene therapies, a more holistic examination reveals numerous obstacles that currently hinder optimal patient access. In the United States (US), these include production and distribution issues, logistical and treatment administration challenges and coverage and reimbursement limitations imposed by US healthcare payers. Several opportunities exist to address these challenges and improve patient access, including the use of appropriate value assessment methods, the development of innovative payment solutions, the removal of inappropriate access barriers imposed by payers and improving education and awareness regarding treatment availability, benefits and risks. Moving forward, we must collectively strive for comprehensive policy and market solutions that are sensitive to the uniqueness of gene therapies to ensure their full potential is realized and these treatments are made available to those in need.accesseducationgene therapypatientspaymentvalue |
| spellingShingle | Tyler D Wagner Lisabeth Buelt Kimberly Westrich Jonathan D Campbell Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States Journal of Comparative Effectiveness Research access education gene therapy patients payment value |
| title | Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States |
| title_full | Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States |
| title_fullStr | Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States |
| title_full_unstemmed | Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States |
| title_short | Improving access to gene therapies: a holistic view of current challenges and future policy solutions in the United States |
| title_sort | improving access to gene therapies a holistic view of current challenges and future policy solutions in the united states |
| topic | access education gene therapy patients payment value |
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