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AMCP Partnership Forum: Preparing for and managing rare diseases

Despite the opportunity for large health gains, there are many challenges associated with rare disease therapies. Among these are striking the appropriate balance between the urgency to respond to patient needs with the uncertainty that is often inherent in rare disease therapy datasets leading to c...

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Detalles Bibliográficos
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Academy of Managed Care Pharmacy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10394175/
https://www.ncbi.nlm.nih.gov/pubmed/33538235
http://dx.doi.org/10.18553/jmcp.2021.20546
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description Despite the opportunity for large health gains, there are many challenges associated with rare disease therapies. Among these are striking the appropriate balance between the urgency to respond to patient needs with the uncertainty that is often inherent in rare disease therapy datasets leading to concerns with developing and interpreting clinical data; uncertainty around financial impact, value determination, and affordability; and variation in approach to coverage and potential effects on access. To discuss these challenges and opportunities to address them, AMCP held a virtual multidisciplinary stakeholder forum on September 8-10, 2020. Forum participants represented diverse sectors of the health care industry, including integrated delivery systems, health plans, pharmacy benefit managers, employer groups, biopharmaceutical companies, patient advocacy organizations, health policy researchers, and consulting firms; they evaluated strategies to plan for and manage rare disease therapies and recommended best practices and next steps.
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spelling pubmed-103941752023-08-03 AMCP Partnership Forum: Preparing for and managing rare diseases J Manag Care Spec Pharm Proceedings Despite the opportunity for large health gains, there are many challenges associated with rare disease therapies. Among these are striking the appropriate balance between the urgency to respond to patient needs with the uncertainty that is often inherent in rare disease therapy datasets leading to concerns with developing and interpreting clinical data; uncertainty around financial impact, value determination, and affordability; and variation in approach to coverage and potential effects on access. To discuss these challenges and opportunities to address them, AMCP held a virtual multidisciplinary stakeholder forum on September 8-10, 2020. Forum participants represented diverse sectors of the health care industry, including integrated delivery systems, health plans, pharmacy benefit managers, employer groups, biopharmaceutical companies, patient advocacy organizations, health policy researchers, and consulting firms; they evaluated strategies to plan for and manage rare disease therapies and recommended best practices and next steps. Academy of Managed Care Pharmacy 2021-03 /pmc/articles/PMC10394175/ /pubmed/33538235 http://dx.doi.org/10.18553/jmcp.2021.20546 Text en Copyright © 2021, Academy of Managed Care Pharmacy. All rights reserved. https://creativecommons.org/licenses/by/4.0/This article is licensed under a Creative Commons Attribution 4.0 International License, which permits unrestricted use and redistribution provided that the original author and source are credited.
spellingShingle Proceedings
AMCP Partnership Forum: Preparing for and managing rare diseases
title AMCP Partnership Forum: Preparing for and managing rare diseases
title_full AMCP Partnership Forum: Preparing for and managing rare diseases
title_fullStr AMCP Partnership Forum: Preparing for and managing rare diseases
title_full_unstemmed AMCP Partnership Forum: Preparing for and managing rare diseases
title_short AMCP Partnership Forum: Preparing for and managing rare diseases
title_sort amcp partnership forum: preparing for and managing rare diseases
topic Proceedings
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10394175/
https://www.ncbi.nlm.nih.gov/pubmed/33538235
http://dx.doi.org/10.18553/jmcp.2021.20546