This article provides key insights and suggested best practices for managed care to improve patient care in MPS disorders, which may also be relevant to other rare and ultra-rare conditions.
JMCP Outsert - IBD
FINDINGS FROM THE AMCP MARKET INSIGHTS PROGRAM | January, 2023
Summary
Hemophilia, sickle cell disease (SCD), and beta thalassemia (β thal) are inherited blood disorders likely to see multiple significant novel therapies approved in the near future, including the possibility for partial or complete cures with gene therapies. Although these are rare blood disorders, the high cost of treatment is an important consideration for payers and employers in ensuring that potentially curative therapies are affordable and accessible to the patients who need them.
For more disease state research, view the Market Insights Program.
For more disease state research, view the Market Insights Program.
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As part of a multi-year RWE initiative, AMCP Research Institute partnered with IQVIA, payers, pharmaceutical companies, and others to design payer-focused RWE standards that were published in JMCP in September, 2025.
AMCP’s guidance translates CMS’s expectations for patient centered, evidence informed formulary decisions into practical steps payers can act on today without adding unnecessary burden to P&T workflows.
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