Burden of illness and unmet needs in patients with erythropoietic protoporphyria and X-linked protoporphyria: A large US nationwide claims analysis
Patients with EPP/XLP experienced substantially higher HRU and costs compared with matched comparators, yet few received EPP/XLP-specific treatment. This highlights the need for new effective and accessible treatments that could improve patient outcomes and alleviate the broader disease burden.
Authors
- Aruna Muthukumar (ORCID: https://orcid.org/0000-0001-7481-8313)
- Wenxu Liu (ORCID: https://orcid.org/0000-0002-1647-9162)
- Melanie Chin (ORCID: https://orcid.org/0000-0002-2771-0518)
- Maral DerSarkissian (ORCID: https://orcid.org/0000-0002-5528-6531)
- Hela Romdhani (ORCID: https://orcid.org/0000-0001-5219-2612)
- Priyanka Bobbili (ORCID: https://orcid.org/0000-0002-1343-8314)
- Chelsea Norregaard
- Ly Trinh
Institutions
- Menlo School (US)
- United Electric Controls (United States) (US)
- Analysis Group (United States) (US)
Publication Details
- Journal
- Journal of Managed Care & Specialty Pharmacy
- Published
- 2025-10-21
- DOI
- https://doi.org/10.18553/jmcp.2025.25132
- Primary Topic
- Porphyrin Metabolism and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00