EXTON, PA, Nov. 21, 2024 (GLOBE NEWSWIRE) -- Spherix Global Insights has released its first annual chart audit study in hematology, Patient Chart Dynamix™ : Paroxysmal Nocturnal Hemoglobinuria (PNH) (US), providing comprehensive, real-world insights into PNH care. Conducted in October 2024, the study analyzes 211 PNH patient charts gathered from 88 U.S.-based hematologists. It offers a detailed look at current treatment patterns, uncovers critical gaps in care, and highlights opportunities for emerging therapies, including Novartis’ Fabhalta, AstraZeneca/Alexion’s Voydeya, and Genentech/Roche’s PiaSky.
PNH is a rare and debilitating hematologic disorder affecting an estimated 10,000-15,000 people in the U.S. Patients diagnosed with PNH are challenged by symptoms of anemia, fatigue, and blood clots. Beyond these physical symptoms, many PNH patients also grapple with comorbidities like anxiety and depression, which may profoundly impact their quality of life. Data from the patient audit spotlight changes in care related to these patient experiences and symptoms at their most recent hematologist visit.
The audit also highlights significant challenges in the journey to diagnosis for many PNH patients. Hematologists report that it takes about a year, on average, many for patients to receive a proper referral and diagnosis, with delays often attributed to misdiagnoses or patients' reluctance to seek care. These delays underscore the critical need for improved patient education and support programs to accelerate diagnosis and enable earlier access to treatment—ultimately driving better outcomes for patients.
Soliris (eculizumab) and Ultomiris (ravulizumab), both from Alexion/AstraZeneca, remain the most commonly prescribed therapies for PNH. However, due to the lifelong nature of the disease and efficacy expectations, physicians turn to Empaveli (pegcetacoplan) from Apellis and Fabhalta (iptacopan) from Novartis when challenged with disease progression. While the uptake of Voydeya (danicopan) by Alexion and PiaSky (crovalimab) by Genentech/Roche is more nascent due to their more recent approval, physicians in the study underline specific clinical scenarios where these therapies could play a valuable role, signaling potential for broader future adoption.
Despite widespread agreement among hematologists that complement and factor inhibitors should be the standard of care for moderate-to-severe PNH patients, a notable proportion of patients remain untreated with these therapies. The study reveals that patient reluctance, along with cost-related barriers—particularly those tied to payer restrictions—are the primary reasons for this gap in treatment.
Looking ahead, the anticipated introduction of biosimilar eculizumab in 2025 has the potential to shift the PNH market. The study explores how physicians may respond to this shift, focusing on current Soliris patients, those already receiving other treatments, and treatment-naïve patients. While physicians acknowledge that payer initiatives will influence biosimilar adoption, many express a strong preference for brand loyalty when possible and are prepared to advocate more assertively for specific patients groups.
As new treatment options gain traction in the market and the introduction of biosimilars approaches, understanding these PNH market dynamics is crucial. This timely study provides companies with invaluable, data-driven insights into the evolving PNH treatment landscape, highlighting real-world treatment patterns, unmet patient needs, and the anticipated impact of emerging therapies. Spherix’s research equips companies with the strategic intelligence necessary to navigate the complexities of the PNH market, uncover opportunities for innovation, and address ongoing barriers in patient care.
Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the “why” behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns.
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NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release.
Tucker Hurtado, Hematology Launch Franchise Head Spherix Global Insights 4848794284 tucker.hurtado@spherixglobalinsights.com
