Rare Diseases
Goodwin B, Dellon E, Liu Y, de los Santos B, Korgaonkar S, Meyers J, Schaeffer-Koziol C, Terreri B, Shah E. Health care burden of eosinophilic esophagitis by disease severity: a retrospective cohort study of US health insurance claims data. Poster presented at the Academy of Managed Care Pharmacy (AMCP) Nexus 2025; October 27, 2025. National Harbor, MD. [abstract] J Manag Care Spec Pharm. 2025 Oct; 31(10-d):S119-20.
Shah ED, Goodwin B, Liu Y, de los Santos B, Korgaonkar S, Meyers J, Schaeffer-Koziol CR, Terreri B, Dellon ES. Using health insurance claims data to determine the severity of eosinophilic esophagitis in patients in the USA: a retrospective cohort study. Poster presented at the American College of Gastroenterology (ACG) 2025 Annual Scientific Meeting; October 27, 2025. Phoenix, AZ.
Abstract not available at this time.
Buck P, Sweeney C, Georgieva M, Kunzweiler C, Tossonian H, Boyle K, Chirila C, Crawford R, Wolowacz S, Pesch M. Family spillover impact of congenital CMV int he United States and Canada. Poster presented at the Congenital Cytomegalovirus (CMV) Public Health & Policy Conference; September 7, 2025. Minneapolis, MN.
Navigating EU Joint Clinical Assessments and Joint Scientific Consultations From January 2025 Onward
What Do We Need to Know About Joint Clinical Assessments (JCAs)?
Joint clinical assessments (JCAs) centralise the clinical domains of the health technology assessment (HTA) process on a European level. Non-clinical HTA domains, which mainly involve economic evaluation, remain with the national HTA bodies of the 27 member states. The national decision-makers also retain the final appraisal of a medicine and its recommendation for reimbursement for subsequent pricing negotiations.
EU Joint Clinical Assessment for Orphan Drugs
Methodological Challenges, Solutions, and Early Learnings
Are you prepared to navigate the complexities of Joint Clinical Assessment (JCA) in the EU for your rare disease product? Join our webinar to hear from our panel of experts with extensive experience supporting clients with their EU JCA strategy, training, PICO prediction, and submission development. With years of deep expertise in orphan drugs, our panel will guide you through the essential steps to develop a successful JCA strategy in rare disease.
Pease S, Mangrum R, Schantz K, Slota C, Rubin L, Martin S, Shu C, Scippa K. Content validity and meaningful change for the FACIT-Fatigue scale in warm autoimmune hemolytic anemia: results from qualitative interview studies with patients. J Patient Rep Outcomes. 2025 Jul 29;9(1):97. doi: 10.1186/s41687-025-00930-0
Delphi Panel to Inform a Health Economic Model for Rare Debilitating Disease
Supporting the value of a disease-modifying therapy in multiple sclerosis
Percy AK, Ryther R, Marsh ED, Neul JL, Benke TA, Berry-Kravis EM, Feyma T, Lieberman DN, Ananth AL, Fu C, Buhrfiend C, Barrett A, Doshi D, Darwish M, An D, Bishop KM, Youakim JM. Results from the phase 2/3 DAFFODIL study of trofinetide in girls aged 2-4 years with Rett syndrome. Med. 2025 Jun 13;6(6):100608. doi: 10.1016/j.medj.2025.100608
Abbatemarco J, Yu J, Kleman M, Myers K, Poulos C, Conway D. Patient preferences for treatment features in Neuromyelitis Optica Spectrum Disorder (NMOSD): results from a discrete choice experiment (DCE). Poster presented at the American Academy of Neurology 2025 Annual Meeting; April 5, 2025. San Diego, CA. [abstract] Neurology. 2025 Apr 8; 104(7_Suppl 1):7-8.013. doi: 10.1212/WNL.0000000000208724
OBJECTIVE: To quantify preferences and predict treatment choices between ravulizumab and other approved treatments (eculizumab, inebilizumab, satralizumab) among US adults with anti-aquaporin-4 antibody-positive (AQP4-Ab+) NMOSD.