At ENDO2022, Endocrine Society’s annual meeting, PHAR’s Ashis Kumar Das, MBBS, MPH, PhD, Cynthia Campos, MPH, and Michael S. Broder, MD, MSHS presented two posters on the ILLUSTRATE study, conducted in collaboration with Recordati Rare Diseases, Inc. ILLUSTRATE is a real-world retrospective chart review of osilodrostat usage in U.S. patients with endogenous Cushing’s syndrome. The first poster describes osilodrostat dosing, titration, and persistence for patients with endogenous Cushing’s disease. The second describes osilodrostat use in a small sample of patients with non-pituitary Cushing’s syndrome. Until now, no information was available describing use of osilodrostat in non-pituitary CS as such use is not yet FDA-approved. The posters can be viewed here, and the abstracts will be published in a supplemental issue of the Journal of the Endocrine Society.
Presentations & Events
PHAR investigators, in partnership with Sunovion Pharmaceuticals, developed clinical consensus on the use of on-demand treatments for Parkinson’s disease (PD) by convening experts to participate in a RAND/UCLA modified Delphi panel. In most evaluated scenarios, panelists agreed on-demand treatment is appropriate for PD patients with OFF episodes, especially for patients experiencing a significant functional impact. A summary of the process and consensus statements were presented as a poster at the 4th Pan American Parkinson’s Disease and Movement Disorders Congress today and can be viewed here. The accompanying abstract was also published in the May supplement issue of Movement Disorders Clinical Practice.
PHAR’s Michael S. Broder, MD, MSHS, Sarah N. Gibbs, MPH, and Irina Yermilov, MD, MPH published an article describing how they adapted the RAND/UCLA modified Delphi panel method, which typically relies on an in-person meeting, during the COVID-19 pandemic. This virtual method can be a cost-effective and efficient alternative for researchers and clinicians. Their work was previously presented as a poster at ISPOR’s annual meeting in May 2022, which can be viewed here. The article can be read on the PHAR publications page and in the Journal of Healthcare Leadership.
PHAR’s research was well represented at ISPOR’s annual meeting with six posters total. PHAR presented a self-funded study describing an adaptation of the RAND/UCLA Delphi panel method for virtual use. PHAR’s outcomes research team also presented a systematic literature review assessing the value of next-generation sequencing in advanced cancer, and a validated claims algorithm to identify patients with severe hemophilia A. PHAR’s real world evidence team presented three secondary data analysis studies, which summarized the burden associated with misdiagnosing neuromyelitis optica spectrum disorders, measured healthcare utilization and costs of diagnosing AL amyloidosis in the inpatient setting, and described treatment patterns in newly treated patients with Parkinson’s disease. All of these posters can be found on the PHAR publications page, and accompanying abstracts will be published in the ISPOR supplement of Value in Health.
PHAR’s President, Michael S. Broder, MD, MSHS, mediated a webinar discussion today with panelists Eric Benchimol, MD, The Hospital for Sick Children in Toronto, Karina Raimundo, MS, HEOR Director at Genentech, and Anil Vachani, MD, MS, of Penn Medicine on best practices surrounding ICD code validation in secondary data research using Medicare or commercial claims. Recently, Dr. Broder wrote on the importance of algorithm validation in claims analyses, which was published on HealthEconomics.com. The webinar attracted more than 80 professionals and academics in pharmacy, medical statistics, healthcare, and health economics insights. The recording of the webinar is available on-demand on InsideScientific.com, and the Q&A report available for download here.
Two studies conducted by PHAR were presented as posters at AMCP 2022. The first study, in partnership with Sunovion Pharmaceuticals, developed clinical consensus on the use of on-demand treatments for Parkinson’s disease (PD) by convening experts to participate in a RAND/UCLA modified Delphi panel. In most evaluated scenarios, panelists agreed on-demand treatment is appropriate for PD patients with OFF episodes, especially for patients experiencing a significant functional impact. The second study, in collaboration with Genentech, presented the interim results of their claims-based algorithm used to identify patients with neuromyelitis optica spectrum disorder (NMOSD). The algorithm helps distinguish patients with NMOSD from patients with multiple sclerosis and other central nervous system inflammatory disorders, allowing researchers and clinicians to better estimate NMOSD burden. The posters can be viewed on the PHAR publications page and abstracts printed in the March supplement issue of the Journal of Managed Care + Specialty Pharmacy.
PHAR, in collaboration with Genentech, presented the interim results of their claims-based algorithm used to identify patients with neuromyelitis optica spectrum disorder (NMOSD) at ACTRIMS 2022. The algorithm helps distinguish patients with NMOSD from patients with multiple sclerosis and other central nervous system inflammatory disorders, allowing researchers and clinicians to better estimate NMOSD burden. The poster can be viewed on the PHAR publications page and the abstract published in the May 2022 supplement of Multiple Sclerosis Journal Online.
PHAR’s Sheila Reiss Reddy, PhD, RPh, Eunice Chang, PhD, and Marian Tarbox, MPP worked with collaborators at Bristol Myers Squibb to conduct an administrative claims study examining treatment patterns, including duration of dasatinib use after pleural effusion, and healthcare resource utilization and costs among patients with chronic myeloid leukemia treated with dasatinib who experienced a subsequent pleural effusion. The poster was presented at the 63rd Annual Meeting and Exposition of the American Society of Hematology and can be viewed on the PHAR publications page, and the accompanying abstract published in the November supplement issue of Blood.
PHAR’s Sarah Gibbs and Michael Broder, in collaboration with Novartis Pharmaceuticals, organized an expert panel to develop guidelines for PIK3CA-related overgrowth spectrum (PROS) severity classification, testing, and medical management. Results were presented at the CLOVES Syndrome Community International Scientific Meeting for PIK3CA Related Conditions and can be read here.
PHAR, with support from Takeda Pharmaceuticals, produced a Monte Carlo simulation model of projected mean costs for treating patients with moderate to severe ulcerative colitis with either vedolizumab or adalimumab. The investigation found that mean estimated total costs per patient treated with either therapy over 1 year were $14,322 lower for vedolizumab than for adalimumab, which may help to inform formulary decision-making. Jesse Ortendahl, Director of Health Economics at PHAR, presented the findings at the American College of Gastroenterology’s Annual Scientific Meeting & Postgraduate Course. See the poster here, and the abstract published in the October supplement issue of the American Journal of Gastroenterology.