PHAR investigators Cynthia Campos, MPH, Hannah Dalglish, MPH, Sarah Gibbs, MPH, and Irina Yermilov, MD, MPH, MS, in collaboration with Sanofi, conducted a systematic literature review on the global economic (healthcare resource utilization and costs) and humanistic (quality of life) burden associated with pediatric onset multiple sclerosis (POMS). They identified and summarized 11 studies on healthcare resource utilization, cost, or insurance coverage, and 36 studies that reported quality-of-life outcomes in patients with POMS. Results demonstrated that healthcare resource utilization and costs are high in patients with POMS and patients reported reduced quality of life as well as significant fatigue compared to healthy children and adolescents. Their review was published in the Journal of Health Economics and Outcomes Research, and can also be found here.
Publications
PHAR Examines Mortality and Costs Associated With Influenza in Elderly Medicare Beneficiaries Treated vs Untreated With Antivirals
PHAR’s Sheila Reddy, PhD, MSc, RPh, Eunice Chang, PhD, Katalin Bognar, PhD, and Marian Tarbox, MPP, in collaboration with Genentech, Inc., identified antiviral treated and untreated patients ≥66 years old with an influenza diagnosis during flu seasons 2016-2018 in a retrospective study of Medicare Fee-For-Service claims from the 100% Research Identifiable Files. Results showed prompt antiviral treatment may be associated with reduced mortality, healthcare resource utilization, and economic burden in elderly Medicare beneficiaries with seasonal influenza. These findings were presented at AMCP Nexus; the poster can be viewed here, and the abstract was published in the October supplement issue of the Journal of Managed Care & Specialty Pharmacy.
PHAR Examines Real-World Treatment Patterns for Chronic Spontaneous Urticaria
PHAR’s Sheila Reddy, PhD, MSc, RPh, Eunice Chang, PhD, and Marian Tarbox, MPP, in collaboration with Genentech, Inc., described treatment patterns, including alignment with guidelines, omalizumab and corticosteroid use, and specialist visits, for patients with chronic spontaneous urticaria in the real world in a retrospective study utilizing Merative™ MarketScan® commercial and Medicaid databases.
These findings were presented at AMCP Nexus; the poster can be viewed here, and the abstract was published in the October supplement issue of the Journal of Managed Care & Specialty Pharmacy.
PHAR Develops Clinical Guidelines to Manage Alpelisib Adverse Events in Breast Cancer Treatment
PHAR investigators, in collaboration with Novartis Pharmaceuticals, presented practical recommendations on the prevention and management of adverse events from Piqray (alpelisib) in the treatment of advanced breast cancer at the ASCO (American Society of Clinical Oncology) Quality Care Symposium.
Earlier this year, PHAR’s Hannah Dalglish, MPH, Sarah Gibbs, MPH, and Michael Broder, MD, MSHS conducted two modified Delphi panels with 20 experts to gather practical, easy-to-implement guidance on the prevention and management of hyperglycemia and rash related to treatment with alpelisib. The Delphi panel methodology provided a systematic and validated approach to creating clinical consensus among the experts, including oncologists, dermatologists, endocrinologists, and patient advocates. The poster can be found on the PHAR publications page, and the abstract was published in the October supplement issue of the Journal of Clinical Oncology.
In addition to finding consensus, the Delphi approach also identified areas of disagreement. Future research may further refine these areas, in addition to testing whether recommendations affect patient outcomes.
PHAR Creates National Estimate of Achondroplasia Burden
PHAR investigators, in partnership with BioMarin, conducted a retrospective analysis of two large, national databases (NIS and NASS), finding that in a typical year, around 2,000 people are admitted to US hospitals with a diagnosis of achondroplasia at a cost of approximately $40 million. The average achondroplasia hospital admission is almost $8,000 more expensive and lasts two days longer than the national average admission. The manuscript was published in the Journal of Comparative Effectiveness Research; both the manuscript and the abstract from ISPOR 2021 can be found on PHAR’s publications page.
PHAR Publishes Analysis of ATTR Amyloidosis Pre-Diagnosis Patterns
PHAR investigators, in collaboration with Akcea Therapeutics and Ionis Pharmaceuticals, conducted a claims analysis on patients newly diagnosed with transthyretin amyloidosis (ATTR) using Medicare Research Identifiable Files, characterizing selected conditions, symptoms, and healthcare utilization during the three years prior to diagnosis. Their findings help fill a gap in the literature on the patient journey prior to diagnosis of ATTR, which may facilitate earlier diagnosis and treatment. The article was published in the Journal of Comparative Effectiveness Research and can be found on PHAR’s publications page.
PHAR Estimates Impact of FDA’s Accelerated Approval Program
In collaboration with Pharmaceutical Research and Manufacturers of America, PHAR analyzed real world utilization of five drugs that were granted accelerated approval and projected the clinical outcomes for those who had access sooner due to the FDA’s Accelerated Approval Program. We found that earlier access to the five drugs sampled impacted over 9 million patients and led to substantial clinical benefits to these individuals. Read the issue brief here.
PHAR Study Assesses Epidemiology of HD Among Medicare and Medicaid Beneficiaries
PHAR’s Sheila Reiss Reddy, PhD, MSc, RPh, Eunice Chang, PhD, and Caleb Paydar, in collaboration with Genentech Inc., conducted an analysis of Medicare and Medicaid claims data to examine the epidemiology of Huntington’s disease (HD), which was published in the August 2022 issue of Neuroepidemiology. Their study is the first to provide a comprehensive assessment of the prevalence and incidence of HD among Medicare beneficiaries and prevalence of HD among Medicaid beneficiaries, populations about which little was previously known. Their study found higher estimates of prevalence and incidence of HD than previously estimated, and highlighting opportunities for earlier diagnosis.
PHAR Studies Compare OSMs for Psoriasis, Psoriatic Arthritis
PHAR investigators Eunice Chang, PhD, Michael S. Broder, MD, MSHS, Caleb Paydar, and Katalin Bognar, PhD, in collaboration with Amgen, published their claims analyses comparing the risk of biologic initiation between apremilast and methotrexate starters among systemic-naive patients with psoriasis (PsO) and psoriatic arthritis (PsA). 2014–2019 claims data from the IBM® MarketScan® Commercial and Medicare Supplemental databases were analyzed; logistic and Cox regression models were used to estimate the likelihood of biologic initiation, and the risk of biologic initiation, during a one-year follow-up, respectively. In both populations, findings show that first-line apremilast users had lower rate of, and longer time to, biologic initiation over the one year following initiation than first-line methotrexate users. The analysis in a PsA population was published in Open Access Rheumatology: Research and Reviews, while the analysis of PsO patients was published in the Journal of Comparative Effectiveness Research.
PHAR Presents Cushing’s Syndrome Medical Records Review Research at ENDO 2022
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.