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.
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’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.
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.
At the 31st Annual Congress of the European Academy of Dermatology and Venereology, PHAR investigators Ashis Das, MBBS, MPH, PhD, Eunice Chang, PhD, Caleb Paydar, and Michael Broder, MD, MSHS, in collaboration with Amgen Inc., presented their retrospective analysis of treatment patterns for patients with psoriasis and psoriatic arthritis initiating apremilast after a telehealth visit versus an in-person visit while access to care was limited during the COVID-19 pandemic. Their findings suggest apremilast initiation can be effectively managed with telehealth visits. The poster can be viewed on the PHAR publications page.
A study done by PHAR in partnership with Prothena Biosciences Inc., was presented at the International Society Of Amyloidosis’s XVIII. International Symposium on Amyloidosis in Heidelberg, Germany. PHAR investigators Eunice Chang, PhD, Katalin Bognar, PhD, Marian Tarbox, MPP, and Michael S. Broder, MD, MSHS presented their retrospective analysis of nationally representative hospital discharge data examining the rate of diagnostic hospitalizations of light-chain (AL) amyloidosis and associated high costs and increased utilization. They found that healthcare utilization and costs were particularly high for patients who had not been diagnosed prior to being admitted for an acute event, and that healthcare costs are about three times higher among diagnostic hospitalizations for AL amyloidosis compared to the average US hospitalization. Their poster can be found on the PHAR publications page.
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 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.
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’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.