PHAR’s Sarah N. Gibbs, MPH, Irina Yermilov, MD, MPH, MS, and Michael Broder, MD, MSHS, conducted a Delphi panel to estimate healthcare utilization in patients with advanced AL amyloidosis at different points in the disease course. Their abstract was published in the November 2022 supplement issue of Blood, the official journal of the American Society of Hematology, and can also be found on the PHAR publications page.
In collaboration with the Pharmaceutical Research and Manufacturers of America (PhRMA), PHAR conducted a real-world analysis in order to calculate the benefits of R&D past initial FDA approval in a subset of previously approved drugs. The recently passed Inflation Reduction Act would allow the government to renegotiate prices for prescription medicines as early as 7 years post approval. PHAR and PhRMA’s study examines how this may impact R&D investment decisions for medicines by investigating the number and timing of additional FDA approvals for new uses or indications for new medicines initially approved by the FDA between 2010 and 2012, and calculating the share of post-approval indications that occurred 7 or more years after each drug’s initial FDA approval. The findings of their analysis speak to the potential impact of recently enacted government policies, which disincentivizes investment and further development of critical post-approval innovations. Read the policy brief here.
PHAR investigators, in collaboration with Prothena Biosciences Inc., conducted a retrospective analysis of nationally representative hospital discharge data estimating the frequency and costs associated with hospitalizations during which systemic amyloid light chain (AL) amyloidosis was diagnosed. This is the first analysis to use an AL amyloidosis specific ICD-10 diagnosis code in a nationally representative database to provide data on hospital admissions for this disease.
Eunice Chang, PhD, Katalin Bognar, PhD, Marian Tarbox, MPP, and Michael S. Broder, MD, MSHS found that diagnostic admissions are more likely to be urgent/emergent, require longer stays, and have higher costs compared with hospitalizations in known AL amyloidosis patients. Their manuscript describing their findings was published online ahead of print in the Journal of Comparative Effectiveness Research and can be found on PHAR’s publications page.
PHAR’s Irina Yermilov, MD, MPH, MS, Cynthia Campos, MPH, and Michael Broder, MD, MSHS, in collaboration with Novartis Pharmaceuticals, convened a RAND/UCLA modified Delphi panel of 10 clinicians to develop consensus on the management of infusion-related reactions presenting with pain in patients with sickle-cell disease receiving crizanlizumab. Their research and findings were orally presented at the 17th Annual Scientific Conference on Sickle Cell and Thalassaemia (ASCAT). The presentation can be viewed here, and the abstract will be published in a supplement issue of HemaSphere.
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.