PHAR, in collaboration with Novartis, published findings from a modified Delphi panel summarizing expert clinical consensus on managing infusion-related reactions (IRRs) in patients with sickle cell disease (SCD) who are treated with crizanlizumab. IRR management in patients with SCD is dependent on key patient characteristics including: prior history of IRRs to other monoclonal antibodies or medications, changes to crizanlizumab infusion rate and patient monitoring, pain severity relative to patient’s typical SCD crises, and severe allergic symptoms. The full manuscript detailing the methods and findings are published in Annals of Hematology, or can be found on the PHAR publications page.
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PHAR Conducts Expert Panel on Management of Pharmacodynamic Breakthrough Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria
PHAR, in collaboration with Novartis, published findings from a modified Delphi panel summarizing expert clinical consensus on how to classify and manage pharmacodynamic breakthrough hemolysis (BTH) in patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with complement inhibitors. The paper presents the group’s agreed-upon recommendations on how to manage BTH caused by a complement-amplifying condition, as well as provides a severity classification system for BTH and strategies to mitigate risk of BTH in special circumstances (e.g., vaccination, planned or unplanned surgery, and pregnancy). In general, as severity of BTH increased, experts agreed more interventions to manage the BTH were appropriate. The full manuscript detailing the methods and findings are published in Hematology, or can be found on the PHAR Publications page.
PHAR Presents Multiple Sclerosis and AL Amyloidosis Research at AMCP Nexus 2023
PHAR presented three posters at AMCP Nexus 2023. One poster presented findings on a multicenter, retrospective medical chart review of adult patients with MS and investigates transitions between multiple sclerosis (MS) phenotypes over time. An encore poster reported on the development of a validated algorithm capable of identifying adult patients with nonrelapsing secondary progressive MS in US-based electronic health records or claims databases. A third poster presented the findings of a retrospective claims analysis which compared healthcare resource utilization and costs in patients with and without AL amyloidosis.
All three posters can be found on the PHAR publications page, and accompanying abstracts are published in the October 2023 supplement of Journal of Managed Care + Specialty Pharmacy.
PHAR Conducts Expert Panel to Estimate Healthcare Utilization Among Advanced and Non-advanced AL Amyloidosis Patients
PHAR, in collaboration with Prothena, published findings from a modified Delphi panel summarizing expert clinical consensus on healthcare utilization among advanced and non-advanced AL amyloidosis patients. Experts estimated advanced patients, who would not be good candidates for hematopoietic stem cell transplant, would have high rates of hospitalization (traditionally the most expensive type of healthcare utilization) and other health service use. Experts concluded that the development of new treatment options to facilitate organ recovery and improve function may lead to decreased utilization. The full manuscript detailing the methods and findings are published in ClinicoEconomics and Outcomes Research, or can be found on the PHAR publications page.
PHAR Assesses Treatment Patterns for Telehealth Patients with Psoriasis & Psoriatic Arthritis
PHAR’s Ashis K. Das, Eunice Chang, Caleb Paydar, and Michael S. Broder recently published a study examining adherence and persistence in patients with psoriasis and psoriatic arthritis who newly initiated apremilast at the beginning of the COVID-19 pandemic. Their analysis, done in collaboration with Amgen, Inc., compared treatment patterns in patients who initiated treatment via telehealth visits versus the in-person setting. The study found similar medication adherence and persistence in this population during a 6-month follow-up period, suggesting that patients initiating apremilast can be as effectively managed with telehealth visits as with in-person visits. The full manuscript can be found in Dermatology and Therapy or on the PHAR publications page.
PHAR Estimates Healthcare Utilization and Direct Medical Costs of Huntington’s Disease Among Medicaid Beneficiaries
PHAR’s Eunice Chang, Caleb Paydar, and Sheila R. Reddy recently published a study estimating healthcare utilization and direct medical spending among Medicaid beneficiaries with Huntington’s Disease (HD). Their analysis, done in collaboration with Genentech Inc, found that Medicaid beneficiaries with HD have higher acute healthcare utilization and costs compared to beneficiaries without HD, with utilization and costs increasing with disease progression, indicating that HD beneficiaries at later disease stages have greater burden. The full manuscript can be found in Journal of Medical Economics or on the PHAR publications page.
PHAR Analysis Examines Optimal Treatment Strategy for Leukemia Patients Experiencing Adverse Event
PHAR, in collaboration with Bristol Myers Squibb, examined treatment patterns, healthcare resource utilization, and costs among patients with chronic myeloid leukemia (CML) who experienced a pleural effusion during treatment with dasatinib, a tyrosine kinase inhibitor (TKI). Their claims analysis using data from the Merative® MarketScan® Commercial and Medicare Supplemental databases aimed to understand the optimal strategy for dasatinib treatment after pleural effusion in patients with CML, which is sometimes managed by modifying the dose or changing the therapy. Their study found that dasatinib dose modification after pleural effusion was not always required, and that patients with dose modifications continued therapy for a longer duration with a lower rate of switching to another TKI versus patients who remained on a stable dose. The manuscript detailing their findings is published ahead of print in Acta Haematologica, and can be found on the PHAR publications page.
PHAR Comprehensive Literature Review Examines Clinical Impact of Next-generation Sequencing Testing in Cancer Management
In a collaboration with Exact Sciences, PHAR’s Sarah Gibbs, MPH, Hannah Dalglish, MPH, Cynthia Campos, MPH, and Irina Yermilov, MD, MPH, MS conducted a comprehensive literature review on the clinical impact of using next-generation sequencing (NGS) tests to guide management of advanced cancer patients in the United States. They examined progression-free survival and overall survival in patients who received treatments selected using NGS tests to those who did not receive matched treatments. The manuscript describing their full methodology and findings is published in the American Society of Clinical Oncology’s JCO Precision Oncology, and can be found on the PHAR Publications page.
PHAR Predicts Potential IRA Drawbacks on Post-Approval R&D of Small Molecule Medicines
PHAR, in collaboration with the Pharmaceutical Research and Manufacturers of America (PhRMA), examined the potential negative impact on post-approval R&D of small molecule medicines. Their analysis describes the new indications made for small molecule medications initially approved between 2006-2012 and demonstrates that a large and diverse number of clinically important indications are approved often many years later. Under the 2022 Inflation Reduction Act, which will allow the federal government to renegotiate pricing of certain medications prescription medicines as early as 7 years post approval, incentives to develop these later indications may be significantly reduced. Read the full policy brief here.
PHAR Conducts Expert Panel to Update Clinical Recommendations in Diagnosis and Management of Rare Genetic Disorders
PHAR, in collaboration with Novartis, just published findings from a modified Delphi panel summarizing expert clinical consensus on the medical management of a group of rare genetic disorders. PIK3CA-related spectrum disorders (PROS) are defined by somatic variants in the PIK3CA gene and have diverse clinical manifestations. PHAR used the modified Delphi panel method, a formal, structured, and validated panel process designed to develop expert clinical consensus, to update clinical recommendations on the severity classification, testing, and medical management of patients with PROS. The full manuscript detailing their methods and findings are published in the June 2023 issue of Journal of Vascular Anomalies, or can be found on the PHAR publications page.