Publications

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.

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’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’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, 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.

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, 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, 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.

In response to a recent American Hospital Association report stating that significant increases in expenses related to drugs create financial challenges for hospitals and health system, PHAR, in collaboration with PhRMA, conducted an analysis of 2015-2020 National Health Expenditure data to determine the increase in hospital expenditures and what percentage is attributable to prescription drug spending. PHAR’s analysis found that national hospital expenditures increased by $280 billion, and that only 1.1% ($3.1 billion) of that increase was attributable to spending on drugs. Not only that, but the share of total hospital spending attributable to medicines has actually decreased from 3.7% to 3.1% over the past 5 years. Read the infographic or the full brief for more details.