Publications

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.

PHAR, in collaboration with Sunovion Pharmaceuticals, used the RAND/UCLA modified Delphi panel method, convening a 12-member international panel, to develop expert consensus on the use of on-demand treatments for OFF episodes in Parkinson’s. Cynthia Campos, MPH, Sarah N. Gibbs, MPH, and Michael S Broder, MD, MSHS developed a 432-item rating form where panelists rated the appropriateness of prescribing on-demand therapies (i.e., inhaled levodopa, apomorphine subcutaneous injections, apomorphine sublingual film) in different clinical scenarios. Panelists agreed that on-demand treatment is appropriate for many patients with OFF episodes. The greater the functional impact of OFF episodes and interference with basic daily activities, the more likely experts agreed that on-demand treatment was appropriate to prescribe. This report can be read on the PHAR publications page or ahead of print in Movement Disorders Clinical Practice.

PHAR investigators, in collaboration with Sanofi, conducted a commercial claims analysis of patients with relapsing-remitting multiple sclerosis (RRMS) who started treatment with teriflunomide using IQVIA PharMetrics^® Plus data. Their study found that high adherence to disease-modifying therapies (DMTs) was associated with fewer relapses and lower healthcare utilization among patients with RRMS. As DMT adherence among RRMS patients is suboptimal, this work may inform decisions on selecting interventions to improve adherence, and therefore, patient outcomes. Their manuscript can be read in ClinicoEconomics and Outcomes Research or on the PHAR Publications page.

PHAR investigators, in partnership with GRAIL, convened a panel of 10 oncologists to develop clinical consensus on which cancers had the greatest likelihood of benefitting from advances in early diagnosis. Investigators used a formal, validated group consensus process that combines systematic literature review evidence and clinical expertise. Panelists were asked to rate current curability and benefit (improvement in curability) of an annual hypothetical multi-cancer screening blood test on 20 solid organ cancers, concluding most solid tumors have a likelihood of benefit from early detection. Read the full manuscript in PLOS One or on the PHAR Publications page.

In collaboration with Prothena, PHAR’s Michael S. Broder, MD, MSHS, Eunice Chang, PhD, and Marian H. Tarbox, MPP published their analysis of costs for hospitalizations among patients with AL amyloidosis using a national database of hospital discharge data. Investigators found higher healthcare utilization and costs associated with patients with an in-hospital death compared with patients discharged alive, likely attributed to greater cardiac and renal involvement. Their research underscores the need for increased disease awareness and early diagnosis, which may lead to earlier treatment and reduced costs. The manuscript was published in the Journal of Comparative Effectiveness Research and can also be found on the PHAR Publications page.

Collaborator Tiffany Quock, PhD, MS gave an interview on the study and its findings with the Evidence Base as part of its Peak Behind the Paper feature, which can be read here.