Publications

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.

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. The manuscript describing their findings was published in the December 2022 issue of Journal of Comparative Effectiveness Research and can be found on PHAR’s publications page.

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.