Publications

Alongside PHAR’s first virtual exhibit presence at Virtual ISPOR 2021, PHAR investigators presented their findings outlining the national burden of achondroplasia in adults and children, done in conjunction with BioMarin Pharmaceutical. Their examination of the National Inpatient Sample, the largest all-payer inpatient healthcare database in the US, provided the first national estimate of the cost or burden of hospitalization and surgery in adults and children with achondroplasia. They estimated that the annual cost of hospitalization alone was $40 million. The iPoster can be viewed here, and the abstract was published in the May 2021 supplement issue of Value in Health.

PHAR’s Sheila Reiss Reddy, PhD, RPh, Eunice Chang, PhD, and Marian Tarbox, MPP, in collaboration with investigators at Akcea Therapeutics, Inc., conducted two studies investigating cardiovascular complications associated with transthyretin-mediated (ATTR) amyloidosis. Their first study analyzed a large US insurance claims database to identify patients with ATTR amyloidosis and cardiomyopathy (ATTR-CM) and to estimate the US prevalence and incidence of ATTR-CM. Their second study examined cardiovascular symptom manifestation and healthcare use leading up to an ATTR diagnosis among Medicare beneficiaries in the US. Their findings were presented as posters at American College of Cardiology’s 70th Annual Scientific Session & Expo and can be found on the PHAR publications page, and published as abstracts in the Journal of the American College of Cardiology‘s May 2021 supplement issue.

PHAR presented research on transthyretin-mediated (ATTR) amyloidosis and Huntington’s disease (HD) at the virtual 2021 Annual Meeting of the American Academy of Neurology. PHAR’s Sheila Reiss Reddy, PhD, RPh, Eunice Chang, PhD, and Marian Tarbox, MPP, in partnership with Akcea Therapeutics, Inc., investigated the feasibility of estimating ATTR amyloidosis prevalence and incidence using IBM® MarketScan® Commercial and Medicare databases. They also presented research on the clinical characteristics and resource use patterns among Medicare beneficiaries with ATTR amyloidosis in the years before diagnosis. In addition, PHAR’s Sheila Reiss Reddy, PhD, RPh, and Eunice Chang, PhD, in partnership with Genentech, Inc., presented their findings on healthcare resource use among US Medicare beneficiaries with late-onset HD. The posters for these studies can be found on the PHAR publications page, and the abstracts were published in Neurology’s April 2021 supplement issue. 

PHAR’s Sheila Reiss Reddy, PhD, RPh, and Eunice Chang, PhD, in collaboration with GRAIL, analyzed 2012-2016 data from the linked SEER registry-Medicare claims database calculating and comparing the cancer-related costs of 17 cancers by stage of diagnosis. Their findings show patients diagnosed at later stages of cancer have higher costs of care (up to 7 times as much) than those diagnosed at earlier stages. Earlier cancer diagnosis may lead to more efficient treatment and decreased management cost. A poster detailing the findings was presented at the Annual Conference of the Hematology/Oncology Pharmacy Association and can be viewed here, and the abstract was published in the March 2021 special feature issue of the Journal of Hematology/Oncology Pharmacy.

PHAR’s Sheila Reiss Reddy, PhD, RPh, Eunice Chang, PhD, and Caleb Paydar, BS, in conjunction with Genentech, Inc., presented the results of two studies of Huntington’s disease in Medicaid beneficiaries. Their first study compared healthcare utilization and costs among US Medicaid beneficiaries with Huntington’s disease to those without Huntington’s disease. They also presented a study on the burden of illness among US Medicare beneficiaries with late-onset Huntington’s disease. The abstracts were published in the April 2021 supplement issue of the Journal of Managed Care & Specialty Pharmacy, and the posters can be found on the PHAR publications page. 

Clinical guidance from a PHAR research study on how to taper thrombopoietin receptor agonists (TPO-RAs) in immune thrombocytopenia (ITP) was presented at the 2020 American Society of Hematology Annual Meeting & Exposition. PHAR developed this important guidance with clinical experts using the RAND/UCLA modified Delphi panel process. The consensus statements presented could serve as a guide for clinical care by identifying patients who could safely taper and discontinue TPO-RAs and help to inform the design and development of future clinical trials. The study was also published in the December 2020 issue of Research and Practice in Thrombosis and Haemostasis. The presentation and manuscript can be found on our publications page.