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.
Presentations & Events
Results from a study done in partnership with Akcea Pharmaceuticals and focusing on the patient journey of patients newly diagnosed with hereditary transthyretin (ATTRv) amyloidosis have been presented at multiple virtual conferences this year, including the XVII International Symposium on Amyloidosis, the European Society of Cardiology’s HFA Discoveries platform, and the Heart Failure Society of America’s Annual Scientific Meeting. This is the first real-world study to examine pre-diagnostic characteristics and healthcare utilization in this population of patients. Using commercial claims data, PHAR and Akcea found that patients with ATTRv amyloidosis experience substantial comorbidity, testing, and hospitalization prior to diagnosis. These patients also face considerable cardiovascular burden in the 5 years preceding diagnosis. These posters can be found on the PHAR publications page. A manuscript further detailing this study is currently planned for publication in a peer-reviewed journal later this year.
Two secondary data analysis studies conducted by PHAR, in partnership with Genentech, Inc, were presented at MDS Virtual Congress 2020. The first study investigated healthcare utilization and costs in Medicare beneficiaries with Huntington’s disease at the end-of-life. This study showed most beneficiaries at the end-of-life had late-stage disease and healthcare utilization and costs were greater in the last three months of life compared to the three months prior. The second study investigated the epidemiology of Huntington’s disease among Medicaid beneficiaries. This study showed that the prevalence of Huntington’s disease in the Medicaid population is higher than previously reported in other populations. The posters for these studies can be found on the PHAR publications page.
Many studies conducted by PHAR were presented at Virtual ISPOR 2020 HEOR: Advancing Evidence to Action and in the journal Value in Health. The topics covered a variety of range, including breast cancer, arthritis, COPD, and many more. One of the studies presented at ISPOR was a secondary data analysis done in partnership with Genentech, Inc., which investigated the healthcare utilization and costs by disease stage in Medicare beneficiaries with Huntington’s disease. This study showed that beneficiaries with late-stage disease had significantly higher healthcare utilization and cost burden compared to those with early- or middle-stage disease. The poster on healthcare utilization and costs as well as the abstracts published in Value in Health can be found on the PHAR publications page.
PHAR research was well represented at AMCP Nexus 2019, with 7 posters from the secondary data analysis and health economics teams presented. The secondary data analysis study topics covered a wide variety of conditions and used multiple data sources, including using insurance claims to examine asthma-related health service use, treatment patterns in narcolepsy, and the epidemiology of hereditary transthyretin amyloidosis; as well as using hospital data to estimate costs associated with diffuse large b-cell lymphoma. The health economics studies were based on the budget impact of perampanel on pediatric and adult patients with partial onset seizures. The posters and related manuscripts can be accessed on the PHAR website.
Jesse Ortendahl, Director of Health Economics at PHAR, recently spoke at the 2019 annual meeting of the American Society of Preventive Cardiology in San Antonio, TX. At the meeting, Jesse joined Dr. Seth J. Baum, MD, FACC, FACPM, FAHA, FNLA, FASPC, in presenting the symposium “Economics 101- What Every Clinician Needs to Know”. This symposium was designed to provide clinicians with essential information to allow them to better understand, critique, and utilize economic evaluations.
Two studies conducted by PHAR were presented at the 24th Congress of the European Hematological Association in Amsterdam. This research, done in partnership with Celgene Corporation, shed light on the treatment patterns and economic burden associated with relapse in patients with follicular lymphoma. The first study showed that over a quarter of second-line treatment regimens for relapsed follicular lymphoma fail, suggesting a need for alternative treatment options. The second revealed a substantial burden of medical costs in the period following relapse. The two posters on treatment patterns and costs can be accessed on the PHAR publications page.
Three studies conducted by teams at PHAR were presented at the AMCP Managed Care & Specialty Pharmacy Annual Meeting in San Diego. One study done in partnership with AstraZeneca illustrated how value framework tools designed by ASCO, ICER, and NCCN can be used in a real-world setting by physicians, pharmacists, and health services researchers to systematically assess therapy value in oncology. The two studies done in partnership with Otsuka Pharmaceuticals Development & Commercialization, Inc. and H. Lundbeck A/S focused on the impact of different antipsychotics on medical costs in patients with schizophrenia. The posters can be accessed on the PHAR website.
At the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Symposium, two of PHAR’s newest studies were presented. One study done in partnership with Akcea Therapeutics estimated helathcare utilization and cost among patients with hereditary transtheyretin amyloidosis (hATTR) by analyzing insurance claims. Another study, in partnership with Otsuka Pharmaceutical, examined the effectiveness of long-acting injectables in reducing psychiatric hospitalization among patients with bipolar I disorder.
Three studies conducted by the secondary data analysis team at PHAR were presented at the AMCP Managed Care & Specialty Pharmacy Annual Meeting held in Boston, MA. The studies focused on different disease areas and outcomes, with one study on the health care utilization and costs of newly diagnosed AL amyloidosis, another study on adherence to atypical antipsychotics among patients with bipolar disorder, and another on the disease burden of hereditary transthyretin amyloidosis. All studies were well-received at AMCP 2018, and the posters can be viewed on the PHAR website here.