PHAR investigators, in partnership with BioMarin, conducted a retrospective analysis of two large, national databases (NIS and NASS), finding that in a typical year, around 2,000 people are admitted to US hospitals with a diagnosis of achondroplasia at a cost of approximately $40 million. The average achondroplasia hospital admission is almost $8,000 more expensive and lasts two days longer than the national average admission. The manuscript was published online ahead of print in the Journal of Comparative Effectiveness Research; both the manuscript and the abstract from ISPOR 2021 can be found on PHAR’s publications page.
Publications
PHAR Publishes Analysis of ATTR Amyloidosis Pre-Diagnosis Patterns
PHAR investigators, in collaboration with Akcea Therapeutics and Ionis Pharmaceuticals, conducted a claims analysis on patients newly diagnosed with transthyretin amyloidosis (ATTR) using Medicare Research Identifiable Files, characterizing selected conditions, symptoms, and healthcare utilization during the three years prior to diagnosis. Their findings help fill a gap in the literature on the patient journey prior to diagnosis of ATTR, which may facilitate earlier diagnosis and treatment. The article was published online ahead of print in the Journal of Comparative Effectiveness Research and can be found on PHAR’s publications page.
PHAR Estimates Impact of FDA’s Accelerated Approval Program
In collaboration with Pharmaceutical Research and Manufacturers of America, PHAR analyzed real world utilization of five drugs that were granted accelerated approval and projected the clinical outcomes for those who had access sooner due to the FDA’s Accelerated Approval Program. We found that earlier access to the five drugs sampled impacted over 9 million patients and led to substantial clinical benefits to these individuals. Read the issue brief here.
PHAR Study Assesses Epidemiology of HD Among Medicare and Medicaid Beneficiaries
PHAR’s Sheila Reiss Reddy, PhD, MSc, RPh, Eunice Chang, PhD, and Caleb Paydar, in collaboration with Genentech Inc., conducted an analysis of Medicare and Medicaid claims data to examine the epidemiology of Huntington’s disease (HD), which was published in the August 2022 issue of Neuroepidemiology. Their study is the first to provide a comprehensive assessment of the prevalence and incidence of HD among Medicare beneficiaries and prevalence of HD among Medicaid beneficiaries, populations about which little was previously known. Their study found higher estimates of prevalence and incidence of HD than previously estimated, and highlighting opportunities for earlier diagnosis.
PHAR Studies Compare OSMs for Psoriasis, Psoriatic Arthritis
PHAR investigators Eunice Chang, PhD, Michael S. Broder, MD, MSHS, Caleb Paydar, and Katalin Bognar, PhD, in collaboration with Amgen, published their claims analyses comparing the risk of biologic initiation between apremilast and methotrexate starters among systemic-naive patients with psoriasis (PsO) and psoriatic arthritis (PsA). 2014–2019 claims data from the IBM® MarketScan® Commercial and Medicare Supplemental databases were analyzed; logistic and Cox regression models were used to estimate the likelihood of biologic initiation, and the risk of biologic initiation, during a one-year follow-up, respectively. In both populations, findings show that first-line apremilast users had lower rate of, and longer time to, biologic initiation over the one year following initiation than first-line methotrexate users. The analysis in a PsA population was published in Open Access Rheumatology: Research and Reviews, while the analysis of PsO patients was published in the Journal of Comparative Effectiveness Research.
PHAR Presents Cushing’s Syndrome Medical Records Review Research at ENDO 2022
At ENDO2022, Endocrine Society’s annual meeting, PHAR’s Ashis Kumar Das, MBBS, MPH, PhD, Cynthia Campos, MPH, and Michael S. Broder, MD, MSHS presented two posters on the ILLUSTRATE study, conducted in collaboration with Recordati Rare Diseases, Inc. ILLUSTRATE is a real-world retrospective chart review of osilodrostat usage in U.S. patients with endogenous Cushing’s syndrome. The first poster describes osilodrostat dosing, titration, and persistence for patients with endogenous Cushing’s disease. The second describes osilodrostat use in a small sample of patients with non-pituitary Cushing’s syndrome. Until now, no information was available describing use of osilodrostat in non-pituitary CS as such use is not yet FDA-approved. The posters can be viewed here, and the abstracts will be published in a supplemental issue of the Journal of the Endocrine Society.
PHAR Develops Clinical Guidelines on the Use of On-Demand Treatments for Parkinson’s OFF Episodes
PHAR investigators, in partnership with Sunovion Pharmaceuticals, developed clinical consensus on the use of on-demand treatments for Parkinson’s disease (PD) by convening experts to participate in a RAND/UCLA modified Delphi panel. In most evaluated scenarios, panelists agreed on-demand treatment is appropriate for PD patients with OFF episodes, especially for patients experiencing a significant functional impact. A summary of the process and consensus statements were presented as a poster at the 4th Pan American Parkinson’s Disease and Movement Disorders Congress today and can be viewed here. The accompanying abstract was also published in the May supplement issue of Movement Disorders Clinical Practice.
PHAR Adapts Delphi Panel Method for Virtual Use
PHAR’s Michael S. Broder, MD, MSHS, Sarah N. Gibbs, MPH, and Irina Yermilov, MD, MPH published an article describing how they adapted the RAND/UCLA modified Delphi panel method, which typically relies on an in-person meeting, during the COVID-19 pandemic. This virtual method can be a cost-effective and efficient alternative for researchers and clinicians. Their work was previously presented as a poster at ISPOR’s annual meeting in May 2022, which can be viewed here. The article can be read on the PHAR publications page and in the Journal of Healthcare Leadership.
PHAR Presents Various Studies at ISPOR Annual Meeting
PHAR’s research was well represented at ISPOR’s annual meeting with six posters total. PHAR presented a self-funded study describing an adaptation of the RAND/UCLA Delphi panel method for virtual use. PHAR’s outcomes research team also presented a systematic literature review assessing the value of next-generation sequencing in advanced cancer, and a validated claims algorithm to identify patients with severe hemophilia A. PHAR’s real world evidence team presented three secondary data analysis studies, which summarized the burden associated with misdiagnosing neuromyelitis optica spectrum disorders, measured healthcare utilization and costs of diagnosing AL amyloidosis in the inpatient setting, and described treatment patterns in newly treated patients with Parkinson’s disease. All of these posters can be found on the PHAR publications page, and accompanying abstracts will be published in the ISPOR supplement of Value in Health.
PHAR Discusses Discrimination in CEAs
In collaboration with Pharmaceutical Research and Manufacturers of America, PHAR developed an issue brief discussing how cost-effectiveness analyses can perpetuate health inequities. Cost-effectiveness analyses are commonly used to compare interventions, but fail to account for a diverse patient population. Read the issue brief here.