Two studies conducted by PHAR were presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS). The first study, done in partnership with Sanofi US, examined the effectiveness of teriflunomide in patients with relapsing multiple sclerosis (RMS) who switched from other DMTs following disease progression. The authors of this retrospective chart review study observed that RMS patients who switched to teriflunomide after progression may have experienced reductions in EDSS scores and stable or reduced relapse rates. In the second study, PHAR collaborated with Genentech to develop a claims-based algorithm to identify patients with neuromyelitis optica spectrum disorder (NMOSD) and distinguish them from patients with multiple sclerosis and other central nervous system inflammatory disorders, allowing researchers and clinicians to better estimate NMOSD burden. The posters can be viewed on the PHAR website publications page and the abstracts were published in the October supplement issue of Multiple Sclerosis Journal.
News
PHAR President, Health Economics Director Discuss SR Automation Best Practices
PHAR’s President Michael S. Broder, MD, MSHS, and Director of Health Economics, Jesse Ortendahl, appeared at a roundtable hosted by Evidence Partners and HealthEconomics.com to address questions around how to best automate systematic reviews. With the increasing number of scientific journals over the last few years, using the most effective and up-to-date methods for conducting literature reviews has become a must. Michael and Jesse shared best practices learned conducting systematic reviews over two decades. The pair will also be presenting at EP’s upcoming Evidence Matters ’21.
PHAR Organizes Expert Panel to Develop Consensus for the Management of Neurotrophic Keratopathy
PHAR, with support from Dompé US, Inc., published their synthesis of clinical recommendations for the management of neurotrophic keratopathy (NK), an uncommon, underdiagnosed degenerative corneal disease, in BMC Ophthalmology. The investigators gathered an 11-member expert panel who reviewed published evidence and independently rated 735 patient scenarios using a validated methodology (a RAND/UCLA modified Delphi panel) to develop consensus on when to screen for and how best to diagnose and treat NK. Diagnosis may be delayed in NK because patients experience few symptoms, however, the best opportunity to reverse ocular surface damage and prevent progression is early in the disease course.
PHAR President and Health Economics Director Speak Out About Racial Inequity in CEAs
Michael S. Broder, MD, MSHS, President at PHAR, and Jesse Ortendahl, Director of Health Economics at PHAR, discuss the inherent racial equity problem in cost-effectiveness analysis and how the history of racial injustice in health outcomes can be unintentionally perpetuated by researchers. Read their post at HealthEconomics.com here.
PHAR Uses Delphi Panel to Develop Order Set for Acute Pain Management for Patients with Sickle Cell Admitted to the ED
PHAR’s David Beenhouwer, MD, Michael S. Broder, MD, MSHS, Sarah N. Gibbs, MPH, and Irina Yermilov, MD, MPH, in collaboration with Novartis Pharmaceuticals, organized a multidisciplinary panel of practicing emergency department (ED) clinicians and used the modified Delphi panel process to develop an order set for managing acute pain in sickle cell disease (SCD) patients in the ED. Painful, incapacitating vaso-occlusive episodes (VOEs) are a hallmark of SCD. Recommended treatment within 30 minutes of triage is rarely achieved in clinical practice; ED order sets may facilitate better VOE management. The description of the process and resulting order set was published in the Journal of the American College of Emergency Physicians Open and can be read here. The resulting order set is presently undergoing review, adoption, and implementation in several NYC EDs.
PHAR Study Shows Medicare Beneficiaries with ATTR Have Considerable Cardiovascular Disease Burden Prior to Diagnosis
PHAR’s Sheila Reiss Reddy, PhD, RPh, Eunice Chang, PhD, and Marian Tarbox, MPP worked with collaborators at Akcea Therapeutics, Inc., to conduct a retrospective analysis using Medicare claims data to identify cardiovascular conditions within 3 years prior to a transthyretin amyloidosis (ATTR) diagnosis. Findings show Medicare beneficiaries with ATTR have considerable cardiovascular conditions prior to diagnosis. Awareness of characteristic cardiovascular symptoms could lead to earlier diagnosis and prompt intervention. Results were presented as a poster at the virtual Heart Failure Congress 2021, and can be viewed here.
PHAR Presents Study on How Nurses Are Well Positioned to Uncover ATTRv in Heart Failure Patients
PHAR’s Sheila Reiss Reddy, PhD, RPh, Eunice Chang, PhD, and Marian Tarbox, MPP, in collaboration with investigators at Akcea Therapeutics, Inc., presented at the American Association of Heart Failure Nurses 17th Annual Meeting. The study found that patients diagnosed with hereditary transthyretin amyloidosis (ATTRv [variant]) have considerable cardiovascular disease burden in the 5 years preceding diagnosis. Heart failure nurses’ recognition of ATTRv cardiovascular manifestations may increase clinical suspicion, leading to earlier diagnosis. The poster can be found here, and the abstract published in Heart & Lung‘s July/August 2021 issue.
PHAR Delphi Panel Estimates Dwell Time in 21 Cancer Types
PHAR’s Michael S. Broder, MD, MSHS, Sarah N. Gibbs, MPH, and Irina Yermilov, MD, MPH, in collaboration with GRAIL, estimated the progression rate of 20 solid cancers and lymphoma during the period before the cancers are diagnosed. As this period can’t be directly observed, the authors organized a RAND/UCLA modified Delphi panel to have experts provide estimates of progression rates. These estimates may be useful when determining the appropriate interval for cancer screening tests. The abstract was published in the May 2021 supplement issue of the Journal of Clinical Oncology and can be read here.
PHAR Medical Record Review Compares Prognosis of RA Patients Treated with Abatacept vs TNFis
PHAR’s Michael S. Broder, MD, MSHS, Eunice Chang, PhD, Sarah N. Gibbs, MPH, and Irina Yermilov, MD, MPH, in collaboration with Bristol-Myers Squibb, conducted a study on real-world treatment patterns of patients with rheumatoid arthritis (RA). The investigators performed a multi-center retrospective medical record review, assessing treatment persistence in adult patients with RA complicated by poor prognostic factors. They found that patients on abatacept, including the subgroup of patients with poor prognostic factors, stayed on treatment longer and had a lower risk of discontinuation due to disease progression than patients on tumor necrosis factor-inhibitors. Their findings were published in the Journal of Health Economics and Outcomes Research and can be read here.
National Burden of Achondroplasia Research Presented at Virtual ISPOR
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.