CER Daily Newsfeed

Video: Understanding the Impact of Health Economics on Cost Effectiveness and Patient Outcomes

(8/14, Don Tracy, PharmExec) “Rob Abbott, CEO, ISPOR, explains how health economics and outcomes research is helping healthcare systems measure the true value of treatments, improve patient outcomes, and address rising drug costs globally.” View Video

Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

(8/15, Rose McNulty, The American Journal of Managed Care) reports “Children with spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec (Zolgensma; Novartis) following initial treatment with nusinersen (Spinraza; Biogen) or risdiplam (Evyrsdi; Genentech) experienced clinically meaningful motor function improvement in a real-world study published in Neuromuscular Disorders.” Full

Duke-Margolis 2025 RWE Policy Series: Addressing Data Quality, Regulatory Standards, and the Role of Technology in Evidence Generation

(8/15, The Evidence Base) reports “...Convened with the Duke-Margolis Real-World Evidence Collaborative, the 2025 public meeting provided a forum to review recent RWE Collaborative activities, strategic RWD/E policy developments, and future applications. Session 1, a fireside chat moderated by Duke-Margolis Director Mark McClellan, aimed to highlight ‘current possibilities – and future visions – for RWD/E’ and hear from experts in policy, regulation, and payment on approaches to advance an efficient and innovative RWE landscape.” Full

Draft MAHA Report Emphasizes Real World Data

(8/15, Ruth Reader, Politico) reports “...The draft report is not finalized and therefore subject to change. However, if this new platform remains part of the agenda, it would pull in claims information from electronic health records and wearables, among more traditional kinds of data, according to the document. Researchers would be tasked with analyzing the information to understand the causes of chronic disease along with potential treatments.” Full

NICE Recommends Benralizumab for Rare Form of Vasculitis

(8/15, Ella Day, Pharmafile) reports “The National Institute for Health and Care Excellence (NICE) has recommended AstraZeneca’s benralizumab (Fasenra) as an add-on treatment for adult patients with relapsing or refractory eosinophilic granulomatosis with polyangiitis (EGPA). This marks the first NHS-funded biologic treatment option for eligible patients in England, with interim funding allowing immediate access.” Full

BioMarin Fights Back After NICE Blocks English Funding For £500K Batten Disease Drug

(8/15, Neena Brizmohun, Pink Sheet) reports “...The health technology assessment institute, NICE, said the price BioMarin was asking for Brineura was too high, even though its appraisal committee applied a cost-effectiveness threshold ten times higher than usual.” Subscription Required

Dutch HTA Calls For “Socially Acceptable” Price For Gene Editing Therapy Casgevy

(8/15, Francesca Bruce, Pink Sheet) reports “...The Dutch health technology assessment body says Vertex and Biogen should slash the prices of their rare disease treatments, Casgevy and Skyclarys, respectively, if they are to be reimbursed in the Netherlands.” Subscription Required