CER Daily Newsfeed

Real-World Data Salvage Biohaven Drug, Paving Way for FDA Filing in Neuro Disease With No Approved Meds

(9/23, Frank Vinluan, MedCity News) reports “...Biohaven did not give up on the drug, and apparently, neither did the FDA. The agency is welcoming more real-world data to generate the evidence for its regulatory decisions. Based on a clinical trial design and endpoints that were blessed by the regulator, this approach for the Biohaven drug, troriluzole, led to more data, new analyses, and positive results in the treatment of the neurological disease spinocerebellar ataxia (SCA).” Full

From Lab to Life: Harnessing Real-World Evidence for Informed Clinical Insights and Improved Patient Outcomes

(9/24, Stephen Deacon, PMLiVE) comments “...Real-world data also plays a crucial role in generating new clinical evidence, especially in discovering new therapeutic uses and benefits of the medicine in people with different medical conditions than the ones the medicine was initially tested for. With the safety profile of the medicine well established over many years – revamped medicines with new indications can get to the market much faster than the 12-year minimum period for a truly new drug.” Full

Wang and Rosenthal Awarded $1.05M for Patient Risk Prediction Model Research

(9/24, Angelina Livigni, Stony Brook Matters) reports “The Patient-Centered Outcomes Research Institute has announced a $1.05 million award to Fusheng Wang, professor in the Departments of Biomedical Informaticsand Computer Science, and his team, including Richard Rosenthal, MD, professor and addiction psychiatrist from the Department of Psychiatry, MPI of the project. Their research is focused on machine learning models to predict patient outcomes.” Full

How To Evaluate The Innovation Center’s Impact On Delivery System Transformation

(9/24, Steven Sheingold, Kaushik Ghosh and Lok Wong Samson, Health Affairs Forefront) comments “...To date, the CMS Innovation Center has mainly been assessed in terms of reducing Medicare expenditures. Recently, the center outlined an initiative, the Quality Pathway, to focus models on improving patient-centered quality goals and designing evaluations that can expand models on the basis of enhanced quality. These budget estimates and patient-centered quality metrics represent one important aspect of an evaluation, but we believe a more useful assessment is one that emphasizes the center’s broad potential to transform health care delivery across the nation writ large. With this article, we outline a comprehensive framework for conducting exactly that kind of assessment.” Full

EU HTA Regulation: Some Member States Could Be ‘Deselected’ From Joint Assessments

(9/24, Eliza Slawther, Pink Sheet) reports “...The EU HTA Regulation will see clinical comparisons of medical products conducted at an EU level. However, the needs of some smaller nations may not be represented, experts from Cencora explain in this second of two articles on the new EU joint clinical assessments.” Subscription Required

UK: NICE and NIHR: An Enduring Partnership

(9/24, Shane Collins, NICE Blog) comments “For nearly 20 years, NICE has worked with the National Institute for Health and Care Research (NIHR) to improve the evidence base for new interventions in health and social care...A relatively recent outcome of this partnership has been a rolling funding call for studies addressing NICE research recommendations. The aim of this is to complement their programme of commissioned calls. The funding call gives researchers flexibility to apply for NIHR funding throughout the year - as and when they identify priorities arising from NICE guidance.” Full