CER Daily Newsfeed

Duke-Margolis RISE Together Workshop Highlights Infrastructure, Ethics, and Collaboration Priorities in Rare Disease Data Sharing

(4/14, Katie McCool, The Evidence Base) reports “At the joint Duke-Margolis Institute for Health Policy and FDA Rare Disease Innovation Hub (RDIH) event, RISE Together: Data Sharing Across the Rare Disease Ecosystem, speakers from regulatory agencies, patient organizations, industry, and academia discussed the technical, governance, and ethical challenges shaping rare disease data sharing. Speakers also highlighted the infrastructure and trust frameworks needed to support faster therapy development.” Full

Weight-Based and Fixed-Dose Pembrolizumab Compared in NSCLC

(4/14, Gargi Mukherjee, Medscape) reports “...Weight-based pembrolizumab dosing demonstrated comparable effectiveness and safety to fixed dosing in patients with advanced non-small cell lung cancer (NSCLC). In a real-world cohort of 414 patients, the median overall survival (OS) was 23.26 months vs 18.43 months with weight-based dosing vs fixed dosing, and rates of laboratory-defined immune-related adverse events (irAEs) were similar between groups.” Subscription Required

Expanding Access to Palliative Care for Patients with Advanced Liver Disease

(4/13, Karuna Meda, Thomas Jefferson University) reports “...In the PAL LIVER trial, a large cluster-randomized study conducted across 19 U.S. centers, researchers evaluated whether hepatologists trained in primary palliative care could match the effectiveness of palliative care specialists in delivering quality-of-life benefits to patients with ALD, including those with decompensated cirrhosis and liver cancer...With patient outcomes and engagement at the forefront of this trial, this study was funded by [PCORI].” Full

CMS Officials Point To MA As Model For LEAD Framework

(4/13, Jalen Brown, Inside Health Policy) reports “...During Monday’s LEAD Kick-Off Event at the National Press Club, which was hosted by several Accountable Care Organization groups, agency officials said the new 10-year model is designed to expand participation in accountable care arrangements within traditional Medicare while incorporating features that have driven MA enrollment and engagement. That framing came through clearly in how officials described the model’s core structure, which centers on assigning patients to accountable providers in a way that more closely resembles MA, while also incorporating financial incentives for beneficiaries and stronger provider accountability for patient outcomes.” Subscription Required

Dems Press More Companies For Terms Of MFN Deals, Tariff Exemptions

(4/13, James Jarvis, Inside Health Policy) reports "Congressional Democrats are escalating pressure on drugmakers that have struck 'most favored nation' pricing deals with the Trump administration, sending a new round of letters to 11 additional companies demanding details of their agreements, which still have not been publicly disclosed despite the administration's push for Congress to codify MFN and a tariff exemption for participating companies." Subscription Required