News from Monday, March 2, 2026
Articles
America Needs to Address Our Health Care Crisis Head-On
(2/27, Congressman Andy Harris) comments “...We should improve interoperability of electric health records (EHR), use social media and digital messaging to enhance health maintenance, screening and literacy and smooth communication among all those involved in patient care delivery. EHRs contain enormous amounts of data that should be used to conduct virtual clinical trials and comparative effectiveness research, develop better quality metrics and screening tools, improve diagnostic accuracy and increase the yield of public health research.” Full
Real-World Data Brings New Insights to Natural History of Disease Studies
(3/2, Sujay Jadhav, PharmExec) comments “...Historically, RWD wasn’t accessible in any practical way because the unstructured data required extensive manual processing. But advances in artificial intelligence (AI) and natural language processing (NPL) technology have made it possible to rapidly extract meaningful data from these sources. Today, RWD readily offers a broad view of the patient experience and several advantages over information typically gathered in clinical trials, including the opportunity to gain longitudinal insights as well as a comprehensive view of diverse patient populations.” Full
What Rare Disease Research Teaches Us About the Future of Precision Medicine
(2/28, Anne Fortier, PhD, TheScientist) comments “...By integrating patient experience into the trial design from the outset, rare disease studies can produce therapies that are more relevant to patients’ needs and more likely to succeed in the real world than treatments developed without meaningful patient input. This patient-centered approach is what is needed to address long-standing gaps in medical evidence, particularly for populations that have been failed by the medical system due to underrepresentation in clinical research.” Full
Nordic Countries Press For Wider Adoption Of Joint HTA Pathway
(3/2, Francesca Bruce, Pink Sheet) reports “...Nordic health technology appraisal bodies are calling on companies to apply for joint HTAs through the JNHB, the Joint Nordic HTA Body.” Subscription Required
Press Releases
New Research Explores Gaps in Health Plan Implementation of FDA Label Revisions
(3/2, National Pharmaceutical Council Press Release) “New research published in Health Affairs Scholar from the National Pharmaceutical Council (NPC) and the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center evaluates how quickly commercial health plans updated specialty drug coverage after new label decisions from the Food and Drug Administration (FDA). ‘The Timeliness of Health Plan Drug Coverage Policy Changes to FDA Label Revisions’ found response times were longer for label expansions than contractions, underscoring the need for policy and operational approaches that better align coverage timelines with evolving scientific evidence. This alignment can ensure timely patient access, notes the study authors...” Full
From Principle to Practice: New Checklist Supports Patient Engagement in Rare Disease Value Research
(2/24, The Center for Innovation & Value Research Press Release) “...The RDPE Guidance and Checklist provides clear, actionable guidance for value researchers and their partners on collaborating with patients and caregivers in comparative effectiveness research (CER), value assessment, and economic modeling. The launch marks the completion of Phase 2 of the Center’s rare disease initiative and represents an important step toward more inclusive, patient-centered evidence generation.” Full
NICE Publishes First Quality Standard to Improve Care for People with Rare Diseases
(2/27, NICE Press Release) “Ahead of Rare Disease Day on 28 February, we've published our first ever quality standard on rare diseases, and the first to have been developed in partnership with an external organisation. Setting out clear expectations for improving the diagnosis, management and treatment of rare conditions across England and Wales, the quality standard outlines eight priority areas for improvement.” Full
Journals
Landscaping Evidence on First-Line Therapies for Infantile Epileptic Spasms Syndrome: An Umbrella Review
Saroj Kundan Bharti, et al.
February 19, 2026, Epilepsy Research
Integrating Patient Engagement into Clinical Pharmacology and Pharmacometrics: Bringing the Patient Voice into Data-Driven Science
Jiawei Zhou, et al.
February 26, 2026, Clinical Pharmacology & Therapeutics
Metformin Provides Superior Neuroprotective Potential Compared to Semaglutide in Preventing Diabetes-Associated Alzheimer's Disease via Dual Actions
Andrea Georgiou, et al.
February 27, 2026, Communications Medicine
Comparative Effectiveness of Lipid-Lowering Therapies in Increasing HDL Cholesterol Levels in Patients with Chronic Kidney Disease
Mohammad Abdullah Al Zubair Naim, et al.
February 27, 2026, Nephron