News from Friday, August 8, 2025
Articles
Few Next-in-Class Cancer Drugs Show Survival Benefit in Head-to-Head Trials
(8/8, Mary Ellen Schneider, Renal & Urology News) reports “Less than one-quarter of next-in-class cancer drugs demonstrated a survival benefit over existing in-class agents in head-to-head trials, according to findings published in JAMA Internal Medicine...‘[These] findings raise important questions about the value of next-in-class oncology drugs,’ the researchers wrote. ‘These agents may offer benefits in specific scenarios, including overcoming resistance variants, such as osimertinib for EGFR T790M variants, or improving safety profiles like alectinib, which compares favorably with crizotinib. However, in cases where head-to-head RCTs were lacking, it is difficult to assess the true therapeutic value of next-in-class drugs.’” Full
New Pirtobrutinib Data Compare Favorably With Ibrutinib in CLL/SLL
(8/7, Jared Kaltwasser, The American Journal of Managed Care) reports “The non-covalent Bruton tyrosine kinase (BTK) inhibitor pirtobrutinib (Jaypirca; Eli Lilly) appears to perform as well as ibrutinib (Imbruvica) in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), according to a new study. The results of the phase 3 BRUIN CLL-314 trial were announced in a press release last week.” Full
Augmenting Brain Power: Real-World Data, Advanced Analytics, and the Future of Clinical Trials
(8/8, Sujay Jadhav, MedCity News) comments “...Life sciences companies use RWD and the evidence derived from it for a wide variety of purposes including retrospective and prospective studies, comparative effectiveness research (CER), health economics and outcomes research (HEOR), and market research and targeting (i.e., commercialization). Meanwhile, the increasing adoption of insights from unstructured RWD in clinical research is supported by FDA guidance and a growing range of use cases.” Full
Germany’s IQWiG Opens Consultation on Methods for Routine Practice Data Collection in Drug Benefit Assessment
(8/8, Katie McCool, The Evidence Base) reports “...The German Federal Joint Committee (G-BA) has tasked IQWiG with developing a scientific concept for the generation and analysis of routine practice data in the context of ‘routine practice data collection.’ This process is used when studies comparing a new drug with the therapeutic standard have not been conducted, particularly for medicines targeting rare diseases or approved under special regulatory pathways.” Full
Press Releases
New Study Reveals Significant Racial and Ethnic Disparities in Prescription Drug Utilization and Spending Across the United States
(8/8, National Pharmaceutical Council Press Release) “Significant racial and ethnic disparities exist in prescription drug utilization and spending in the U.S. Utilization rates vary widely by state and payer, with Black populations experiencing underutilization of medications after adjusting for age and disease prevalence, according to a new study in JAMA Health Forum from the National Pharmaceutical Council in partnership with the University of Washington...‘Addressing the disparities highlighted in our study requires tailored policy interventions that consider the unique challenges faced by different racial and ethnic groups,’ stated Tyler Wagner, PharmD, PhD, study co-author and NPC Director of Research. ‘Finding ways to ensure coverage, address pharmacy deserts, and enhance preventive care are critical steps to helping all individuals, regardless of race or ethnicity, have access to the appropriate medications.’” Full
Study of Surgical Interventions for Children, Adolescents with Kidney Stones Supports Precision Treatment Options
(8/8, Children's Hospital of Philadelphia Press Release) “Researchers at Children's Hospital of Philadelphia (CHOP), along with several academic partners, have reported the primary results of the Pediatric KIDney Stone (PKIDS) trial, the largest comparative effectiveness study of surgical interventions for children and adolescents with kidney stones.” Full
Journals
Oncology “Me-Too” Drugs Compared With Original Drugs in Randomized Clinical Trials
Timothée Olivier, MD; Calvin Smith, BS; Vinay Prasad, MD, MPH; et al
July 14, 2025, JAMA Internal Medicine
Comparative Effectiveness of 5-Fluorouracil Dissolving Microneedle Patch vs. 5-Fluorouracil Intralesional Injection for the Treatment of Keloid Scars: A Randomised, Single-Blinded, Split-Scar Study
Jeerapond Leelawattanachai, et al.
July/August 2025, Wound Repair and Regeneration
Phase III Trial of Pirtobrutinib Versus Idelalisib/Rituximab or Bendamustine/Rituximab in Covalent Bruton Tyrosine Kinase Inhibitor-Pretreated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (BRUIN CLL-321)
Jeff P Sharman, et al.
August 2025, Journal of Clinical Oncology
Ureteroscopy vs Shockwave Lithotripsy to Remove Kidney Stones in Children and Adolescents: A Nonrandomized Clinical Trial
Gregory E. Tasian, MD, MSc, MSCE; David I. Chu, MD, MSCE; Caleb P. Nelson, MD, MPH; et al
August 7, 2025, JAMA Network Open
Prescription Drug Utilization and Spending by Race, Ethnicity, Payer, Health Condition, and US State
Maitreyi Sahu, PhD; Tyler D. Wagner, PharmD, PhD; Azalea Thomson, MPH; et al
August 8, 2025, JAMA Health Forum