News from Wednesday, December 11, 2024
Articles
Real-World Evidence Improving, Not Replacing, Traditional Studies
(12/11, Bridget Silverman, Pink Sheet) reports “...As RWE advances, a multiplicity of roles are emerging, but case studies show how it is more effective filling evidence gaps during drug development and improving surveillance than creating a randomized controlled trial alternative.” Subscription Required
Elacestrant Shows Real-World Effectiveness in Metastatic Breast Cancers
(12/11, Cameron Santoro, The American Journal of Managed Care) reports “Patients with ER-positive/HER2-negative metastatic breast cancer who received elacestrant experienced a significant reduction in disease progression or death compared with standard-of-care endocrine therapy.” Full
New Research Shows Better Ways To Help Young People With OCD
(12/10, PCORI Blog) comments “Findings from a Patient-Centered Outcomes Research Institute-funded systematic review conducted by the Agency for Health Care Research and Quality have provided important insights into diagnosing and treating obsessive compulsive disorder in children and teens.” Full
Press Releases
NPC Announces Takeda’s Jen Norton as Board Chair for 2025
(12/11, National Pharmaceutical Council Press Release) “...‘Jen’s deep expertise on the biopharmaceutical industry landscape has helped countless leaders better understand how this industry works and what levers will help patients access medicines,’ said John M. O’Brien, PharmD, MPH, NPC President and Chief Executive Officer. ‘She also understands the importance of timely, clear, policy-relevant research, which in 2025 will be more important than ever. I am grateful to her for her past leadership and excited for her continued leadership as Board Chair.’” Full
ISPOR Launches “HEOR Explained” Microsite
(12/10, ISPOR Press Release) “...‘HEOR Explained is an exciting new initiative designed to help anyone with an interest in healthcare to better understand how the field of HEOR can help improve healthcare decisions and the lives of patients,’ stated ISPOR CEO Rob Abbott. ‘As healthcare decision making becomes increasingly complex, HEOR insights can help cut through the noise to inform the best possible choices for patients. The HEOR Explained microsite, and initiatives like this, are in service of our vision of a world where healthcare is accessible, effective, efficient, and affordable for all.’” Full
New Patient-Reported Outcome Insights from Target RWE: NASH-CHECK Reveals Stability Over Time and Worse Scores Among Those with Decompensated Cirrhosis of Patients with MASLD
(12/11, Target RWE Press Release) “Target RWE, real-world evidence leader, shared two studies at The Liver Meeting 2024 that revealed insights on the NASH-CHECK1 patient-reported outcome measure (PROM) tailored for patients with metabolic dysfunction-associated steatotic liver disease (MASLD).” Full
Interprofessional George Mason Researchers Awarded More than $1 Million to Improve Outcomes for Patients with Depression
(12/10, George Mason University Press Release) “AI will soon receive a dose of empathy with the goal of helping to match people with depression to their best-fit medication. A team led by Farrokh Alemi, a professor in the College of Public Health, and Kevin Lybarger, an assistant professor in the College of Engineering and Computing, received $1,049,998 in research funding from the Patient-Centered Outcomes Research Institute to continue their work on developing an AI system that helps patients find the right depression medications.” Full
New Price Deal Paves Way for NICE Approval of Treatment for Rare Genetic Condition
(12/10, NICE Press Release) “Vamorolone (also called Agamree and made by Santhera), is an alternative to currently available corticosteroids used to reduce muscle inflammation and maintain muscle strength and function in people with DMD...New analysis presented by the company, together with an improved discount to the price of vamorolone, mean that NICE can now recommend it as a cost-effective use of NHS resources for people 4 years and over.” Full
Study Reveals Inequities and Inadequacies in Access to Medications and Treatment for Cardiovascular Conditions in Canada
(12/11, Elsevier) “...‘Can the Present Canadian Health Care System Provide Evidence-Based Pharmacare? Consideration of Two Important Cardiovascular Clinical Contexts’ compared all provincial drug formulary reimbursement criteria for medications recommended to treat heart failure with a reduced ejection fraction and antiplatelet therapies in acute coronary syndromes...The study assessed whether reimbursement criteria were concordant with listing recommendations from the Canadian Agency for Drugs and Technology in Health (CADTH) and with CCS cardiovascular therapy guidelines.” Full
Journals
Evaluating the Cost-Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B Treatment in the USA
Jyotirmoy Sarker, et al.
December 2, 2024, Applied Health Economics and Health Policy
Can the Present Canadian Health Care System Provide Evidence-Based Pharmaceutical Care? Consideration of Two Important Cardiovascular Clinical Contexts
Morgane Laverdure, MD, et al.
December 10, 2024, Canadian Journal of Cardiology