News from Thursday, March 13, 2025
Articles
Contributor: The Role of Dynamic Pricing in Health Technology Assessment for MDD
(3/13, Dominique Seo, MPH, Larragem Raines, MS, Rick Chapman, PhD, The American Journal of Managed Care) comments “...Incorporating dynamic pricing allows cost-effectiveness models to more accurately reflect drug costs in economic evaluations, ultimately improving resource allocation and treatment access for diverse patient groups. Although challenges remain—including data gaps and uncertainties with novel treatments—dynamic pricing has the potential to enhance efficiency in health care.” Full
Video: Emerging ADC Clinical Trial Evidence in Breast Cancer Treatment
(3/13, Heather Moore, BCOP, CPP, PharmD, Jodi Taraba, PharmD, MS, BCOP, Rodney Hunter, PharmD, BCOP, Jordan Hill, PharmD, BCOP, Rose DiMarco, PharmD, BCPS, BCOP, Pharmacy Times) reports “Panelists discuss how emerging antibody-drug conjugates (ADCs) in metastatic breast cancer (mBC) treatment demonstrate innovation through optimized drug-to-antibody ratios, novel payloads, and targeted approaches to different breast cancer subtypes. Key trials explore combinations with established therapies and potential for improved tolerability profiles. Additional notable studies include comparative effectiveness research between approved ADCs and investigation of biomarker-driven patient selection strategies to maximize therapeutic benefit while minimizing adverse effects.” Full
Uniform Global Standards, Processes Could Accelerate CGT Approvals, Access
(3/13, Rose McNulty, The American Journal of Managed Care) comments “...‘Our comparative analysis of CGT product submissions to the FDA and EMA supports the crucial need for harmonization efforts in CGT product development, including improved standardization of trial design and reporting,’ the authors wrote. ‘While some variance in clinical efficacy reported across applications may be expected due to differences in regulatory requirements, risk tolerance, and submission timing, a better understanding of factors resulting in large differences in outcomes is needed to ensure that regulatory decisions are based on robust and consistent evidence.’” Full
VBID, Heading Into a Third Decade, Looks to Promote Personalization and Access
(3/12, Christina Mattina, The American Journal of Managed Care) comments “...Looking forward, [Adam Beck, MS, JD] suggested that provider buy-in and patient knowledge of VBID need to grow for its continued evolution. [Andrew Chasin, JD] added the necessity of building on personalized VBID, because the same service that’s high value for one person can be low value for another. Finally, Martin emphasized the importance of setting realistic expectations for VBID; these efforts may not always save money, if high-value care is being used in place of low-value care, but they should help get closer to the ideal of paying for the right care for the right patient at the right time.” Full
England Reimburses Ultra-Rare Disease Drug Joenja, While EU Regulatory Review Drags On
(3/13, Neena Brizmohun, Pink Sheet) reports “...England has become the first European country to agree to reimburse Joenja (leniolisib), Pharming’s ultra-rare immune disorder drug, which received UK marketing approval last September but is undergoing a protracted regulatory review in the EU.” Subscription Required
Press Releases
First Licensed Treatment for Ultra-rare Immune Disorder Recommended
(3/13, NICE Press Release) “In final draft guidance published today, we have recommended leniolisib (also called Joenja and made by Pharming) for treating activated phosphoinositide 3-kinase delta syndrome (APDS), an ultra-rare inherited immune system disorder, in people 12 years and over.” Full
First Daily Pill for Endometriosis Approved for NHS Use
(3/13, NICE Press Release) “Today we've recommended the first long-term daily pill to treat endometriosis that could transform how this painful condition is managed...The treatment, called relugolix–estradiol–norethisterone (also known as relugolix combination therapy or Ryeqo), works by blocking specific hormones that contribute to endometriosis, while also providing necessary hormone replacement in a single daily tablet.” Full
Journals
Psoriasis Risk Is Lower in Type 2 Diabetes Patients Using Dipeptidyl Peptidase-4 Inhibitors or Thiazolidinediones Compared to Sulfonylureas
Wei-Sheng Chen, et al.
March 2025, Clinical and Translational Science
Lived Experience Is Essential for Innovation in Biomedical Research
Jonathan Haydak, et al.
March 13, 2025, Nature Reviews Nephrology