News from Friday, January 17, 2025
Articles
CMS Drug List Commentary
(1/17, John O’Brien, LinkedIn Post) comments “...The 2027 list is 100% small-molecule, has four oncology drugs on it, and averages 60% fewer Part D enrollees per drug than the first list. The time since FDA approval also shrank by nearly 3 years. My fear is that we will continue to see signs demonstrating what our research found: The unintended consequences of the IRA will lead to delayed launches, fewer subsequent indications, less post-approval research, all with uncertain impact for the patient.” Full
Novo's Semaglutide Among 2nd Batch of Drugs Selected for IRA Price Negotiations
(1/17, Angus Liu, Fierce Pharma) reports “...According to the CMS, the selected meds together accounted for about $41 billion in Medicare Part D gross spending between November 2023 and October 2024, or about 14% of the insurance plan's total costs for covered prescription drugs...In a statement Monday, Stephen Ubl, CEO of the industry group PhRMA, again called the IRA process ‘price setting’ that's ‘dangerous for millions of Americans who rely on innovative treatments.’ ‘In rushing out this list in their final days, the Biden administration once again fails to address the true challenges facing seniors and Medicare,’ Ubl said. Full
IBAT Inhibitors Have Potential to Boost Life Quality for Patients
(1/17, Lindsey Shapiro, PhD, Liver Disease News) reports “Alagille syndrome comes with a high clinical, economic, and quality-of-life burden for patients and their families, but new treatments such as Livmarli (maralixibat) and Bylvay (odevixibat) could change the future outlook, according to a recent review study...The study, ‘The burden of Alagille syndrome: uncovering the potential of emerging therapeutics – a comprehensive systematic literature review,’ was published in the Journal of Comparative Effectiveness Research.” Full
The Quintuple Aim: Redefining Patient-Centered Care in the Digital Age
(1/17, Prashila Dullabh, HIT Consultant) comments “...Advancements in digital technology in health IT offer great potential to enhance patient-centered care outcomes, but it is essential to implement appropriate safeguards to maintain and foster patient trust amid these exciting developments. As the velocity of these developments continues to accelerate in healthcare contexts, more research from NORC and other research organizations is needed to discern the necessary safeguards that will protect patient data, earn trust, and provide greater care outcomes in the years to come.” Full
Press Releases
International Collaboration Provides New Guidance on the Use of Surrogate Endpoints in Cost-Effectiveness Analysis
(1/17, NICE Press Release) “NICE, in collaboration with other international health technology assessment agencies, has published a new report which offers guidance on best practice for using surrogate endpoints in health economic models to guide health technology assessment (HTA) decisions...The new report outlines how and when these surrogate endpoints can best be used to help judge the value of technologies to patients.” Full
Journals
The Burden of Alagille Syndrome: Uncovering the Potential of Emerging Therapeutics – A Comprehensive Systematic Literature Review
Philip Bufler, et al.
January 14, 2025, Journal of Comparative Effectiveness Research
Journal of Comparative Effectiveness Research
Selection of Patient-Reported Outcome Measures in Pulmonary Arterial Hypertension Clinical Trials: A Systematic Review, Meta-Analysis and Health-Related Quality of Life Framework
Frances L Varian, et al.
January 17, 2025, medRxiv
Enhancing Therapy Adherence: Impact on Clinical Outcomes, Healthcare Costs, and Patient Quality of Life
Urszula Religioni, et al.
January 17, 2025, medicina
Reports
Surrogate Endpoints in Cost-effectiveness Analysis for Use in Health Technology Assessment
January 2025