CER Daily Newsfeed

Perspectives from IVI’s 7th Annual Methods Summit: Scientific Spillovers, Cell and Gene Therapy, and the Challenge of Measuring Broader Societal Value

(4/9, The Evidence Base) reports “...[PCORI’s Nakela Cook] emphasized that PCORI does not define value directly, but generates evidence to inform more patient-centered decisions. Drawing on examples such as sickle cell disease, Cook highlighted the importance of identifying outcomes that matter most to patients and caregivers, including quality of life, emotional and mental health, access to care, and financial stress. She also stressed that engagement must be continuous and meaningful throughout the research process, reflecting a broader shift from involving patients as participants to supporting them as co-leaders.” Full

FDA, Device Makers Near MDUFA Deal Tying Funding To NEST RWE Program

(4/9, James Jarvis, Inside Health Policy) reports “...For months, device makers and FDA had gone back and forth over staffing transparency, fee structure and how user-fee dollars would be allocated, as companies sought greater visibility into the agency’s review capacity following significant workforce reductions across FDA, including the termination of many medical device reviewers...Transparency around staffing was not explicitly addressed in the March 18 meeting minutes...The meeting instead focused on finalizing key elements of the agreement, including how industry funding would support FDA’s real-world evidence efforts. Industry said its agreement to fund additional staff tied to real-world evidence is contingent on those resources being used specifically for premarket review activities and governed through the NEST framework, reflecting a broader push to ensure user-fee dollars are narrowly targeted.” Subscription Required

How to Use Real-World Data to Improve Drug Development, Starting with the Patient Journey 

(4/9, Stephanie Baum, MedCity News) reports “...A new eBook from PurpleLab and MedCity News discusses how real-world data (RWD) can illuminate hidden obstacles in the NSCLC patient journey. This can include transportation access to disparities in molecular testing. States have passed a variety of legislation requiring that insurance companies cover biomarker testing. This can lead to earlier detection and improve the effectiveness of more targeted interventions and equitable outcomes.” Full

Levels, Growth, And Semantics: The Role Of Prices In Driving Health Care Spending

(4/9, Michael E. Chernew, Health Affairs Forefront) comments “...New products, particularly high-priced ones, present a particular challenge for the price/quantity decomposition—they are both. Sometimes the value on average justifies the high price; sometimes not. Sometimes the use diffuses to populations beyond where value would justify the price. The question of how to balance drug prices with innovation matters, but labelling the spending consequences of innovation as specifically price effects or specifically volume effects is not necessary. We can simply refer to these effects as innovation effects and debate the appropriate policies related to price or use of innovation.” Full

England’s HTA Body Reverses Rejection For Seriver’s Voranigo

(4/9, Neena Brizmohun, Pink Sheet) reports “...The health technology assessment institute, NICE, said its appraisal committee considered additional benefits identified during a consultation for the drug, which is used to treat certain types of low-grade glioma.” Subscription Required