CER Daily Newsfeed

Dupilumab Efficacy in Young Children With Severe AD Confirmed by Real-World Data

(2/18, Colby Stong, Dermatology Advisory) reports “...‘The outcomes of this EAP complement the controlled environment of randomized controlled trials and provide data beyond the typical 16-week period of randomized trials. The results from this EAP can be more generalizable to the broader patient population of 6 months-5 years of age, as they reflect real-world use across diverse clinical settings,’ the researchers concluded.” Full

Updated ISPOR HEOR Competencies Framework Outlines Skills for a Changing Evidence Landscape

(2/19, Katie McCool, The Evidence Base) reports “...ISPOR links the update to the evolution of HEOR over the past two decades, stating that ‘the field of health economics and outcomes research (HEOR) has grown substantially in the past 20 years’ and that, given the discipline’s ‘dynamic nature’ and external influences on healthcare decisions, there is a need to provide guidance to professionals so they have the tools ‘to create, implement, and evaluate the different aspects of HEOR.’ The society also states that the framework ‘promotes research excellence by outlining a comprehensive list of knowledge and skills important and relevant for HEOR professionals,’ and that it indicates ‘both technical and soft skills are necessary for professionals to be capable of improving healthcare decision making.’” Full

Explore the Medical Care Special Article Collection on PCORnet

(2/18, PCORI Blog) comments “...Taken together, the collection underscores the scale, reach and impact of PCORnet. One paper, ‘PCORnet®: 10 Years of Research Innovation,’ discusses PCORnet links to 47 million individuals across thousands of care sites. Of these individuals, the most common chronic conditions presented were hypertension, anxiety disorders, Type 2 diabetes and asthma. The paper also highlights plans to further develop the PCORnet® Front Door, the access point for all investigators, funders and individuals within the broader healthcare community to begin interacting with the PCORnet infrastructure.” Full

Cochrane Launches Innovative Study to Assess AI Tools for Evidence Synthesis

(2/19, Martha Powell, Cochrane) comments “Cochrane has launched a study to test whether artificial intelligence (AI) tools can support or enhance evidence synthesis. The initiative will use an innovative platform study design and will adhere to robust and responsible criteria, upholding Cochrane’s rigorous standards for evidence.” Full

Navigating Cell And Gene Therapy Risk In Medicaid: Lessons From The States

(2/19, William H. Shrank, Bruce Greenstein, Hannah Katch, Health Affairs Forefront) comments “...Adjudication of value-based contracts requires long-term collection of outcome data. CMS is developing plans to support this data collection, but will most certainly require partnership with private organizations to collect, integrate and analyze that data. In short, the federal model builds a necessary scaffold for the administration of value-based contracting for these transformative therapies in Medicaid. However, CMS and states still need to partner with the private sector to address long-term cost growth for the full scope of CGT while operationalizing the CMMI model’s principles.” Full

Ultra-Rare Disease Drug Loargys Wins English Funding Ahead Of US FDA Verdict

(2/19, Neena Brizmohun, Pink Sheet) reports “...Immedica’s Loargys is set to become the first disease modifying treatment for arginase 1 deficiency to be reimbursed in England. Meanwhile, the US regulator is reviewing a resubmitted marketing application for the product and has set a target action date of Feb. 23.” Subscription Required

UK: NICE Backs Changes to NHS' Diabetes Care Pathway

(2/19, Phil Taylor, PharmaPhorum) reports “...The new NICE guidance recommends that most people should now be offered metformin along with an SGLT-2 inhibitor from the start, while also advising that they get a slow-release formulation of metformin, which can cause fewer gastrointestinal side effects like stomach upset than the regular, standard-release form. Douglas Twenefour, head of clinical at the Diabetes UK charity, said the guidance will ‘transform treatment for people living with type 2 diabetes across the UK [and reduce] the harm caused by this relentless condition.’” Full