CER Daily Newsfeed

IRA Leaves Orphan Drugs Stranded Even as Regulators Promise Progress

(6/23, Heather McKenzie, BioSpace) reports “...Indeed, a National Pharmaceutical Council (NPC) Policy& Evidence Brief, published May 15, found that the number of drugs receiving a first orphan designation each year has remained largely consistent since the passage of the IRA, while those that went on to secure a second designation decreased by 48%. ‘Patient groups, policymakers, and other leaders have all said they wanted to know if these early signals are showing up—and they are,’ Jon Campbell, chief science officer at NPC, told BioSpace in an email. ‘We believe good policy starts with good evidence.’” Full

Semaglutide May Improve Stroke Risk Vs. Empagliflozin for Diabetes

(6/23, Scott Buzby, Cardiology Today) reports “Diabetes treatment with semaglutide was associated with significantly reduced risk for stroke compared with empagliflozin, researchers reported. Risk for any CV events was not significantly different among patients with diabetes treated with dulaglutide (Trulicity, Eli Lilly) compared with empagliflozin (Jardiance, Boehringer Ingelheim), according to new data published in the Annals of Internal Medicine.” Full

Health Insurers Promise to Improve Coverage Reviews That Prompt Delays and Complaints

(6/23, Tom Murphy, Associated Press) reports “...UnitedHealthcare, CVS Health’s Aetna and dozens of other insurers say they plan to reduce the scope of health care claims subject to prior authorization, standardize parts of the process and expand responses done in real time...Insurers said Monday that they will standardize electronic prior authorization by the end of next year to help speed up the process. They will reduce the scope of claims subject to medical prior authorization, and they will honor the preapprovals of a previous insurer for a window of time after someone switches plans.” Full

Duke-Margolis Policy Brief Outlines Regulatory and Ethical Best Practices for Synthetic Data Generation Using Generative AI

(6/23, Katie McCool, The Evidence Base) reports “...To support regulatory acceptance, the brief highlights the need for structured evaluation frameworks to assess the credibility of synthetic datasets. It recommends tools such as the HARmonized Protocol Template to Enhance Reproducibility (HARPER) and the ISPOR SUITABILITY Checklist to help ensure fidelity, reproducibility, and fitness for use. In line with the FDA’s draft guidance on AI, the brief proposes a seven-step risk-based credibility assessment framework tailored to synthetic data generated by generative AI.” Subscription Required