CER Daily Newsfeed

Orphan Cures Act Re-Added to Trump Tax Bill as Senate Prepares to Begin Vote-a-Rama

(6/30, Angus Liu, Fierce Pharma) reports “...The bipartisan Orphan Cures Act was designed to fix what the biopharma industry views as one of the many unintended consequences of the IRA. By offering exemptions exclusively to drugs with just one rare indication, the original IRA disincentivizes companies from conducting follow-on tests of rare disease therapies, the industry has argued. An analysis by the National Pharmaceutical Council recently found that after IRA's passage, the percentage of drugs with an initial orphan designation that went on to receive a second designation decreased by 48%—from 12.1% to 6.3%—despite the industry seeing a steady increase before the law.” Full

Value Viewpoint: June 27, 2025

(6/27, Kimberly Westrich, LinkedIn) comments “...A new article published in The Evidence Base recaps a recent ISPOR 2025 session on efforts to improve HTA across borders, featuring my NPC colleague Jon Campbell. He spoke about the relationship between HTA methods and patient access, emphasizing the importance of striking a balance between decision-making and scientific integrity, as well as context-sensitive judgment, framing ‘HTA methods as, “helpful scientific tools, but not rules.”’ Full

Second-Line Treatments for Women With Epilepsy Show Comparable Outcomes

(6/30, Jessica Nye, PhD, Neurology Advisor) reports “Among women with idiopathic generalized epilepsy (IGE), there are no significant differences between add-on therapy and substitution monotherapy as a second-line treatment, according to study results published in JAMA Network Open.” Full

Real-World Evidence Helps Accelerate Formulary Decisions for Biologics

(6/27, Muhammad Cheema, PharmD, MPBA, Pharmacy Times) comments “...A recent survey of US payers found that 85% use RWE to guide formulary placement in oncology, primarily to compare treatment effectiveness when head-to-head trials are lacking and to validate guideline recommendations. The most frequently used RWE sources were administrative claims data (79% of payers), clinical/EHR data (69%), prospective observational cohorts (60%), and disease registries (37%).” Full

Video: Effective Real-World Evidence for Adding Value to a Clinical Trial

(6/30, Ed Schoonveld, PharmExec) “In response to the rising importance of real-world evidence (RWE) in showing value to payers beyond traditional clinical trials, key opinion leaders emphasize finding cost-effective approaches, considering diverse patient populations, and using AI to pinpoint which patients will benefit most from treatment.” View Video

CMS Continues to Make a Bad Law Worse

(6/27, Matthew Norawong, PhRMA Blog) comments "...The guidance continues to send the message to innovators that investments to explore new indications, dosage forms, strengths or formulations for medicines are unwelcome, despite the value they bring to patients. For instance, CMS’s definition for determining if a drug is eligible for price setting (a ‘qualifying single source drug’ or QSSD) is so broad that even newly launched treatments could be price set. Moreover, rare disease drug development remains at risk. CMS has chosen to stop taking feedback and continue to implement the IRA’s Orphan Drug Exemption in a manner that unnecessarily punishes innovators who wish to invest in new rare disease indications." Full

Experts Skeptical of Trump's So-Called 'Gold Standard Science'

(6/30, Rachael Robertson, MedPage Today) reports “...The evidence-based medicine framework, he explained, holds that some research is higher quality than other research, that systematic evidence reviews are vital, and that evidence never tells a clinician what to do; rather, it must be interpreted in the context of a patient's values and preferences. [McMaster University professor of medicine Gordon Guyatt, MD] said that much of HHS Secretary Robert F. Kennedy Jr.'s views and initiatives ‘would be completely discredited by the standards of evidence-based medicine.’” Full