CER Daily Newsfeed

Rare Disease Drugmakers Shift Plans After Win in Trump Megabill

(7/23, Nyah Phengsitthy, Bloomberg Law) reports “...The brief provision in Republicans’ bill passed this month exempts drugs with multiple rare disease indications from the Medicare Drug Price Negotiation Program...Now, the change is prompting drugmakers that develop rare disease treatments to ease back into exploring additional rare indications, drug industry groups and attorneys say...An analysis published in May by the National Pharmaceutical Council found the percentage of drugs with a first orphan designation that later received a second designation decreased from about 12% to 6% following the passage of the 2022 Inflation Reduction Act that created the negotiation program.” Subscription Required

GLP-1 RAs More Effective Than Metformin for Reducing Dementia Risk in T2DM

(7/22, Abigail Brooks, MA, HCPLive) reports “GLP-1 RAs significantly reduce the risk of overall dementia and all-cause mortality compared to metformin in type 2 diabetes patients. The study utilized a decade of data from the TriNetX database, employing propensity score matching to balance patient characteristics.” Full

GLP-1 Receptor Agonists Tied to Decrease in T2DM-Related Dementia Risk

(7/23, Elana Gotkine, HealthDay) reports “...‘Despite limitations such as residual confounding, the findings remain robust and suggest GLP-1 RAs could redefine T2DM-related dementia prevention strategies, warranting further randomized controlled trials to confirm these results,’ the authors write.” Full

Deloitte’s Latest RWE Benchmarking Survey Highlights Biopharma’s next Steps in Real-World Evidence

(7/23, The Evidence Base) reports “...Of the C-suite leaders surveyed, 40% now consistently discuss RWD/E within their organizations. At the same time, the range of data sources being utilized has expanded well beyond traditional electronic health records and claims data. Companies are increasingly seeking more comprehensive, longitudinal views of patient experiences, as well as diverse RWD types such as genomics, digital health and wearables, patient-generated health data, and social determinants of health (SDOH).” Full

Roche’s Flatiron Health Triples Cancer Network Size Across UK, Germany and Japan

(7/23, Darren Incorvaia, Fierce Biotech) reports “...‘The unprecedented expansion of our global network will continue to support improvements to local cancer patients’ care and novel multinational research—fundamentally changing what’s possible for our partners and the patients they serve,’ Nathan Hubbard, Flatiron Health’s chief business officer, said in the release. ‘Our partners are already unlocking answers to complex research questions—answers that traditional data sources can’t support.’” Full

UK: NICE Recommends ALK’s Immunotherapy to Target Underlying Cause of Hay Fever

(7/23, Emily Kimber, PMLiVE) reports “The National Institute for Health and Care Excellence (NICE) has recommended ALK’s once-daily immunotherapy tablet to target the underlying cause of hay fever caused by birch tree pollen. The health technology assessment agency has recommended in final draft guidance that the under-the-tongue drug, Itulazax (12 SQ-Bet), be used on the NHS in England and Wales to treat adults with moderate-to-severe, uncontrolled allergic rhinitis or conjunctivitis caused by birch tree pollen.” Full

Researchers Sequence Complex Parts of Human Genome, Expanding the Future of Precision Medicine

(7/23, Veronica Paulus, STAT+) reports “Complex regions of the human genome remained uncharted, even after researchers sequenced the genome in its entirety. That is, until today. Researchers decoded DNA segments involved in the development of diseases like diabetes and spinal muscular atrophy that had previously been considered too complicated to sequence. Their work, published in Nature on Wednesday, could expand the future of precision medicine.” Subscription Required