Clinical Practice

What steps is the FDA taking to accelerate rare disease treatments? The FDA’s new Rare Disease Framework Initiative aims to streamline regulatory pathways, enhance collaboration with sponsors, and improve patient access to innovative therapies. Explore how this could reshape drug development for underserved patient populations. #SyenzaNews #regulatoryaffairs #drugdevelopment #innovation #healthcarepolicy

Researchers and Allegheny County plan a $13.3 million trial of weekly semaglutide to reduce opioid and alcohol use and justice involvement.

Social prescribing is not yet part of Portugal's national health system. Economists are asking whether it works, and who pays for it.

BioNTech's AHEAD-MERIT trial did not reach significance on progression-free survival. The overall survival read-out is still to come.

The EMA has authorised Denosumab Ascend, a biosimilar of Xgeva. Here is what the assessment shows on safety, efficacy and use. #SyenzaNews #biotechnology #MarketAccess

OHE's A Dose of Economics explores why medicines made for one patient challenge regulation, payment and HTA.

AI is entering every stage of medical training. A new npj Digital Medicine Perspective maps how to integrate it responsibly across UGME, GME and CME.

The EU's ACT EU advice pilot for clinical trial applications becomes permanent in January 2027, with updated sponsor guidance.

A University of Minnesota model fine-tuned on routine clinical notes separated epileptic from psychogenic seizures with an AUC of up to 0.875, and helped neurologists lift their own accuracy by up to 14.3 percentage points. #SyenzaNews #epilepsy #AI #digitalhealth #MarketAccess

Novartis has won the first FDA approval for symptomatic dermographism. Rhapsido (remibrutinib) is now cleared for adults whose hives and itch stay uncontrolled on H1 antihistamines, with 29.3% of patients achieving complete hive resolution at Week 12 against 14.0% on placebo. #SyenzaNews #dermatology #MarketAccess

Genmab and AbbVie report topline Phase 3 results in which epcoritamab plus R-CHOP cut the risk of progression or death by 51% in newly diagnosed diffuse large B-cell lymphoma, the first positive readout for a bispecific combination in the first-line setting. #SyenzaNews #DLBCL #bispecifics #oncology #MarketAccess

Vaxcyte's VAX-31 met every prespecified primary endpoint in the OPUS-1 Phase 3 trial in adults, including superiority for the three serotypes unique to the vaccine. The 31-valent candidate now heads toward a BLA filing in 2028. #SyenzaNews #vaccines #pneumococcal #MarketAccess