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FDA Expands Real-World Evidence Framework to Accelerate Drug Approvals and Support Regulatory Decisions

Real World EvidenceDrug Approval
  • The FDA launched a comprehensive Real-World Evidence Program in December 2018 under the 21st Century Cures Act, allowing RWE to support drug approvals for new indications and population expansions.
  • Real-world evidence studies cost significantly less than randomized controlled trials, with postmarket cardiovascular outcomes trials for diabetes drugs costing around $250 million compared to much lower RWE study costs.
  • Electronic health record adoption has dramatically increased from 31% of hospitals in 2003 to 99% currently, and from 11% of office-based doctors in 2006 to 90% in 2017, creating vast new data sources for RWE generation.
  • The biopharmaceutical industry must establish rigorous standards for regulatory-grade data quality, standardized definitions, and new statistical models to ensure broader RWE implementation and regulatory acceptance.

FDA Approves First Treatment for Primary Hemophagocytic Lymphohistiocytosis, Marking 24-Year Breakthrough

Monoclonal AntibodyDrug ApprovalRare DiseaseOrphan Drug
  • The FDA has approved emapalumab-lzsg (Gamifant) as the first treatment specifically indicated for primary hemophagocytic lymphohistiocytosis (HLH), an ultra-rare and life-threatening immune disorder.
  • In a pivotal phase 2/3 study of 27 patients with refractory disease, 63% demonstrated overall response and 70% proceeded to hematopoietic stem cell transplant.
  • The monoclonal antibody targets interferon gamma, representing the first significant improvement in primary HLH induction therapy in 24 years.
  • Primary HLH typically affects children within the first year of life and has a median survival of less than two months without treatment.

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

NCT01818492CompletedPhase 2
Swedish Orphan Biovitrum
Posted 7/1/2013

FDA Approves First Direct-to-Consumer Pharmacogenetic Test from 23andMe

Drug Approval
  • The FDA approved 23andMe's Personal Genome Service Pharmacogenetic Reports, marking the first direct-to-consumer genetic test to help patients understand how their genetics may affect responses to over 50 common prescription and over-the-counter drugs.
  • The test analyzes 33 genetic variants associated with drug metabolism, including responses to blood thinners like clopidogrel and mental health treatments, but comes with strict limitations prohibiting its use for treatment decisions or medical advice.
  • Medical experts express caution about the clinical utility of pharmacogenetic testing for general consumers, noting that while genetic variants affect drug metabolism, the clinical interpretation and prescription implications remain unclear for most patients.

Novartis's Kymriah: Breakthrough CAR-T Therapy Priced at $475,000 Sparks Value Debate

CAR-TDrug ApprovalOncology
  • Novartis's Kymriah, the first FDA-approved CAR-T cell therapy for pediatric acute lymphoblastic leukemia, demonstrates an impressive 83% remission rate in patients who failed traditional treatments.
  • The therapy's $475,000 price tag has ignited debate among healthcare experts, with some defending it as comparable to bone marrow transplants while others argue it's excessive despite its breakthrough status.
  • Novartis has implemented innovative value-based pricing models, including outcome-based contracts where payment is contingent upon patient response and indication-specific pricing for future approvals.

FDA Approves Two New Ustekinumab Biosimilars, Bringing Total US Biosimilar Count to 65

Monoclonal AntibodyDrug Approval
  • The FDA has approved Biocon's Yesintek (ustekinumab-kfce) and Celltrion's Steqeyma (ustekinumab-stba) in November and December 2024, expanding treatment options for autoimmune conditions including Crohn's disease and psoriasis.
  • Of the 65 FDA-approved biosimilars, 14 have received interchangeable designation, allowing pharmacists to substitute them for reference products without prescriber intervention, enhancing patient access to more affordable treatments.
  • The biosimilar landscape continues to evolve with products spanning five major classes: insulin, granulocyte colony-stimulating factors, monoclonal antibodies, TNF-alpha inhibitors, and VEGF inhibitors.

FDA Approves Sanofi's Multaq for Atrial Fibrillation Patients, Marking First New Treatment in Two Decades

Drug Approval
  • The FDA has approved Sanofi's Multaq (dronedarone) to reduce cardiovascular hospitalization risk in patients with atrial fibrillation or atrial flutter, representing the first new treatment option in this therapeutic area in twenty years.
  • The approval is based on five clinical studies involving 6,300 patients, with the landmark ATHENA trial demonstrating a 24% reduction in cardiovascular hospitalization or death from any cause compared to placebo.
  • Multaq showed significant clinical benefits including a 28% reduction in total hospital days and 35% decrease in heart-related hospitalization time in atrial fibrillation patients.
  • The drug is contraindicated in patients with severe heart failure, following results from the ANDROMEDA trial that showed increased mortality risk in this population.

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