Sponsor Overview
Explore verified public information about Novartis Pharmaceuticals's expanded access programs, compassionate use policies, and patient eligibility pathways. This report aggregates official statements, independent research, and regulatory data to help patients, providers, and sponsors collaborate efficiently.
Unique references curated across 4 content categories.
ClinicalTrials.gov data sources power this status.
Dataset refreshed continuously through Right2Hope ETL pipelines.
Expanded Access Intelligence
Official Statements
Score contribution: 100 — 6 supporting sources.
“Within Novartis, we refer to such provision of investigational or pre-approval products as “Managed Access.” The Novartis “Managed Access” terminology covers all locally defined pre-approval access mechanisms and programs such as “Compassionate Use”, “Expanded Access”, “Named Patient Supply”, “Special Access Schemes/Programs”, “Autorisations temporaires d’utilisation (ATU)” and others.”
“The Novartis “Managed Access” terminology covers all locally defined pre-approval access mechanisms and programs such as “Compassionate Use”, “Expanded Access”, “Named Patient Supply”, “Special Access Schemes/Programs”, “Autorisations temporaires d’utilisation (ATU)” and others.”
“At Novartis, we are providing a lifeline of access to innovative medicines under review or not yet approved locally when no other treatment options are available. When this is not available or the patient is not suited, another option is access via ‘compassionate use’ (CU) programs, at Novartis referred to as ‘Managed Access Programs’ (MAPs), which enable access to locally unlicensed medication (generally free of charge) for patients with serious or life-threatening medical conditions.”
“The Zolgensma® (onasemnogene abeparvovec) global Managed Access Program (gMAP) was created in 2020, the first of its kind for a one-time gene therapy, as a pathway while we worked to broaden global access to Zolgensma. The program was available and provided our gene therapy free of charge to eligible patients in countries where it was possible to make Zolgensma available, where it had not yet received approval, or where no access pathway existed.”
“We are committed to providing patients who have no comparable or satisfactory alternative therapy a Novartis product prior to regulatory approval.”
“GEMS is a global cloud-based system for the submission and ongoing management of all Managed Access Program (MAP) requests. All initial and resupply requests must be submitted via the GEMS online portal accessed from www.novartis.com. GEMS makes it easy for you (the physician) to submit and manage your request and for Novartis to speedily review and make a decision.”
Active EAPs on ClinicalTrials.gov
Score contribution: 60 — 18 supporting sources.
Conditions: Sickle Cell Disease
Conditions: Carcinoma, Non-Small Cell Lung, Non-Small Cell Lung Cancer, Non-Small-Cell Lung Carcinoma, Nonsmall Cell Lung Cancer
Conditions: Atherosclerotic Cardiovascular Disease, ASCVD
Conditions: C3 Glomerulopathy (C3G), Paroxysmal Nocturnal Hemoglobinuria (PNH)
Conditions: Thrombocytopenia
Conditions: PIK3CA-Related Overgrowth Spectrum (PROS)
Past EAPs on ClinicalTrials.gov
Score contribution: 40 — 24 supporting sources.
Conditions: FMS-Like Tyrosine Kinase 3 (FLT3)-Mutated Acute Myeloid Leukemia, Acute Myeloid Leukemia, Aggressive Systemic Mastocytosis, Mast Cell Leukemia, Systemic Mastocytosis With an Associated Hematologic Neoplasm
Conditions: HR+ Advanced or Metastatic Breast Cancer
Conditions: Acute Lymphoblastic Leukemia (ALL)
Conditions: Chronic Myeloid Leukemia
Conditions: Acromegaly
Conditions: Melanoma, Adjuvant
Reagan-Udall Foundation Insights
Single-Patient EA Policies/Criteria Novartis considers granting managed access to investigational or pre-approval products via single patient access provided the Managed Access criteria are fulfilled. Disease/Category-Specific EA Policies/Criteria Product Indication Capmatinib MET dysregulated Non-Small Cell Lung Cancer (NSCLC) Ceritinib Non-small cell lung cancer: ALK positive tumors, previously pre-treated with ALK inhibitors or naive Clofazimine Mycobacterial infection Iptacopan C3 Glomerulopathy (C3G) Tisagenlecleucel Acute lymphoblastic leukaemia; Diffuse large B-cell lymphoma refractory
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