Sponsor Overview
Explore verified public information about Novartis'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.
“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.”
“Managed Access Programs offer us a mechanism to provide our novel innovative therapies in a responsible, ethical, speedy and safe manner to eligible patients with no other treatment options, especially when there is no time to lose and enrolment in a clinical trial is not possible. It’s a true privilege to be able to address unmet patient needs in this area, and I’m proud of our teams who are constantly seeking new and compliant ways to further improve the design and execution of this important access approach.”
“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: Anaplastic Lymphoma Kinase (ALK)- Positive Tumors, Non-small Cell Lung Cancer (NSCLC)
Conditions: Chronic Spontaneous Urticaria
Conditions: Atherosclerotic Cardiovascular Disease, ASCVD
Conditions: Thrombocytopenia
Conditions: C3 Glomerulopathy (C3G), Paroxysmal Nocturnal Hemoglobinuria (PNH)
Past EAPs on ClinicalTrials.gov
Score contribution: 40 — 26 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: Chronic Myeloid Leukemia
Conditions: Melanoma, Adjuvant
Conditions: Non-small Cell Lung Cancer (NSCLC)
Conditions: Acute Lymphoblastic Leukemia (ALL)
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