Celgene

Multiple Myeloma

This study is designed to evaluate the safety and efficacy of nonconforming idecabtagene vicleucel (ide-cel) in participants with multiple myeloma per the approved prescribing information. This is an expanded access protocol (EAP) to be conducted at Risk Evaluation and Mitigation Strategies (REMS) qualified sites approved for commercial administration of idecabtagene vicleucel and where the EAP is authorized to be conducted for use of nonconforming idecabtagene vicleucel. Non-conforming idecabtagene vicluecel is idecabtagene vicleucel that does not meet commercial release specifications but may be acceptable for use as an investigational product in the Expanded Access Protocol setting.

Acute Myelogenous Leukemia (AML)

This is an expanded access program (EAP) for eligible participants designed to provide access to CC-486.

Multiple Myeloma

To provide pomalidomide access to relapsed/refractory multiple myeloma subjects with a likelihood of benefit from the pomalidomide treatment while the medication is not commercially available

Myelodysplastic Syndrome

This is an expanded access program (EAP) for eligible participants designed to provide access to ACE-011.

Myelofibrosis

This is an expanded access program (EAP) for eligible participants designed to provide access to fedratinib. Expanded access is only available in markets where fedratinib is not yet approved.

Bristol-Myers Squibb

This is an expanded access designed to provide access to abatacept for eligible participants.

This is an expanded access designed to provide access to fixed-dose combination of nivolumab plus relatlimab for eligible participants.

This is an expanded access designed to provide access to Relatlimab for eligible participants.

This is an expanded access designed to provide access to Anti-IL8 for eligible participants.

This is an expanded access designed to provide access to apixaban for eligible participants.

This is an expanded access designed to provide access to dasatinib for eligible participants.

Renal Transplantation

To make belatacept available for recipients of a renal allograft who are currently intolerant to or have contraindications to CNIs and/or m-TOR inhibitors and are either: - unable to construct an adequate immunosuppression regimen due to non-renal toxicity / contraindication (and withdrawing the causative agent would lead to renal graft loss) OR - at imminent risk of losing the allograft kidney due to nephrotoxicity and have no other options for renal replacement therapy

Multiple Myeloma

The objective of this expanded access program is to provide treatment with elotuzumab in combination with lenalidomide and dexamethasone for patients with relapsed or refractory multiple myeloma at U.S. sites where licensed physicians determine clinical need.

Multiple Myeloma

The objective of this expanded access program is to provide treatment with elotuzumab in combination with lenalidomide and dexamethasone for patients with relapsed or refractory multiple myeloma at Japanese sites where licensed physicians determine clinical need.

Multiple Myeloma

At BMS, we work with physicians/investigators to make investigational products available to patients with life-threatening diseases that have exhausted other treatment options and where there is a reasonable expectation of benefit over risk. When contacted by a treating physician, BMS will consider requests for providing early patient access to Elotuzumab in patients with multiple myeloma who are residents of Belgium, Thailand, Turkey, Argentina, and Colombia.

Pediatric Cancer

At BMS, we work with physicians/investigators to make investigational products available to patients with life-threatening diseases that have exhausted other treatment options and where there is a reasonable expectation of benefit over risk. When contacted by a treating physician, BMS will consider requests for providing early patient access to Nivolumab in pediatric patients exhibiting a high mutational load.

Boehringer Ingelheim Pharmaceuticals

Solid Tumors

Gene fusions are defined as two separate genes joined together (gene 1, gene 2), generating a novel fusion gene. NRG1 fusions are rare and complex with regard to the fusion/fusion partner. The specific NRG1 gene fusion will be identified by a specialized molecular testing lab.

Lung Diseases, Interstitial

This Expanded Access Program in Belgium is open to people with different lung diseases. This program provides a medicine called nintedanib to people who have no alternative treatment options. They can participate if they have a type of lung disease called non-IPF ILDs (chronic fibrosing interstitial lung diseases with a progressive phenotype other than idiopathic pulmonary fibrosis). Participants take 2 capsules of nintedanib a day. The treating physician checks the health of the participants and notes health problems that could have been caused by nintedanib. Participants receive nintedanib as long as they benefit or until nintedanib becomes commercially available in Belgium. For a patient to participate in this program, their treating physician should apply to Boehringer Ingelheim.

HIV Infections

To provide early access to tipranavir and evaluate the safety and tolerance of tipranavir combined with low dose of ritonavir in patients with progressive, HIV-1 disease who have failed or are intolerant to currently approved treatments for HIV infection, who are unable to participate in another tipranavir controlled clinical trial and have an urgent need for anti-HIV treatment.

HIV Infections

This study was designed to provide early access to and evaluate the safety of TPV/r in PI-experienced patients with HIV-1 infection.

HIV Infections

The purpose of this Open Label Safety Study is to provide access to and evaluate the safety and tolerability of TPV/r in treatment-experienced patients with advanced HIV-1 infection who have failed at least two PI-containing regimens, and have limited treatment options.

Carcinoma, Non-Small-Cell Lung

The program will provide early access to the investigational drug BIBW 2992 to treat patients with advanced NSCLC who have failed at least 12 weeks on erlotinib or gefitinib. The Compassionate Use Programme will also provide additional safety and efficacy information on BIBW 2992 use. Named Patient Use (NPU)

Carcinoma, Non-Small-Cell Lung

This is an open-label, multi-center, single-arm trial, designed to provide early access to afatinib and to provide additional information on the safety and efficacy of afatinib in advanced NSCLC patients who harbor an EGFR mutation.

Carcinoma, Non-Small-Cell Lung

To provide expanded access and to evaluate the safety, tolerability and efficacy of afatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring Epidermal Growth Factor Receptor (EGFR) mutation(s) and have never been treated with an EGFR tyrosine kinase inhibitor (TKI)

Idiopathic Pulmonary Fibrosis

To provide early access and to evaluate the safety and tolerability of nintedanib in patients with idiopathic pulmonary fibrosis (IPF).

Lung Diseases, Interstitial

This Expanded Access Program is intended to facilitate the availability of OFEV to patients suffering from non Idiopathic Pulmonary Fibrosis-Interstitial Lung Disease (non IPF-ILD) with a progressive clinical course despite Standard of Care treatment and for whom no satisfactory authorised alternative therapy exists or who cannot enter a clinical trial.

Blueprint Medicines Corporation

Advanced Systemic Mastocytosis
Aggressive Systemic Mastocytosis
Systemic Mastocytosis With an Associated Hematological Neoplasm
Mast Cell Leukemia

This is a US, multicenter, open-label expanded access program designed to provide access to avapritinib in eligible patients with AdvSM until such time that avapritinib becomes available through other mechanisms, or the Sponsor chooses to discontinue the program.

GIST

This is a US, multicenter, open-label expanded access program to provide access to avapritinib until such time that avapritinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.

Non-Small Cell Lung Cancer
Medullary Thyroid Cancer

This is a global, multicenter, open-label pre-approval access program to provide access to pralsetinib (BLU-667) until such time that pralsetinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.

Biogen

Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism in order to address a high unmet medical need in this population.

Infantile-onset Spinal Muscular Atrophy

To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.