Jazz Pharmaceuticals is committed to the advancement of clinical knowledge generated from various types of research, including non-interventional studies. Like clinical trials, these studies are executed within an established framework of applicable regulations, industry best practices, and internal policies. The integrity of our sponsored research is further supported by the highest standards of transparency and reproducibility that allow researchers, regulators, and patients access to important medical evidence generated from the non-interventional studies below.

ASSESSMENT OF UK HEALTHCARE PROVIDER (HCP) EXPERIENCE WITH THE TOLERABILITY AND OUTPATIENT DELIVERY OF INTENSIVE CHEMOTHERAPIES FOR ACUTE MYELOID LEUKAEMIA (AML)

This study will collect aggregated data on HCP experience with the tolerability and outpatient delivery of intensive AML chemotherapies. The objectives of this assessment are:

• Describing the perceptions of HCPs regarding the tolerability of Vyxeos liposomal vs DA 3+10 and FLAG-Ida. The following adverse reactions are of primary interest, although some others will be also assessed:
  • Fatigue
  • Nausea
  • Alopecia
• Evaluating HCP understanding of the outpatient delivery of Vyxeos liposomal, DA 3+10 and FLAG-Ida during both induction and consolidation treatment.

Study Type: Observational survey
Survey Responses: 31 (15 Haematologists and 16 Cancer Nurse Specialists)
Start date: January 2022
Estimated completion date: June 2022

The data are collected by means of a web-based tool, with each assessment taking approximately 30 minutes to complete. To ensure sufficient experience with Vyxeos liposomal and to minimize selection bias, pre-identified AML treating centres (large AML practice workload and experience in AML 18 and AML 19) will be systematically invited to participate in the study.

A REAL-WORLD STUDY OF TREATMENT PATTERNS AND CLINICAL OUTCOMES FOR SECONDARY AML PATIENTS IN ENGLAND TREATED WITH SYSTEMIC ANTI-CANCER THERAPIES (SACT)

This study makes use of data from the National Cancer Registration and Analysis Service (NCRAS) in Public Health England. NCRAS collects data on all patients diagnosed with cancer in England, which are then linked with other data to create the Cancer Analysis System (CAS). Acute myeloid leukaemia (AML) is a rare type of blood cancer and it includes a specific subtype of disease known as ‘secondary AML’. Secondary AML includes AML occurring following prior exposure to cytotoxic agents and/or radiation and AML evolving from preceding blood disorders i.e., myelodysplastic syndromes (MDS) or myelodysplastic/ myeloproliferative neoplasms (MDS/MPN).

The objectives of this study are:

• Describe the demographic and clinical characteristics of SACT-treated secondary AML patients at diagnosis
• Describe treatment patterns of SACT-treated secondary AML patients
• Describe the survival of Vyxeos-liposomal treated AML patients, overall and by subgroups of interest, including:
  • Early mortality post diagnosis (30- and 60-day survival)
  • Yearly survival
  • Median overall survival

Study Type: retrospective cohort design
Estimated study size: 2000+ AML patients
Estimated completion date: December 2022

Real-world data on the use of cannabidiol in patients with Lennox-Gastaut syndrome or Dravet syndrome in the UK early access programme

This study will obtain data on a sample of patients aged 2-17 years old with Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) who were prescribed cannabidiol as part of the Early Access Program. It will provide real-world insights into the disease, current treatment patterns, clinical outcomes, and healthcare resource use within the UK healthcare environment.

The objectives of the study are:

• To describe demographics and clinical characteristics of patients prescribed cannabidiol for DS and LGS in the UK for Jazz Pharmaceuticals’ Early Access Program from November 2018 to January 2020
• To describe treatment patterns following prescription of cannabidiol, including duration of treatment, dosage, treatment discontinuation and switching from cannabidiol
• To describe clinical outcomes of patients with DS and LGS following prescription of cannabidiol, including type and frequency of seizures, percentage change from baseline in the frequency of seizures and duration of seizure-free days

Study type: Multi-centre non-interventional observational study (retrospective)
Study size: 26 patients (9 patients with Dravet Syndrome and 17 patients with Lennox-Gastaut Syndrome), across two sites
Start date: October 2021
Estimated completion date: April 2023