Chapter 1 28 On the 12th of December 2022, tabelecleucel was registered by the European Medicines Agency under the tradename Ebvallo® for the treatment of relapsed/refractory post-transplant lymphoproliferative disease for patients who test positive for Epstein-Barr Virus.2 Setup and methodology of the Expanded Access Program In addition to the trials, an ‘Expanded Access Program’ was initiated by the biotech company Atara (the sponsor) and myTomorrows (the service provider) for ineligible trial patients in Europe. The primary goal was to provide treatment to tabelecleucel to patients. Patients were first triaged for participation in clinical trials for tabelecleucel. If participation was impossible, patients were assessed for eligibility for expanded access and determined if treatment could potentially be of benefit to the patient. If appropriately matched product was available and the patient was eligible, the patient could participate in the expanded access program to receive tabelecleucel. In addition to PTLD-patients, patients with different types of diseases driven by the Epstein-Barr Virus could also be considered in the expanded access program, such as primary immunodeficiency‑driven lymphoproliferative disease (LPD), acquired immunodeficiency (AID)‑driven LPD, smooth muscle tumors (sarcomas including leiomyosarcoma) in line with the clinical development program. A separate observational study (ATA129-EAP-902) was set-up to describe the patient population, tabelecleucel usage, treatment outcomes, and safety in patients with Epstein-Barr Virus driven diseases treated with tabelecleucel under the expanded access program in Europe (see Figure 3). This observational study required additional consent, as it yearly followed-up on the survival status of patients after the start of tabelecleucel – even when patients were no longer under treatment in the expanded access program. Presented at EAN Virtual Congress, 2020 Patients All patients scheduled to be treated with tabelecleucel in the EU EAP that have consented to research Data Collection Limited to what is required for treatment to provide access and a minimum of 2 annual survival follow up Regions Europe: UK, DE, ES, FI, FR, IT, AT, NL, BE, CH, SE, FI, PT, DK Day 28 Assessment Treatment as per Treatment Plan or Clinical Practice Minimum 2 Year Survival Follow Up Study Objectives • Primary: Demographics and Disease Characteristics • Secondary: Outcomes (ORR, OS), Dosing Pattern and Safety Patient Consent for Inventory Check End-of-Treatment and 30-day safety follow-up after last dose Additional Treatment Expanded Access Use Request by HCP Clinical Trial Eligibility Check Triage to clinical trial Patient Consent for Research Inventory Check Patient Consent for Treatment Data Collection Observational Study ATA129-EAP-902 An observational study to describe the patient population, tabelecleucel usage, treatment outcomes, and safety in patients with EBV driven diseases treated with tabelecleucel under the Expanded Access Program in Europe Expanded Access Program Expanded Access Eligibility Check Figure 3: Flowchart for the expanded access program and data collection study for tabelecleucel. Figure adapted from poster publication.56 2 https://www.ema.europa.eu/en/medicines/human/EPAR/ebvallo
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