1594P Harmonising patient-access programmes

Abstract

In oncology, there is a great need for timely access to novel anticancer drugs. When encouraging clinical data are available, pharmaceutical companies often initiate ‘Expanded Access Programmes’ (EAP) or Compassionate Use Programmes (CUP). In addition, health care professionals do regularly request access to these drugs for individual patients as part of Named Patient Programmes (NPP). This fragmented off-label use of anticancer drugs or experimental compounds can lead to individual opinions on efficacy rather than solid data. To address this, we propose that patient-access programmes should be harmonised on a national level under supervision of medical societies. We assume that by coordinating all the programmes we can not only facilitate a fast and efficient access to novel anticancer drugs, but we can also pursue research in order to obtain additional data on safety and effectiveness in a real-world setting. Such data are valuable for both healthcare professionals (e.g. for clinical judgement and guideline development) and payers and national health authorities (e.g. for pharmaco-economic evaluations). In addition, the market authorisation holders can use these data to address regulatory requirements or develop local or global launch strategies. In the Netherlands, we have recently implemented a national programme for regulated patient-access in oncology and haemato-oncology, known as DRUP-Access. DRUP-Access is an addition to the existing national study platform, the so-called DRUP* trial. DRUP-Access platform can also be used in the post-launch period, if additional data collection is required; for instance when the dossier evaluation by the payers or Health Technology Assessment (HTA) bodies has led to a negative reimbursement decision due to insufficient clinical data. By integrating all peri-approval programmes into this platform, we ensure a structured data collection that not only contributes to a better understanding of how novel anticancer drugs perform in real life but also results in a faster and more substantiate reimbursement by healthcare authorities.