Harmonising patient-access programmes: the Dutch DRUG Access Protocol platform

• Published in: The lancet oncology 2022-02, Vol.23 (2), p.198-201
• Main Authors: Zeverijn, Laurien J, van Waalwijk van Doorn-Khosrovani, Sahar Barjesteh, van Roy, Anke A M G Pisters, Timmers, Lonneke, Ly Tran, T H, de Boer, Jolanda E, de Wit, Gijsbrecht F, Geurts, Birgit S, Gelderblom, Hans, Verheul, Henk M W, Blijlevens, Nicole, Wymenga, A N Machteld, Eskens, Ferry A L M, Smit, Egbert F, Bloemendal, Haiko J, Voest, Emile E
• Format: Article
• Language: English
• Subjects: Antineoplastic Agents - therapeutic use; Antitumor agents; Cancer therapies; Clinical trials; Collaboration; Cost analysis; Data collection; Decision making; Drugs; FDA approval; Health insurance; Health Services Accessibility; Humans; Insurance companies; Monoclonal antibodies; Netherlands; Oncology; Patients; PD-1 protein; Pharmaceutical industry; Reimbursement; Safety; Squamous cell carcinoma; Tumors
• ISSN: 1470-2045; 1474-5488
• DOI: 10.1016/S1470-2045(21)00707-5

In Europe, an EAP is often considered an open-label extension study aiming to provide access to a medicine and generate additional safety data, whereas the FDA's definition of an EAP is similar to that of a European Compassionate Use Programme (CUP), which is not designed to obtain additional safety or efficacy data, but instead to provide access to patients in need, pending FDA approval.2 In Europe, CUPs are designed to facilitate early patient access to unauthorised drugs. The EMA's Committee for Medicinal Products for Human Use (CHMP) might also provide recommendations to Member States on how to administer, distribute, and use medicines for CUPs.3 Other access schemes to fulfil the needs of an individual patient are Named Patient Programmes (NPPs) for non-authorised compounds and free-of-charge access for authorised drugs.4 Unlike EAPs and CUPs, there are no strict inclusion criteria and no requirements regarding registration of data on either safety or efficacy. Cemiplimab, a human monoclonal antibody against PD-1, received EMA approval in 2019 after demonstrating promising results in treating cutaneous squamous-cell carcinoma.7 Due to uncertainties regarding its real-world effectiveness and value to the individual patient, however, it received no health-insurance coverage until February, 2021, when it was incorporated in DAP. For authorised indications, we have implemented a uniform personalised reimbursement scheme with a transparent structure in contrast to confidential managed-access agreements. [...]to the best of our knowledge, this is the first national anticancer drug access platform of its kind.