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

Abstract

Over the past decades, many new targeted anticancer drugs have been introduced, and many more are expected to follow. Many of these drugs are only being tested in non-randomised studies, as they target small and newly defined patient populations. This approach is concerning, given that non-randomised studies are susceptible to bias and confounding and there is no consensus on critical appraisal tools or review methodologies. 1 Quigley JM Thompson JC Halfpenny NJ Scott DA Critical appraisal of nonrandomized studies—a review of recommended and commonly used tools. J Eval Clin Pract. 2019; 25: 44-52 Google Scholar Health-care authorities and payers, therefore, often raise concerns about the clinical effectiveness and cost-effectiveness of these therapies, which leads to lengthy dossier evaluation and price negotiations. The DRUG Access Protocol: access inequality and European harmonisationWe commend the initiators of the DRUG Access Protocol (DAP) for their efforts to combine earlier access to medicine with structured data collection.1 Although this is a novel programme in the Netherlands, similar programmes covering compassionate use, evidence generation, and reimbursement are already in effect in England (Early Access to Medicines Scheme) and France (L'Accès Précoce).2,3 The benefits of the DAP in providing conditional reimbursement of registered drugs and thereby creating access are evident. Full-Text PDF The DRUG Access Protocol: access inequality and European harmonisation – Authors' replyWe would like to respond to the comments of Tobias Polak and colleagues on the establishment of the DRUG Access Protocol (DAP) in the Netherlands.1 First, we fully agree that there is a clear, unmet need to systematically collect data and prevent delayed and unequal access to effective drugs; these were the most important reasons for initiating DAP. Therefore, the concerns of Polak and colleagues surprised us. First, DAP is indeed a national solution, simply because each country has its own system for evaluating drugs and reimbursement. Full-Text PDF