Nieuwe geneesmiddelen sneller beschikbaar voor juiste patiënt

Pharmaceutical companies are under increasing scrutiny because of their strategy for gaining market access and reimbursement authorisation for novel drugs. The tool most often used is that of a randomised controlled trial (RCT) in a highly selected population that has a high chance of responding on the treatment but a low chance of developing side effects. This population differs to a large extent from real-life patients, who have diverging characteristics that can influence effectiveness and safety; these include co-morbidity, age and disease severity. The ultimate consequence is that evidence resulting from RCTs is not immediately transferable to clinical practice. This paper illustrates examples of drugs developed for rheumatoid arthritis and hepatitis C. We discuss research designs that can complement findings from RCTs, such as pragmatic trials, enriched trials, adaptive pathways, early access programs and patient registries. The aim is to stimulate debate among different stakeholders so that they answer the right question at the right time using a suitable research methodology.

Conflict of interest and financial support for this article: J.P.H. Drenth has received several research grants, honoraria and other payments, including those from Celltrion. ICMJE forms provided by the authors are available online along with the full text of this article.