New drugs available more quickly for the right patient

Kievit, Wietske

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. This leads to the paradox that medicines are being used in daily practice for situations and patients which are not investigated in the original trials.

Senior research Wietske Kievit (department for Health Evidence) and PhD candidate Floor Berden (Gastroenterology and Hepatology) (photo below) have illustrated this paradox in a recent paper published in PlosOne. They researched the situation with protease inhibitors telaprevir and boceprevir for hepatitis C patients. Nearly half of real world hepatitis C patients would have been excluded from registration trials, and these patients developed more serious adverse events than eligible patients .

Better research
In a publication in the journal ‘Nederlands Tijdschrift voor Geneeskunde (NTvG)’Kievit and Berden 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.

PlosOne: Limited Generalizability of Registration Trials in Hepatitis C: A Nationwide Cohort Study
NTvG: Nieuwe geneesmiddelen sneller beschikbaar voor juiste patiënt
Te streng deurbeleid registratiestudies


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