Often medicines are being used in daily practice for situations and patients which are not investigated in the original randomized controlled trials (RCT). Wietske Kievit and Floor Berden have illustrated this paradox in a recent PlosOne paper.
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 researcher 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.
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
Floor Berden Wietske Kievit
Related news items
650,000 Euro funding for research into the phasing out of medication for leukaemia patients30 July 2020
With a 650,000 euro funding from ZonMw, researchers from the Haematology and Pharmacy departments can develop a medication phasing out strategy for patients with chronic myeloid leukaemia. This strategy will be tested in practice.read more
Increased role of patients after bowel cancer treatment30 July 2020
Approximately 14,000 patients get colorectal cancer every year. Almost all patients are operated on and monitored afterwards (follow-up). In 2019 Radboudumc started a new approach to follow-up research after the treatment of stage II/III colorectal cancer.read more
ERC Proof of Concept grant received by Ronald van Rij30 July 2020
Ronald van Rij, theme Infectious diseases and global health, received an ERC (European Research Council) Proof of Concept grant of 150,000 euros, in order to make arbovirus vaccines even safer.read more
Hypatia fellowship Call is open30 July 2020
The Hypatia fellowship round with the deadline 31 May has been canceled. Therefore, the next available deadline will be 27 September 2020. Radboudumc researchers are invited to scout young potentials to fill the strategic gaps within the research themes imbedded in RIHS and RIMLS.read more
Mihai Netea and colleagues published two papers back-to-back in The Journal of Clinical Investigation.29 July 2020
These back-to-back articles investigated the effect of BCG vaccination on trained immunity. The first article shows that BCG vaccination inhibits systemic inflammation, depending on gender. The second article demonstrates that the circadian rhythm influences the induction of trained immunity.read more
Genetic mutation reveals how coronavirus strikes TLR7 plays essential role in disease process28 July 2020
''Does a congenital immune defect play an important role in the defense against Coronavirus?'' This was published by Cas van der Made, Frank van der Veerdonk and Alexander Hoischen.read more