Implementation of n=1 trial methodology in the assessment of health care reimbursement (WC2014-003)


Starting date: 01/01/2014

A previous project explored the need for n-of-one trials and what preconditions are necessary, according to stakeholders involved in medical care as well as assessment of medications. The focus was on evaluating treatments for rare diseases. Systematic literature review addressed two indications suitable for n-of-one trials, 3,4-diaminopyridine and ephedrine for myasthenia gravis. A clinical protocol for an n-of-one trial was developed. Both the project results and more recent developments show that the n-of-one design is a necessary addition to the current set of designs used for regulatory decisions on reimbursement and/or marketing authorization of (off-label) medications for rare diseases.

The current implementation project aims to deepen insight in the usefulness of n-of-one trials for regulatory decisions. This is in line with the Dutch Health Care Insurance Board’s (CVZ) development of policy for obtaining ‘fitting’ evidence via a ‘feasible information trajectory’ in situations where classic randomized controlled trials are not (financially) feasible (Heymans et al., 2013).