Table 1 Questions submitted to the reimbursement and licensing authorities for scientific advice
National Health Care Institute | Medicines Evaluation Board |
|---|---|
Could the aggregated data from the series of N-of-1 trials, as presented in the briefing document, play a role within the framework of a reimbursement advice, in case a medical professional society requests such an advice at a later date? | Are the data in the briefing document sufficient for a benefit/risk evaluation (for licencing) and if not, what else is required? |
If so, in what way could it play a role and what might the reimbursement advice be? | Â |
If not, why not? What level of precision in the data is needed for a reimbursement advice at the population level? | In case the level of precision in the data is insufficient for a benefit/risk evaluation (for licensing), how many more patients would need to be included in the aggregated N-of-1 trial to enable MEB to make a judgement? What specific outcome or type of analysis would be recommended as the basis of a new sample size calculation? |
| Â | What would be the recommended regulatory route to get this indication on-label? Which would be the preferred route: ephedrine tablets imported from Spain or a possible future product to be compounded by a Dutch GMP-certified pharmacy (LUMC)? |