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Table 1 Questions submitted to the reimbursement and licensing authorities for scientific advice

From: Aggregated N-of-1 trials for unlicensed medicines for small populations: an assessment of a trial with ephedrine for myasthenia gravis

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?  
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?
  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)?