The FDA believes that:
- because of the rarity of the disease or condition, it is generally infeasible or highly resource or time intensive to generate extensive clinical evidence premarket; and
- there is an unmet medical need that is addressed by the device, such as there are no available therapeutics or diagnostics for that patient population.
This approach could be applied when the device is not eligible for the Breakthrough Device Program or the HDE pathway. This would give the sponsors of eligible devices options for how they could meet the reasonable assurance of safety and effectiveness standard where greater uncertainty may be appropriate under the circumstances (e.g., smaller premarket data collection with larger postmarket data collection and other postmarket controls, greater premarket data collection with smaller postmarket data collection and no or fewer other postmarket controls, or even greater premarket data collection with no or little postmarket data collection and other postmarket controls). While there is not a specific number of patients that would be considered a “small patient population,” this approach could be used for patients with a rare disease or condition or for patients within a clinically meaningful subset of a broader population. Sponsors may submit a Q-submission to discuss with the appropriate review team.