Bulletin #05 Request for Proposal Clarifications File #C-2401280 Rare Disease Registry

In particular, the following will be considered when evaluating the Proposed Objectives and Workplan and the Alignment of Proposal to RFP Objectives sections: • Data quality domains: Does the proposal specify which domains of the data quality enhancement framework are being covered? • Registry gaps and enhancements: Does the proposal identify and describe existing registry gaps or limitations and. [...] Examples: • Enhanced data collection procedures and policies: Implementing standardized data collection forms and procedures to improve the completeness and accuracy of registry data; improving registry policies and governance to become more equipped for HTA analyses. [...] Proposal Evaluation Data quality domains: Does the proposal specify which domains of the data quality enhancement framework are being covered? Registry gaps and enhancements: Does the proposal identify and describe existing registry gaps or limitations and how they will be addressed? Does the proposal demonstrate how the enhancements align with historical regulatory and HTA data requiremen... [...] Scalability: Does the proposal describe and demonstrate the capability of enhancements to lead to sustainable long-term improvements, particularly with reference to the evolving landscape of innovative rare disease therapies and the RWE landscape? RFP Funding Categories We have established standardized funding categories to clarify the expectations and scope of proposal submissions. [...] Examples: Enhanced data collection procedures and policies: Implementing standardized data collection forms and procedures to improve the completeness and accuracy of registry data; improving registry policies and governance to become more equipped for HTA an...