Understanding the perspective of different stakeholders and their information needs from the earliest stages of the drug life cycle can both enhance and streamline decision-making processes. Early engagement is particularly important for rare diseases because trials are often small due to the rarity of the condition and few patients, which can make study outcomes uncertain.
In response to this need, CADTH is initiating multistakeholder dialogue at key timepoints in the life cycle of drugs for rare diseases. This would better inform the decision-making process and meet the needs of key stakeholders.
The main objectives of this strategy are to develop guidance for multistakeholder dialogue on drugs for rare diseases, define a process for CADTH to engage patients and health care providers in multistakeholder dialogue, and establish processes to leverage the stakeholder input strategies currently in place at CADTH.
The project deliverables include a literature review and multistakeholder meeting reports from learning projects about drugs for rare disease to inform the proposal. These will be used to develop a final multistakeholder dialogue service proposal and guidance document.