Determine the societal value for the treatment of rare diseases.
Create a priority-setting and decision making framework for expensive drugs for rare diseases.
Develop and initiate an ongoing research agenda related to policy and decision-making for expensive drugs for rare diseases.
Mentor future researchers and clinician scientists on the ethical, moral, clinical, legal, and policy and decision-making implications.
Develop and implement knowledge translation strategies that will facilitate the integration of evidence into policy making, and the evaluation of the decision-making framework, in the short and long-term.