ISSUE:

Caregiver engagement in valuing new treatments in rare diseases is limited, and few HTA agencies incorporate caregiver burden when evaluating new treatments.

Is the role of the caregiver considered a vital resource when measuring the value of new treatments for rare diseases? What specific ways can caregivers inform rare disease drug development and approval?

OVERVIEW:

Olivier Chassany will introduce the path to acknowledgment of the value of clinical outcome assessments (COAs) as study endpoints. Studies highlight the caregiver burden, but this burden in all physical, mental, emotional, social, and productivity domains is like an “invisible labor that is not captured”. It is time to value the caregiver’s perspective of health products in HTA.

Selena Daniels will discuss leveraging the use of caregiver input in rare diseases to inform the COA measurement strategy in medical product development and regulatory decision-making.

Terry Jo Bichell will talk about the potential to approve a drug based on the improvement in quality of life (QoL) of the family and whether there is proof that the QoL of the patient has improved. When it is difficult to measure improvements in patients, measuring the indirect improvements on the family could be an alternative way to evaluate biomarkers in the patient.

Laurie Lambert will discuss CADTH’s Learning by Doing Period on optimizing the integration of RWE into decision-making in rare diseases. Exploring how the perspectives of a variety of stakeholders, including caregivers, can be used to assess the value of both drugs and health technologies. CADTH is establishing guidance for the reporting of RWE that will be submitted for the purposes of regulatory and HTA submissions and the generation of RWE. She will discuss the lessons learned from a recently published multistakeholder report concerning the treatment of pediatric cancer.

The audience will interact throughout the session.