BMC’s Submission on the National Strategy for Drugs for Rare Diseases — Additional Considerations (A

Principles and pillars: Entrenching fit-for-purpose and patient benefit • A comprehensive and effective strategy must first and foremost focus on saving and transforming patients’ lives. [...] • While the principles and strategic pillars presented have merit, the central and fundamental concept of improved patient outcomes – saving and transforming the lives of patients – is not given the prominence necessary to truly guide the framework and its elements. [...] • Processes like Health Canada’s Priority Review and the aligned review process with the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d’excellence en santé et en services sociaux (INESSS) are helpful in moving the dial, but we also need the pan-Canadian Pharmaceutical Alliance (pCPA) and private payers to work in step with these processes, and there is no. [...] Regarding ensuring a safe and secure supply, the framework as proposed ignores the need for drug and vaccine development and manufacturing inside Canada and the need for investments to build capacities. [...] 6 www.bestmedicinescoalition.org About the Best Medicines Coalition The Best Medicines Coalition (BMC) is a national alliance of patient organizations which seeks timely access to a comprehensive range of medically necessary, safe, and effective drugs and other treatments, informed by patient-driven evidence and values, and delivered equitably and affordably to all patients in Canada.