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Canada’s National Strategy for Drugs for Rare Diseases Needs a Holistic Approach

December 8, 2021

International Report Summarizes Drugs for Rare Disease Best Practices and Recommendations

TORONTO, ON – December 8, 2021 –– Takeda Canada Inc. has released a Report on the global rare diseases policy landscape at the Forum de l'Industrie de la Santé de Québec (FISQ). Takeda’s longstanding commitment to Drugs for Rare Diseases (DRD) and extensive global footprint provides a unique opportunity to assess and summarize best practices on how different countries have developed their own tailored approaches to expediting and expanding access to new treatments for rare diseases.

Many jurisdictions have adopted a holistic approach to treating rare diseases – one that embeds a set of specific protocols and processes within an overarching policy framework that incorporates multiple measures to support people with rare disease needs.1 These countries recognize that drugs for rare diseases are only one essential part of a robust rare disease strategy. The strategy must also address improved screening, timely diagnosis, data collection and patient and caregiver support.

Rute Fernandes, Takeda Canada’s General Manager, announced the release of the Strategies for Rare Diseases: International Landscape Report at the Forum de l'Industrie de la Santé de Québec, where she was invited to give a presentation on best practices in rare disease strategies.   

“In anticipation of Health Canada’s consultation on a National Strategy for Drugs for Rare Diseases, we engaged a wide range of experts from across Takeda’s network of international affiliates to provide a foundational understanding of multiple policy approaches,” explained Rute Fernandes, General Manager, Takeda Canada. “Canada has the unique opportunity to learn from other jurisdictions to create real and meaningful change for Canadians living with rare diseases. Takeda is proud to partner and collaborate on the urgent work the Federal and Provincial/Territorial governments are doing in building their rare disease strategies.”  

In analyzing practices from 16 comparator countries, seven key elements were identified as instrumental to the success of a comprehensive national rare disease strategy, including:

  1. Identifying an Objective and Harmonized Definition of “Rare Disease”
  2. Incorporating DRDs into a Holistic Rare Disease Strategy
  3. Reflecting Disease Rarity in Market Exclusivity & Investment Support
  4. Creating Accelerated Regulatory and Early Access Pathways
  5. Maximizing Shared Value Through HTA, Pricing and Reimbursement
  6. Leveraging the Benefits of Accessible Data Collection, Diagnostic Screening, and Patient Registries
  7. Improving and Extending Networks of Researchers, Clinicians and Patients

To read the full report, visit: takeda.com/en-ca/newsroom/.

About Takeda Canada Inc.

Takeda Canada Inc. is the Canadian marketing and sales organization of Takeda Pharmaceutical Company Limited, headquartered in Japan. Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Rare Diseases, Oncology, Gastroenterology (GI), and Neuroscience. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

Additional information about Takeda Canada is available at www.takeda.com/en-ca


Media Contact: Amanda Jacobs, Amanda.Jacobs@takeda.com, +1-647-798-2231.


1 EUROPLAN Project. http://www.europlanproject.eu/CapacityBuilding?idCapacityBuilding=5&idCapacityBuildingChild=9