Takeda Releases New Report to Support Health System Readiness for Rare Diseases in Canada

• Globally over 1,100 novel drugs for rare diseases are in developmentⁱ, with nearly 200 expected to launch in Canada within the next 10 years.ⁱⁱThe impact of rare diseases is significant with approximately one in 12 Canadians, affected – two-thirds of whom are children.ⁱⁱⁱ

TORONTO, Aug. 22, 2024 /CNW/ - Today, Takeda Canada Inc ("Takeda") released its Enhancing Diagnosis, Access, Care and Treatment: Recommendations for Health System Readiness for Rare Disease in Canada report. This report found that with the rapid innovations in novel drugs for rare diseases (DRDs), there are over 1,100 DRDs currently in development globally^iv, with nearly 200 expected to launch in Canada within the next 10 years.^v The report examines several key factors that should be considered to ensure Canada's healthcare system is adequately prepared for this anticipated influx of DRDs.

The report assesses the development pipeline for DRDs, outlines the associated implications for the Canadian healthcare system, and highlights the urgent need for innovative funding models and collaboration that will help accelerate Canada's National Strategy for Drugs for Rare Diseases. 

The report identifies six key recommendations to be considered to ensure Canada's healthcare system is adequately prepared for this anticipated influx of DRDs:

1. The Need for Medical Education:  While education is essential for all rare diseases, particular attention should be given to first-in-class DRDs, which offer novel treatment opportunities. Education for physicians and other healthcare providers will be key to ensure these treatments reach patients.
2. The Role of Genetic Testing: Focus on an equitable pan-Canadian approach to investment in, and implementation of, genetic testing across Canada.
3. Delayed Diagnosis and Access Challenges: Investment and policy development that reduces barriers for those living in rural or underpopulated regions is imperative to facilitate healthcare access for all Canadians affected by rare diseases.
4. Address Infrastructure Challenges: As Canada prepares for an influx of DRDs, the demand for cold chain storage and transport may rise. This critical infrastructure requires investment, policy work, and the development of protocols to prevent medication waste.
5. Adopt Innovative Funding Models and Increase Collaboration: Collaboration will be key to ensuring access to treatments for patients with rare diseases. Innovative funding models, including amortization over time, outcome-based agreements (OBAs), and modifying heath technology assessment (HTA) requirements, will be important in ensuring patients can access treatment.
6. Patient Engagement and Partnership: Patient participation is crucial to ensuring that appropriate care reaches patients. Collaborating with patient groups throughout the healthcare planning process is vital to meeting the needs of patients with rare diseases.

The report leveraged the IQVIA^TM Pipeline Intelligence database to identify DRDs in the drug development pipeline. The Pipeline Intelligence database is a comprehensive source of drug information that provides the most up-to-date research and development insights across the entire drug development lifecycle, from early-stage research to commercialization.

"Innovation is at the forefront of how we serve the needs of Canadian patients. The global scale of DRD development is encouraging and hopeful," says Dr. Jefferson Tea, Vice President Medical and Scientific Affairs at Takeda. "We are proud to present this report to help health policy makers across Canada strategically prepare for the introduction of new DRDs so that patients and families living with rare diseases have fewer barriers in accessing these important treatments when they become available. Takeda will continue to invest in research and medical discoveries to support and benefit Canada's rare disease communities."

To view the report, click here.

Takeda is committed to driving forward rare disease policy in Canada and seeing effective implementation of these policies for the benefit of all Canadians. Takeda has released a series of reports looking at rare disease policy both in Canada and globally including a 2022 report Defining Rare Disease in Canada: Lessons from G20 Nations and a 2021 report Strategies for Rare Diseases: International Landscape Report.

Quotes

"Canadians living with rare diseases have every reason to be optimistic," says Durhane Wong-Rieger, President & CEO, Canadian Organization for Rare Disorders (CORD). "Hundreds of new therapies are being developed, many targeting the 95% of rare diseases with no known treatment!  We must leverage the $1.5 billion Rare Disease Drug Strategy, being implemented across the provinces, to invest in timely diagnosis, specialists, and comprehensive care in addition to drug funding to ensure optimal patient outcomes."

"Many Canadians with rare blood disorders require life-saving medications, and they cannot afford to see delays in accessing current or new treatments," says Jennifer van Gennip, Executive Director, Network of Rare Blood Disorder Organizations (NRBDO). "We are pleased to see this report as it provides relevant data and important recommendations to help Canada's healthcare system be prepared to provide timely and equitable access to new treatments that will benefit people with rare blood disorders and other rare diseases."

"Innovation is at the forefront of how we serve the needs of Canadian patients. The global scale of DRD development is encouraging and hopeful," says Dr. Jefferson Tea, Vice President Medical and Scientific Affairs at Takeda. "We are proud to present this report to help health policy makers across Canada strategically prepare for the introduction of new DRDs so that patients and families living with rare diseases have fewer barriers in accessing these important treatments when they become available. Takeda will continue to invest in research and medical discoveries to support and benefit Canada's rare disease communities."

Rare Disease Impact in Canada

A "rare" disease is any disease that affects a very small number of individuals. It is often genetic, chronic throughout a patient's life and life-threatening.^vi With rare diseases affecting a relatively small set of patients, innovative treatments are often unavailable. It's estimated only 5% of rare diseases have an approved treatment.^{vii, viii} This reality has a distressing effect on patients and their families: studies suggest that at least two-thirds of rare diseases affect children^ix, 30% of whom die before their fifth birthday.^x, xi, xii

The journey toward appropriately managing a rare disease is long and challenging. On average, it takes 6-8 years before a patient receives a correct diagnosis; in this time, a patient will see an average of eight physicians and receive two to three misdiagnoses.^xiii The treatment delays caused by these diagnostic challenges often result in avoidable disease progression, which only amplifies the effect of the diseases on the patient and their caregivers.^xiv

About Takeda Canada Inc.

Takeda Canada Inc. is the Canadian organization of Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK), a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discovering and delivering life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). 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. For more information, visit: https://www.takeda.com/en-ca/

______________________________________

i	IQVIA Pipeline Intelligence. 2023.
ii	Ibid.
iii	Canadian Organization for Rare Disorders, "Now is the time: A strategy for rare diseases is a strategy for all Canadians," 2015.
iv	IQVIA Pipeline Intelligence. 2023.
v	Ibid.
vi	Critical Care Services Ontario. RARE DISEASES WORKING GROUP REPORT. March 10, 2017. [Cited 2022 Apr 6]. Available from: RARE DISEASES WORKING GROUP REPORT (gov.on.ca)
vii	National Organization for Rare Disorders. Rare Disease Day. Frequently Asked Questions. [Cited 2022 Apr 6] Available from: https://rarediseases.org/wp-content/uploads/2019/01/RDD-FAQ-2019.pdf
viii	Clarke, S., Ellis, M. & Brownrigg, J. The impact of rarity in NICE's health technology appraisals. Orphanet J Rare Dis 16, 218 (2021). [Cited 2022 Apr 6]. Available from: https://doi.org/10.1186/s13023-021-01845-x
ix	Canadian Organization for Rare Disorders, "Now is the time: A strategy for rare diseases is a strategy for all Canadians," 2015.
x	EDITORIAL. Spotlight on rare diseases. The Lancet Diabetes & Endocrinology. | VOLUME 7, ISSUE 2, P75, FEBRUARY 01, 2019. DOI: https://doi.org/10.1016/S2213-8587(19)30006-3
xi	Batshaw ML, Groft SC, Krischer JP. Research into rare diseases of childhood. JAMA. 2014;311(17):1729-1730. Available from: 10.1001/jama.2013.285873
xii	The United Kingdom Department of Health & Social Welfare. The UK Rare Diseases Framework. January 9, 2021. [Cited 2022 Apr 6]. Available from: https://apcp.csp.org.uk/news/2021-07-29-uk-rare-diseases-framework
xiii	Shire. Rare Disease Impact Report: Insights from patients and the medical community. April 2013. [Cited Apr 6]. Available from: https://globalgenes.org/wp-content/uploads/2013/04/ShireReport-1.pdf
xiv	Sawyer SL et al. Utility of whole-exome sequencing for those near the end of the diagnostic odyssey: time to address gaps in care. Clin Genet. 2016 Mar;89(3):275-84. doi: 10.1111/cge.12654. [Cited 2022 Apr 6] Available from : https://pubmed.ncbi.nlm.nih.gov/26283276/

SOURCE Takeda Canada Inc.

Media Contacts: Amanda Jacobs, [email protected], [phone]