By the Numbers: Rare Diseases and Conditions

July 16, 2021

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A rare disease is, by definition, uncommon. Taken collectively, however, rare diseases touch millions of Canadians—which means that timely and equitable access to the right treatments can have an enormous impact on Canadian lives.

Rare disease realities

7,000: the approximate number of rare diseases identified in the world to date.1

80%: the proportion of rare diseases that have a genetic origin.1

One in 12: The estimated proportion of Canadians who live with a rare disease.1

50%: Proportion of rare diseases that begin in early childhood and involve progressive, life-threatening deterioration.1 In fact, rare diseases account for 30-40% of neonatal deaths.2

5%: Approximate percentage of rare diseases with a currently available treatment.2

7: Number of curative therapies for rare diseases and conditions, 3 of which 3 are currently available in Canada.4


The rare drug market

40%: percentage of pipeline drugs in pre-registration phase that target rare diseases.5

300: Approximate number of cell and gene therapies in development, collectively targeting more than 100 diseases.6

32%: Growth of the Canadian market for expensive drugs for rare diseases (EDRD) from 2012 to 2019—more than 6 times the growth rate for all prescription drugs in Canada.7

9.4%: Share of total drug-spend in Canada devoted to expensive drugs for rare diseases (EDRD), including oncology and non-oncology medications.7

$2.1M: Approximate cost of Zolgensma, a medication for spinal muscular atrophy, approved in Canada in 2020 and dubbed the “most expensive treatment in the world.”8,4

$200,000: minimum annual cost of 76% of all EDRD approved in Canada, as of 2019.9


Access status and setbacks

50%: Approximate proportion of EU-approved drugs for orphan diseases (neglected rare diseases) that end up approved in Canada.10

18-24 months: Delay between Health Canada approval and public reimbursement of orphan-disease drugs.10

99%: Price discount that would be required if applying new Patented Medicine Prices Review Board (PMPRB) regulations to two new cystic fibrosis drugs.11,12

26: Number of European Union member countries (out of 28) who have their own framework for reviewing rare disease drugs.13

$500M: Funds earmarked in the Canadian government’s 2019 federal budget to a program for rare diseases.14

0: Frameworks for rare diseases in Canada. This may change with the government’s recently released consultation document on “Building a National Strategy for High-Cost Drugs for Rare Diseases.”9


References

  1. About drugs and rare diseases in Canada. Health Canada. Last Modified Aug. 14, 2018. https://www.canada.ca/en/health-canada/services/licences-authorizations-registrations-drug-health-products/regulatory-approach-drugs-rare-diseases/about-drugs-rare-diseases.html

  2. Jump-start planning and implementation of rare drug strategy. Webinar 1: Every 18 minutes. Canadian Association of Rare Diseases. May 20, 2021. https://www.youtube.com/watch?v=2tU-gdPPPKw

  3. Maldonado R et al. Curative gene therapies for rare diseases (Table 3). J Commun Genetics 2021;12:267.

  4. 20Sense original research.

  5. Meds pipeline monitor 2020. Patented Medicine Prices Review Board. https://www.canada.ca/content/dam/pmprb-cepmb/documents/npduis/analytical-studies/meds-pipeline-monitor/2020/MPM-2020-en.pdf

  6. The Inside Story June/July 2019. Greenshields Canada. https://bit.ly/3B8Atvk

  7. Patented Medicine Prices Review Board Annual Report 2019. https://www.canada.ca/content/dam/pmprb-cepmb/documents/reports-and-studies/annual-report/2019/pmprb-ar-2019-en.pdf

  8. Successful market access for gene therapies—strategic challenges and possible solutions (white paper). SKC Beratungsgesellschaft 2020. https://skc-beratung.de/whitepaper/GentherapieWhitepaper_ENG.pdf

  9. Building a national strategy for high-cost drugs for rare diseases. Government of Canada. January 2021. https://www.canada.ca/content/dam/hc-sc/documents/services/health-related-consultation/National-Strategy-High-Cost-Drugs-eng.pdf

  10. Benchmarking study: access to therapies for rare disease. Novartis. April 15, 2021.

  11. Cystic Fibrosis Canada calls on prime minister and health committee to investigate patented medicine prices review board. cystic fibrosis canada. June 4, 2021. https://www.cysticfibrosis.ca/news/cystic-fibrosis-canada-calls-on-prime-minister-and-health-committee-to-investigate-patented-medicine-prices-review-board

  12. House of Commons Standing Committee on Health. Friday, Jun 4, 2021. Government of Canada. https://bit.ly/3kmg7ZE

  13. Benchmarking study on public policies for rare diseases. Montreal InVivo. https://www.montreal-invivo.com/wp-content/uploads/2021/03/rare-diseases-synopsis-mars-2021-final.pdf

  14. Letter to the Canadian Government. Canadian Association of Rare Diseases. April 26, 2021. http://www.raredisorders.ca/content/uploads/CORD-PM-MOH-Rare-Drug-Strategy-0422021v2.pdf

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Rare Disease Update: Status, Strides, Setbacks