Drugs for Rare Diseases Flashcards
(17 cards)
What is the definition of a rare disease?
Generally viewed as a life-threatening, seriously debilitating, or serious and chronic condition that affects a relatively small number of people (less than 1 in 2000)
What is the general etiology of rare diseases?
80% genetically-based and more than 50% appear in early childhood
What is the prevelance of rare diseases combined?
6000-7000 rare diseases identified worldwide
1 in 12 Canadians affected by a rare disease
What is an orphan drug?
A drug (pharmaceutial or biological) used to treat a rare disease that is typically the first drug treatment for a life-threatening or chronic debilitating disease
How do Canadians access orphan drugs?
Traditionally, access to many orphan drugs has been through the Special Access Program (accessed more often by motivated and well-connected patients and clincians, equity issue)
What percentage of drug costs come from orphan drugs?
10.4% of drug costs in 2020
Recent growth in the number and cost of orphan drugs has raised concerns about financial sustainability
Do third-party payers cover orphan drugs?
Usually not, but when they do offer coverage there are a lot of restrictions (indications, spending caps, large co-pays)
What are some factors that may influence a provincial drug plan to cover an orphan drug?
- Cost of drug
- Drug budget
- Genetic factors that may make some rare diseases more common
Does Canada have robust policies to address issues associated with orphan drug therapy?
No, but most other developed nations have policies to specifically address issues associated with orphan drugs
What are some ways the US supports orphan drug development?
Offers a number of incentives via the Orphan Drug Act
- Targeted research grants
- Tax credits (50% of R&D costs)
- Waiving application fees
- Regulatory assistance to navigate approval process
- Market exclusivity (blocks other from selling the drug for the same indication for 7 years)
What are some concerns about the Orphan Drug Act?
Orphan Drug designation has been given to number of existing medicines to reward companies that would not otherwise see a return on their investment
This has resulted in drug costs for these ODA designated drugs to rise (100x) due to granted market exclusivity
Some question whether the ODA has merely created a new path to excessive profits
Why was a regulatory framework for orphan drugs rejected in Canada?
Due to easy access to orphan drugs developed in other jurisdictions (ex. USA)
What consequences do Canadians with rare diseases face due to the lack of a regulatory framework specifically for orphan drugs?
Orphan drugs are eventually approved in Canada, it just takes on average 6 years longer (delayed access to care)
How have government agencies attempted to improve delays in access to orphan drugs?
The CDR reviews orphan drugs following Health Canada approval under an enhanced process (more specialist engagement, patient input, more dialogue with manufacturers, but no formal distinction)
See slide 33
What is the new National Strategies for Drugs for Rare Diseases (DRD) aka orphan drugs?
In 2023, the first phase was launched
- $1.4 billion through 3-year agreements to improve coverage and access
- Work towards improving screening and diagnosis or rare diseases
- Creating a common list of orphan drugs
- Increase access to eligible First Nations via NIHB
See slide 38 for orphan drugs takeaways
Review slide 41 to answer questions from the orphan drugs study
