Module 6: Drugs for Rare Diseases Flashcards
What is an orphan drug? (3)
- A drug (pharmaceutical or biological) used to treat a rare disease.
- Typically, the first drug treatment for a life-threatening or chronic debilitating disease.
- Treatment with DRD are usually life-long and in 2015 was estimated to cost more than $300,000 per patient annually.
How does access to DRDs differ in Canada vs Europe and the USA?
Fewer DRDs approved for use in Canada - around 65% vs. 100% and 84% respectively
Traditionally, access to many orphan drugs has been through Canada’s _______ ______ __________
Special Access Programme (SAP)
What are the concerns about SAP and equity? (2)
- Motivated and well-connected patients and clinicians are more likely to pursue and obtain access through SAP.
- Inequity further exacerbated when access requires significant private financial resources and/or travel to another province to obtain the drug therapy.
Overall, the number of DRDs being approved is __________
increasing
What is the problems with more DRDs?
- The cost of DRD as a share of total drug expenditures in Canada has risen steadily.
– 10.4% of drug costs in 2020 - PMPRB 2022 - More recent growth in the number and cost of DRD has raised concerns about financial sustainability.
Do third-party payers cover DRDs? (3)
- Often not covered by insurers due to a lack of efficacy data and higher costs.
- Coverage often only obtained after media campaigns portraying desperate patients fighting heartless payers/governments. - When coverage is provided, third-party payers often put restrictions on conditions for use, and/or yearly spending caps, and/or large co-pays.
- Coverage for DRD varies across the country due to provincial public plans that decide which drugs are funded.
– Variation associated with:
* The cost of the drug, drug budgets, and genetic factors that are more common in certain communities and locations
– e.g. Fabry’s Disease in Nova Scotia
In 2015, five provinces reported as having specific programs for DRDs. They include Alberta, NB, BC, Ontario, and Sask. What do these programs consist of?
Coverage plans to improve access to a defined set of DRD
- Either as a formulary listing (AB and NB) or a dedicated decision-making process (ON, BC, SK)
Why is Canada a rare exception when it comes to DRD policy?
Most developed countries have specific policies to address issues associated with drug therapies for rare diseases
America and Europe have well established procedures for DRDs. Such as? (3)
- Designating a drug as a DRD
- Regulator support (advising) for drugs and protocol development
- Financial Incentives such as:
- Grants, tax credits, market exclusivity, fee reductions
Many DRDs are not marketed in Canada, and when they are, it takes much longer. How much longer?
~6 additional years until DRD approved in Canada
In 2008, EMA and FDA began to work together to allow applications for an orphan drug ___________ to be submitted __ ________
designation; in parallel
What was the plan in 2012 for Canada to join in on the FDA EMA joint orphan drug framework? (3)
- After 2012, Canadian collaboration with EMA and FDA was anticipated.
- Expected to start with aligning orphan drug designation application processes, and advise to manufacturers on trial design - DRD framework proposed in 2012 in attempt to deal with a ‘patchwork’ policy landscape:
– Ensure more timely access to orphan drugs
– Encourage/facilitate clinical research in rare diseases. - Would use a lifecycle approach that would align its regulations as much as possible with the FDA (USA) and EMA (Europe).
– Criteria for determining rare diseases and orphan drugs.
– Provide support and advise on trial design.
– Criteria for post-market monitoring and risk-management.
What happened in 2017 with the DRD joint framework with the FDA and EMA? (3)
- In 2017, Health Canada removed all references to a planned framework for DRD from its web-site.
- Opts for a review of all drug regulatory policies including those for rare diseases. - No longer pursued a unique DRD framework.
- Use the same review process as other drugs - No longer on a path that would align with FDA and EMA practices.
How did the CDR respond to the DRD joint framework thingy in 2017?
- Federal role only one aspect of the drug assessment process.
- Once a drug is available for sale in Canada, CDR must still review the drug on behalf of public plans. - DRD reviewed by CDR under an enhanced process:
- More engagement of specialists; more patient input, and more opportunities for more dialogue with manufacturers…BUT no formal distinction of DRD compared to a non-DRD.
CDR enhanced framework used when there is an absence of ____________ ___________
alternative treatments
(Majority of DRD decisions based on clinical parameters - most of those approved receive a conditional cost recommendation due to uncertainty of clinical efficacy)
Go though the timeline of the new national strategy for DRDs
(2019, 2021x2, 2023)
- 2019 Federal Budget proposes funding to support patients on a DRD
- Up to $1 billion over two years starting in 2022, with up to $500 million per year ongoing. - In January 2021, Health Canada launches a national online engagement with Canadians to share their views and ideas on next steps.
- Report on public engagement process completed in July 2021.
- Work toward a national strategy continues. - 2023: Launch of the first phase of Canada’s National Strategy for Drugs for Rare Diseases.
- Funding agreements with provinces & territories:
* $1.4 billion through 3-year agreements (April 1, 2024, to March 31, 2027) to provide better coverage and access.
* Work toward improving screening and diagnosis of rare diseases and create a common list of DRD.
- Increase access to eligible First Nations via NIHB. - Lessons learned from agreements and other partner activities to inform future phases.
