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Is Equitable Access for Rare Disease Drugs Within Sight?

A doctor holding a patient's hand
A doctor holding a patient's hand

Durhane Wong-Rieger

President & CEO,
Canadian Organization for Rare Disorders

I believe we all support the principle that every patient should get access to the medicine that best meets their needs. We all probably also agree on two key issues: the importance of finding the right drug for the right person and determining the right amount that should be paid for each drug. These challenges are especially acute for so-called “orphan” drugs, as developing a drug for a small and little-known disease group takes a lot of time and money.

The good news is that there is a strategy that can not only promote equitable access by directing the right patient to the right drug, but can also promote cost-effective drug expenditure by assuring outcome-based payment. This strategy encompasses two components: managed access and risk-sharing. Managed access acknowledges that orphan drugs are often the first therapy to address a specific life-threatening or progressively debilitating disease — because of urgent need, these drugs may be approved without absolute certainty as to how well they will work for each patient.

A managed access plan defines the criteria for access based on available evidence and sets up a monitoring program to collect additional, real-world evidence. The risk-sharing component of the strategy calls upon the drug developer to share the financial risk of treating patients in a monitored setting while additional evidence is being collected.

The very good news is that we know this strategy works because we have been employing it in Canada for over a decade. For selective cancer and rare disease drugs, patients have been getting access to promising therapies in clinic settings where payment is based on a risk-sharing agreement. So why hasn’t Canada implemented this strategy for all orphan drugs?

The (potentially) very, very good news is that the Provincial/Territorial Expensive Drugs for Rare Diseases working group, set up in Sept. 2014, just proposed a supplemental process for complex and specialty drugs, including those for rare diseases, in October of this year. This process includes expedited reviews through Health Canada and the Canadian Agency for Drugs and Technologies in Health and has, at its core, both managed access and risk-sharing components.

Implementation of the process is expected next spring. It is too bad that the Spinal Muscular Atrophy patients didn’t have this in place sooner — it would have saved a lot of hardship.

Nevertheless, as we continue to fight for patient access to rare disease drugs, we will remain steadfast in believing advocacy works, partnership rules, and miracles do happen.

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