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blood cancer treatment becomes first to benefit from canada's drug agency fast-track program
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when the wait time can be anywhere from 12 to 24 months, those living with grave diseases like cancer may not have that kind of time.
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canada has historically lagged behind other countries when it comes to the public reimbursement of approved medications. to address this, canada’s drug agency (cda) introduced the time-limited recommendation program in september, designed to shorten the time between drug approval and patient access.
now, this initiative is set to deliver its first breakthrough with the blood cancer treatment epkinly, making it the first therapy under the program to be fast-tracked for patients with diffuse large b-cell lymphoma (dlbcl).
dr. david macdonald, a hematologist who focuses on treating and developing new treatments for lymphoma and chronic lymphocytic leukemia, sees patients every day who can benefit from the initiative and is excited that something is now being done to improve access to life-changing drugs for blood cancers and beyond.
“introducing this new reimbursement pathway or expedited reimbursement pathway is beneficial to patients that need it, and it’s not putting patients at additional harm because the confirmatory tests are not yet done on these medications,” he said. “so, it’s really going to benefit not just relapsed diffuse large b-cell lymphoma patients, but it’ll benefit any patient for whom a drug is developed that can be subjected to this pathway.”
what is a time-limited recommendation?
a time-limited recommendation allows for the public funding and reimbursement of a drug or treatment for a specific period of time, with the condition that the manufacturer continue to conduct clinical trials to address any gaps in the existing evidence of the treatment’s efficacy. the cda will also review the additional data and reassess the drug, eventually making a final decision on long-term reimbursement. only drugs that meet specific eligibility criteria can qualify for the time-limited recommendation category.
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current drug access pathways
currently, for a drug to get into the hands of patients, it has to go through rigorous testing and approval processes. these methods ensure that any medications available are safe to use and work as intended. however, the process can sometimes take months or even years to complete, leaving those who need those new drugs now out in the cold.
“they look at the clinical data, they look at the situation with that disease and what other medications are available for that disease, so, the unmet need sort of question,” said dr. macdonald. “they receive patient feedback, so patient groups can provide input into the reimbursement review to make the argument that this is a drug that’s really needed in their disease.”
after being developed, clinical data is collected to prove those safety and efficacy requirements, and it’s often not until the third trial that drugs are submitted or approved for use by canadians. the clinical trial process has three phases: the first assesses safety and dosage in a small group of volunteers, the second evaluates the drug’s effectiveness and side effects in a larger patient group, and the third further confirms the drug’s efficacy while monitoring adverse reactions in a broader population.
“the problem lies in the fact that health canada’s notice of compliance with conditions is usually based on phase ii data, but there are many examples where the reimbursement review has recommended not to fund a drug because there is no phase iii data to support its use,” said dr. macdonald.
according to dr. macdonald, this new category of recommendations will allow drugs to be given the go-ahead a lot sooner. when the wait time can be anywhere from 12 to 24 months, those living with grave diseases like cancer may not have that kind of time.
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darren bessette, a loving husband and father of three, was diagnosed with dlbcl for the first time in 2016. after going through the treatment motions, he relapsed again in 2019. he is now free of symptoms after receiving car-t cell therapy, a new therapy that wasn’t officially available at the time he needed it.
luckily for him, he could access it and now advocates for people living with various blood cancers. however, the same cannot be said for many other patients who need different types of up-and-coming therapies.
when asked what would have changed for him if this new reimbursement program was up and running at the time he was diagnosed, he said that he “wouldn’t have had to wait as long” to get the treatment he needed to go into remission.
“i wouldn’t have to go through a trial process. i’d likely would be able to get it more locally instead of having to drive to toronto,” he said. “luckily for me, i had an oncologist who was very knowledgeable and had a lot of connections who were able to get me into this. if i were somewhere else, like if i was living up north where i grew up in north bay, i probably wouldn’t have known this and not have known about this treatment, and who’s to say i would be here right now.”
this goes to show that while some people can still access newer drugs, many canadians are left in the dark, and this new program hopes to shine a light on that.
epkinly, the first drug on the program
the drug epkinly is the first on the program, primarily because of the unmet need for people living with dlbcl.
roughly 3,500 people are diagnosed with dlbcl in canada every year. the disease itself is aggressive and requires prompt treatment. while there are several therapies available, and those with the disease have adequate access to them, about 40 per cent of patients will not respond to the drugs available or relapse and require more treatment with a different drug.
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in many cases, that drug could be epkinly, so putting it on the program for faster access is a saving grace for almost half of those diagnosed with dlbcl.
“things can be happening very quickly, and while there are standard treatments now available for those patients, like autologous stem cell transplant or car-t therapy, not all patients can have access to those treatments, either because their disease is progressing too rapidly (or) possibly related to logistics of getting to a centre that can provide an autologous stem cell transplant or car-t therapy,” said dr. macdonald. “so, for a portion of those patients with refractory diffuse large b-cell lymphoma, a disease that can be behaving very aggressively, there’s a need for drugs that can be accessed quickly.”
bessette, who knows just how emotionally taxing it can be to be diagnosed not only once but twice after a treatment doesn’t take, believes that the new program is a step in the right direction for those that require effective treatments now rather than later.
“at least this gives them (patients) another avenue to pursue that could provide them with the necessary treatments and/or cure that they need in order to progress forward,” he said.
the time-limited recommendation program is not geared toward providing a permanent solution but rather a temporary one that grants access to drugs after gathering enough data from a phase ii clinical trial that shows a drug works and is safe to use.
however, in many cases, that temporary approval will give people access to the drugs they need sooner when the phase iii data will only solidify its place in canada’s collection of medications for hard-to-treat diseases.
spreading awareness for treatment options
according to bessette, one of the most critical aspects of this new program isn’t the drugs themselves but rather spreading awareness of its existence and the medications that can be accessed now because if people don’t know that they have more options, it won’t matter whether or not a drug is approved.
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doctors in canada must follow specific protocols in the system, which often means recommending first-line therapy and going down the list as the medications fail to act. in some cases, they aren’t even able to suggest or tell patients about the newer drugs that are still in trial phases. due to this, patients who are aware of the new drugs are the only ones who can request them.
“it is the patient who has to make the request for these advanced drugs. so, making these drugs more visible to the public so they can make themselves more available, give the patient all the information you need in order to seek them out,” said bessette. “because right now, what i’ve found is that a lot of these new drugs that are potentially available, unless you know where to look and how to find them, you may not see them, and because your doctor can’t suggest them to you based on how the system works now, you may not ever know about them and missed out an opportunity to take advantage of this.”
according to dr. macdonald, the best place to access information about the new program is directly on the time-limited reimbursement page on the canada drug agency’s website. it explains the time-limited reimbursement program and how it will help improve access.
as for finding out which drugs are available, that may take more work. for blood cancer, specifically, bessette suggests going to the leukemia and lymphoma society of canada website for more information on drugs, but patient-centric organizations for other diseases are also more likely to stay on top of these things.
that said, with the new program in place, drug companies will be able to promote their latest products more effectively, which will be very helpful in informing those dealing with debilitating diseases that could be remedied or helped with an up-and-coming medication or therapy.
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