Patient support programs essential in rare diseases
Jonathan Soong says the Canadian healthcare system struggles to address individual needs of patients with rare diseases (Issue #324, 1,210 words, 6 minutes)
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The landscape for Canadian rare disease treatment is challenging, according to Jonathan Soong. On an episode of the NPC Podcast, he highlighted the lack of specific legislation or designation for rare diseases in Canada, which can delay or prevent treatments from reaching patients. He is currently the commercial vice president of BioScript Solutions.
Soong pointed out that the Canadian healthcare system is designed for broad populations and has problems addressing the individual needs of rare disease patients. “On a positive note, there is some promise of progress,” he said. “Rare disease funding was first announced in 2019 and re-announced in 2023 with a commitment to support rare disease therapies and patients. It is encouraging to see the recognition of the resources gap, and I’m hopeful for the future.”
According to Soong [pictured below], a core aspect of addressing these challenges is ensuring that patient voices shape clinical trial design and that patient input is integrated throughout the process. “It often begins with patient roundtable discussions and advisory boards to ensure the relevance of projects and activities at the start of a clinical program,” he said. “Insights are gathered on the burden of disease and the patient journey.” These discussions help researchers understand the disease burden and patient journey and identify the outcomes to be measured with the patient’s priorities in mind. Soong emphasized that this collaboration extends beyond trial design—the findings are shared with patient organizations to maintain transparency and engagement.
Beyond drug development, Soong discussed the broader issue of access, noting that regulatory approval alone does not guarantee that a therapy is available for patients. “All the research, good work and even regulatory approvals are meaningless if people can’t access the medicines,” he said.
He stressed that the high cost of rare disease treatments presents a societal challenge, as these conditions often stem from genetic mutations, not lifestyle factors. He noted that Amicus works closely with public health systems and private insurers, working on sustainable solutions that balance treatment value with affordability to improve accessibility. Soong also acknowledged the complexities of this process, emphasizing the need for creative approaches to ensure patients can receive their therapies without undue financial burden.
Patient support programs also play a critical role in addressing access barriers. Soong noted that a key element in advancing rare disease care is using real-world evidence (RWE). He explained that RWE is even more important in rare diseases, given how individualized the therapies and responses can be and the low patient numbers. He added that traditional clinical trials often lack comprehensive long-term data due to the limited patient populations for many rare conditions. RWE, derived from patient registries, electronic health records, and real-world treatment experiences, helps fill these gaps by providing insights into treatment effectiveness and safety over time. He emphasized that integrating RWE into regulatory and reimbursement decisions can enhance access and ensure that rare disease patients benefit from ongoing medical advancements.
THIS WEEK 03/25/25
The U.S. FDA has approved Celltrion’s omalizumab-igec (Omlyclo) as a biosimilar designated as interchangeable with omalizumab (Xolair) for the treatment of moderate to severe persistent asthma, chronic rhinosinusitis with nasal polyps, Immunoglobulin E (IgE)-mediated food allergy, and chronic spontaneous urticaria.
Biogen Canada announced that Health Canada has approved omaveloxolone (Skyclarys) for treating Friedreich’s ataxia in patients 16 and older.
Kye Pharmaceuticals’ ferric maltol (AccruFeR) is now available across Canada to treat adults with iron deficiency anemia who are unresponsive or intolerant to other oral iron formulations.
Health Canada has approved Acutis’ roflumilast cream 0.15% (PrZoryve) for treating mild to moderate atopic dermatitis.
Health Canada has issued a Notice of Compliance for Johnson & Johnson’s lazertinib (Lazcluze) in combination with amivantamab (Rybrevant) for the first-line treatment of adult patients with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 19 deletions or exon 21 L858R substitution mutations. Janssen Inc., a Johnson & Johnson company, is the market authorization holder for Lazcluze and Rybrevant in Canada.
NOW LISTEN UP: HERE’S LISA MULLETT OF SUMITOMO PHARMA
In season 13 of the NPC Podcast, Paul Levesque, President and CEO of Theratechnologies Inc., gives insights into biopharma innovation, tariffs' effects on supply chain and pharma manufacturing, and cross-border business development. Hear him in conversation with podcast hosts Mitch Shannon, Jim Shea, and Mark McElwain.
HEALTHBIZ REWIND
This feature of NPC Healthbiz Weekly looks back at some of the most insightful moments from previous NPC Podcast episodes. You can listen to the podcast here.
Jaclyn Katelnikoff
Clinical Pharmacist
Saskatoon Family Pharmacy
Season 12, Episode 06
Listen to this episode here
(This episode was recorded in May 2024)
What is the most fulfilling part of your role as a clinical pharmacist?
Well, it’s really about helping people. I have so many examples. This weekend, a young gentleman came in, someone I'd interacted with before, dropping off a prescription for his brother. He told me he had a terrible experience at the hospital’s emergency room when getting the prescription. I asked what happened, and he said the doctor told them, ‘I don’t help people like you.’ I was shocked—such a horrifying thing to hear. This family is trying to help their brother with addiction, and he’s trying to get help for himself.
I spent about an hour with this gentleman, trying to assist with his brother's situation. He mentioned that his brother admitted to being depressed, and I suggested, ‘Maybe that's where you start—focus on the mental health part first, and the addiction part can follow.’ He seemed to appreciate that insight. Then, I learned they were struggling financially. I told him there was funding available for medications, and I gave him the application form. He nearly broke down in tears. But to me, I wasn’t doing anything extraordinary. I was helping a person on the other side of the counter. Sometimes, a few simple things you say can make a big difference, and that’s truly why I love my job.
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