Beyond clinical trial data: The emerging role of real-world evidence in drug approval
Canada is aligning with global trends and incorporating RWE into our Rx approval process (820 words, 4 minutes)
Regulatory agencies increasingly recognize the importance of real-world evidence (RWE) in evaluating and approving new pharmaceuticals, according to research published by the International Journal of Environmental Research and Public Health. This trend represents a departure from the exclusive reliance on randomized controlled trials and emphasizes the need to consider data derived from everyday clinical practice.
JCO Clinical Cancer Informatics notes that while clinical trial data is reliable, it sometimes falls short of capturing the complete picture of a drug’s performance in real-world settings. It’s estimated that up to 50% of trials are incomplete because of insufficient enrollment, making real-world evidence sourced from electronic health records, claims databases, and patient registries even more valuable. Pharmaceutical Medicine notes that RWE offers a more comprehensive understanding of a drug’s safety profile, efficacy, and use patterns.
Canada is aligning with this global trend and has incorporated RWE into its drug approval process, recognizing its potential to enhance decision-making, according to the feds. The University of Alberta also notes that Health Canada has been actively working to integrate RWE at various stages of drug development and evaluation.
One significant application of RWE in Canada is in the post-market surveillance of drugs. Ottawa notes that Health Canada collects and evaluates reports of suspected adverse reactions after products are approved for sale in Canada. This means that RWE plays a crucial role in assessing the effectiveness and value of new drugs in the Canadian healthcare system.
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THIS WEEK 11/28/23
The U.S. FDA approved Evive Biotech and Acrotech Biopharma’s efbemalenograstim alfa (Ryzneuta) for chemotherapy-induced neutropenia in adult patients with non-myeloid malignancies.
Jazz Pharmaceuticals received Health Canada approval for a cannabidiol oral solution (Epidiolex) for treating seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex in patients two years and older.
Vertex Pharmaceuticals announced that the European Commission approved the label expansion of their ivacaftor/tezacaftor/elexacaftor product (Kaftrio) in a combined regimen with ivacaftor for the treatment of children with cystic fibrosis who are between two and five years of age and have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.
Apotex launched teriparatide injection, a single-patient-use pre-filled pen for the treatment of osteoporosis, in the United States.
LISTEN UP
In season 11 of the NPC Podcast, Dimitris Polygenis, President of Biopharma and Payer Solutions at McKesson Canada, gives insights using technology and AI to decrease prescribing and distribution times, virtual and real-time care, and targeted gene therapies. 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. In Healthbiz Rewind, you’ll read bold life sci predictions made during the renowned “Prognostication Korner” segment of the NPC Podcast.
Bob McLay
Vice President and General Manager
Swedish Orphan Biovitrum (Sobi Canada)
Stratford, ON
Season 04, episode 05
Listen to this episode here
Canada’s federal government’s budget had promised a billion dollars for rare disease treatments. How’s that going to play out? (This episode was recorded in May 2021.)
I would like for that billion dollars to help patients be earmarked for patient care and new innovative treatments. There is a massive unmet medical need for many of these patients, and a lot of them are children, so a lot of families are very much affected. We see the delays and the gaps, where other countries get these products months or years ahead of when we do. Imagine you have a child who has a rare disorder, and you know there’s a therapy available in the U.S. or somewhere else, and you’re waiting to get it. You’re calling the company and the government and doing everything you can, yet our system is not patient-centric.
In Germany, for example, when a new product comes out, it’s instantly approved and paid for by the government. Then what they do is a year later, they say, “Let’s look at what that price was, let’s look at what the real-world outcome was, and we can decide if their value for money was there, if they were at a reasonable price, and renegotiate.” That’s putting the patient first, especially in a rare disease situation with massive unmet medical needs. Here, we’re relying on samples having to come under a particular authorization program. It’s not that easy.
Thank you for attending the 17th Annual National Pharmaceutical Congress on Wednesday, November 22, 2023.
The 17th Annual National Pharmaceutical Congress brought the best of Canadian Pharma together. The Congress was held on November 22, 2023, at the Mississauga Convention Centre and was attended by 250 industry professionals. Stay tuned for more information about upcoming events, including the 2024 NPC Winter Webinar.
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