A ‘game-changing moment’: using real-world evidence to approve drugs

New guidance on using real-world evidence could change how some drugs are approved and evaluated. It’s the first in the world to share how that information should be reported in a way that both drug approvals and health technology assessments can use. The guidance, from Health Canada and the Canadian Agency for Drugs and Technologies in Health (CADTH), includes recommendations for how real-world evidence should be reported in a consistent, transparent way that aligns with global standards. The guidance was developed by a working group co-chaired by Mina Tadrous, assistant professor at the University of Toronto’s Leslie Dan Faculty of Pharmacy. “Using real-world evidence means we’re going to have better evidence where there are gaps, and we’re adapting to the changing landscape of how drugs are evaluated,” Tadrous said in a story on the university’s website. “This guidance lays the foundation for the use of RWE in regulatory and HTA decision-making and will help develop a pathway to support the evaluation of newer drugs.” Read: University of Toronto launches two new courses on biosimilars Real-world evidence includes data from patient healthcare records, insurance plans and patient registries. It’s an important addition to randomized controlled trials because those often exclude certain groups, such as pregnant people or those with other health conditions. Real-world evidence can also be used when finding enough participants for a large trial isn’t possible, for example, with drugs for rare diseases and highly targeted medicines. “It’s becoming more challenging to rely only on RCTs in some scenarios, and this is limiting and slowing down our ability to get drugs to patients,” says Tadrous. “Real-world evidence will never and should never replace RCTs. But real-world evidence is a way to augment or add to a drug’s body of evidence that will support better decisions about what drugs are going to be approved and paid for.” Organizations worldwide, including the U.S. Food and Drug Administration (FDA) and the European Union’s European Medicines Agency, have shifted towards using real-world evidence. In 2021, the U.S. FDA approved an immunosuppressant drug for people with lung transplants based on real-world evidence. The increasingly important role of real-world evidence was part of what drove the need for this guidance. “The use of real-world evidence will be a change in how we approve and pay for drugs, and this guidance is the foundation of making that change happen by ensuring the highest-quality evidence is used,” says Tadrous. “We haven’t had a big shift in how we approve drugs in a long time, and this is one of those game-changing moments.” Read: CADTH is launching a new Post-Market Drug Evaluation Program

A ‘game-changing moment’: using real-world evidence to approve drugs

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