Tightly-controlled studies used to test the safety and effectiveness of new drugs don't always tell the whole story of how well a drug works for the thousands and sometimes millions who later take it.
These drug trials often don't often include women, minorities, and patients with additional conditions who are taking different drugs, leaving doctors unsure how a new drug will affect many of their patients.
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To address this problem, regulators, insurers and drug companies are now trying to gather more information about how drugs work in the real world. The idea is that this data will help them make more-informed decisions on who will likely benefit from medication — and who won't.
Tom Hubbard, vice president of policy research at the Network for Excellence in Health Innovation, a policy institute focused on health care quality and costs, recently discussed this with The Associated Press. The interview has been edited for length and clarity.
Q: What is real-world evidence?
A: Data that is collected routinely in health care. The setting could be virtually anywhere — hospitals, physician offices, pharmacies. It can include data generated by patients themselves ... how they're reacting to a treatment, whether they're experiencing side effects. It could include data from wearable devices like a Fitbit or smart watch.
Q: Who is collecting this data?
A: Virtually every big sector in health care. Many of the larger insurers have formed units to create real-world evidence through examining data they have. Increasingly, drug companies are collecting real-world data in anticipation of questions about drug effects from insurers, patient groups and clinicians. When they get accelerated approvals based on studies including few patients, they are obligated to do real-world studies and report to the FDA.
Q: What's driving this trend?
A: There's much more ability to capture data, partly because so many physicians now have electronic health records.
Q: Is this data likely to help doctors and patients?
A: In theory. A doctor will be able to match treatments more closely to patients. Real-world evidence should ferret out more quickly what the side effects might be. We're talking about a shift from a data system focused on billing to one focused on answering questions of medical effectiveness.
Q: Will this evidence reduce waste in the health care system and save money?
A: That's absolutely one of the great hopes for this, that as treatments become more effective, guided by real-world evidence, it would reduce costs for everybody. It could also help improve the clinical research process.
Q: When will this really kick in?
A: I think we'll see more decisions informed by analysis of real-world evidence within the next five years.
Follow Linda A. Johnson at @LindaJ_onPharma.