Learn on pragmatic randomized clinical trials and real-world evidence.
ABSTRACT:
Governments, their regulatory agencies and health technology assessment (HTA) bodies informing the healthcare payers have the ambition of providing patients with fast access to evidence-based innovations in healthcare. The historical split in objectives and responsibilities of the regulators and the HTA bodies can be seen as one of the reasons for the growing evidence gap remaining after the private sector has performed the trials needed to bring their product to the market. Regulators and HTA bodies differ in their recommendations on patient population, comparators and trial endpoints. The confirmatory trials asked by the regulators may not be fit for purpose to perform HTA nor to inform physicians wanting to practice evidence-based medicine. A better alignment of evidentiary requirements of regulators and HTA/payers could accelerate patient access to evidence-based innovations. An additional option is to perform the missing comparative effectiveness trial in the post-marketing phase. Most informative to healthcare decision-makers are practice-oriented, also named pragmatic trials. Such trials have patient relevant outcomes, recruit a broad patient population and have usual care as comparator. Practice-oriented trials lead to real-world evidence and have proven to be practice-changing. A clear pathway towards the realization of such efficient trial methods is needed.
KEY TAKEWAYS:
- Patient access to evidence-based innovations can be accelerated if practice-oriented trials are timely conducted.
- Such trials have patient relevant outcomes, recruit a broad patient population and have usual care as comparator.
- Facilitators would be an alignment of the evidentiary requirements of regulators and HTA/payers as well as a framework for an efficient conduct of trials based on coded electronic health records.
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