The value of pragmatic trials to clinicians

October 2019 | Written by Mark Cziraky, PharmD, CLS, FAHA, FNLA

A reliance on real-world evidence for optimal decisions

Clinicians are always striving to improve the quality and the precision of care that they provide to their patients. Oftentimes, they’re lacking the information needed to make an optimal decision for an individual patient because much of the information they have at hand was either derived from a very broad and diverse population, or the population studied was demographically different than the population they are managing.

The evidence generated by randomized controlled trials (RCTs) may establish the potential efficacy, or clinical impact, of a drug, but it does not necessarily tell us how safe and effective it is for any given individual, or in whom it works best. This issue, coupled with rapidly escalating costs in care delivery, makes it ever-more critical to generate high-quality, real-world evidence of value in an efficient manner.

Pragmatic clinical trials provide the optimal environment to address a broad spectrum of real-world issues, and generate evidence synergistic and complementary to RCTs. For clinicians, pragmatic trials generate evidence that enables better-informed decision making.

What are pragmatic trials?

Pragmatic trials are studies designed for the primary purpose of informing decision-makers regarding the comparative balance of benefits, burdens and risks of a pharmaceutical, biomedical, surgical, or care management intervention at the patient level (Califf and Sugarman).

What are the benefits?

• Pragmatic trials can enable rigorous scientific evaluation of the safety and effectiveness of a product or intervention, in a relatively short timeframe, using designs that provide strong external validity to ensure that findings apply to real-world patient populations and care environments to payers, prescribers and patients.
• Pragmatic trials can address the growing demands of clinicians and payers for high-quality evidence of the clinical outcomes, safety and economic value from drugs, devices and care management programs for different demographic groups of individuals.
• Pragmatic trials can better inform healthcare decision-making processes at the regulator, payer, physician and patient level.

How can clinicians engage with pragmatic trials?

Clinicians can use pragmatic trials with several levels of engagement. They can become participating investigators, choose to enroll their patients in trials, or simply incorporate the learnings from the trials, through scientific publication, into their clinical practice.

Carelon Research is among the early innovators and key promulgators of pragmatic trials on a national basis. We have conducted multiple studies within our research environment.

Thought leadership originally published in June 2017.