The value of pragmatic trials to life science product developers
October 2019 | Written by Mark Cziraky, PharmD, CLS, FAHA, FNLA
The need for higher levels of real-world evidence
Healthcare stakeholders are increasingly demanding higher levels of real-world evidence. As the volume and price tag of novel therapies continue to grow within a resource-limited environment, the decisions of payors and policy-makers must be even more well-informed and precise.
Life science product developers must therefore evolve their post-approval evidence portfolio to further emphasize the combined safety, effectiveness and value of their products in real-world patient populations in order to sustain or advance the success of their products in the marketplace.
For the biopharmaceuticals industry, pragmatic trials uniquely offer solutions to address the growing demands of healthcare payers and policy makers for high-quality evidence of therapeutic safety, effectiveness and economic value.
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 biomedical or behavioral health intervention at the individual or population level (Califf and Sugarman).
What are the benefits?
Pragmatic trials can provide evidence to:
- Support reimbursement and pricing by demonstrating real-world product performance and economic value increasingly required by payers.
- Demonstrate product effectiveness and safety including greater insight into sub-group variations in outcomes in post-approval medical practice sought by regulators.
- Enhance competitive position by demonstrating product benefits to prescribers and patients, often relative to current standards of care.
- Increase research efficiencies by applying new health data sources and advances in information technology to answer research questions pertaining to real-world performance faster, and at reduced cost.
Where can pragamatic trials be conducted?
We believe pragmatic trials will be a large part of the future of outcomes research – to understanding life science products in real-world settings, to evaluate comparative effectiveness, and to help to improve patient care.
Today, evolving health systems, where there is good collaboration between providers and payors in the conduct of research, are among the primary recipients of real-world evidence and the ideal facilitators of pragmatic trials in the United States. By their nature, these studies seek to replicate routine clinical practice and patients’ experiences in real-world, community settings – many aspects of which are both documented and managed by the policies, processes and care delivery channels maintained by health systems. As such, evolving health systems are uniquely positioned to provide the most suitable real-world environment to conduct pragmatic trials.
Thought leadership originally published in May 2017.