1. Pragmatic RCTs and the Learning Healthcare System
Cory E. Goldstein
Department Here

2. Objectives Understand the Learning Healthcare System
Obstacles and a novel ethical framework
Concerns and criticisms
As we have just heard from Jeremy, there is a recent history of doing trials that are real-world in nature. These trials help not only patients and physicians make informed decisions, but they also help to inform the decisions made by health systems, and there is a social imperative that we do this type of research.
Within the last 10 years in the U.S., we have seen a rise in the conduct of pragmatic trials in conjunction with a transition to, what is called, the Learning Healthcare System (LHS).
Pragmatic RCTs and the LHS

3. Learning Healthcare System (LHS)
Institute of Medicine (2007) defines the LHS:
“[One that] is designed to generate and apply the best evidence for the collaborative healthcare choices of each patient and provider; to drive the process of discovery as a natural outgrowth of patient care; and to ensure innovation, quality, safety, and value in health care.”
Pragmatic RCTs and the LHS

4. LHS’s objectives
LHS main objective is to reduce medical errors and avoidable shortcomings in treatment due to inadequate evidence for decision-making 
Electronic health records (EHRs) and central data warehouses used to answer pragmatic questions
The Learning Healthcare System proposed approaches for systemically improving healthcare nationwide, attempting to rectify medical errors and avoidable shortcomings in treatment due to longstanding concerns that much of the decision-making surrounding clinical care in the United States—even when guided by clinical practice guidelines—was inadequately supported by high-quality evidence.
At the same time, the emergence of electronic health records (EHRs) and central data warehouses (which collect and combine data from across entire health systems) point toward a future in which data routinely captured at the point of patient care could ultimately be used to answer questions about quality of care, and the comparative effectiveness of different therapies.
Pragmatic RCTs and the LHS

5. Pragmatic RCTs and the LHS
Greene et al. Ann Intern Med. 2012;157(3):
Pragmatic RCTs and the LHS

6. Ethics as an obstacle
Research ethics committee review is time consuming, costly, and overly complex
Consent and other regulatory requirements impede socially important research
This makes little sense for low-risk pragmatic research aimed at improving care delivery
Global frustration that research ethics committee review is time consuming, costly, and overly complex, and that consent and other regulatory requirements impede socially important research.
Pragmatic RCTs and the LHS

7. Addressing the obstacle
Pragmatic research calls into question the conceptual distinction between research and practice
The traditional ethics principles cannot govern the new “research-practice” domain
Faden and colleagues (2013) propose a new ethical framework designed for the broad range of activities within a LHS
Pragmatic RCTs and the LHS

8. Novel ethical framework
Respect the rights and dignity of patients and families
Respect the judgement of clinicians
Provide optimal clinical care to each patient
Avoid imposing non-clinical risks and burdens on patients
Address health inequalities
Conduct continuous learning activities that improve the quality of care and health care systems
Contribute to the common purpose of improving the quality and value of clinical care and health care systems
Pragmatic RCTs and the LHS

9. How this will work Ethics oversight panels
Decide when consent is required using a “threshold of negative effect”
Notify patients, community and stakeholders about research via “transparent mechanisms”
Pragmatic RCTs and the LHS

10. But there are problems with this view…
Pragmatic RCTs and the LHS

11. Patient obligations
“Just as health professionals and organizations have an obligation to learn, patients have an obligation to contribute to, participate in, and otherwise facilitate learning… This obligation is justified by what we call a norm of common purpose.” (Faden et al., 2013)
No consent or disclosure of information needed for low-risk pragmatic research
Pragmatic RCTs and the LHS

12. Do patients have an obligation?
Faden and colleagues provide a prima facie obligation for patients to participate in research integrated into clinical care
Patients when asked for their consent should agree, unless they have a reason for not participating
It does not relieve researchers of the obligation to seek and obtain patient consent
Why should we think there’s an obligation to participate in research at all? There is a human right to be free of experimentation.
Let’s say there is a prima facie obligation, that doesn’t get is to where they want to go (namely, no consent or disclosure).
Pragmatic RCTs and the LHS

13. Current oversight committee
Current oversight and regulatory system is burdensome
“In learning health care systems with [ethics-oversight panels and transparent mechanisms], it will be ethically acceptable for some randomized CER studies, having no or only minor effects on important patient interests, to proceed without informed consent from or specific notification to individual patients.” (Faden et al., 2014)
Pragmatic RCTs and the LHS

14. Will the new system be burdensome?
“[A streamlined process] could be designed to be respectful of patients and less burdensome for them and for clinicians than the lengthier process entailed by current informed-consent requirements.” (Faden et al., 2014)
Do the obstacles come from consent and other regulatory requirements, or from human factors (such as interpretation, control, and bureaucratization) or both?
Why should we think the new system will be less burdensome?
Surely some of the problems come from the rules and regulations, but is this the only place they come from? Could it also not be from the committees themselves, how they interpret the rules and regulations, or the over obsession with bureaucratization?
So why should we think that replacing research ethics committee with ethics oversight panels (a simple name change) will makes this process any easier?
Pragmatic RCTs and the LHS

15. Lack of detailed policy proposal
“Going forward, the next step will be to specify the framework’s implications for oversight policies and practices, including prior review and informed consent, and to determine precisely how the framework will interact with the current human subjects regulations and institutional review board system.” (Faden et al., 2013)
How will the proposed framework interface with existing ethical regulations?
Will existing regulations need to be replaced?
With what precisely?
Lack of any specific proposals. This was published 4 years ago, but no policies have yet to be proposed.
Pragmatic RCTs and the LHS

16. Conclusion Novel framework is not the way forward
Not clear we need to jettison the traditional ethics principles
Going forward, developing substantive ethical guidance remains an urgent priority for pragmatic RCTs
Pragmatic RCTs and the LHS

17. Thank you
Cory E. Goldstein
Pragmatic RCTs and the LHS