New Provision Bolsters Relevance, Usefulness of PCORI's Work

When Congress reauthorized PCORI’s funding for another 10 years, it gave us and the healthcare community we serve a remarkable opportunity to continue to fund critical research to inform a more effective, efficient, and patient-centered healthcare system.

With this new opportunity came a provision designed to ensure that our work will be even more relevant and useful to our stakeholders by considering the full range of clinical and patient-centered outcomes that meet the needs of patients, clinicians, purchasers, and policy makers. This includes, as appropriate, capturing the potential burdens and economic impacts of various healthcare services, along with the relative health outcomes and clinical effectiveness measures our work has captured to date.

Examining Wide Range of Impacts

PCORI’s new provision covers a wide range of impacts at the center of the ongoing public and professional discussion about how our healthcare system can provide the greatest value to all. Patients, their families, caregivers, clinicians, policy makers, and others across the healthcare community know these well—out-of-pocket medical costs; health plan benefit and formulary design; nonmedical costs to patients and families such as transportation and childcare, workplace productivity, and absenteeism; and healthcare utilization. Several of these impacts, such as those affecting patients and their families most directly, are often overlooked, both in research and in conversations about value.

We know that patients, caregivers, and healthcare stakeholders are making tough choices every day about which treatment works best for them, or how to present options to a patient that align with their values, or which benefit design is most suitable for their beneficiaries. Too often, they lack sufficient evidence to make the most informed decision, especially as it relates to the cost burden and economic impact of those choices.

PCORI understands that discussions of healthcare cost burdens and economic impact have a number of diverse perspectives that we and others must consider and balance. We embrace our role in informing this discussion as the nation’s premier funder of comparative clinical effectiveness research and patient-centered outcomes research.

After all, PCORI’s vision from our earliest days is that patients and the public have information they can use to make decisions that reflect their desired health outcomes. And PCORI’s mission is to help people make informed healthcare decisions and improve healthcare delivery and outcomes, by producing and promoting high-integrity, evidence-based information that comes from research guided by patients, caregivers, and the broader healthcare community.

Four Principles to Guide Our Way

So we embark on this latest chapter in our history in a way familiar to the communities we have served since 2010—soliciting input from a broad range of stakeholders on a set of principles that will inform how we will implement this new provision in our legislation while ensuring that PCORI-funded research continues to be patient centered and impactful. These principles, on which we seek public input, will serve as a basis when developing guidance for future applicants and reviewing applications for research funding. The principles are:

PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts.
PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients.
The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study.
Beyond the collection of burden and economic impact data, PCORI may support the conduct of certain types of economic analyses as part of a funded research study, to enhance the relevance and value of this information to healthcare decision makers.

Even with this expanded provision, our authorizing law still does not allow developing or employing a dollars-per-quality adjusted life year as a threshold to establish what type of health care is cost-effective or recommended. We cannot mandate, nor will we recommend, coverage, reimbursement, or other policies for any public or private payer. Thus, we will not fund studies that seek to conduct a formal cost-effectiveness analysis or whose results will include coverage recommendations, payment or policy recommendations, or clinical practice guidelines.

What we will do is ensure that in all the work we do, we remain true to our overarching mandate to generate authoritative clinical evidence that will help patients, clinicians, purchasers, and policy makers in making informed health decisions.

Stakeholder-Guided Approach

In seeking comment on and finalizing the principles that will inform our work, we will follow a deliberate and transparent process with opportunities for stakeholder input at each stage.

The Board of Governors’ approval of the release of our draft principles on September 14 begins a 60-day public comment period that closes November 13. You can see the complete draft principles document here along with a form you can use to provide comment. We also plan to host two town hall webinars on October 5 and October 6 to provide more opportunities to solicit your comments. Once we have all your input, we will analyze it, revise our draft principles as needed, and use it to guide our instructions to applicants. We will finalize the draft principles for Board consideration early next year.

PCORI’s stakeholder-guided approach to patient-centered outcomes research makes us unique among funders. It served us, and the healthcare community, very well during our first decade. We are confident it will continue to do so in the years ahead.