Over a decade ago, I founded the Partnership to Improve Patient Care with a basic principle: Patients are best served when they are informed and empowered to decide which care options are best for them; they are poorly served when policymakers in Washington dictate which options are best.
In 2010, I noted that the Affordable Care Act (ACA) “recognizes not only the importance of strong clinical evidence to support decision-making but also the need to use the research to support doctors’ and patients’ decisions, not to dictate what those decisions should be.”
That principle is again under debate — and under threat — as Congress considers new government standards to regulate drug prices. I’m all for improving drug affordability, but it is vital to pursue solutions that reinforce this core principle of empowering patient choice, not undermining it.
During the ACA debate, we fought for two things — strong patient protections against government misuse of comparative effectiveness research, and an equally strong solution for patients through creating the Patient-Centered Outcomes Research Institute (PCORI). I’ve stayed in this fight because of my own experience as a person with epilepsy, and my career spent championing the concerns of people with disabilities.
The creation of PCORI was essential because existing research entities devalued people with disabilities, serious chronic conditions, rare diseases and older adults with research focused on broad populations. Too often, researchers made conclusions about treatment effectiveness based on averages as opposed to understanding the differential treatment impact among subpopulations. And payers used — and still use — that information to justify restrictive coverage.
When PCORI was established in 2010, advocates fought for provisions in their statute that would preclude comparative clinical effectiveness research from being used against Medicare beneficiaries by restricting access through coverage decisions while generating useful information for patient and provider decision-making. Congress struck the balance between members of Congress who wanted the institute to be a federal entity whose research was used to restrict Medicare coverage and generate cost savings versus members who referred to a comparative effectiveness research institute as a death panel that would deem people with disabilities and serious chronic conditions as too expensive to treat with the newest innovations. PCORI’s statute ensured that comparative clinical effectiveness research would not be used for one-size-fits-all coverage determinations in Medicare and would be used to improve health decisions.
As Congress considers new strategies for informing Medicare reimbursement and coverage, the bipartisan history of PCORI’s creation should provide some lessons.
First, a bipartisan group within Congress struck a balance by allowing comparative effectiveness research to inform decision-making, without it being used to restrict patient care through reimbursement, coverage or utilization management programs. This balance is important, whether in the context of access to existing or future treatments.
Second, nondiscrimination was an explicit goal of PCORI’s work, with a statutory bar on the use of metrics that discriminate to compare the effectiveness of treatments, such as quality-adjusted life years (QALYs). This provision assured that PCORI’s work did not violate the Americans with Disabilities Act or other disability rights laws, a lesson learned from Oregon’s 1992 Medicaid waiver application that demonstrated how QALYs lead to disability discrimination when used to inform its prioritized list of services.
Third, experience overseas shows why it is so important to build on PCORI's work and get to better, more patient-centered approaches to comparative effectiveness research. For example, even in Germany physicians find that their ability to prescribe effective treatments is harshly restricted under their government's one-size-fits-all comparative effectiveness standard. When a standard devalues better treatments, it devalues the lives of patients with unmet needs.
Policymakers can demonstrate their continued support for patients and people with disabilities by making a few key changes: one, Congress should strengthen existing safeguards in statute against CMS misuse of one-size-fits-all comparative clinical and cost-effectiveness standards; two, strengthen PCORI’s role in driving affordable, patient-centered care by supporting the Institute’s ongoing work to collect patient-centered cost data, establishing a public database of patient-centered outcomes, and ensuring they deliver relevant summaries of research when and where they are needed.
Patients and people with disabilities support the conduct of comparative clinical effectiveness research that is truly centered on the needs, outcomes and preferences of patients and people with disabilities. We want to see improved methods for assessing the value of treatments that better reflect outcomes that matter to patients and people with disabilities. We do not want to move backward by using this information to make one-size-fits-all reimbursement and coverage decisions.