Congress Moves to Import Discriminatory Health Policies

Healthcare costs seem to be a constant discussion point both in Washington and in our state capitals. Policymakers are on a quest for a silver bullet that they can trumpet as a win to constituents, but this debate is complex, and nuanced; and will have enormous ramifications for American patients, like my daughter, Marissa, who lives with sickle cell disease and who has a new lease on life from innovative treatments.

For now, the health care debate is myopically focused on the price of pharmaceuticals, without consideration of what we pay holistically in terms of caregiving, hospitals, and other inherent costs of being sick. I cringe at the idea of referencing “cost effectiveness” as the basis for how this country pays for pharmaceuticals because people like me and my Marissa – and the burdens both economic and personal that we shoulder – are not considered in that equation. I have testified to my concerns in state legislatures and am actively working to raise awareness of the danger of policies being considered at the federal level as well that could conclude my family is not worth treating.

Let me be blunt. Looking to outside entities like the Institute for Clinical and Economic Review (ICER) or importing foreign pricing models, as seen in H.R. 3, a bill passed by the House of Representatives in 2019, is not the answer. Though this might sound like an easy solution, but ICER and the referenced countries rely on the discriminatory Quality-Adjusted Life Year (QALY), as confirmed by a recently released CBO report acknowledging that the bill scored savings because government would reference QALYs.

To determine what they will cover and whether they will pay for a treatment, many foreign countries rely on cost-effectiveness analyses based on QALYs and studies that fail to represent people like Marissa. We’ve known for over 30 years that the QALY fails patients and people with disabilities, as it is well known to place a lower value on the lives of people with disabilities and chronic illnesses and on the improvement in quality of life needed to make treatment “worth it.” For this reason, in 2019, the National Council on Disability, an independent federal agency, released a report finding the utilization of the QALY in public policy, whether directly or by referencing foreign countries, is in direct violation of United States disability policy and civil rights laws.

Also, the data that fuels the QALY is from surveys and randomized clinical trials that primarily represent populations who are white and male. In this moment, when our country is reckoning with systemic racism and having robust discussions about health equity, relying on the QALY would be a drastic step in the wrong direction.

As we learned early on with my daughter, there is a deep stigma associated with sickle cell disease. Pain from sickle cell disease crises is frequently undertreated due to racial biases – too often these patients are treated as drug seekers, not patients in need of attention and care. Historically, research funding for sickle cell disease has been significantly less than that for other rare genetic diseases. We finally got a long overdue win for sickle cell patients, when, in the past 3 years, three new drugs were approved, with 35 more in the pipeline.

My daughter is now on two of these drugs – prior to treatment she was spending weeks at a time in the hospital and accruing hundreds of thousands of dollars in medical bills. She now is able to live her life more fully, is not consistently in and out of the hospital, and has drastically reduced her holistic healthcare costs. I see the benefit this treatment has brought her and others, but I also see the incredible access issues that QALY-based assessments can present for patients. Shortly after the approval of these new treatments, ICER conducted a QALY-based assessment (similar to those used abroad) of these new treatments. ICER’s report found these therapies not worth the cost, and patients have suffered.  Payers rely on this report to justify making patients fight tooth and nail for access to the drug that has been revolutionary to my daughter. This is just a snapshot of what we will see if QALY-based assessments begin to be used widely within the United States.

Though, I haven’t painted a rosy picture of the current landscape, there is hope. There is a better way to determine actual value to patients and society by conducting high quality, patient-centered comparative clinical effectiveness research. This type of research determines the right treatment for the right patient to provide higher value in the healthcare system writ large.  We have an entity created by Congress in the United States designed to conduct this exact type of research, the Patient Centered Outcomes Research Institute (PCORI).  Just last week, PCORI’s Board approved new principles to collect and analyze a full body of outcomes data, including economic and cost data. This will allow PCORI to be a vital part of the value discussion by developing alternative methods for collecting and analyzing cost data that reflects the real-world costs to patients and their families. Even better, PCORI’s statute bars it from using QALYs. I have incredible optimism that this is a huge step forward toward health equity and robust, high value patient care.

If we as a nation want to have a serious and nuanced discussion about getting to a fair and equitable health system, we must reject reliance on QALYs and other discriminatory metrics and embrace PCORI as part of the solution.

Adrienne Shapiro is the Executive Director of Axis Advocacy