By Steve Pearson
(This commentary is being co-posted by The Donaghue Foundation

In the United States, consequential decisions around prescription drug pricing and patient access are generally made based on limited evidence and without patients in the room. In confidential negotiations, drugmakers and insurers make financial tradeoffs that seriously affect the health and wealth of all Americans. Drugmakers charge as much as they can, insurers react by restricting care and raising copays, and patients are none the wiser.

Inarguably, this system is broken. One remedy is to bring these pricing and coverage decisions more into the open, where we all can wrestle with how to understand the benefits drugs and other health care services bring to patients and their families, and how we should try to align our spending to make sure we get the most health we can out of the dollars available. The first step has to be guided by patients and patient groups who know the condition, know the diversity of patient experience, and who can bring a truly comprehensive view to the table on what matters most to patients. Only when that input is made central to any assessment of value can we usefully judge the evidence on how well drugs improve the quality and length of patients’ lives. If this input and deliberation on the evidence occurs through clear methods – and in a public forum open to all – only then can we have a more honest discussion about whether the prices set by drugmakers are fair.

But don’t be fooled; this honest discussion isn’t easy for anyone. Bringing discussions about evidence, pricing, and coverage into the open requires facing the demons of the US health care system: that there are limits to the amount we can and should spend on health care; that the impact of high prices falls disproportionately on the sick; that insurers often try to control costs by limiting access to care; and that all these tensions and flaws have become entrenched in a health care system dominated by powerful corporate interests. Under the circumstances, it’s understandable that many patients and patient groups worry that the end result of any discussion of “value” or “fair pricing” will tend towards an answer of “not covered for you.” Thus, any effort to engage patients and patient groups to talk more honestly about weaknesses in the evidence for new drugs, about prices that may not fairly reflect the amount of benefit patients receive, or about insurance coverage limitations that may make sense, can seem to threaten their access to new drugs. Patients and patient groups may worry that, in seeking to do the right thing, they may ultimately contribute to the access restrictions they fear. Nonetheless, we can’t create positive change without them. We can’t even begin to understand the value of new drugs without hearing from patients, and we will never be able to have the honest, mature, and difficult conversation about prices and coverage unless patients are at the table.

My organization, the Institute for Clinical and Economic Review (ICER), is an independent nonprofit research organization striving to create a public process of determining what a “fair” price would be for a new drug – with patients’ perspectives serving as the guiding structure. ICER takes an iterative publishing approach to each of our assessments, reaching out to patient groups even before we announce a topic, and then engaging with them over the entire 8-month review process. Where possible, we have in-person meetings, and through these interactions, phone calls, and emails, we share an ongoing dialogue about what matters to patients and what evidence is available. We invite formal written comments at three distinct times throughout our timeline; patients are also invited to be front and center at our public meetings where an independent appraisal committee deliberates on the strength of the evidence. At the public meeting, patients help the appraisal committee hear first-hand about the true burden of their disease, what health outcomes matter most to them, if a treatment has any benefits and disadvantages that may not have been adequately captured in a clinical trial, and the broader context policymakers should consider when evaluating the fairness of a price.

How has all the input from patients affected ICER’s drug value assessments? Here are a few examples:

ICER’s goal of improving transparency, along with our ongoing efforts to incorporate constructive feedback as our methods evolve, improves the science behind our work. Just as importantly, it binds us to a commitment to engage with the patient community to help guide our work. While we’ve made improvements in how we engage with patients over the years, and had notable success stories of how patient engagement has made our work better, we’re well aware we need to continue to learn how to be more effective when working with different kinds of patients and patient groups. Over the next month, we’ll be announcing a series of new enhancements, and we hope these further embed patients at the heart of our assessment efforts, and of any discussion of health care value. Stay tuned.