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How to Include Patients When Measuring Value | AMCP 2024


Panelists discussed reaching beyond large patient advocacy groups for input and how clinical trial endpoints may paint an incomplete picture of a drug's true value to patients.

Sarah Emond

Sarah Emond

Sarah K. Emond, M.P.P., president and CEO of the Institute for Clinical and Economic Review (ICER), said the quiet part out loud: Providers and healthcare payers make trade-offs when deciding what drugs to cover and for what patients.

“There is a real tension when making decisions for a particular condition or particular patient population with all the other priorities we have as a healthcare system or as a payer. That tension is real,” said Emond during an education session on patient engagement at the annual meeting of the Academy of Managed Care Pharmacy in New Orleans.

ICER is an independent group that conducts cost-effectiveness studies of drugs and makes recommendations on how they should be priced. Emond said the tension between particular patients and the other priorities is a consequence for having overpaid for the health benefits provided by some prescription drugs: “We do real harm by not answering the hard questions. It’s an arms race with higher prices, but clients then can’t afford an increase premium. How do we make this more affordable?”

Emond said the tension can be eased with hard conversations about paying for value, including the price of drug, the value patients receive, and what fair access looks like. “We're starting to have a better conversation about to get to a fair price,” she said. “If we want to pay for value, we have to understand what value is.”

Eleanor M. Perfetto, Ph.D.

Eleanor M. Perfetto, Ph.D.

Value is not always captured by the endpoints measured in clinical trials, noted Eleanor M. Perfetto, Ph.D., M.S., a professor at the University of Maryland, Baltimore.

“For patients, it’s not just a blood test; it can be ‘now I can go back to work’ or ‘now I can take care of my kids,’” she said. “We're now asking patients what value is and we’re learning and working to find that value for patients.”

In her presentation, Perfetto said it’s impossible to answers questions about the impact of a treatment or disease without first knowing what’s important to patients.

“If a new treatment does not have as dramatic an impact as expected, should it be developed? Should it be approved by the FDA? Should it be prescribed or used at all? Should we pay for it?” she asked. “We can't even begin to address these kinds of questions because we don’t have the data.”

The patients’ lived experience is an important element and informs how ICER approaches its cost-effectiveness reviews, Emond said.

Health technology assessments are important because of limited resources in the healthcare system. Patients, Emond said, are often caught in the middle of uncontrolled pricing and payer access restrictions. Health technology assessments can provide evidence to ensure pricing matches patient benefit.

To do that effectively, data is needed about patients’ experience with a disease and the impact of various treatment. But patient data has been a missing element in health assessment. In the past, Emond said if the organization did not have any data on the patient experience from the clinical trial data, they would input a zero into their modeling.

“We’ve made a lot of strides in this area for patient-focused drug development, and there is more data coming from clinical trials,” she said. “We are doing additional things to supplement what we see from the clinical trial program from the manufacturer when we develop our models.”

Over the last few year, ICER has incorporated new methods and procedures to help it more accurately assess the impact impacts. For example, ICER in July 2023 established a patient council that meets quarterly. The goal of the council is to evaluate ICER’s processes and identify gaps to ensure inclusivity.

ICER has also deployed a new method where they can compute the value for patient and caregiver productivity and the impact on patients but based on the data in the clinical trial.

“This is an evolution of our methods, but it is responding to something that was missing in the ecosystem,” said Emond, a longtime ICER employee who succeeded Steven Pearson, M.D. M.Sc., as CEO at the beginning of this year. “We also know that an effectiveness model and a comparative effectiveness of review can feel incomplete in terms of all the things that might matter to a community about a new treatment. That's why we emphasize benefits beyond health and social and ethical priorities.”

ICER has incorporated small focus groups of patients as part of their assessment and include reports the patient and caregiver perspective. Part of this effort includes both large patient groups and patients not affiliated with these organizations.

“I’ll say this out loud," said Emond, "sometimes the large patient advocacy organizations are a reflection of white privilege, because those who have the ability to maybe like quit their job and raise money to bring attention to a rare condition already come from a place of privilege. If we only talk to people from organization advocacy organizations, we might be missing a lot of the lived experience of people who don't actually have that kind of privilege in terms of access to healthcare system.”

The focus on patient diversity has been extended include an assessment of diversity in drug trials. ICER has developed a new framework that considers disease prevalence based on race and ethnicity, sex and age. They plan to evaluate whether a clinical trial’s patient population mirrors the disease population.

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