Value Assessments in the Age of Personalized Medicine May Require a Cultural Shift, Panel Says

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On the closing day of the International Society for Pharmacoeconomics and Outcomes Research 23rd Annual International Meeting, in Baltimore, Maryland, stakeholders gathered to grapple with the role of value assessments in a healthcare landscape that is increasingly focused on the use of precision medicine in treating disease.

Moderating the panel was Kristen Migliaccio-Walle, director of Global Health Economics and Outcomes Research at Xcenda. Migliaccio-Walle opened by pointing out that, among the barriers to the increased use of personalized medicine is a lack of value recognition, both clinical and economic. Value assessment frameworks have the potential to encourage the use of personalized medicine, but that can only happen if frameworks incorporate the appropriate elements to demonstrate value.

Speaking from the perspective of the health technology assessor (HTA) was Daniel A. Ollendorf, PhD, CSO of the Institute for Clinical and Economic Review (ICER). Ollendorf began his remarks by disabusing the audience of what he said was a common perception that “…HTA bodies have a natural bias against personalized treatment…we actually feel that nothing is further than the truth.”

He explained that organizations like ICER must base their assessments on evidence available to them, and “Oftentimes, a personalized approach is just emerging—or may even be a thought—at the time of FDA approval," but “We are certainly open to the idea of identifying the right population for treatment [to] make the value proposition a solid one.”

Ollendorf gave the example of ICER's recent assessment of vesicular monoamine transporter-2 inhibitors that treat tardive dyskinesia, an involuntary movement disorder that is caused by the long-term use of antipsychotic drugs that treat schizophrenia, bipolar disease, and other conditions. Clinical studies of these drugs were powered to assess involuntary movement, but other stakeholders raised the possibility of the drugs’ having additional benefits: treating tardive dyskinesia effectively could allow for better control of underlying psychiatric disorders because patients could potentially be more adherent to their antipsychotic treatment.

Because no data were collected on this potential benefit during clinical trial, ICER worked with one drug’s manufacturer to create a set of threshold analyses to try to assess whether this benefit could affect the result of the value assessment. (Eventually, even with this information, ICER determined that the drugs would need to be discounted substantially to fall within its threshold value range.)

“We are certainly open to the idea of identifying the right population for treatment [to] make that value proposition a solid one,” he said. When manufacturers say there are benefits to treatments that are not shown in clinical trial, his response is, “Why didn’t you collect information in the study? There are standardized instruments to use.”

Sara Traigle van Geertruyden, JD, executive director of Partnership to Improve Care, said that ICER’s experience with tardive dyskinesia therapies “underscores some of the challenges that patients are having in the value assessment world,” because data that are important to patients are not always part of clinical trials, and subpopulations are not necessarily analyzed.

While value assessments are not always aligned with the outcomes that matter to patients, they do drive patients’ options and choices, however.

“What it boils down to is culture,” said van Geertruyden. The current culture in the United States, she said, is structured upon a fail-first approach and on limiting access to new, expensive treatments. A cultural transition should focus on building evidence to know which treatments work for which patients, and when. Such an approach would help to save on costs by preventing adverse events or nonresponse to treatment. One key to achieving this new reality is to invest in real-world evidence earlier, so that payers have more information to work from.

Robert W. Dubois, MD, PhD, chief science officer of the National Pharmaceutical Council, added that value assessments range from simple to complex; if the FDA has approved a product with a companion diagnostic, value assessment is relatively straightforward, he said. But in other cases, including cases in which outcomes that matter most to patients have a great deal of nuance, “It’s a nightmare to figure out how you develop a value assessment.”

Dubois, too, felt that the United States faces a cultural challenge, saying “I think we all want more data...I don’t think this is a methods issue; I think this is a social and cultural issue.” He pointed to recent passage of Right to Try legislation—calling it a “uniquely American problem”—as a clue that the US culture at large is in favor of an approach to treatment access that is not evidence-based in nature.

“In the absence of data, what is the right approach? Until you get the answer to that question, I don’t think you can ask our value assessors to do more than what society is doing.”