Health Plans Juggle the Hope, High Costs of Rare Disease Drugs

As more rare disease therapies come to market, health plans are often evaluating products for conditions they have never encountered before, Susan M. Wescott, RPh, MBA, told The American Journal of Managed Care^® in an interview at the Academy of Managed Care Pharmacy Nexus 2026 meeting.

Wescott, a panelist in the session “Payer Evidence and Decision-Making in Rare vs Common Disease Products,” described the current moment as “exciting” for patients with these conditions, who now have reason for hope. However, it is also a demanding time for payers, as many of these conditions, which may affect only 1000 to 10,000 patients in the US, previously had no treatment. As a result, plans must “dig deep” into their research, she said.

The evaluation process begins with education and data gathering, followed by expert opinions to guide decision-making, Wescott explained. She emphasized that expert input is particularly important given the specificity of these diseases and patient situations, as plans must understand how a therapy works and how it will help patients.

On cost, Wescott was candid that plans have limited room to maneuver. She said the US health care system is approaching a difficult turning point, as some plans will be unable to afford these products, and self-insured plans could face bankruptcy after receiving a high-cost claim. In response, payment models are evolving, with plans increasingly underwriting across larger populations by joining bigger groups.

Despite these financial pressures, Wescott emphasized that coverage decisions center on whether a drug makes a significant difference in patients’ lives. She identified bringing the patient voice to the table as another important part of the process, with plans assessing a treatment’s impact based on input from providers representing patients.

Ultimately, Wescott said, evaluating these therapies comes down to asking the “so what?” question.

“So, it does this,” she concluded. “What does that mean to the patient, and how does it change health care cost and quality of life exactly?”