As medicine continues to evolve and solutions for rare diseases come in greater frequency, so to does the way stakeholders address the costs associated and begin to think about value, according to a recent article in the American Journal of Managed Care (AJMC).
Panelists at the 23rd annual International Society for Pharmacoeconomics and Outcomes Research (ISPOR) convention discussed the challenges associated with the high-cost of drugs for rare diseases through the eyes of the payer, patient and economist.
Clifford Goodman, PhD, senior vice president and director of the Center for Comparative Effectiveness Research at The Lewin Group, presented the payer’s perspective on the panel. Goodwin said rare disease treatments priced in the millions has driven the U.S. to rethink its stance on value.
But there are challenges to conducting health technology assessments for the drugs, Goodwin said. Including evidence limitations and value assessments. In addition, the number of effective treatments has increased significantly, has made it much more difficult to pay for them.
Patient perspective panelist Paul Melmeyer, MPP, director of federal policy at the National Organization for Rare Disorders (NORD), said discussions of value should take into account what value means to patients. Clinical outcomes are only as important as tangible experiences to patients. Melmeyer also said the “value of hope” for patients, their families and caregivers were markers to consider but more difficult to measure.
Economist panelist Louis P. Garrison, professor emeritus of the Comparative Health Outcomes, Policy and Economics Institute at the University of Washington, said that value boils down to its most basic components for the economist: the amount someone is willing to pay for something. However, Garrison said that value is not consistent among patients, making it difficult to measure.