This week is Rare Disease Week—seven days to acknowledge the more than 25 million Americans who suffer from one of 7,000 conditions that, on their own, affect fewer than 200,000 people. You’ve probably never heard of the vast majority of these conditions and, traditionally, these diseases attract fewer research dollars than diseases like cancer or Alzheimer’s. Patients fight for their lives—and for a cure, often hitting the ground to advocate for more money.
To help bring light to the need for cures, this week the EveryLife Foundation will bring more than 800 advocates to Capitol Hill to meet with members of Congress.
Lawmakers are not the only ones rare disease advocates are petitioning, however. Groups like the EveryLife Foundation also are fighting the Institute for Economic and Clinical Review, or ICER, the John and Laura Arnold-backed group that has taken it upon itself to determine the “value” of various treatments, including those used to manage or cure rare diseases. Using the hashtag #NumbersThatMatter, patients and physicians took to Twitter yesterday to call into question ICER’s methods. These advocates say they are worried ICER’s determinations are a “danger to diseases like cystic fibrosis and other chronic diseases.”
Specifically, the rare disease community focused yesterday on ICER’s evaluation of Trikafta, a drug approved last year by the U.S. Food and Drug Administration (FDA). In a press release announcing the approval, Acting FDA Commissioner Dr. Ned Sharpless said: “At the FDA, we’re consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review. Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options ...”
Rare disease advocates are worried ICER is trying to get insurance companies (which, as we explain here, also provide significant financial backing to ICER) not to cover the drug. Indeed, advocates have less than a month to response to ICER’s evaluation of Trikafta before the organization releases a final report on the drug in May.
In a new podcast, Gunnar Esiason, a cystic fibrosis advocate with the Cystic Fibrosis Engagement Network (CFEN), explains why ICER’s model for determining drugs’ value has patients worried. He should know. In an op-ed published last month in The Hill, Esiason said Trikafta “saved my life.” “In a matter of days after” taking the treatment, Esiason “was allowed to look toward my future, a future that until then had to seem elusive.” He says the drug is the reason he is now able to pursue an MBA and Master of Public Health at Dartmouth. He says life with Trikafta is “a far cry from the days of not having the strength to walk up a flight of stairs.”
In that op-ed, Esiason also asked, “Would the analysts at ICER prefer I return to a state of recurrent hospitalizations, traumatic medical procedures, bouts of hemoptysis (coughing up blood), and the fast track towards end-stage illness and disability income?”
A video posted on the CFEN website by the Institute for Patient Access also examines ICER’s methodology. It argues ICER takes a “one size fits all” approach that is incomplete and not reflective of patients’ personal experiences. It concludes doctors and patients working together—not economists at ICER—should get to determine what treatment works best.
Of course, as we reported here, ICER has told patients, their parents, and their advocates that their voices actually matter very little. When the mother of a child who suffers from Duchenne muscular dystrophy, a rare and often fatal disease that causes muscle deterioration, asked how she was supposed to quantify the value of her child’s life, ICER President Stephen Pearson said, “[T]hat’s why we don’t have you vote.”