Cystic fibrosis sufferers, their families and physicians are fighting back against proposals that they claim could curb access to treatments.
The Institute for Clinical and Economic Review, a research organization that evaluates the clinical and economic value of prescription drugs and other health care products, has stated its analysis suggests certain medications for sufferers should be much cheaper.
“Our analysis suggested that discounts of up to 77 percent would be needed to bring the prices into alignment with their clinical value to patients,” ICER's chief scientific officer, Dan Ollendorf said in a statement earlier this month.
Ollendorf was referring to Vertex's cystic fibrosis drugs, which cost more than $200,000 a year.
But advocates for cystic fibrosis sufferers are pushing back against this analysis, and warning that it could have a "chilling effect" on innovation while causing insurance companies or other parties to drop coverage of the treatments.
Former NFL quarterback Boomer Esiason, whose son was diagnosed with cystic fibrosis 26 years ago, said the debate over cost-effectiveness "upsets me."
"The criticism ignores the vast time and money invested in developing these treatments, and....the proposals advanced by the critics rarely account for the damaging impact on actual patients," Esiason, co-chairman of the Boomer Esiason Foundation, which has raised $115 million to support research into the disease, said.
"People could use these findings to limit what treatments they cover or to justify price caps on these treatments—both of which would restrict patients’ access to these treatments.”
In a conference call ahead of an ICER meeting in Minneapolis today, Dr. Stanley Fiel, a pulmonologist and member of the American College of Chest Physicians expert panel, said there has been great progress in medications that have allowed cystic fibrosis sufferers much longer lives.
Fiel pointed out that when he began practicing 25 years ago few sufferers were expected to live to adulthood. They now can expect to live into their mid-40s, largely because of advances in medications..
Protein modifiers, also known as transmembrane conductance regulator (CFTR) modulators, have changed the face of treatment.
ICER, Fiel said, is attempting to put a value on the treatment of individuals. He questioned how they were able to do that type of measurement.
He argued that ICER, when evaluating the drug, failed to take into account the wider cost benefit of improving a person's quality of life, going to school, working, avoiding hospitalizations and generally adding value.
"That does not come into play," Fiel said. "As a caregiver, (this) limits my ability to offer all treatments available."
Brian Callanan, the founder and executive director of the Cystic Fibrosis Life Foundation, was diagnosed with the condition at birth 42 years ago. His life expectancy at birth was less than 10 years.
There has been "tremendous progress, but (we're) still waiting for a cure," Callanan said, adding that the gene that leads to cystic fibrosis has been identified and the potential is there for a cure.
Callanan vowed at an early age to live long enough to see that cure in his life time. Medications and exercise are key to keeping him alive, he said.
He described the disease as "chronic, terminal, and aggressive," and one that leads to associated psychological issues, including anxiety and depression.
The drugs in question have allowed him to reduce that anxiety because he does not have to worry that a simple cold may lead to hospitalization, then pneumonia and even death. He has not been hospitalized in five years.
"When I hear that a group of people have decided that the cost of this medication should be 77 percent less in value, it’s hard not to take that personally and feel like my life value is worth 77 percent less," Callanan said. "What is the value of well-being? What is the value of my life to you? I’m not just a number or a cost."