Almost exactly a year after the US Food and Drug Administration approved a revolutionary new genetic treatment for spinal muscular atrophy, most patients with the rare disease are still waiting to get the life-saving medicine. Demand for Spinraza, which is sold by Cambridge-based Biogen Inc., quickly overwhelmed health care providers. Hospitals, many of which had to buy Spinraza up front before seeking reimbursement, weren’t sure whether insurers would pay.
And their concerns were well-founded. Some insurers have balked at the staggering price-tag: $750,000 per patient for the first year and $375,000 annually after that. The Globe looked deeply at one family’s quest to get the drug. The paper also examined the impact on the nation’s health care system as a wave of six-figure treatments for rare and deadly diseases move toward approval.
The Globe published four stories:
“The new price of hope,” the main story, focused on the struggles of the English family, of Orem, Utah, and their desperate effort to get the medicine for 12-year-old Colin English. He had recently been diagnosed with spinal muscular atrophy, the same inherited disease that killed two of his sisters.
There were three sidebars: “At a UMass lab, a eureka moment,” which focused on the circuitous path of Spinraza from auniversity laboratory to the market; “Five mils of this stuff is $125,000,” a you-are-there account of an infusion of Spinraza in a Johns Hopkins Hospital operating room; and, “No miracle cure for rising drug costs,” which described potential remedies for the soaring cost of life-saving medicines.
Judges comments: The New Price of Hope is a remarkable and touching series about a new drug for spinal muscular atrophy. Wonderful balance of personal stories, science, business and politics. The amount of research and legwork involved was obvious and impressive. The stories also demonstrated well the unusual and often cruel and counterproductive nature of the US healthcare system. Congratulations on inspiring work of journalism!