One girl’s hope, a nation’s dilemma
A Cambridge firm’s drug worked wonders, but was hugely costly – more than Costa Rica thought it could spend on one child among so many
Every two weeks, Tania Gonzalez receives a two-hour treatment with a drug called Cerezyme in a San Jose hospital. (Tito Herrera for The Boston Globe)
By Stephen Heuser
Globe Staff / June 14, 2009
Costa Rica – At the time, he had no way to know it would trigger a high-stakes controversy that reached all the way to Boston, but Jose Antonio Gonzalez remembers clearly the day he first heard that there might be a drug to help his little daughter.
To Jose, it sounded like a miracle. As a toddler, Tania had been a bright girl with a vivid smile and a penchant for dancing. But by age 8 she was in a strange and frightening decline. She struggled on frail limbs to carry her swollen abdomen. As other children rode their bikes on the tiny fishing village’s dirt roads, Tania lay on the sofa in her orange cinderblock house, inert.
No one understood what was wrong. Specialists had run tests on Tania to rule out common diseases, then unusual ones. When Jose’s phone finally rang with an answer, the doctor told him Tania had a genetic defect so rare that it strikes only a tiny scattering of people around the world.
“The first thing he said was, she could die,” said Jose, a somber and powerfully built man. “The strength I was supposed to carry with me just vanished.”
But the doctor had another piece of news. There was a drug that might halt Tania’s suffering and perhaps even reverse the toll of her disease. The drug was called Cerezyme.
For Jose and his family, it was as though a hand had reached down to answer their prayers. But in that moment, something else had happened as well: The Cambridge drug company Genzyme had just found its first potential patient in Costa Rica. And now that it had found one, it would supply the drug to Tania, but at an astonishing cost – $160,000 a year, possibly for the rest of her life.
This was far more money than the Costa Rican government had ever paid for a drug, and Genzyme would not bend on the price. The country’s health officials were forced to weigh the prospect of a healing gift for one girl against the needs of a nation struggling to care for millions.
Should Tania get the drug?
What unfolded in that village was a dramatic example of the hard choices often forced by the inventions and ambitions of the biotechnology industry, an increasingly important part of global healthcare and a critical growth sector for Massachusetts. Its high-priced cures are creating both great wealth and great moral dilemmas, one new drug, one new patient at a time.
The greatest dilemma of universal healthcare, with a single payer: the federal government
will result in political intervention for affected individuals. We have already witnessed Barney Frank pick up the phone and threaten the new CEO of GM. The politically connected with whomever is in power will get preferential treatment. Do you really want to go down this road. Please don’t offer the rationale that it won’t happen. It already does.