Patients hope sale won’t sever bonds

Sanofi’s CEO says Genzyme practice of fostering communities to continue

Linda Rubenstein, who has Gaucher disease, wondered if Genzyme’s relationships with patients will change. Linda Rubenstein, who has Gaucher disease, wondered if Genzyme’s relationships with patients will change. (Dina Rudick/Globe Staff)
By Erin Ailworth
Globe Staff / February 17, 2011

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To its patients, Genzyme Corp. is more than a drug maker. It is the lifeline that offered them hope in the face of fatal illnesses, sat with them through doctor appointments, and fought battles with insurance companies to cover treatments that cost hundreds of thousands of dollars a year.

That’s how the Cambridge biotech built its business of selling very expensive remedies for enzyme deficiencies. It fostered close ties with many of the few thousand people worldwide who suffer from these rare diseases. But with Genzyme’s sale to French pharmaceutical giant Sanofi-Aventis SA, patients wonder what will become of those relationships, which many called vital to their well-being.

“I’m a little nervous,’’ said Linda Rubenstein, a Carlisle resident with Gaucher disease who has been on Genzyme treatments for about two decades. “I’m going to think positive.’’

Today, nearly 8,000 people take Genzyme’s drugs for Fabry, Gaucher, and Pompe diseases, all of which cause waste to build up in the body, shutting down vital organs and weakening muscles and bones. Genzyme’s drugs, which can cost as much as $300,000 a year, help end the retention of waste.

Since they depend on the drugs to keep them alive, patients said, they also depend on Genzyme as more than just a pharmaceutical supplier.

When Rubenstein was diagnosed in 1991 — the year the Gaucher drug Cerezyme was introduced — Genzyme helped with insurance paperwork, encouraged her to attend meetings of the National Gaucher Foundation, and eventually enlisted her to help educate new Genzyme employees about the disease. Later, her sister got a job at the company.

“Everyone was always in the loop,’’ she said. “I’m hoping it stays like that.’’

In an interview yesterday, Sanofi chief executive Christopher A. Viehbacher said he recognized Genzyme is a leader in reaching out to people suffering from rare genetic disorders. He said Sanofi plans to continue Genzyme’s practice of building patient communities around the conditions its products treat.

Viehbacher said Sanofi, too, communicates closely with patients who take its drugs for cancer, epilepsy, depression, and other conditions.

“I think a more patient-centered model is important,’’ he said. “Patients are almost forgotten in the whole health care system.’’

Patients need to remain at the heart of Genzyme’s work, said Tiffany House of Texas, who was diagnosed with Pompe just before her 12th birthday. The disease impaired her breathing and mobility, and put her in a wheelchair. But Genzyme’s Pompe treatment, Myozyme, helped her regain much of her health and attend law school.

Now 28, House is president of the Acid Maltase Deficiency Association, a nonprofit that advocates for people with Pompe disease. She said she will push Sanofi to retain the relationships patients have built with Genzyme.

“It’s taken, oh shoot, 15 or 16 years to get to this point. And if we have to start over, then that’s what we’ll do,’’ House said. “All we can hope is that the best interests of the patients are kept in mind and that whatever happens, a relationship stays.’’

Rhonda P. Buyers, chief executive of the National Gaucher Foundation, said Genzyme’s approach to patients has long been atypical, but new players, such as rival drug maker Shire PLC of Ireland, are now emulating Genzyme in the hope of building their business.

And that, she said, means patients have options if they don’t feel Sanofi is taking care of their needs.

“As long as the patients are taken care of, that’s really the most important factor,’’ Buyers said. “It’s probably going to be a while before we know if there are going to be any repercussions.’’

Kevin R. Kline of New Jersey, a longtime Genzyme patient, recently switched from Cerezyme to Shire’s Gaucher drug therapy. He made the move after a virus found at Genzyme’s Allston Landing plant two summers ago caused shortages of Cerezyme and the company’s Fabry drug, Fabrazyme. Kline said he has been satisfied with the switch, in large part because Shire followed Genzyme’s approach and reached out to patients.

“What separates one company from the next is that we’re not treated like the customers that we are, but like actual patients,’’ Kline said. “If I know that I can make one phone call to my case manager and have that person take care of all the administrative stuff, that’s extremely important.’’

Robert Weisman of the Globe staff contributed to this report. Erin Ailworth can be reached at