This story is from BostonGlobe.com, the only place for complete digital access to the Globe.
Scott Martin’s son was still a newborn when nurses began to suspect the baby had hemophilia. The diagnosis sent Martin and his wife into a tailspin for about six months—then they began searching for treatment.
Three years later, frustrated by the lack of options, Martin, a Texas energy executive, pooled his wealth with John Taylor, a New York financial services executive whose own son is a hemophiliac, to launch a biotechnology firm, now based in Cambridge, focused on discovering a cure for the bleeding disorder.
“I can’t tell you how many times we’ve gone out to try to raise money from third parties who said, ‘You guys are crazy,’ ’’ said Martin, noting his and Taylor’s sons are constant motivators. “[But] you kinda gotta be crazy.’’
Martin and Taylor are among the many patients and their families taking an increasingly larger role in drug development, inspiring new treatments, working closely with pharmaceutical companies, matching patients with clinical trials, paying for research, and lobbying to speed drug development.
In doing so, patients are pushing their diseases into the limelight and fundamentally changing the way the biotechnology and pharmaceutical industries, as well as the government, approach drug creation and approval, and patient care.
Patients and patient groups have taken high profiles at the Biotechnology Industry Organization convention in Boston this week, appearing on panels, protesting regulatory policies, and hobnobbing with pharmaceutical executives to press for more investment and research into new drugs.
The companies appear to be listening. Major firms such as Biogen Idec of Weston, Pfizer Inc. of New York, and French pharmaceutical giant Sanofi, the parent of Genzyme Corp. of Cambridge, have agreed to partner on several initiatives with patient groups such as The Michael J. Fox Foundation for Parkinson’s Research.
“There has not been a time that companies paid as much attention to these patient groups and patients,’’ said Margaret Anderson, executive director of FasterCures, a think-tank in Washington with a goal of accelerating medical developments. “There is a stark realization that no one is going to get where they need to go unless there is collaboration.’’
For decades, patients and their families were largely spectators as pharmaceutical companies, regulators, and research foundations decided which drugs would be developed and which diseases would be targeted. But that changed with the AIDs epidemic of the 1980s and ’90s, when victims and activists took to the streets and the halls of Congress to press government and pharmaceutical companies to do more to treat the disease, then a virtual death sentence
The activists bolstered their case by becoming experts on HIV and AIDS, able to converse with doctors and drug developers about clinical trials and the federal approval process.
“What the AIDS activists did was they broke the barriers down to let [patient] groups and this type of expertise into the room,’’ Anderson said.
The AIDS activists paved the way for advocates like Pat Furlong, whose two sons died of Duchenne muscular dystrophy in the mid-1990s. When they were first diagnosed with Duchenne at ages 4 and 6, Furlong borrowed $100,0000 to finance research, pretended to be a doctor to nab meetings with experts on the disease, and staked out pharmaceutical executives to beg them to find treatments.
In 1994, she founded Parent Project Muscular Dystrophy, a nonprofit advocacy group that invests in drug research and clinical trials for the disorder, a genetic defect that causes muscles to weaken over time for lack of a certain protein. In the last decade, the nonprofit has raised and spent about $20 million to build a registry of patients, press companies to develop better drugs, and design clinical trials.
“The human spirit will do what it needs to do to triumph,’’ Furlong said. “I protect the people that I love. It just so happens that my family has grown to include the whole Duchenne community.’’
Drug companies increasingly see such patient communities as important components of their business strategy. Genzyme pioneered patient-pharma relations as it created drugs to treat rare enzyme deficiencies. Genzyme works with patient groups to lobby government officials in various countries to make sure its expensive drugs—which can cost hundreds of thousands a year to treat a single patient—are covered by insurance or subsidized when necessary.Continued...