Daily journalists often have a unique perspective on health. Their constant search for intriguing copy exposes them to issues that physicians and scientists may never encounter in the clinic or the lab. Yet what they seldom have is time – the critical months, perhaps even years that are needed to fully reveal stories that might have the power to change the lives of millions. That’s why Amy Dockser Marcus, a 2006 Robert Wood Johnson Foundation (RWJF) Investigator, who tells her story in this month’s Health Affairs, credits her term at RWJF as key to her groundbreaking reports on patient advocates spurring the search for treatments for rare diseases.
“You can’t underestimate the value of time—especially when you’re a beat reporter,” Dockser Marcus said. “It’s very hard to dig in. As an Investigator, I was able to follow people for months or a year, to see their challenges and progress.” As the only journalist in her Investigator class, Dockser Marcus also appreciated, “getting access to a very wide academic community,” she said. “The Foundation brought us together and many of us were interested in bringing patients together and learning how they could impact policy.”
A writer for more than 20 years, this time out, Dockser Marcus set out to do a great deal more than report on people with rare diseases. Her work was also deeply personal. In 2005, she won a Pulitzer Prize for her series on patients living longer on targeted cancer drugs while working for The Wall Street Journal. A few months later, as she received her RWJF Investigator Award, Dockser Marcus’ beloved Mom lost her battle with gallbladder cancer, a rare disease. “At one point, I had to decide if I had the emotional energy to do research with people who were going through what my mother went through,” she said.
But as Dockser Marcus learned more about the circumstances faced by people with rare diseases, she became committed to their cause. “I came to see this as a civil rights issue,” she said. “When 25 million people have rare diseases, individually their numbers are small, but as a group they are very significant and their medical needs are not being met.” She found that patient groups were struggling to raise money to hire their own researchers and finding that even basic data on their cancers was unavailable because there was very little interest by drug companies or academic research centers in focusing on rare tumors. And, there were usually no existing advocacy groups focused on their rare disease so patients were faced with the challenge of establishing a new entity. They were also often shut out of various forms of advocacy because they did not have the same legal heft as foundations or medical organizations.
“I came to the conclusion that there should be changes in certain policies to make it easier for these groups to get involved in venture philanthropy. Patient groups should be able to apply for grants for research, help with project management, creating databases and other methods of working together to drive research into new treatments,” said Dockser Marcus, who has been following emerging efforts to create online networks of patient groups.
“The National Institutes of Health (NIH) are trying to work with patient groups on this now, so there may be some way to help without huge policy changes, but a lot needs to be done,” said Dockser Marcus, who hopes to continue her work on the subject. “After my mother died, I decided to move forward because this is a topic that goes far beyond an individual and any one loss,” she said. “My mother was not an activist, she was a very private person, but she was happy that I was able to use her experience to help others.”
The Investigator Awards in Health Policy Research are designed to support the work of innovative scholars from a wide range of fields who undertake ambitious, cutting-edge studies of significant health policy challenges facing America.