Autism research welcomes patients, parents' input

Jonathan Kratchman, a 16-year-old with autism from Cherry Hill, likes participating in medical research studies.

"They explain everything to him," says his mother, Amy Kratchman. "For once he feels that someone is listening to him. He's always asking, 'Mom, when's the next study?' "

Over the next few years, both mother and son will be spending even more time on the front lines of autism research. Amy Kratchman is part of a research team at Children's Hospital of Philadelphia that recently received a $668,000 federal grant to help doctors understand the treatment outcomes that children and parents really want.

Clinical trials are critical because they test new treatments and help improve care. And while it may seem obvious to take the views of parents and children into account, that hasn't been the case.

Patients and health professionals often "speak a very different language," says Katherine Bevans, lead researcher on the project.

For example, she says, physicians treating autism spectrum disorder tend to "focus on things like aggressive behavior, lethargy, fatigue," which parents and children rarely mention.

"They ask, 'How does it affect the family? How does it help [the children] adapt better to new events and people?' " Bevans said. "Researchers often don't live in the real world."

The Children's grant was one of 50 nationwide made this year by the Patient-Centered Outcomes Research Institute, a Washington agency established under the 2010 Affordable Care Act. Its goal is to support comparative effectiveness research, which seeks to determine which interventions work best for particular conditions.

Despite its innocent-sounding name, comparative effectiveness has drawn fire from some critics who see it as leading to rationing of care. Some are Republican lawmakers. Others are physicians sold on procedures that researchers find have little value.

Its first grants, totaling $30 million over two years, stressed two of the agency's less controversial goals: improving communications and patient involvement.

Getting patients and clinicians on the same page is a key part of this effort.

"We see the patient as the north star," says chiropractor Christine Goertz, a member of the institute's board of governors. "They want to know, 'What are my options? What outcomes can I expect? What can I do to improve the outcome?' " (The 21-member board includes consumers, physicians, researchers, and pharmaceutical and health care executives.)

"The research has to answer the questions that real patients and doctors ask," says Lisa Simpson, a pediatrician who is president and chief executive officer of AcademyHealth, a leading health research group. "Too little research has involved Choice A or Choice B."

The institute's first funded projects included:

Involving nursing-home residents and families in decisions about transferring to acute hospital care.

Understanding consumer reactions to recent recommendations on mammography and prostate cancer screenings.

Patient and provider perspectives on what causes hospital readmissions.

Patient-centered counseling to reduce unnecessary diagnostic tests.

The agency is reviewing applications for an additional $100 million in grants, to be awarded this year, and the Obama administration has projected a $3 billion investment in comparative effectiveness over the next decade.

As part of the two-year Children's grant, researchers will develop an interactive computer program in which parents and children make a series of choices about what their treatment goals are. "Children's outcomes may be different," Bevans said. "They're more likely to talk about peer interactions. 'Will I be able to play sports, go to the prom?' "

"The technology isn't new," she says, "but the application to health research will be new."

While the grant will center on autism, the research will also deal with asthma, epilepsy, and obesity so the tool can later be adapted for those conditions. A family advisory group at Children's, made up of parents whose children have medical diagnoses, will help guide the research, as will children.

"We've assumed children can't provide information about their health needs, what they want from health care," Bevans says. "We need to know how to talk to kids about these things." Children as young as 6 can provide helpful input, she says.

Jonathan Kratchman didn't talk until he was past 3, his mother says, and her first goal and that of his doctors was to "bombard" him with speech and occupational therapy.

"What I didn't realize was that this was just a first step," she says. "The clinicians were saying, 'If he could verbalize, we'd have it down pat,' but there's a lot more to it. Realistically, is he going to be able to hold down a job if he misinterprets everything people say? We had very different ways of looking at the outcomes."

Kratchman cites epilepsy treatment as another example where patient outcomes can get complicated. "If your kid isn't having 10 seizures a week, that's great," she says. "But what if the medication makes him so drowsy that his quality of life is lousy? You can read all the studies you want, but unless [some of] it comes from a parent, a piece is missing."

Jill Conti, a member of Children's family advisory council, says that "there's a real difference in dealing with a physician who has children and the physician who doesn't," but that any physician can become more sensitive to how a treatment affects a family.

Conti's 4-year-old daughter, Andrea, suffered a stroke while in utero and one aftereffect has been epilepsy, which can cause seizures. "The doctor has to be thinking about how a medication affects her whole life," she said. "There might be a side effect some families can handle and some can't. Some kids are home all day, some are in day care.

"You're not treating a condition, you're treating a child."