New voices in medical advocacy are often patients

Parent-advocates of children with chronic conditions have long worked toward finding cures; adult self-advocates are shifting the focus to goals of independent work and living.

A New Generation of Disability Advocates: The Patients Themselves. Children with disabilities or chronic medical conditions, including autism and muscular dystrophy, have grown up and are taking on the mantle of advocacy from their parents. WSJ’s Amy Dockser Marcus joins Lunch Break with details on the next generation of disabilities advocates. Photo: Shanda Venneau. Wall Street Journal, Youtube Jul 25, 2016

By Amy Dockser Marcus, The Wall Street Journal July 25, 2016

In the world of advocacy for children with disabilities, life-threatening conditions and chronic disease, a new generation of advocates is emerging: the patients themselves.

Christopher “Buddy” Cassidy Jr. was in a difficult spot last year as the sole patient on a Food and Drug Administration panel advising regulators who were considering whether to approve an experimental drug for a rare disease. He spent hours before the hearing reading company and FDA documents, then listened carefully to parents’ testimony that the drug was helping their children and slowing down the progression of the disease, Duchenne muscular dystrophy, which affects primarily males and leads to progressive weakening of muscles and premature death.

But despite the compelling personal testimony, Mr. Cassidy, a 26-year old from Annandale, Va., who has Duchenne, says he worried about the drug’s safety profile. Finding himself on a different side of the issue than many of the parents at the hearing was “wrenching,” he says.

One of those parents was Debra Miller, co-founder and chief executive of CureDuchenne, which among other things raises money for research and helped fund development of the drug discussed that day, BioMarin Pharmaceutical Inc.’s Kyndrisa. Ms. Miller’s 19-year-old son, Hawken, has Duchenne but wasn’t in the BioMarin trial. She says the results convinced her that the drug worked for some and should have been approved. “We had families standing up testifying that their son did really well on the drug…” she said. “A patient voting these families should not have the drug, I was surprised about that.”

Advocacy has often been dominated by parents of children with the disorders. One of their greatest successes is that, through better daily care, funding, and advocacy, even in cases of lethal illness, the children generally are living longer, better lives. Some of those children now are at an age where they are forcing advocacy groups and their own parents to take their views into account, even when opinions diverge.

Many advocacy groups, including Parent Project Muscular Dystrophy, were set up by parents and grandparents to raise funds and support research toward finding cures, says Annie Kennedy, the group’s senior vice president for legislation and public policy. The growing population of adults living with a disease or condition, she says, is insisting the focus also include social issues, such as insurance reimbursement for personal aides so that patients can live independently and work.

Last year, Parent Project set up an adult advisory council comprising young adults affected by Duchenne. Mr. Cassidy, who is on the council, says the experiences he and others had with anxiety and depression led the group to launch a new initiative gathering data on the mental health of people with Duchenne in an effort to better identify and treat them.

Kenneth Hobby is president of CureSMA, a group founded by families affected by spinal muscular atrophy, a genetic disease that affects voluntary muscle movement. In the past, the group has focused on the most common and severe pediatric form for the disease, in which patients diagnosed in infancy often die within two years. There is no cure for SMA but with a number of drugs in the pipeline expected to extend lifespan, the board has created a subcommittee of adults living with the disease to expand programming to issues such as transportation and reproduction, Mr. Hobby says.

Aaron Carter Bates, a 36-year-old litigator at Bates Law Group in Jacksonville, Fla., is a rights advocate for people with disabilities who has spinal muscular atrophy. He says he often speaks to advocacy groups about a more even distribution of time and resources. “A cure is important,” he says, but he wants equal attention paid to the issue of making sure individuals with disability are on “level footing with individuals who are healthy.”

In the autism community, some parent-run advocacy groups have squared off with self-advocacy organizations over recent federal rules on what counts as community housing.

Self-advocacy groups supporting the rules say people with disabilities do better when living with and integrated into the wider community. Parent groups opposing the rules say their result is to restrict options for those who want to live with larger numbers of other people with disabilities, typically with greater supervision and safety measures.

Alison Singer, co-founder and president of Autism Science Foundation, is the mother of an 18-year-old daughter with autism who she says is minimally verbal- and cognitively-impaired. Ms. Singer says self-advocates’ voices are crucial, but as a parent, she says, she understands her daughter’s needs better. Self-advocates “talk about equal rights and self-actualization,” Ms. Singer says. “We as parents are worried about safety.”

The debate illustrates the complexities as a new generation of advocates speaks out. Some parent-run groups support the position taken by Autistic Self-Advocacy Network, a prominent national disability rights group created by and for people with autism, says Ari Ne’eman, the group’s president and co-founder. On a number of other issues, he adds, many of the Network’s strongest partners are “parent organizations in the broader disability world.” Nonetheless, Mr. Ne’eeman said groups like his are a response to the “emphasis on young children and causation to the near exclusion of everything else.”

Samantha Crane, ASAN’s legal director and director of public policy, says very little federal research money has been spent on the needs of adults with autism, such as disparities in access to health care and communication technology.

In the case of BioMarin’s drug, the FDA earlier this year said it wouldn’t approve Kyndrisa for Duchenne. A BioMarin spokeswoman says the company isn’t developing Kyndrisa for Duchenne any longer, and is focusing on moving other compounds for the condition forward.

Ms. Miller of CureDuchenne said members of the community are still waiting to hear whether a Duchenne drug from Sarepta Therapeutics Inc. gets FDA approval. Her group is also funding companies pursuing other treatment options for the disease and complications. CureDuchenne sends physical therapists to meet and train other physical therapists and caregivers of Duchenne patients, she says; scientists hold workshops for parents who want to learn about trials, research, and care issues.

She said her son Hawken, a college student and editor at a university newspaper, went on a road trip this summer with friends. The voices of self-advocates like Hawken and Mr. Cassidy are important, she says, adding, “The patient is the expert.” But even as groups expand their focus, she doesn’t want anyone to forget a cure hasn’t yet been found. “There are still a lot of boys we are losing very early,” she says.

Source The Wall Street Journal