By Belonging to ATS PAR, Members Can ‘Punch Above Their Weight’ and Provide Patients with Hope
Editor’s Note: this the second and last part of a series about how the ATS integrates the patient perspective into its mission and activities. Read Part 1.
Most ATS Public Advisory Roundtable members represent orphan diseases; their organizations often started around a kitchen table. Joining PAR gives them visibility and can bolster their efforts in a way that passion alone may not. “By joining the ATS, we now are aligned with a much larger organization that really cares about issues like access to health care,” Ms. Rosbeck says. “ATS has also given us access to medical experts and researchers that we did not have before.”
Several current PAR representatives mention how important it is to their organization that they have a central location on the International Conference Exhibit Hall each year. “People know us when they see our booth at the International Conference Exhibit Hall, and they see us as friends and colleagues and vice versa,” Ms. Appell says.
PAR representatives appreciate the opportunity to serve on ATS committees. Mr. Porta says that serving on the Drug/Devise Discovery and Development Committee helps him talk to industry about the needs of children with interstitial lung disease. “I still have kids being given adult cannulas,” he adds.
Educating Patients Everywhere
Ms. Manion is the new patient representative on the Patient and Family Education Committee and is particular happy with the assignment. “The Patient Education Series is fantastic and a model for patient groups when they are developing their own materials,” she says. “And because ATS makes it freely available, we mail it to our communities and send it all over social media.”
Ms. Manion says that she wants to help build on the success of the series by “constantly thinking about it from the patient perspective--about topics to be covered, ways to present the materials that are very useful to families.” She believes that a particular strength of the series is that so many topics covered, like pulmonary function testing, breathlessness, and oxygen therapy, are resources for many different groups of patients with chronic respiratory disease. She believes that some of the “day-to-day” issues patients and their families must deal with—from cleaning their equipment to setting up oxygen tanks—might be presented most effectively in how-to videos.
PAR organizations also find that being part of the ATS helps them reach people with the disease their organizations is dedicated to helping. Each year, the Meet the Expert event held on Saturday during the International Conference draws about 250 patients and family members to learn about advances in treating a broad range of respiratory diseases, and more than 17,000 people visit the Lung Disease of the Week webpages.
“Because children’s interstitial lung is ‘ultra rare,’—there may be 6,000 patients altogether in the U.S.—the chILD Foundation hosts a regional event, we may have 20 families in attendance,” Mr. Porta says. “The last Lung Disease of the Week webinar we had 87 patients and families members join live.”
Research Partners
From the very beginning, the relationship between the ATS members and PAR has been mutually beneficial. In no area has that been more apparent than in supporting research. The Society is widely regarded as the leading scientific respiratory organization and among its members are some of the most prominent basic, translational, and clinical researchers in the world. At the same time, supporting research to improve the quality of life of patients and, ultimately, to find a cure for the particular disease they are dedicated to is central to the mission of every PAR organization.
The first Research Program grants awarded were partnership grants, with PAR organizations providing half the funding and the ATS matching those grants. These partner grants continue to be important to early career investigators. In 2016, 10 of 28 grants by the ATS Research Foundation were partnership grants. Last year, the Primary Ciliary Dyskinesia Foundation funded its first partnership grant with the ATS. The PAR group also connected ATS with two families affected by PCD, who funded two additional partnership grants. Ms. Manion says that the rigorous review process that the ATS Scientific Advisory Committee conducts of research proposals was instrumental in gaining the additional funding.
Dr. McCormack, who is now director of the pulmonary, critical care, and sleep program at the University of Cincinnati, says the partnership between ATS’s professional members and its PAR members will be crucial, particularly when it comes to testing potential therapies for the diseases the groups represent. His work with the LAM Foundation led to a randomized controlled trial, and ultimately FDA approval, of sirolimus for the treatment of LAM. The young women who participated in the trial made enormous sacrifices—most notably, knowing that they might be assigned to the placebo arm of the trial and miss out on a promising therapy, available off-label, for two years.
PAR builds trust between health care professionals and patients and that trust is necessary to recruit patients to clinical trials. An accompanying editorial to Dr. McCormack and colleagues’ New England Journal of Medicine report on the sirolimus trial notes the importance of having “patients with such a rare disease” who are “willing to put themselves at risk in order to find a treatment or a cure.” One of the things you can learn from patients with a disease like LAM, Dr. McCormack says, is courage. For patients and families, courage is easier to muster when the ATS and its member researchers offer hope.
Challenges and Opportunities
This is not to say that the relationship between ATS and PAR member organizations is without its challenges. Just getting patients involved in the ATS can be difficult because many have to travel with oxygen. “For someone not on oxygen, an hour or two drive to attend a meeting isn’t a big deal,” Mr. Porta says. “But for someone on oxygen it can be a hurdle, so imagine how much more difficult it is for people on oxygen if they have to fly to attend a meeting.”
Former ATS president Atul Malhotra, MD, a strong supporter of PAR, notes that patients and their advocates are understandably biased towards their disease. “This is perfectly human nature, especially if you have a rare disease: you want everyone talking about it,” he explains. “But the scope of ATS is quite broad—adult and pediatric pulmonary, critical care, and sleep—so you sometimes have to step back and ask what is best for the Society.”
PAR member organizations face an additional challenge in gaining—and keeping attention—focused on their disease within a professional society known for its broad medical and scientific scope. PAR organizations routinely rotate off the roundtable to make room for other patient and public health organizations. Off PAR, Ms. Appell says, “it’s harder to stay connected to the family and to stay abreast of everything going on in the ATS.”
From a patient perspective, the ATS’s “laudably high standards” can prevent the Society from giving practical advice to patients, Ms. Manion says. Without rigorous scientific studies, for example, the ATS cannot produce airway clearance therapy guidelines, so patients and their families must decide on their own what seems to work best. This situation is unlikely to change, Ms. Manion adds, because airway clearance has been done for so long that it would be unethical to ask patients to stop and serve as a control in a properly conducted clinical trial.
There is also the challenge of relying solely on PAR, whose member leaders are highly knowledgeable about the diseases they represent, when it comes to producing patient education materials. “PAR cannot cover all the conditions and diseases that we need patient input from,” Dr. Sockrider says. “Plus the patients who do volunteer often know more that the average person diagnosed with the condition.” Dr. Sockrider adds that some patients may come to believe that their experience of a disease is universal, when it fact it may not be representative of all, or even most, patients.
The answer to these problems, she believes, is to have multiple patient reviewers—just as there are multiple expert reviewers of patient education pieces. She would also like to see ATS adopt an Amazon-style review system to gather up as much information about the strengths and weaknesses of a patient education piece so that it can be improved. “We really want to tap into the target audience who will read these pieces,” she says.
For the most part, the health care professionals, patients and patient advocates within the Society have found ways to overcome obstacles and resolve differences that have strengthened the partnership and made it more productive. The result, says Ms. Appell, is that as ATS PAR nears the end of its second decade, the biggest challenge may be that “there are more great opportunities for collaboration than the patient organizations have the bandwidth to get involved with--and that is an enviable model of patient engagement for any organization."
