Among all efforts made to advance research and treatment development in Friedreich’s ataxia (FA), one stands out: the Friedreich’s Ataxia Global Patient Registry run by the Friedreich’s Ataxia Research Alliance (FARA).
This FA registry aims to collect data from patients worldwide to make clinical trial recruiting a more efficient task in a rare disease — giving the trials, and the potential treatments they test, a better chance at succeeding.
“We have used the registry to recruit for [more than] 30 clinical trials (Phase 1, 2 and 3) and research studies. All our trials have met or exceeded their enrollment goals; we have not had a single trial fail due to lack of participation,” Jen Farmer, executive director of FARA, said in an email exchange of questions with Friedreich’s Ataxia News.
The registry’s benefits extend well beyond study enrollment. Because it collects basic demographic and disease information, its data helps capture a sense of how common FA is in different parts of the world, what age groups are most affected, and the disease’s impact on quality of life.
All this, in turn, can identify needs for additional research, added Katie Schultz, the group’s patient engagement director who works with FARA’s global advocacy partners and patient population to enroll participants.
Knowledge and numbers count, a lot
Friedreich’s ataxia is quite rare, affecting an estimated 15,000 or so people worldwide. But for research to be meaningful, relatively big numbers are needed.
A clinical trial or research study has to enroll enough participants to allow for a robust interpretation of results. The smaller a study, the larger the risk of results being influenced by chance.
But can adding information into a database really promote Friedreich’s ataxia research?
“Absolutely,” Schultz said. “The FA Global Patient Registry is our primary tool for understanding who has FA, where they live, and basic elements about their FA diagnosis, disease and level of disability. We then use this information … to inform and recruit for FA clinical trials, with the ultimate goal of helping scientists identify meaningful treatments.
“Patient participation is critical to helping the research community deliver on that promise.”
The FA Global Patient Registry is the only registry for patient recruitment available in Friedreich’s ataxia, with 65 countries represented among its 2,900 registrants.
Most are currently from the U.S., but Brazil has the second-largest patient group in the registry, largely thanks to a volunteer’s incredible efforts, Schultz said. Canada, Australia, and the U.K. are not far behind.
Greater worldwide participation is needed and welcome, however, to bring greater depth to the registry and the studies it supports.
FARA, in fact, is working closely with its global advocacy partners in Australia, France, Germany, Ireland, Spain, and the U.K. toward that goal, Schultz said.
“A recent meeting of the group confirmed its strong and collective commitment to supporting the registry as well as industry and academic partners who are actively engaged in FA research,” she commented.
Participation worldwide is critical because “most FA patients are not in the U.S.,” Schultz said, and because “collecting data from people in other parts of the world will help us better understand the overall prevalence of the disease, and may lead to insights about population differences, which can be important from a research perspective.”
Global trials also need global participants. For instance, Reata Pharmaceuticals — which FARA describes as a leader in FA clinical development — is currently recruiting 100 patients for part two of a possibly decisive Phase 2 trial, (NCT02255435), of omaveloxolone as a potential FA treatment, with test sites in Austria, Australia and the U.K., as well as in the U.S.
Multiple trials could also soon be “enrolling simultaneously, so we will need even more participants,” Schultz said. And researchers and pharmaceutical companies “are more likely to be involved in places where there is a critical mass of patients.”
Schultz said that FARA’s goal for the FA Global Patient Registry is “to expand outreach and register people as quickly as possible, with the hope of at least doubling the number of registrants to over 5,000 patients worldwide in the next few years.”
Better trials, especially for pediatric patients
Since the registry’s launch in 2006, it has been used to enroll patients into more than 30 clinical trials and research studies.
“Recently we had a Phase 3 trial enroll 90 subjects, with age and disability criteria, in less than 1 year,” Schultz said, adding this trial had “no screen failures.”
A screen failure occurs when an interested patient fails to meet study criteria and cannot be enrolled.
“Zero screen failures for a trial is almost unheard of — this saves time, money, and overall reduces burden on patients and research teams,” she added, emphasizing that fewer companies would be working in trials for FA treatments “if we did not have this registry. It demonstrates to them that there is an engaged and supportive patient community,” she said.
While all Friedreich’s ataxia patients are encouraged to join the registry, FARA is particularly looking to enroll more pediatric patients.
“We are very interested in collecting more information about young children with FA since it is a pediatric disorder, and currently this population is underrepresented in the FA Global Patient Registry,” Schultz said.
She encourages parents or caregivers to register their children even if they do not want them to participate in a research study or clinical trial.
“The information you provide enhances scientists’ understanding of the disease in young children, and the registry is a valuable resource for families to keep abreast of what is happening in the research community,” she said.
Importantly, FARA administrators are the only people who can access patient information, Schultz added, with data stored on a secure, password-protected server and encrypted. Personal information will never be released to anyone not affiliated with FARA or FA clinical research studies.
So, Schultz and her colleagues continue to work — using approaches as disparate as word of mouth and advertising — toward the common goal of recruiting people who will be instrumental in finding new treatments for Friedreich’s ataxia.
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