Patient groups and industry a good match for drug development

ORLANDO – From the kitchen table volunteer groups of the past trying to convince government and industry of the need for more research, to the modern patient advocacy organizations taking a role in everything from clinical trial design to underwriting new drug studies, the role of patient groups in the drug development process has undergone a dramatic transformation.

Brandon Kashfian That’s good news not only for patients, but also for industry, which has much to gain from the insights and relationships that patient groups bring to the drug development process – particularly when the focus is on rare disorders.

That was the message delivered by AltaThera Pharmaceuticals President and CEO Brandon Kashfian, who addressed a gathering of clinical trial experts at the Cambridge Healthtech Institute’s Sixth Annual Summit for Clinical Ops Executives (SCOPE) in Orlando, Fla. Feb. 24-26.

Kashfian told the group that, among other benefits, the involvement of patient groups in the drug development process has the potential to reduce the enrollment and retention hurdles that prevent more than half of all Phase II and Phase III drug studies from meeting their timelines.

By contrast, Kashfian noted, when the Friedreich’s Ataxia Research Alliance partnered with a drug company in pursuit of the rare condition’s first drug application, the organization used its patient registry to recruit volunteers and managed to fill all 60 slots at three sites in under three hours.

Kashfian also cited the work of the Cystic Fibrosis Foundation, whose patient registry includes 90 percent of the cystic fibrosis patients in the United States and was key in the development of Kalydeco, the first drug to address the underlying cause of cystic fibrosis in certain patients.

Likewise, a partnership with the Tuberous Sclerosis Alliance gave Novartis the platform it needed to spread the word about clinical trials for the drug Afinitor, which treats brain tumors associated with the disease.

Such partnerships are “a good marriage,” Kashfian said, because patient advocacy groups have a personal investment and want to be involved in the development of new therapies for their members.

“Patient groups have made clinical trials a part of their mission,” he said.


About AltaThera: AltaThera Pharmaceuticals is a specialty pharmaceutical company focused on critical care and alternative therapeutics for the unmet medical needs of patients with severe, and often rare, disorders for which few effective treatments are available. AltaThera’s commitment to improving patients’ lives includes working with patient advocacy organizations serving those communities.

Patricia Dunnigan
Manager, Communications & Marketing
AltaThera Pharmaceuticals
200 S. Wacker Drive
Suite 3100
Chicago, IL 60606
[email protected]

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