‘Every patient counts’: Pharm-Olam on orphan disease trial that led to approval

By Maggie Lynch

- Last updated on GMT

(Image: Getty/IR_Stone)
(Image: Getty/IR_Stone)

Related tags Orphan drug Clinical trial Fda Ema marketing authorization Clinical trial management

After Pharm-Olam conducted an orphan disease trial for therapy for a rare blood disorder, the European Commission granted it marketing authorization, and FDA is to give it priority review.

Pharm-Olam, a full-service contract research organization (CRO) specializing in oncology, infectious diseases, and vaccines, conducted Hercules, a Phase III pivotal study for the now approved therapy, Cablivi.

Cablivi is for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP), a rare, autoimmune-based blood clotting disorder that can be fatal without treatment.

The trial was global, multi-site, with complexities beyond the orphan disease status it held.

John Colby, executive director of Global Marketing and Communications at Pharm-Olam told us, the company provided regulatory, site identification, start-up, clinical monitoring, project management, and medical monitoring for this trial.

The CRO completed enrollment for the study ahead of schedule. The trial extended over 92 sites, 16 countries, and 145 patients.

One of the key components of the trial according to Colby was, “Providing flexible support from the CRO perspective was also a critical component to early enrollment completion.”

Another critical component was supplying centralized solutions that could be adjusted to local circumstances when dealing with a global study.

“Understanding the patient landscape and how they present in the various health care systems spanning 16 involved countries was critical to this study’s success,”​ said Colby. “This is a rare disease and every patient counts.”

Most of the study participants were identified in the emergency room setting, so making the trial available to them and providing them with local resources was imperative.

Pharm-Olam worked with 92 hematologists in 16 countries, and coordinated local investigative sites to support the study, explained Colby.

In 2015, Pharm-Olam was contracted to conduct the study, along with a three-year follow-up study to characterize any long-term effects of the drug and assess repeated use.

Ablynx, a subsidiary of Sanofi, will launch its newly approved therapy, Cablivi (caplacizumab), in the German market after the European Commission granted it marketing authorization.

Following the European authorization, the US Food and Drug Administration (FDA) will give priority review to the drug.

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