Orphan drug CROs look to capitalize on influx of biotech cash

By Zachary Brennan contact

- Last updated on GMT

Orphan drug CROs look to capitalize on influx of biotech cash

Related tags: Orphan drug

As small biotech companies continue to cash in on new investments, IPOs and increasing approval rates of orphan and rare disease drugs, CROs are cashing in too and helping the companies navigate the regulatory and clinical landscape.

Although many large and mid-size CROs claim to feature service offerings directed at rare or orphan disease companies, only a couple devote their entire offerings to such companies.

One such small CRO dedicated to the orphan drug cause is PSR Orphan Experts, which recently won in partnership with Agility Clinical, the second consecutive award for “Best Orphan Drug CRO”​ for offering consulting and full clinical trial services.


Roger Legtenberg, CEO of PSR Orphan Experts, told Outsourcing-Pharma.com of the recent influx of orphan drug projects, noting that such trials require a “completely different mindset​” because of the difficulties of finding patients and ensuring that “every single data point is captured for every single patient​.”

A survey of clinical trial decision makers from last year by Premier Research, which also offers services for orphan drugmakers, found that more than two-thirds (69%) of respondents said that among the most difficult factors in recruiting patients into a rare disease clinical trial was not only finding and motivating patients to join and remain in trials, but identifying and setting up investigative sites for studies.

Premier gave the example of flying patients from the Middle East to a trial in the UK, which required the oversight of arranging for transportation of patients and parents, to visa requirements, language issues, and ongoing follow up with patients upon their return home.

We help companies think outside the box for recruitment tools, IT solutions, and ways to use the Internet in the right way to reach out to patients​,” Legtenberg said. He added that the company works with patient advocacy groups to make sure there’s enough awareness around a specific condition, and it’s upcoming clinical trials, which is crucial for successful recruitment for rare disease studies.

The key is figuring out how to make a study “as easy as possible for patients with rare diseases to participate​,” which often involves sorting out travel arrangements, logistics and patient home support, he added. “Our job is to help clients develop programs and make programs work in an efficient way by interacting with regulators​” on everything from adaptive licensing to innovative clinical trial designs.

Cost Issues

The cost of developing a clinical program also must have some leeway so that trials don’t have to be repeated if something goes wrong the first time around.  “The small and virtual companies aren’t coming to us for the cheapest deal​,” Legtenberg said. “It’s not the right approach. These companies come for expert advice, which can mean that they need to invest money upfront on proper designs and study logistics in order to save money in the long run. I know several companies that had to redo trials​” for example, because of inferior study designs upfront.

The standard way of conducting clinical trials will not fly in the rare disease space…the out of the box, innovative approaches and  close partnerships between all stakeholders is what must be offered​,” he added.

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