Improving Orphan Drug Patient Recruitment

Despite the substantial difficulties associated with orphan drug trial recruitment, this segment of the drug development market is booming.  By 2020, orphan drug sales are expected to make up 19% of prescription drug sales, excluding generics.  In ISR’s Orphan Drug Market Dynamics and Patient Recruitment Best Practices report, ISR provides an overview of the orphan drug development market as well as how to best drive patient recruitment in an area of drug development in which drug developers do not want to be left behind. 

In the Orphan Drug Market Dynamics and Patient Recruitment Best Practices report, ISR interviewed 10 orphan drug development experts and collected quantitative data from 96 survey respondents from pharma companies that develop orphan drug compounds.  One of the takeaways featured in the report and consistently mentioned by interviewees is the varying preference for orphan drug experience.  The degree of this preference is driven by the type of work that is being outsourced.  The more logistically driven the outsourcing components, the more advantageous it is to seek third-party providers with orphan drug experience.  However, if the components being outsourced are more medically driven, targeting providers with therapeutic expertise is advantageous--particularly since regulations are therapeutically focused.

“If your company were to outsource an orphan drug/ rare disease trial, would it prefer a CRO that….”  

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In all cases, orphan drug experience was preferred by a lower percentage of respondents than the alternative (therapeutic experience and global experience).  The takeaway for sponsors is that they should consider exactly what aspects of the clinical trial are being outsourced.  If the sponsor plans to outsource the advertising, marketing, and recruitment of an orphan drug trial, ISR found that it may be worth exploring the option of working with a company that specializes in orphan drug trial recruitment--a company that knows the best practices for reaching these patients--as opposed to the medical expertise required to treat them.  What this means for CROs and recruitment specialty providers is not that orphan drug experience does not matter, but that therapeutic expertise, and global operations capabilities should not be abandoned from your positioning in exchange for orphan drug experience.  Framing orphan expertise almost as if it is an additional therapeutic area is one way ISR found companies have included orphan drug experience as a service offering.

The recruitment of patients for orphan drug trials adds levels of complexity to the already difficult process of patient recruitment.  Navigating the space as a sponsor, CRO, or specialty provider requires careful consideration of what activities need to be outsourced, which recruitment outlets need to be utilized, and how to navigate an often complicated regulatory environment.  ISR’s Orphan Drug Market Dynamics and Patient Recruitment Best Practices report explores these topics and further provides industry recommendations and best practices for those involved in the intrinsically onerous task of orphan drug trial patient recruitment.