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This piece was co-authored by  Marie McDonald, Vice President, Quintiles Advisory Services  

 
As pharma companies pursue more targeted therapies to treat new, rare and niche diseases, they are being forced to rethink their clinical operation strategies. These trials generally target highly specific populations of patients who are less prevalent, challenging to identify or find, and/or are often spread out across diverse geographies, creating potentially significant difficulties in timely and successful execution.

In new, rare, and niche diseases and populations, data insufficiencies are most acute and insidious in their ability to limit the selection of optimal sites. And in trials in which the condition, patients, and/or investigators are poorly understood, there are additional challenges to finding sites that will be able to access the right patients, investigators, and infrastructure needed for study success. This all adds time, cost and risk to the trial process, especially if sponsors rely on traditional methods of site selection.

And, while traditional methods of site selection often include utilizing sites that are familiar to the sponsor or their study partner, executing trials in new, rare, and niche disease areas may involve expanding potential site options to naïve or less commonly utilized sites.  

Where are all the patients?

Relying on a pre-existing network of well-established sites that have demonstrated an ability to recruit patients in the past is a logical approach for mainstream development of drugs that target broad patient populations, but it is less successful with targeted patient populations. In these cases, developers are finding more success when they begin by studying the disease and the treatment pathways so they can understand profiles of patient types, how patients present with the disease, the kinds of specialists they may see, and common treatments and outcomes. These data help the developers map patient density and characterize the populations in different geographies to locate target patient clusters, overlay with treaters and investigators, and ultimately identify sites where recruiting efforts might be most successful.

Gathering these data up front can directly inform how developers identify and prioritize existing sites in their networks, and determine whether they need to build or support new sites in areas when no site infrastructure exists. It also gives them greater insight into their protocol and inclusion/exclusion criteria, ensuring the right patients are recruited to the trial, and opportunities to include patients in the study are not forfeited.

Don’t wait for failure

Ideally this targeted data gathering and analysis will be done before site selection begins, though in many cases biopharma companies only use these methods after their traditional site networks fail. In these cases, they may have already spent many months operating sites that are unable to recruit enough patients because decisions were made based on assumptions that are inaccurate. In diseases where there are no current treatments, for example, patients may not be closely followed by specialists, or they will only travel to treatment centers for initial diagnosis, but then remain with their primary physician for ongoing care. Developers who assume these patients will be consistently seen by the specialist and/or be clustered around treatment centers, will be surprised when they are not there for recruiting.

Ultimately, no matter how talented or experienced a site team is, if there are no patients to recruit, the trial will fail. When developing drugs for new, rare and niche markets, sponsors need to anticipate these recruiting challenges and be willing to think more strategically about how they are going to recruit enough patients to meet the trial goals. Taking a patient-centric approach can minimize this risk of failure, and speed the time to recruiting and bringing new drugs to market.