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W-22: Leveraging Physician Referral Networks in Rare Disease Genotyping

Poster Presenter

Nariman Nasser

Vice President, Site Engagement
Continuum Clinical
United States

Objectives

Attendees will learn how a referral network can positively impact enrollment for a rare pediatric disease.

Method

Engaging a physician referral network resulted in more than 1000 referrals in a 6 month period. We will demonstrate best practices of how referring physicians empowered their patients to self-enroll in a clinical trial and complete home genetic/test kits.

Results

The data collected was related to the feasibility of understanding the size of the potential patient pool for pediatric patients with a rare genetic deficiency leading to obesity through the establishment a physician referral network. The data collected included: • Number and location of potential physicians as determined by diagnosis data • Number of physicians contacted/visited • Number of physicians confirming the patient population and agreeing to refer • Number of in-services conducted at physician offices • Number of referrals • Age, gender and birth year of patients referred • Number of referrals eligible/ineligible to receive genetic testing kit • Number of kits completed and returned The data proved the hypothesis that this population could indeed be identified in relatively large numbers using diagnosis data to identify a physician referral network, that physicians were highly engaged to assist their patients in completing the process, and that the population to support a formal clinical trial does exist.

Conclusion

We visited local physicians identified through diagnosis data targeting the study population to obtain referrals. Eligible patients would receive a buccal swab, instructions, and return packaging via mail. The goal was to obtain a minimum of 400 referral forms in 6 months. Focusing on physicians only in Florida & New Jersey, the team of two specialists obtained more than 1000 referrals in 9 months. Through regular follow-up the team ascertained that referring physicians were highly engaged, performing the buccal swab test for their patients or confirming that the patient completed and returned the swab to the central site. Of these referrals those who ultimately qualified to receive the genetic screening was higher than 50%. These efforts clearly demonstrated that a rare genetic pediatric disease population could be identified and referred at a high rate of eligibility using an engaged physician referral network. Implications for the positive outcome include sponsor confidence in the ability to enroll a pivotal clinical trial in the rare pediatric indication within a reasonable amount of time. As a result of these efforts the sponsor now has a registry of patients who have already been confirmed to carry the genetic marker specific to the therapy for which they are developing potential treatments, which will greatly reduce the time required to recruit clinical trials in this population. Continuum has built lasting relationships with pediatric physicians in Florida and New Jersey that are actively requesting new clinical trial opportunities for their patients, which are integrated into our database. Most importantly, patients with rare pediatric obesity disorders were able to receive genetic testing otherwise not available free of charge which will positively impact the depth of care that they receive from their physicians in the future, as well as opportunities to participate in future clinical trials testing new potential therapies where none currently exist.