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Clinical Development

How to Identify, Enroll, and Retain Rare Disease Populations in Clinical Trials

  • February 20, 2018
Waiting Room

This post is the second of a three part blog series. Click here to read Part 1.

Enrolling enough subjects into a rare disease study can seem like an insurmountable challenge, especially when you’re looking at a subset of a rare disease, or an ultra-rare disease population. By mapping out your path, you can mitigate many potential issues. There are two key concepts: A patient-centric approach and referral networks.

Patient centricity is more than a buzzword

One important driver of success is taking a patient-centric approach at the earliest stages of the trial—even before the protocol is finalized. It should be emphasized that patient-centricity is not a buzzword—it should be baked-in to every aspect of your study. You need to understand what’s important to your patient and how study burden can be reduced. And you want to make sure that your study design and endpoints are appropriate and that your patients are going to be happy with the treatment that you’re providing.

It is critical that patients fully understand the risk/benefit profile of your study. Great care should be taken here. A well-written consent, as well as consent aids such as a video aid for what type of procedures will be done, or even illustrations of procedures especially in the pediatric population, will help minimize any fear for participating in studies.

A strategy for reducing patient burden will enhance both recruitment and retention. Reducing time away from home is critical as it often translates in time lost at work, either for the caregiver or the patients themselves. And if that’s not possible, try to make travel as easy as possible by providing concierge-level service from door to door. You can also achieve reduced time away with home-based nurse support and delivery of study medication (depending on the drug’s safety profile.) Flexibility in visit windows can help patients manage the study within their schedule. Early family/caregiver engagement is also important as accommodations for their other dependents may be necessary to obtain subject participation in the study.

Once you’ve recruited your patient population, how do you keep them engaged? Patient boards—with patient, caregiver and advocacy group representation—can provide excellent insights for protocol design and potential study challenges. The relationship with the PI/CRC is critical; support must be readily available to maximize the time dedicated to the patients. Keeping the patient as comfortable as possible is an important component of patient centricity. Personalized support products that are designed for their specific disorder can help with physical comfort while access to counseling and networking and even transport to advocacy meetings can help with emotional comfort and feelings of isolation.

Referral networks are key to successful recruitment

It’s important to build referral networks before you actually need them because they take time to set up and maintain. Getting connected with the right people ensures a higher awareness of your study. Because you’re already working in very small populations, a global approach allows patients access to clinical trials regardless of geography.

Building a referral network requires resourcefulness. Often there is registry data available which provides insights into who is treating your patient population and where. Advocacy groups can provide intelligence into all of the pockets of patients and their key physicians. Investigators typically have strong ties within their own community and can provide input on potential additional investigators. Working with a CRO with prior experience in rare diseases gives you access to many different patient populations and sites. This list can complement your own well-vetted list of sites and doctors to build into a referral network. Some rare diseases may already have a referral network in place.

Working with referral networks to access patients requires a strong communication plan. One important step is to train local staff on sponsor-approved messaging to raise awareness about the study. In-person visits, phone calls, email updates and newsletters are effective tactics. Most critical is frequent engagement with investigators to stay top of mind. If a patient needs to go to a different facility, it’s important to facilitate a smooth transition between the transferring physician with the new treating physician, with a focus on the patient’s well-being. Clear communication about the patient’s disposition and any important social considerations should be shared. There is usually a strong willingness between the investigators because they are looking to enhance their patient’s treatment options and typically take an altruistic view. A well-defined patient transfer process can help ensure that the patient has a good experience to increase retention. This should include an expansive checklist to ensure all critical items are covered before the patient is moved including that visas are in place, and any special needs are accommodated.

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