Essential Tips for Enrolling in Rare Disease Clinical Trials

by Dan McDonald

The last decade has seen an explosion in rare disease clinical trials for therapies focused on these diseases. PhRMA reports that there are more than 700 orphan drugs currently in development or regulatory review. While rare diseases are rare in a singular sense, when combined, 1 in 10 Americans has a rare disease. According to the PhRMA, 90 percent of rare diseases have no approved medicines. Fifty percent of people affected by rare diseases are children, 30% of whom will not survive until their 5th birthday. So, the growth of company pipelines and the development of government strategies to fight this war are welcome signs for millions.

How do you find people with a rare disease to volunteer for enrolling in your rare disease clinical trial? That is a question facing the clinical operations teams at many biopharmaceutical companies. With years of evidence that most trials take much longer than expected to enroll, including those with broad-based chronic conditions, it seems like enrolling in a rare disease-focused trial would be an insurmountable task. Not true. The key is knowing where to look, who to collaborate with, and asking a simple question: How can I add value to the lives of individuals with a rare disease and with their support network?

Two things to keep in mind when trying to recruit study subjects for rare disease clinical trials:

Enlist Advocacy and Support Groups

When it comes to rare diseases, the advocacy and support community often owns the relationship with the patient. Meaning, those individuals are heavily dependent on these groups as an information source, whether that be about the best physicians and care available, new treatment, or quality of life support. As such, it is often very important to recruit these organizations. It always surprises me when we work with a biopharmaceutical company on a recruitment project, and the company does not have a relationship with the top advocacy groups for the disease they are looking to treat. Advocacy groups are often hungry for information on new treatments in research, and part of their credo is to deliver such information to their members and other stakeholders.

Keys to making advocacy and support groups an ally in your trial:

  • Build a list of organizations and key contacts.
  • Call them to share more about the trial and to gauge their interest in receiving information.
  • Email and/or mail information about the study.
  • Request distribution of your information to members in their newsletters, on their website, and on bulletin boards.
  • Some advocacy groups have a clinical trials page or a database. Request that they include your trial in their listings.
  • Offer to provide other helpful information and support. Ask, how can we help you?
  • Check in regularly.

Get Local Through Event Participation

There are thousands of fundraising events taking place across the globe during any given week. As the COVID-19 pandemic eases, and life begins to return to normal, races, walks, galas, and other events will again bring people together in support of a cause. The advocacy groups that support rare and orphan indications are no different. Visit their websites and make a list of events taking place in the year ahead. Look for ways to get involved. Most have an opportunity to sponsor the event. Some will let you set up a table and distribute information. Others may offer post-event timeslots for hosting a gathering. While you might not be able to put people on the ground in such instances, you can support your study site with participation through your centralized efforts. Making the calls, booking tables, sending information for distribution, getting the word out, and more.

There are additional resources and approaches you can take for your rare disease clinical trials beyond the two noted here. You can purchase databases for direct mail campaigns. There are many digital-only support groups, like those found on Facebook Groups. You can organize your own events or start your own groups. We have found that the secret sauce for success is persistence, a desire to help and add value to the groups, providing good quality information, and meeting stated commitments.

Imperial has managed hundreds of community outreach efforts involving advocacy groups for enrollment in clinical trials. Contact us, we would love to speak with you about your trial and discuss custom ways that we can help you identify, educate, enroll, and retain rare disease patients in your study.

Updated 17 February 2023

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1 comment

PGDCR May 5, 2021 - 10:28 am

Great post about rare disease enrollment. I am doing internship it is very helpful for me. I am currently working on Clinical Research and I found this post and it’s most useful. This would be the best place to source all the necessary information for beginners.


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