Expert Insights

Expert Insights

14 Ways to Increase Recruitment in Rare & Orphan Disease Clinical Trials

14 Ways to Increase Recruitment in Rare & Orphan Disease Clinical Trials
11 July 2019

Rare diseases and orphan indications are gaining prominence because public incentives and facilitations are now making drug development for these health issues more financially viable.

A rare disease is a disease that affects a small percentage of the population. In the U.S. any disease affecting fewer than 200,000 people is considered rare; in Europe, a disease is rare when it affects less than 1 in 2000. These numbers may seem small, but with over 7,000 rare diseases already identified and more being discovered each day, rare diseases collectively make a large impact. It is estimated that 350 million people worldwide suffer from rare diseases. 

Orphan indications, on the other hand, have no definitive and convincing treatment. A frequently-occurring disease can be orphan, if there is no treatment. Both of them have some unique characteristics, which we addressed in this blog post on “7 Key Challenges of Drug Development for Rare & Orphan Diseases,” not the least of which is recruitment.  In this post we will look at ways to proactively recruit so as to overcome some of these challenges.

14 ways to conduct proactive recruitment for rare and orphan studies

We recommend a proactive, visible, recruitment strategy.

  1. Conduct database searches at study sites to pre-identify potentially eligible patients.
  2. Partner with advocacy groups, special interest groups, and self-help groups to spread awareness.
  3. Make presentations at medical congresses. Some doctors may tell their patients about the study or may even want to participate in the study as an investigator.
  4. Contact universities with medical schools as they may have patients with early diagnosis coming to them to find out what treatments are available.
  5. Consider developing a companion diagnostic to help identify mis-diagnosed and under-diagnosed populations.
  6. In cases of children’s diseases, make sure that pediatricians are aware of the study. Children see their pediatricians regularly in the early years, and pediatricians may be able to refer patients to the study.
  7. For rare diseases that are genetic in origin, ensure discussions and collaboration are taking place with the geneticist at each site.
  8. Cover the costs of genetic screening for patients and their family members if the disease of interest is genetic in nature. 
  9. Create a study website, using search engine optimization, so that when prospective patients are searching for information about their illness, your study will come up.
  10. Advertise in suitable media. Even if the patients don’t see it, a friend or relative may.
  11. Use social media. The viral nature of this medium may be what is needed to spread the information far and wide.
  12. Make it easy for patients to participate by taking the trial to them if possible. Visiting nurses who can come to the patient’s home to handle an infusion, for example, would help to boost recruitment of patients who may be too sick to travel.
  13. Use technology assistance. Virtual trials and other technology solutions can help reduce the burden of trial participation. 
  14. Contact Synteract to help you recruit!

Mental agility required by investigators and trial clinicians

Rare diseases and orphan indications present distinct challenges that require biotechs and contract research organizations to approach clinical studies with mental flexibility, creative thought processes, and unique skills. Not only do they conduct extensive research on diseases that show a similar pathophysiology and response, but they also must be able to assess each situation on a case-by-case basis. The ability to deal with diverse illnesses is perhaps more important than experience in the particular indication. After all, when a disease is rare, it is less likely that a team will have previous experience in it. Look for a team that knows how to think outside the box and that has had a diverse range of experience in multiple indications.

At Synteract, we have conducted more than 130 rare and orphan clinical trials across a wide range of therapeutic indications in in the past five years alone.

Download this free, handy checklist with ideas to maximize recruitment in rare and orphan disease trials orcontact usfor more information on drug development for rare and orphan diseases.

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