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Orphan Indications and Clinical Trials - Recruiting: Part 2 of a 2-part series

Orphan Indications and Clinical Trials - Recruiting: Part 2 of a 2-part series
21 June 2016

By Dr. Stephan de la Motte, Chief Medical Advisor, Synteract

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.

Rare diseases are those for which effective treatments may be available but that affect a relatively small segment of the population (defined as roughly .07% in the U.S. and .05% in the European Union).  Orphan indications, on the other hand, have no definitive and convincing treatment. A frequent disease can be orphan, if there is no treatment.

Both of them have some unique characteristics, which we addressed in part 1 of this blog post topic. Today we look at ways to proactively recruit so as to overcome some of these challenges.

Ways to conduct proactive recruitment for rare and orphan studies

We recommend a proactive, visible, recruitment strategy.

  • Conduct database searches at study sites to see if there are patients known to the site who might fit.
  • Contact special interest groups, self-help groups and Internet forums to inform them of the study; they will spread the word among themselves.
  • 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.
  • Contact universities with medical schools as they may have patients with early diagnosis coming to them to find out what treatments are available.
  • 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 they may be able to refer patients to the study.
  • Create a study website, using search engine optimization, so that when prospective patients are searching for information about their illness, your site will come up.
  • Advertise in suitable media. Even if the patients don’t see it, a friend or relative may.
  • Use social media. The viral nature of this medium may be what is needed to spread the information far and wide.
  • 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.

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 all kinds of diverse illnesses is perhaps more important than experience in the particular indication. After all, when a disease is rare, how much experience could a team have in it anyway? Look for a team that knows how to think outside the box and has had a diverse range of experience in multiple indications.


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