Challenge #2: Identify, recruit and retain patients

Rare disease patient identification, recruitment, and retention

We often start with patient advocacy groups. They can be very helpful getting the word out to qualified patients, and it helps to establish relationships with them well before a study starts. We often do, and our first job is to show them how the study will benefit the patients they support.

Once we identify potential study subjects, we prepare patient-friendly educational materials to help them and their families understand the purpose of the study and the benefits of participating — to their sick child and others just like him.

Enrolling one new patient often means a whole new study in a new country, and because there are so few people with the disease, you absolutely need that patient. We have the global resources to satisfy local regulatory requirements wherever and whenever you need to obtain approvals.

For some diseases, where treatments are of relatively short duration and few follow-up visits are needed, we may fly patients and their families from, say, the Middle East to a treatment center in the U.K., and fly them home again when the treatment is completed. The logistics are complicated, but we can get it done. In fact, when patients have to travel long distances to see specialists, we actually increase retention by providing extensive travel and lodging assistance.

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