{"id":67228,"date":"2020-07-01T11:33:41","date_gmt":"2020-07-01T15:33:41","guid":{"rendered":"https:\/\/www.imperialcrs.com\/blog\/?p=67228"},"modified":"2023-12-09T08:24:48","modified_gmt":"2023-12-09T08:24:48","slug":"rare-disease-enrollment","status":"publish","type":"post","link":"https:\/\/www.imperialcrs.com\/blog\/business-insights\/rare-disease-enrollment\/","title":{"rendered":"Essential Tips for Enrolling in Rare Disease Clinical Trials"},"content":{"rendered":"\r\n

The last decade has seen an explosion in rare disease clinical trials for therapies focused on these diseases. PhRMA<\/a> 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<\/a>, 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.<\/p>\r\n\r\n\r\n\r\n

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?<\/p>\r\n\r\n\r\n\r\n

Two things to keep in mind when trying to recruit study subjects for rare disease clinical trials:<\/h2>\r\n\r\n\r\n\r\n

Enlist Advocacy and Support Groups<\/strong><\/h3>\r\n\r\n\r\n\r\n

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.<\/p>\r\n\r\n\r\n\r\n

Keys to making advocacy and support groups an ally in your trial:<\/p>\r\n\r\n\r\n\r\n