By Vickki Brandi, PA DHSc
It is essential to attract potential participants to rare respiratory disease clinical trials. But, that can be easier said than done. Here, we look at some ways of meeting those all-important enrollment deadlines by putting ourselves in the patient’s or caregiver’s shoes to see how we can make it easier for them to participate.
In allergy and respiratory, there are many indications that qualify as a rare disease including, but not limited to: cystic fibrosis (CF), idiopathic pulmonary fibrosis (IPF), primary ciliary dysplasia (PCD), alpha 1 antitrypsin disease (AATD) and hereditary angioedema (HAE).
There are many clinical trials worldwide seeking identical patient populations to participate in industry-funded and academic clinical trials. Those of us responsible for managing clinical trials – study site, sponsor or Clinical Research Organization (CRO) – are well aware of the significance of meeting or exceeding study timelines.
One of the most important elements for meeting timelines is ensuring that enrollment is completed on time. It is very important for the team, including study site, sponsor and CRO, to collectively think of ways to make the study more interesting to potential patients (and caregivers, where relevant) and also to reduce, if not eliminate, barriers to recruitment.
Although these methods may be specific to the study design and protocol, there are some common themes. Patient recruitment materials can be of assistance and these may include general information regarding participating in a clinical trial and study-specific “talking points” for the staff to help explain the nuances of the study.
From a patient or caregiver perspective, taking part in a study can be worrying – it possibly signals a deterioration of their disease. These materials must be linguistically and culturally relevant and cater to the appropriate age range. Importantly for rare diseases, patients may not wish to receive bags and other items that may label them as having a disease.
Another important aspect is that patients and their caregivers are likely to travel long distances to the specialized tertiary care center that serves as the study site. A reasonable travel reimbursement plan that takes into account local regulations should be outlined prior to the study start.
FDA’s “Patient Panel” meetings have provided useful insights about the patient experience and barriers to study participation. It is important for the study site, sponsor and CRO team to visualize what participating in a study is like from a patient’s perspective. Involving patients’ input, in addition to patient advocacy organizations, early in the process will reap many benefits.
To learn about our experience in respiratory clinical trials, click here.
About the Author
Vikki Brandi has more than 30 years’ experience in allergy/respiratory clinical research in addition to seven years practicing as a physician assistant. She has earned two Bachelor of Science degrees in addition to a Master’s of Science and a doctorate in health science. She has worked for large pharmaceutical companies and biotech companies in addition to CROs and has managed Phases 1-4 clinical global trials. Therapeutic areas of expertise includes CF, COPD, asthma, AATD, IPF, RSV and allergic rhinitis. Additionally, she has authored abstracts, posters and journal articles for many of these indications.
